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05: Patient Engagement [clear filter]
Sunday, June 23
 

9:00am

SC22: #22: Protocol Co-Design with Patients and Advocates
Component Type: Tutorial
CE: ACPE 3.25 Application UAN: 0286-0000-19-502-L04-P; CME 3.25; IACET 3.25; RN 3.25

This short course will allow participants to experience multiple activities which encourage active collaboration among patients, trial sponsors, and advocates. Following a brief introduction outlining the benefits of interactive activities with multiple stakeholders, you will engage in several activities:
  • Protocol red lining (individual/partner activity)
  • Design your own protocol (small group activity)
  • Walk the protocol (large group activity)
The first activity asks you to react to a draft protocol individually. You will then share your reactions with a partner representing a different stakeholder group. The second activity encourages collaboration among multiple stakeholders as table teams work to design a protocol that meets the needs of all stakeholders. The third and final activity will have the entire group reacting individually to a sample protocol (represented by posters on the wall), then discuss their reactions and seek consensus. Each activity will be debriefed to not only assess the experience, but to discuss how a similar activity might be applied to your needs. We will conclude by presenting a case-study that outlines the successful application of these methods for trial design. These exercises were designed by clinicians, instructional designers, and experts in patient literacy. They have been used successfully in numerous workshop settings to obtain input on protocol design features and trial recruitment and retention methods. Feedback on the experience from past participants has been very positive. It is our hope that you will leave the workshop with an understanding of how these types of exercises can be designed and executed to encourage collaboration and to design clinical trials that meet the needs of all stakeholders. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for individuals responsible for clinical trial design and protocol development; clinical trials operations professionals; advocacy leaders who wish to collaborate with trial sponsors; CRO professionals; patient engagement professionals; advocacy engagement professionals.

Learning Objectives

At the conclusion of this course, participants should be able to:
  • Evaluate valuable and detailed input from patients and advocates on clinical trial design;
  • Design patient interactions that encourage collaboration among patients, sponsors, and advocacy groups;
  • Critique clinical trials to ensure they meet the needs of all stakeholders.



Speakers
avatar for Kelly Franchetti

Kelly Franchetti

RN, CCRN, CEN Vice President, Global Head Patient Insights & Engagement, ICON plc.
Kelly has a diverse background comprised of nursing, advocacy & clinical research. She is also the mother of a child with a rare disease. This gives her a unique 360 degree view of patients’ & caregiver’s perspectives in the realm of disease management, treatment and burden. As... Read More →
avatar for Maggie Adamski

Maggie Adamski

Senior Director, Global Patient Insights, ICON plc.
Maggie brings over 25 years of marketing and clinical research experience within pharma, CROs and agencies. Her work spans patient recruitment, advocacy engagement, patient insights, site training and patient engagement technology. Currently, as Senior Director, Global Patient Insights... Read More →
avatar for Ariel Rosen

Ariel Rosen

Director, Global Patient Insights, ICON plc.
Ariel has over 15 years of wide-ranging experience in clinical research including tenures as a study coordinator at large academic medical centers. She has an MS in Clinical Neuropsychology and spent a large portion of her career focusing on CNS indications. Ariel has extensive experience... Read More →


Sunday June 23, 2019 9:00am - 12:30pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

9:00am

SC42: #42: Patient Preferences: Using Conjoint Analysis and Stated Preferences in Drug Development and Regulatory Decision Making
Component Type: Tutorial
CE: ACPE 6.50 Application UAN: 0286-0000-19-519-L04-P; CME 6.50; IACET 6.50; RN 6.50

The need to understand patient preferences for health and healthcare has become well established and demand for stated preference studies has grown exponentially in recent years. This short course is designed as an introduction to a range of stated-preference methods and the application of these methods in drug development, regulatory decision-making, and patient advocacy. The short course will also provide an overview of good research practices and principles that are broadly applicable to all stated-preference methods and describe how good research practices can be applied to discrete choice experiments and several other stated-preference methods. Topics to be covered will include designing a survey, developing an experimental design, analyzing data, and presenting results. This short course will include hands-on exercises and detailed case studies of recent empirical examples to illustrate concepts. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This introductory to mid-level course designed to acquaint attendees with the current state of the art in the use of methods for the development of evidentiary patient preference information. Expected attendees may include: industry, regulators, payers, patients and patient advocacy representatives.

Learning Objectives

At the conclusion of this course, participants should be able to:
  • Identify when & how to successfully develop and present patient preference information for use in a range of applications, including regulatory interactions such as new drug applications;
  • Discuss specific methodologies frequently used in the development of patient preference information.



Speakers
avatar for Rachael DiSantostefano

Rachael DiSantostefano

Senior Director, Benefit Risk, Epidemiology, Janssen Research & Development, LLC
Rachael L. DiSantostefano, MS PhD, is a Senior Director of Benefit-Risk in the Epidemiology Department within Janssen Pharmaceuticals, R&D, LLC. She has 25 years of pharmaceutical research experience across the quantitative disciplines of epidemiology, biostatistics, and health outcomes... Read More →
avatar for Brett Hauber

Brett Hauber

Senior Economist, Vice President of Health Preference Assessment, RTI Health Solutions
Brett Hauber is Senior Economist and Vice President of Health Preference Assessment at RTI Health Solutions and Affiliate Associate Professor in the School of Pharmacy at the University of Washington. His is an expert in stated-preference methods. He was principal investigator for... Read More →
avatar for Carol Mansfield

Carol Mansfield

Senior Economist and Head, Health Preference Assessment, RTI Health Solutions
Carol Mansfield, PhD, is a Senior Economist and Head in the Health Preference Assessment group at RTI-HS, where she conducts stated-preference studies for pharmaceutical applications. She has 25 years of experience conducting research related to health and the environment. She has... Read More →
CP

Christine Poulos

Senior Research Economist and Head, Health Preference Assessment, RTI Health Solutions
avatar for Kristin Bullok

Kristin Bullok

Benefit-Risk Management Scientist, Global Patient Safety, Eli Lilly and Company
Kristin Bullok, PhD, is a research scientist in benefit-risk management at Eli Lilly and Company, Global Patient Safety. Since joining Lilly, she has years of combined experience in conducting structured benefit-risk assessments, consulting on patient preference trade-off studies... Read More →


Sunday June 23, 2019 9:00am - 5:00pm
Room 11B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

1:30pm

SC37: #37: Patient Literacy 101: Practical Strategies for Improving Your Patient Materials
Component Type: Tutorial
CE: ACPE 3.25 Application UAN: 0286-0000-19-516-L04-P; CME 3.25; IACET 3.25; RN 3.25

This short course is designed to teach participants a step-by-step approach to designing patient materials and educational initiatives that effectively communicate concepts at a level appropriate for their audiences. Patient literacy (or health literacy) is certainly not a new topic, yet isn’t getting its much-deserved attention these days. Materials developed for patient communications or education often miss the mark. The application of literacy and numeracy principles is now more critical than ever, due to the increased tendency of patients to seek out disease state and treatment-related information on their own. In this interactive short course, participants will learn principles of patient literacy, receive a number of tools to assist them in developing their own patient-literate materials, and learn about excellent resources that can be used to help them continue to develop their skills in this area. Interactive exercises will provide opportunities to practice these skills. This will include evaluating existing materials, editing to improve literacy, and development of new materials. We will address both written and oral communication. Finally, an active discussion will provide strategies on how participants can work to encourage a culture of patient literacy within their own organizations, becoming champions of this approach and bringing about true cultural change. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for individuals responsible for the design and development of patient-facing materials; clinical educators; developers of patient educational materials.


Learning Objectives

At the conclusion of this course, participants should be able to:
  • Define patient literacy, numeracy and health literacy, describe how each affects the quality of patient communications and educational efforts;
  • Identify common mistakes in patient-facing materials;
  • Develop a strategy for improving your organization’s patient-facing communications;
  • Develop a culture that embraces patient literacy principles.



Speakers
avatar for Kelly Franchetti

Kelly Franchetti

RN, CCRN, CEN Vice President, Global Head Patient Insights & Engagement, ICON plc.
Kelly has a diverse background comprised of nursing, advocacy & clinical research. She is also the mother of a child with a rare disease. This gives her a unique 360 degree view of patients’ & caregiver’s perspectives in the realm of disease management, treatment and burden. As... Read More →
avatar for Valerie Martin

Valerie Martin

Associate, Global Patient Insights and Engagement, ICON plc.
Valerie’s career has been centered around leading and managing large-scale, global patient recruitment and retention programs. She also has extensive experience crafting individualized site support initiatives to accelerate enrollment. She prioritizes the needs of each program’s... Read More →
avatar for Ariel Rosen

Ariel Rosen

Director, Global Patient Insights, ICON plc.
Ariel has over 15 years of wide-ranging experience in clinical research including tenures as a study coordinator at large academic medical centers. She has an MS in Clinical Neuropsychology and spent a large portion of her career focusing on CNS indications. Ariel has extensive experience... Read More →


Sunday June 23, 2019 1:30pm - 5:00pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Monday, June 24
 

11:00am

#112: Collecting Better Patient Experience Data: Lessons Learned from Patient Organizations
Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-526-L04-P; CME 1.00; IACET 1.00; RN 1.00

We will present methods and success stories from three rare disease patient organizations that have collected meaningful patient experience data. Panelists will share their insights and challenges for data collection including surveys and patient registries.

Learning Objectives

Describe the importance of participating in registries and the methods by which data is collected; Identify strategies for overcoming the challenges of patient engagement to help provide pathways towards accelerating rare disease research.

Chair

Laura Trutoiu, PhD

Speaker

Panelist
Monica Weldon

Panelist
Pamela Mace, RN



Speakers
PM

Pamela Mace

Executive Director, Fibromuscular Dysplasia Society of America
At the age of 37, Pam Mace was diagnosed with a rare disease called Fibromuscular Dysplasia (FMD). She struggled with her health for the first few years after her diagnosis and realized that medical professionals had no real understanding of the disease. Pam became an advocate for... Read More →
LT

Laura Trutoiu

Director of Research, The Association For Creatine Deficiencies
Laura is a computer scientist and the Director of Research for the Association for Creatine Deficiencies. She holds a PhD in Robotics from Carnegie Mellon University and she has conducted research in several top industry labs including Disney Research, Industrial Light and Magic... Read More →
avatar for Monica Weldon

Monica Weldon

President/Chief Executive Officer, Bridge the Gap - SYNGAP Foundation
In November of 2012, Monica's twin son, Beckett, was the first to be diagnosed at Texas Children's Genetics Clinic with the gene mutation SYNGAP1 (6p21.3). When Beckett was 4 months old, she noticed he was not meeting the same milestones as his twin sister. Monica then began a journey... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

11:00am

#114: Patient Perspective Diversity: Taking Cultural Differences in Patient Views Into Consideration in US, EU, and Japan
Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-528-L04-P; CME 1.00; IACET 1.00; PDU 1.00 PMI 2166ASOAHB; RN 1.00

This session will discuss potential cultural impacts on various patient engagement processes, especially in US, UK and Japan. Presenters will briefly overview the contextual differences, such as medical system, provide actual cases, and facilitate the participants to identify tips to listen to patients globally, in order to incorporate those into global drug development.

Learning Objectives

Describe general patient practice and value differences (including in patient-doctor relationships) between Eastern culture and Western culture, and also understand the background to those differences; Identify potential opportunities and limitations to leverage patient voices in different cultures; Discuss further incorporating global diverse patient voices into drug development.

Chair

Atsushi Tsukamoto, PhD

Speaker

Nurse Point of View
Noriko Fujiwara, MS, RN

Intercultural Point of View (UK)
Gareth Julian Monteath, PhD, MBA, MS

USA Point of View
Robert Hilke, MA



Speakers
avatar for Noriko Fujiwara

Noriko Fujiwara

Research Nurse, Project Researcher, Department of Palliative Medicine, IMSUT Hospital, The Institute of Medical Science, The University of Tokyo
Ms. Noriko Fujiwara is a research nurse and a project researcher of the department of palliative medicine, the Institute of Medical Science, the University of Tokyo. She has over 10 years' experience in research field. She is a Certified Nurse Specialist in Cancer Nursing with the... Read More →
avatar for Atsushi Tsukamoto

Atsushi Tsukamoto

Vice President, New Drug Regulatory Affairs, Daiichi Sankyo Co., Ltd.
Atsushi is VP of New Drug Regulatory Affairs (RA) at Daiichi Sankyo. He is also Head of Specialty Medicine RA in R&D division, to lead gobal specialty medicine regulatory strategies. As Head of Japan New Drug RA, he is responsible for leading new drug RA activities for all projects... Read More →
avatar for Robert Hilke

Robert Hilke

Chief Executive Officer, Hilke Communications Corporation
Robert Hilke is the CEO of Hilke Communications Corporation, a firm specializing in providing intercultural communications and TOEIC training for global businesspeople, many of whom work in the pharmaceutical industry. Mr. Hilke's primary research interest is the management of diverse... Read More →
avatar for Gaz Monteath

Gaz Monteath

Executive Director, Link Global Solution Inc.
Dr. Gareth ("Gaz") Monteath is Executive Director at Link Global Solution Inc., an HR and OD consulting firm based in Tokyo. In his position, Gaz is responsible for the overall content and quality of programmes. LGS has long experience of working with pharmaceutical and medical instruments... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 15AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

11:00am

#120: FDA Payer Communication Guidance, Twenty Years in the Making: Now What?
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-533-L04-P; CME 1.00; IACET 1.00; RN 1.00

The FDA Payer Guidance released in 2018 has provided some clarity to industry and payers. The FDA explicitly noted the important difference between information required for medical product approval and what is needed by payers to make decisions about population-based coverage and reimbursement. The FDA Payer Guidance is largely pragmatic regarding HCEI communications and advances industry’s proactive dialogue with payers. However, there remains areas of uncertainty within the guidance. A panel composed of the relevant stakeholders will have an open dialogue on the intention of the guidance, the challenges with developing a guidance for industry and payers, the practical challenges of operationalizing aspects of the guidance, the remaining informational needs of payers from industry to inform access decisions.

Learning Objectives

Discuss how to decrease the anxiety in proactively communicating relevant coverage and reimbursement information from industry to payers for population-based coverage decisions.

Chair

Sissi Pham, PharmD

Speaker

FDA Perspective
Sheila Ryan, MPH, RAC

Panelist
Chris Leo Pashos, PhD

Panelist
Mitchell Higashi, PhD

Panelist
Denise Sanchez, JD, MPH, MS



Speakers
avatar for Mitchell Higashi

Mitchell Higashi

Vice President, Health Economics and Outcomes Research, Bristol-Myers Squibb
Mitch is the Vice President of HEOR at Bristol-Myers Squibb. Prior to joining BMS, he was the Chief Economist for GE Healthcare, where he led global efforts in health economics, pricing, reimbursement, and health policy. Mitch is a published author, including JAMA, and his work in... Read More →
avatar for Chris Pashos

Chris Pashos

Vice President, Global Evidence Strategy, AbbVie Inc.
Dr. Pashos oversees AbbVie’s Real-World Evidence Generation and Patient Centric Research Center of Excellence, demonstrating the value of medicines in addressing needs perceived by regulators, health technology assessment agencies & payers, the medical community and patients. He... Read More →
avatar for Sissi Pham

Sissi Pham

Chief Executive Officer, AESARA
Sissi Pham is a founder and CEO of AESARA, Inc. and AESARA Foundation, both established in 2016. AESARA, Inc. was established with the commitment to absorb the administrative cost of the AESARA Foundation to enable the charity to give 100% of the donations to directly to humanitarian... Read More →
SR

Sheila Ryan

Acting Director, Division of Supply Chain Integrity, Fda/cder/Office Of Compliance/Office of Drug Security, Integrity and Response
Sheila Ryan, PharmD, MPH, RAC is currently the Acting Director of the Division of Supply Chain Integrity in the Office of Drug Security, Integrity and Response within CDER's Office of Compliance at FDA. She has over 15 years of FDA regulatory experience, including over 10 years in... Read More →
avatar for Denise Sanchez

Denise Sanchez

Executive Director, Regulatory Affairs Advertising and Promotion, Allergan
Denise joined Allergan with 15 years of combined industry, government and law firm experience. Prior industry roles include Regulatory overview of commercial promotion and practices at Ironwood Pharmaceuticals, Celgene Corporation, and Cubist Pharmaceuticals. While at the law firm... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

1:15pm

#129 EE: The Potential Value of Shared Decision Making (SDM) in Clinical Trial Consideration and Participation
Component Type: Workshop
Level: Intermediate

Shared decision making is a form of collaborative communication to help patients and their providers consider various care options and potential clinical trials. Through facilitated discussion and small group activity, participants will examine the value of SDM from the point of view of people living with different disease types, from common, chronic diseases, such as diabetes, to various stages of cancer and rare diseases. They will also consider a study design with patient-centered outcome measures related to decision-making and patient experience.

Learning Objectives

Explore the application of SDM in discussions including clinical trials as options; Examine examples from clinical development operations, assess them for their suitability for decision support; Discuss the challenges of implementation Explain a study design that could help demonstrate the potential of SDM to transform traditional clinical trial recruitment into patient-centered decision support.

Chair

Mary Stober Murray, MBA

Speaker

Facilitator
Diana Pankevich, PhD

Facilitator
T.J. Sharpe, PMP



Speakers
avatar for Mary Murray

Mary Murray

Associate Director, Diversity and Patient Engagement, Bristol-Myers Squibb Company
As an Associate Director for Diversity and Patient Engagement at Bristol-Myers Squibb, Mary facilitates collaborations with disease-specific and community-based organizations to bring relevant clinical trial options to patients and representative populations. Mary has co-chaired the... Read More →
DP

Diana Pankevich

Director,Innovation Policy, Pfizer Inc
avatar for T.J. Sharpe

T.J. Sharpe

Patient Advocate, Starfish Harbor LLC
T.J. Sharpe is a Stage IV melanoma patient who shares his journey through cancer in the Patient #1 Blog.  He was diagnosed in August 2012 with melanoma tumors in multiple organs, only four weeks after his second child was born. Since then, he has undergone six surgeries and four... Read More →


Monday June 24, 2019 1:15pm - 2:00pm
E and E Area Exh Hall San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:15pm

#142: Show Me the Money! Patient and Caregiver Roles and Compensation in Research, Development, and Innovation
Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-542-L04-P; CME 1.00; IACET 1.00; RN 1.00

With advancements in stakeholder engagement prompted most recently by the 21st Century Cures Act, PCORI, and FDA’s Patient-Focused Drug Development initiatives, patients and caregivers have increasingly shifted from being passive subjects to more active partners in research and healthcare innovation. Groups like the Clinical Trials Transformation Initiative have established best practices for how and when to engage patients in research and development, NIH’s NCATS has created toolkits to support patient engagement in therapy development, and PCORI has put forth recommendations that patients be included as valued research team members who are appropriately compensated for their time. However, no framework to date has put a spotlight on patient and caregiver motivations and expectations around involvement and compensation nor mapped competencies and support needs from their standpoint. As we work towards embracing patients and caregivers as valued partners, it is important to understand challenges as well as best practices to ensure we can pave a path forward.Through our research we will illustrate the motivations, needs and expectations of patient and caregiver involvement in research and healthcare innovation.

Learning Objectives

Discuss the types of roles patient/caregivers serve in research, development, and healthcare innovation; Describe the various mechanisms for patients and caregivers to get engaged and their respective barriers/facilitators to involvement from the patient/caregiver perspective; Discuss patient/caregiver views on ethics, competency, and support required for each role; Illustrate patient/caregiver motivations and expectations around involvement and compensation.

Chair

Sarah Krug, MS

Speaker

Panelist
Bray Patrick-Lake, MS

Panelist
Veronica Todaro, MPH

Panelist
Jaye Bea Smalley, MPA



Speakers
avatar for Sarah Krug

Sarah Krug

Chief Executive Officer, CANCER101
Sarah Krüg is the CEO of CANCER101, a patient advocacy organization whose mission is to help patients and caregivers navigate the cancer journey and partner with their healthcare team to make informed decisions. Sarah is also Founder of the Health Collaboratory, a global innovation... Read More →
avatar for Bray Patrick-Lake

Bray Patrick-Lake

Director of Stakeholder Engagement, Duke Clinical Research Institute
Bray Patrick-Lake supports efforts to actively engage patients, health advocacy organizations, and other stakeholders in Duke Clinical Research Institute’s local and national research programs. She has led extensive efforts through the CTTI to incorporate patient voice into clinical... Read More →
avatar for Jaye Bea Smalley

Jaye Bea Smalley

Director, Global I and I Patient Advocacy and Life Cycle Management, Celgene Corporation
Jaye Bea joined Celgene as Global Director, Patient Advocacy and Life Cycle Management, Inflammation & Immunology in March 2018. Her twenty-five-year career in health care includes extensive experience in the public and private sector focused on advancing the needs and interests of... Read More →
avatar for Veronica Todaro

Veronica Todaro

Chief Operating Officer, Parkinson's Foundation
Veronica (Ronnie) Todaro, M.P.H., has led the national programs of the Parkinson’s Disease Foundation (PDF) since joining the organization in 2004. In this role, she has pioneered national programs focused on patient engagement both at PDF and in the broader community. She has advocated... Read More →
avatar for Veronica Todaro

Veronica Todaro

Chief Operating Officer, Parkinson's Foundation
Veronica (Ronnie) Todaro, M.P.H., has led the national programs of the Parkinson’s Disease Foundation (PDF) since joining the organization in 2004. In this role, she has pioneered national programs focused on patient engagement both at PDF and in the broader community. She has advocated... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#158: Enhancing Patient-Focused Outcome Assessment in Medical Product Development
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-568-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will discuss the evolution of patient-focused outcome assessment and how current FDA efforts and resources support measuring what matters most to patients in medical product development. Join the Study Endpoints Community for a follow up Round Table discussion on Tuesday, June 25, 9:30-10:30AM in the DIA Community Zone 2, Sails Pavilion.

Learning Objectives

Describe FDA's activities to promote patient voice in outcome measurement; Describe FDA resources to support the science of patient input in medical product development.

Chair

Elektra Johanna Papadopoulos, MD, MPH

Speaker

Update from CDRH
Michelle Tarver, MD, PhD

Panelist
Telba Irony, PhD

Industry Perspective
Robyn T. Carson, MPH

Patient Perspective
Bray Patrick-Lake, MS



Speakers
EP

Elektra Papadopoulos

Associate Director, Clinical Outcome Assessments Staff, OND, CDER, FDA
Dr. Papadopoulos serves as the Associate Director of the Clinical Outcome Assessments Staff in the Office of New Drugs in the Center for Drug Evaluation and Research (CDER). The staff provides consultation to CDER’s Review Divisions as well as other FDA Centers on clinical outcome... Read More →
avatar for Michelle Tarver

Michelle Tarver

Director of Patient Science and Engagement, OST, CDRH, FDA
The Patient Science and Engagement Program fosters innovative approaches to collecting, analyzing and integrating the patient perspective in the development, evaluation and surveillance of medical devices, including digital health technologies. Under her leadership, CDRH is making... Read More →
avatar for Robyn Carson

Robyn Carson

Head, Patient-Centered Outcomes Research, Global Evidence and Value Development, Allergan
Robyn T. Carson has worked in the pharmaceutical industry for 12 years and leads the Patient-Centered Outcomes Research (PCOR) function at Allergan. In this role, Robyn is responsible for the development of innovative strategies to evaluate meaningful outcomes to patients and consumers... Read More →
avatar for Telba Irony

Telba Irony

Deputy Director, Office of Biostatistics and Epidemiology, CBER, FDA
Telba is Deputy Director of the Office of Biostatistics and Epidemiology at CBER. She joined FDA to implement the use of Bayesian statistics for the regulation of medical devices and led the Decision Analysis initiative including Bayesian statistics, benefit-risk determinations, and... Read More →
avatar for Bray Patrick-Lake

Bray Patrick-Lake

Director of Stakeholder Engagement, Duke Clinical Research Institute
Bray Patrick-Lake supports efforts to actively engage patients, health advocacy organizations, and other stakeholders in Duke Clinical Research Institute’s local and national research programs. She has led extensive efforts through the CTTI to incorporate patient voice into clinical... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 10 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#161: Making Trials Work for Special Populations
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-558-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session discusses a variety of ways to address patient needs that help recruit and retain special populations in clinical trials.

Learning Objectives

Identify useful guidelines and regulations regarding special populations in various countries; Discuss useful models of patient engagement that focus on special populations; Discuss case studies that show how some clinical trials have attracted special groups of patients.

Chair

Deborah E. Collyar

Speaker

Therapeutic Needs of Older Patients in the Era of Mobile Health
Dinah Duarte, MSc

Implementation of a Peer Advocate Model to Optimize Trial Enrollment, Diversity, and Retention
Caroline Donovan, MPH

Site Awareness and Best Practices for Inclusion of Diverse Populations in Research
Diana Foster, BSN, PhD, MSN



Speakers
avatar for Deborah Collyar

Deborah Collyar

President, Patient Advocates In Research (PAIR)
Deborah Collyar has applied a wealth of leadership skills from her successful IT business career to help create patient-centered medical research and healthcare, after her first cancer diagnosis at the age of 32. She founded Patient Advocates In Research (PAIR) in 1996, “where research... Read More →
avatar for Caroline Donovan

Caroline Donovan

Manager of Patient Engagement, LuCIN, Lupus Therapeutics, LLC
Caroline Donovan, MPH is the Manager, Patient Engagement for Lupus Therapeutics, an affiliate of Lupus Research Alliance. She is responsible for the development and implementation of a peer education program in which prospective lupus clinical trial patients are matched with a trained... Read More →
avatar for Dinah Duarte

Dinah Duarte

Scientific Evaluation Unit, Directorate of Medicinal Products, INFARMED
Dr. Dinah Duarte is a senior assessor at the Scientific Evaluation Unit at the Directorate of Medicinal Products, in the Portuguese regulatory authority for medicines and health products (INFARMED). She is an expert member at the European Medicines Agency (EMA); the current Committee... Read More →
avatar for Diana Foster

Diana Foster

CEO, Society For Clinical Research Sites
Dr. Foster serves as a consultant to the Society for Clinical Research Sites as the Vice President of Strategy and Development. In her role she is responsible for building relationships with industry that help amplify the voice of the clinical research site. She has been integrally... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#170: Making Early Access for Patients Happen
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-19-565-L04-P; CME 1.00; IACET 1.00; RN 1.00

Research costs are increasing exponentially and patient populations are becoming more targeted. Healthcare systems throughout the world are struggling to cope with the costs of ever more specialized medicines. While these drug candidates hold the promise of slowing, or even curing disease progression, these innovations also often have limited evidence on long-term impacts. Regulators might wrestle with decisions on early access, but payers may also be reluctant to grant coverage for eligible patient populations. Early access may mean continued evidence generation once the drug is on the market, but traditional payment methods may not be well suited to address these issues, and their use may result in reduced coverage and patient access. This forum will identify challenges associated with early access, as well as coverage and reimbursement decisions associated with novel drug types, where there may be incomplete evidence or uncertainty of long-term value. Participants will focus on whether the current system is equipped to handle upcoming, novel therapies, and if not, what approaches would be best to pursue.

Learning Objectives

Recognize the difficulties in balancing patient access with incomplete understanding of a drug's benefits and long-term impacts; Analyze factors that contribute to coverage and reimbursement decisions; Discuss how to consider novel payment approaches to support appropriate patient access.

Chair

Monika Schneider, PhD

Speaker

Early Access: Life Saver for Patients or Unacceptable Weakening of Evidential Requirements?
Stella Blackburn, MD, MA, MSc, FFPM, FISPE, FRCP

Optimizing Coverage, Access, and Reimbursement for Drugs that Target Early-Stage Alzheimer’s Disease
Monika Schneider, PhD

Cell and Gene Therapy Development and the Impact on the Payer System
Dan Tierno, MA, MBA

Patient Perspective
François Houyez



Speakers
avatar for Stella Blackburn

Stella Blackburn

Vice President, Global Head of Early Access and Risk Management, IQVIA
Dr Stella Blackburn. MB BS, MA, MSc, FRCP(Ed), FISPE, FFPM, DLSHTP, Dip Pharm Med.VP, Global Head of Early Access & Risk Management, Real World Insights at IQVIA. Stella studied medicine at Cambridge and Guys and worked in hospital medicine before joining the pharmaceutical industry... Read More →
MS

Monika Schneider

Research Associate, Duke-Margolis Center For Health Policy
Monika Schneider is a Research Associate within the biomedical innovation team at the Duke-Margolis Center for Health Policy. Her work focuses on incentives for antimicrobial product development, value-based reimbursement policy, and payment reform for medical products. Prior to Duke-Margolis... Read More →
avatar for Dan Tierno

Dan Tierno

Associate Director, Global Clinical Data Sciences and Analytics, Bayer
Mr. Dan Tierno has over 20 years of experience in the pharmaceutical and biotech industry, specializing in drug development, data sciences and analytics for clinical trials, and commercialization of novel small molecule and cell based therapeutics. Mr. Tierno has spoken on training... Read More →
avatar for François Houyez

François Houyez

Treatment Information and Access Director, Health Policy Advisor, European Organisation for Rare Diseases (EURODIS)
François Houÿez is working at the European Organisation for Rare Diseases EURORDIS where he is Director of Treatment Information and Access, Policy Advisor. He has always been working as a patient advocate since the early 90s, first in the HIV/AIDS advocacy, and in rare diseases... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

5:15pm

Community Meet Up: Patient Engagement
Component Type: Forum



Chair

Mary Stober Murray, MBA


Speakers
avatar for Mary Murray

Mary Murray

Associate Director, Diversity and Patient Engagement, Bristol-Myers Squibb Company
As an Associate Director for Diversity and Patient Engagement at Bristol-Myers Squibb, Mary facilitates collaborations with disease-specific and community-based organizations to bring relevant clinical trial options to patients and representative populations. Mary has co-chaired the... Read More →


Monday June 24, 2019 5:15pm - 6:15pm
TBD San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Tuesday, June 25
 

8:00am

#205: Driving Enrollment with a Patient-Centric Focus to Artificial Intelligence
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-587-L04-P; CME 1.25; IACET 1.25; RN 1.25

Artificial intelligence, blockchain, natural language processing and real world evidence have been all the rage lately in industry news. Hear perspectives on these new technologies from a panel of experts from sponsor, technology vendor, patient advocacy group, and patients on what's working and what's not.

Learning Objectives

Describe how to drive patient enrollment in clinical studies with a patient-centric approach, allowing patients to pre-screen themselves and retargeting them if excluded; Identify how to address patient recruitment issues associated with pre-screening, screening and dropout to reduce the current average conversion ratio from 10:1; Discuss how to reduce pre-screening burden via mining of EMR records.

Chair

Rob Wynden, PhD

Speaker

Introduction to Patient-Facing Trial Technology
Rob Wynden, PhD

A Bulls-Eye Clinical Trial via Patient's Point- of-View
Abhit Singh, MD, MHA

User-Driven Markets Data that Help Clinicians Assess Treatments and Cure Diseases
Collin Powell, MBA

Turning Clinical Trial Recruitment on its Head Using EHR-Connected Applicants
Scott Cressman

Pragmatism and AI in Research
Mike Nolte, MBA



Speakers
avatar for Scott Cressman

Scott Cressman

Business Development and Strategic Partnerships, Human API
Scott Cressman leads business development & strategic partnerships in clinical research and digital health for Human API. He spends much of his time working at the intersection of life sciences + digital technologies with startups and Fortune 500s alike. His focus is on improving... Read More →
avatar for Mike Nolte

Mike Nolte

Chief Executive Officer, Signant Health
Mike Nolte is the CEO of CRF Bracket. Mike is a seasoned operational leader in healthcare and technology who is passionate about the patient experience. Before CRF Bracket, Mike was CEO at Influence Health, President and COO at MedAssets, and he held several leadership roles in healthcare... Read More →
CP

Collin Powell

Founder, Chief Executive Officer, The Health Exchange Market (THEM)
The Health Exchange Market (T.H.E.M.) is an online marketplace for de-identified healthcare data that allows organizations to realize new revenue streams through data commercialization, while also being able to obtain access to prospective and retrospective data from a variety of... Read More →
avatar for Abhit Singh

Abhit Singh

Chief Medical Advisor, Medical Advisory Board, WeHeal Foundation
Dynamic (Global) Physician Leader, Speaker and Visionary- specializing in the Medical Advisory, Transactional and Transformational management of Healthcare Advisory, Biotech & Biopharma organizations. Among many, a Medical Advisor for WeHeal Inc, a 'CrowdHealing' Therapeutic Options... Read More →
avatar for Rob Wynden

Rob Wynden

Chief Executive Officer, Aloha Health Network
Rob has been a computer scientist and programmer for over 30 years in the scientific programming, e-commerce, biotech, and health care industries. He implemented study startup and CTMS functionality at 9 bay area biotech companies, and invented a method that allows medical and biotech... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 10 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#208: Patient Focus as Part of the Regulatory Affairs DNA: Opportunities and Challenges
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-576-L04-P; CME 1.25; IACET 1.25; RN 1.25

Patients are at the heart of everything we do. Integrating patient focus into the regulatory mindset can be a challenge. Panelists will discuss their experiences, share best practices, and describe challenges in achieving this goal.

Learning Objectives

Identify best practices for integrating a patient focus mindset into the regulatory affairs organization; Recognize challenges faced by leaders in achieving this goal; Describe experiences with implementing patient focused practices as part of regulatory strategies.

Chair

Max Wegner, PharmD, RPh

Speaker

FDA Perspective
Theresa Mullin, PhD

Patient Representative
Marc M. Boutin, JD

Industry Perspective
Miu Chau, PhD

Patient Representative
Jill Bonjean, MSc



Speakers
avatar for Jill Bonjean

Jill Bonjean

Corporate Relations Advisor, Rare Diseases International
Jill Bonjean is Corporate Relations Advisor for Rare Diseases International - the global alliance of rare disease patient organizations. Jill works with RDI and other patient organizations to build effective relationships with companies and other partners. Previously, she's managed... Read More →
avatar for Marc Boutin

Marc Boutin

Chief Executive Officer, National Health Council (NHC)
Marc M. Boutin, JD, is the Chief Executive Officer of the National Health Council. He has been a leading voice for greater patient involvement at every stage of the health care continuum. Boutin has been actively involved in patient advocacy organization management, health advocacy... Read More →
MC

Miu Chau

Senior Regulatory Program Director, Genentech, A Member of the Roche Group
Miu joined Genentech in 2008 and held different roles. Currently she is a Senior Regulatory Program Director responsible for the global regulatory strategy and execution of projects and leading a matrix team of regulatory professionals in oncology. Miu had been the Global Head of... Read More →
avatar for Theresa Mullin

Theresa Mullin

Associate Director for Strategic Initiatives, OCD, CDER, FDA
Principal advisor on strategy including CDER international cooperation and harmonization, application of decision science, and other initiatives. Leads FDA Patient Focused Drug Development, and heads the FDA delegation to ICH. Previous work includes leading FDA negotiations for 2002... Read More →
avatar for Max Wegner

Max Wegner

Head Regulatory Affairs, Bayer AG
After studying pharmacy at the University of Bonn, Max Wegner commenced his PhD in pharmacology at Bayer and started in Regulatory Affairs as a Dossier Manager in 1996 in Wuppertal. Since then, Max has been employed in various functions within Product Development at Bayer and also... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

9:30am

#224 RT: Round Table Discussion: Enhancing Patient-Focused Outcome Assessment in Medical Product Development
Component Type: Session
Level: Basic

Round table discussion tied to session #158: Enhancing Patient-Focused Outcome Assessment in Medical Product Development (Monday, June 24, 3:30-4:30PM). Seating is limited. To include special guests from the session: Elektra Papadopoulos, Telba Irony, Michelle Tarver.

Chair

Emuella Flood


Tuesday June 25, 2019 9:30am - 10:30am
Community Zone Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#236: Our Doors are Open! Pathways and Programs for Patient Stakeholders to Engage with FDA
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-608-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will explore and discuss agency-wide patient engagement programs and initiatives that provide patient stakeholders (patients, caregivers and advocates) opportunities to engage with FDA and participate in regulatory discussions that inform medical product development and review.

Learning Objectives

Describe how FDA has established organizational components to ensure patients are a priority; Describe avenues for patient stakeholders to engage with the agency; Explain key initiatives where patient stakeholders can share their experiences to help inform medical product development; Discuss the value of patient stakeholder participation.

Chair

Samir Shaikh, MBA

Speaker

FDA Perspective
Patroula Smpokou, MD

Patient Perspective
Debbie Drell

Patient Perspective
Sabina A. Kineen



Speakers
avatar for Debbie Drell

Debbie Drell

Director of Membership, National Organization for Rare Disorders (NORD)
Debbie joined the National Organization for Rare Disorders (NORD) in 2017 with more than 18 years of nonprofit experience in public health education, awareness and advocacy. Most recently, she worked for the Pulmonary Hypertension Association, a NORD member organization. Debbie says... Read More →
avatar for Sabina Kineen

Sabina Kineen

Fabry Disease, Patient Advocate
Diagnosed with Fabry Disease in her early teens, Sabina provides a unique perspective as both a patient and caregiver. Over the years, she has shared her experiences living with a rare genetic condition, as well as helping manage the complicated and multi-faceted healthcare needs... Read More →
PS

Patroula Smpokou

Clinical Team Leader, Office of New Drugs, CDER, FDA
Patroula Smpokou, MD, FACMG is a clinical team leader (lead physician) in the Division of Gastroenterology and Inborn Errors Products (DGIEP) at FDA’s Center for Drug Evaluation and Research where she is involved in the regulation of products intended for the treatment of rare genetic... Read More →
avatar for Samir Shaikh

Samir Shaikh

Deputy Director, Patient Affairs Staff, Office of the Commissioner, FDA
Samir Shaikh works at the U.S. Food and Drug Administration (FDA) and serves as the Deputy Director for the Patient Affairs Staff (PAS) in the Office of the Commissioner. PAS leads and enhances patient engagement activities across the medical product Centers—facilitating dialogue... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Beginner
  • Featured Topics Patient Focused,Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

10:30am

#235: Walking the Talk: Using Home Nursing as a Patient-Centric Service in Clinical Trials - From Multiple Perspectives
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-595-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session examines the use of home trial support services from the perspectives of the study sponsor, CRO partner and the potential patient participant. During this session we will discuss the logistics of home trial support, its benefits and pitfalls, when it is most appropriate to use and the patient perception of the service and how it may affect their willingness to participate.

Learning Objectives

Describe the benefits of use of home nursing (specifically, home trial support) in clinical trials from the sponsor, CRO and patient perspectives; Identify barriers to implementation of home trial support; Discuss scenarios where the greatest return on investment for home trial support is realized.

Chair

Ellyn Getz

Speaker

Academic Perspective
Ellyn Getz

Industry Perspective
Todd McGrath

Study Sponsor Perspective
Nour Ziyadeh, MS

Patient Perspective
Mary Koestler

CRO Perspective
Kelly Franchetti, RN



Speakers
MK

Mary Koestler

Assistant Professor, Clinical Trials Adminstrator, Memory and Aging Center, University of California San Francisco
avatar for Nour Ziyadeh

Nour Ziyadeh

Clinical Trial Oversight Lead, Alnylam Pharmaceuticals
Nour Ziyadeh is a Clinical Trial Oversight Lead ensuring compliance for Alnylam’s outsourced clinical trials. She has 15 + years of industry experience including third party auditing for the FDA, CRO , SMO and Pharma. She has held roles including Clinical Research Coordinator, Clinical... Read More →
avatar for Kelly Franchetti

Kelly Franchetti

RN, CCRN, CEN Vice President, Global Head Patient Insights & Engagement, ICON plc.
Kelly has a diverse background comprised of nursing, advocacy & clinical research. She is also the mother of a child with a rare disease. This gives her a unique 360 degree view of patients’ & caregiver’s perspectives in the realm of disease management, treatment and burden. As... Read More →
avatar for Ellyn Getz

Ellyn Getz

Associate Director, Development and Community Engagement, CISCRP
Ellyn Getz is the Associate Director of Development and Community Engagement at CISCRP. She is responsible for directing CISCRP’s educational and outreach initiatives, including AWARE for All: Clinical Research Education Days, MT Pharmacy and Journey to Better Health mobile experiential... Read More →
TM

Todd McGrath

Vice President Operations A/O, Medical Research Network
Todd has over 15 years' experience in the clinical trial and life science industry; beginning his career in pharmaceutical and biotech consulting and then holding operational positions at a clinical research organization. As Vice President Operations Americas Oceania at MRN, Todd... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 11B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#246: How Employers are Reinventing Healthcare and What it Means for Research Participation and Evidence
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-603-L04-P; CME 1.00; IACET 1.00; RN 1.00

Employers are contracting directly with vendors to provide innovative healthcare services for their employees. Featuring patients, advocates and vendors, this panel shows how to engage employers and measure their impact in drug development.

Learning Objectives

Explain the experience of patient/employee balancing career, health care and benefits; Describe how employer benefits are changing; Identify collaborative opportunities between payers, providers, employers and advocates to facilitate employee/patient access to quality, affordable care; Illustrate how stakeholders can align on data requirements to capture and report key metrics to support care quality and cost management in hemophilia.

Chair

Mary Stober Murray, MBA

Speaker

Improving Collaboration and Data Reporting for Integrated Hemophilia Care: Metrics for Quality Improvement/Cost Management
Kollet Koulianos, MBA

Personalized Care Management
Marc M. Boutin, JD

Patient Perspective
Jacqueline Smith



Speakers
KK

Kollet Koulianos

Senior Director Payer Relations, National Hemophilia Foundation
avatar for Mary Murray

Mary Murray

Associate Director, Diversity and Patient Engagement, Bristol-Myers Squibb Company
As an Associate Director for Diversity and Patient Engagement at Bristol-Myers Squibb, Mary facilitates collaborations with disease-specific and community-based organizations to bring relevant clinical trial options to patients and representative populations. Mary has co-chaired the... Read More →
JS

Jacqueline Smith

Associate Director, Oncology Advocacy and Policy, Bristol-Myers Squibb
avatar for Marc Boutin

Marc Boutin

Chief Executive Officer, National Health Council (NHC)
Marc M. Boutin, JD, is the Chief Executive Officer of the National Health Council. He has been a leading voice for greater patient involvement at every stage of the health care continuum. Boutin has been actively involved in patient advocacy organization management, health advocacy... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#262: Measuring the Impact of Patient Engagement Activities in Medicines R&D: A Way to Sustain the Cultural Change?
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-731-L04-P; CME 1.25; IACET 1.25; RN 1.25

Measuring the impact of patient engagement in medicines R&D provides a means of demonstrating effectiveness of the engagement and thus support for sustaining it. In this session, early results of IMI’s PARADIGM, other recent work on assessing value and measuring impact, and their role in supporting sustainability are discussed.

Learning Objectives

Describe the IMI PARADIGM Project’s overall goal and objectives for patient engagement (PE), high level project structure and methods, and early results related to measuring PE impact; Identify the benefits of assessing value and measuring impact of PE; Discuss the importance of PE sustainability and the role of metrics in supporting it.

Chair

Mathieu Boudes, PhD

Speaker

Panelist
Nicholas Brooke, MBA

Panelist
Debra Michaels, MS

Panelist
Lidewij Eva Vat, MS



Speakers
avatar for Mathieu Boudes

Mathieu Boudes

PARADIGM Coordinator, European Patients' Forum
Mathieu Boudes, PhD, joined the European Patients’ Forum (EPF) in 2018 as coordinator of the PARADIGM project, a public-private partnership financed by the Innovative Medicines Initiative and co-led by EPF and EFPIA (European Federation of Pharmaceutical Industries and Associations... Read More →
avatar for Nicholas Brooke

Nicholas Brooke

Executive Director, Patient Focused Medicines Development (PFMD), The Synergist.org
Nicholas Brooke is Founder of The Synergist, a collaboration platform incubator that brings key players together with the express aim of solving significant societal problems through collective action. Under Nicholas’ leadership, The Synergist acts as a backbone, providing vision... Read More →
avatar for Debra Michaels

Debra Michaels

Associate Director, Scientific Programs & Patient Engagement Lead, DIA
In her content-focused role at DIA, Debra Michaels provides guidance on educational content strategy and development across the DIA Americas portfolio of educational programs as well as subject matter expertise in selected areas. She is the lead for DIA’s Patient Engagement thought... Read More →
avatar for Jaye Bea Smalley

Jaye Bea Smalley

Director, Global I and I Patient Advocacy and Life Cycle Management, Celgene Corporation
Jaye Bea joined Celgene as Global Director, Patient Advocacy and Life Cycle Management, Inflammation & Immunology in March 2018. Her twenty-five-year career in health care includes extensive experience in the public and private sector focused on advancing the needs and interests of... Read More →
avatar for Lidewij Vat

Lidewij Vat

Researcher and Lecturer Meaningful and Sustainable Patient Engagement, Vrije Universiteit (VU)
Lidewij (Eva) Vat is a researcher and trainer in patient and public engagement. Her work focuses on creating partnerships between patients, professionals and researchers to drive meaningful innovation. Her research aims to better understand the key conditions needed for patient engagement... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

2:00pm

#261: Patient Experience Data: How Could this Data Enhance Decision-Making at Different Stages of Medical Product Development?
Component Type: Forum
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-613-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will provide an opportunity for key stakeholders to discuss examples of various types of patient experience data, and how this data could potentially inform decision-making at different stages of medical product development.

Learning Objectives

Define patient experience data; Describe types of patient experience data that could inform medical product development; Discuss how patient experience data can inform decision making at different stages of medical product development.

Chair

Meghana Chalasani, MHA

Speaker

FDA Perspective
Michelle Campbell, PhD

Panelist
Elizabeth Hart, MD

Panelist
Isabelle Lousada, MA

Panelist
Kristina Bowyer, LPN



Speakers
avatar for Kristina Bowyer

Kristina Bowyer

Executive Director of Patient Advocacy, Ionis Pharmaceuticals
Kristina Bowyer is the Executive Director of Patient Advocacy & Engagement at Ionis Pharmaceuticals. Kristina has worked at Ionis for over 26 years and has spent the last seven years successfully building Ionis’ internal patient engagement program to ensure that the patient perspective... Read More →
MC

Michelle Campbell

Division of Neurology Products, Office of New Drugs, CDER, FDA
Michelle Campbell is the Sr. Clinical Analyst for Stakeholder Engagement and Clinical Outcomes for the Division of Neurology Products, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Previously, Dr. Campbell was a... Read More →
avatar for Meghana Chalasani

Meghana Chalasani

Senior Research Analyst, Office of the Center Director, CDER, FDA
Meghana Chalasani currently serves as a senior research analyst for the Patient-Focused Drug Development (PFDD) Program in FDA’s Center for Drug Evaluation and Research (CDER). She works closely on CDER’s various PFDD initiatives and provides strategic, regulatory, program, and... Read More →
avatar for Isabelle Lousada

Isabelle Lousada

Chief Executive Officer and President, Amyloidosis Research Consortium
As a former patient, Lousada has spent 20 years dedicated to patient advocacy, accelerating drug development, and promoting awareness of rare diseases. Lousada founded the Amyloidosis Research Consortium (ARC) in 2015 to address the critical needs in clinical trials and related research... Read More →
EH

Elizabeth Hart

Physician, Office of Tissues and Advanced Therapies, CBER, FDA


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#272: Aligning Facilitated Regulatory and Access Pathways: Observations from the North American Experience
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-622-L04-P; CME 1.25; IACET 1.25; RN 1.25

The purpose of this session is for agencies, industry, and other organizations to present their observations around the types of facilitated pathways that are available in North America to speed access to medicines that address an unmet medical need.

Learning Objectives

Describe initiatives that the US and Canada has implemented to streamline the process for approval of and access to therapies that address unmet medical need (UMN); Illustrate opportunities and challenges with aligning regulatory and access pathways; Discuss if these pathways may have a broader remit than just for therapies for UMN.

Chair

Lawrence Eugene Liberti, PhD, RPh, RAC

Speaker

Market Access on the Two Sides of the Atlantic: Barriers and Opportunities
Maria Isabel Manley, LLM

Aligning Approval and Access: The US Experience
Sean Tunis, MD, MSc

Patient Perspective
Julie Eller



Speakers
avatar for Lawrence Liberti

Lawrence Liberti

Executive Director, Centre for Innovation in Regulatory Science (CIRS)
Dr Liberti has worked in pharmaceutical regulatory affairs, communications and clinical R&D for the past four decades. Since 2009 he has served as the Executive Director of CIRS (the Centre for Innovation in Regulatory Science, Ltd, forming part of Clarivate Analytics). He has been... Read More →
avatar for Maria Manley

Maria Manley

Partner, Head of the UK Life Sciences, Sidley Austin LLP
Marie advises on a broad spectrum of matters, both contentious and non-contentious, that arise in the conduct of a bio pharmaceutical business. Marie represents pharma companies before UK and EU institutions (EMA, MHRA, NICE, PMCPA DOH and the European Commission). Marie is Chairperson... Read More →
avatar for Julie Eller

Julie Eller

Manager, Grassroots Advocacy, Arthritis Foundation
Julie leads a patient network of more than 100,000 patient Advocates as they share their story to effect change. Julie has had arthritis since she was seven years old and the Arthritis Foundation has helped her foster a passion for translating the patient experience into advocacy... Read More →
ST

Sean Tunis

Senior Strategic Advisor, Center For Medical Technology Policy (CMPT)


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#289: Impact of Patient Engagement on the Biopharmaceutical Industry's Business and Organization
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-631-L04-P; CME 1.25; IACET 1.25; RN 1.25

Many life science companies are creating new functions or are re-organizing existing patient advocacy roles/departments to support expanding expectations for patient involvement in the research, development, launch, and delivery of new medical products. These newly defined roles require an understanding of and active communication with internal and external stakeholders. They are often on the leading edge of cultural change within their organizations, to help spread both the concept of patient engagement and practice of patient-centricity beyond early adopters. In this session, individuals serving in these positions will share lessons learned, thoughts on factors that contribute to success, describe hard and soft skills needed to be effective, and discuss how they're tracking progress within their companies. We'll compare reporting structures, pros/cons of centralized versus diffused models, and forecast what's ahead from a human-resource planning standpoint as patient-focused drug development matures.

Learning Objectives

Discuss the new patient engagement function within life science companies; Examine differences in where the function is housed within the organization and requirements of the position as well as helpful experience/training for the role; Identify experiences that contribute to success in balancing needs and expectations of internal and external stakeholders alike.

Chair

K. Kimberly McCleary

Speaker

Panelist
Jan Nissen, BSN, MBA, MS

Panelist
Jessica Riviere, MHS

Panelist
Angie Wilson

Panelist
Nikki Levy

Panelist
Joel Beetsch, PhD



Speakers
avatar for Joel Beetsch

Joel Beetsch

Vice President, Global Patient Advocacy, Celgene Corporation
Dr. Beetsch, VP of Patient Advocacy in Celgene’s Corporate Affairs Dept., leads the global development & execution of a coordinated patient-focused Advocacy strategy working w/multiple patient, provider, payer, & policy organizations around the world to foster safe & effective solutions... Read More →
avatar for Nikki Levy

Nikki Levy

Vice President, Patient Engagement, Alkermes
Since April 2015, Nikki has been the VP of Patient Engagement at Alkermes plc, managing a variety of partnerships to incorporate the patient perspective into the company's programs in an effort to improve the health and lives of people affected by serious central nervous system (CNS... Read More →
avatar for K. Kimberly McCleary

K. Kimberly McCleary

Founder and Chief Executive Officer, The Kith Collective, LLC
Kim McCleary has been at the forefront of patient engagement for nearly 30 years. She is a nationally regarded subject matter expert on patient-focused medical product development, patient-centered benefit-risk assessment, and organizational change. McCleary serves on numerous advisory... Read More →
avatar for Jan Nissen

Jan Nissen

Vice President, Patient Innovation & Engagement, Merck & Co., Inc.
Jan leads Merck’s Patient Innovation & Engagement organization comprised of Patient Insights, Patient & Consumer Experience, and Patient and Caregiver Alliances & Advocacy globally. She has been in the pharmaceutical industry over 30 years, serving in sales, training, marketing... Read More →
avatar for Jessica Riviere

Jessica Riviere

Senior Director of Global Patient Advocacy, Biogen
Jessica Riviere leads the Global Patient Advocacy for Biogen overseeing a broad neuroscience portfolio. Jessica’s advocacy experience spans diabetes, HIV/AIDS, immunology and oncology with several companies including AbbVie, Bristol Myers Squibb and Novo Nordisk. Early in her career... Read More →
AW

Angie Wilson

Director, Alliance and Advocacy Relations, BioOncology, Genentech, A Member of the Roche Group
Angie leads patient advocacy at Genentech for Immunology and Ophthalmology. Her role at Genentech since 2011 has focused on building substantive, ongoing partnerships with non-profits built on transparency and respect.


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Wednesday, June 26
 

8:00am

#308: Identifying High-Value Patient Engagement Opportunities: A Collaborative Three-Step Process for Sponsors and Patient Groups
Component Type: Workshop
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-649-L04-P; CME 1.25; IACET 1.25; RN 1.25

This workshop brings together sponsors and patient groups for facilitated discussion of high-value engagement opportunities. Using evidence-based resources, attendees apply a simple prioritization process to identify new activities and evaluate ROI.

Learning Objectives

Discuss how to apply a straightforward process for identifying engagement opportunities with high value for both sponsors and patient groups; Describe the diverse factors that sponsors and patient groups consider in evaluating the benefits and investments associated with engagement opportunities; Identify new patient engagement and relationship opportunities & new ways to partner across the R&D continuum.

Chair

Jaye Bea Smalley, MPA

Speaker

Facilitator
Linda Brennan, MPH



Speakers
avatar for Linda Brennan

Linda Brennan

Director, Community Partnerships, Cystic Fibrosis Foundation
Linda Brennan is Senior Director, Community Partnerships at the Cystic Fibrosis Foundation (CFF), which brings the perspectives of people affected by cystic fibrosis (CF) into all the Foundation’s activities, including research and clinical development, and provides opportunities... Read More →
avatar for Jaye Bea Smalley

Jaye Bea Smalley

Director, Global I and I Patient Advocacy and Life Cycle Management, Celgene Corporation
Jaye Bea joined Celgene as Global Director, Patient Advocacy and Life Cycle Management, Inflammation & Immunology in March 2018. Her twenty-five-year career in health care includes extensive experience in the public and private sector focused on advancing the needs and interests of... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#319: How to Solve the Problem of Access for Rare Diseases
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-663-L04-P; CME 1.25; IACET 1.25; RN 1.25

This panel will share learnings and build on outcomes from the recent Rare Access to Critical Therapies (ACT) Stakeholder Summit convened by Global Genes and the Child Neurology Foundation, two patient advocacy organizations which brought together more than 200 individuals representing key stakeholders in the rare disease drug development space to build awareness and understanding about access to and pricing of rare disease therapies.

Learning Objectives

Discuss critical access issues related to rare disease drug development; Describe lessons learned from recent experiences to improve communication and collaboration among stakeholders and ensure the patient and caregiver needs are truly understood, considered and incorporated into access and pricing decision-making.

Chair

Sissi Pham, PharmD

Speaker

Panelist
Kari Luther Rosbeck

Panelist
Nicole Boice

Panelist
Scott Demarest, MD

Panelist
Sarah Pitluck, MSc

Panelist
Sean Tunis, MD, MSc



Speakers
avatar for Sissi Pham

Sissi Pham

Chief Executive Officer, AESARA
Sissi Pham is a founder and CEO of AESARA, Inc. and AESARA Foundation, both established in 2016. AESARA, Inc. was established with the commitment to absorb the administrative cost of the AESARA Foundation to enable the charity to give 100% of the donations to directly to humanitarian... Read More →
avatar for Kari Rosbeck

Kari Rosbeck

President and Chief Executive Officer, Tuberous Sclerosis Alliance
Kari joined the TS Alliance in June 2001 and became President and CEO in November 2007. Kari has been involved in nonprofit fundraising and volunteer management for nearly 30 years. During Kari’s tenure as President and CEO, the TS Alliance established a comprehensive research program... Read More →
avatar for Sarah Pitluck

Sarah Pitluck

Head, Global Pricing and Reimbursement, Spark Therapeutics
Ms. Pitluck is the Head of Global Pricing & Reimbursement (P&R) at Spark Therapeutics. In her current role, Ms. Pitluck priced the first FDA-approved gene therapy for a genetic disease, LUXTURNA®. She is now working to ensure market access for all US patients in need of LUXTURNA... Read More →
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Nicole Boice

Founder, Global Genes Project
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Scott Demarest

Assistant Professor, Departments of Pediatrics and Neurology, University of Colorado School of Medicine; Children's Hospital Colorado
Scott Demarest received a Bachelor of Science in biology from the University of Texas at Austin before going on to medical school at the University of Texas Health Science Center in San Antonio. He completed his residency in Pediatrics and child neurology at Children's National Health... Read More →
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Sean Tunis

Senior Strategic Advisor, Center For Medical Technology Policy (CMPT)


Wednesday June 26, 2019 8:00am - 9:15am
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Beginner
  • Featured Topics Patient Focused,Rare Disease
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

8:00am

#318: Opportunities and Challenges of Collecting Data in a Pre-Approval Access Setting: A Multi-Stakeholder Perspective
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-658-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session reviews the benefits and risks of collecting real world data through single-patient requests (SPRs)/expanded access programs (EAPs) in a pre-approval access (PAA) setting and discusses what may confer the highest value to an organization from a multi-stakeholder perspective.

Learning Objectives

Recognize relevance and value for an organization of collecting real world data: Discuss the value of real world data from a pre-approval access (PAA) setting as it relates to regulatory and access strategies; Describe benefits and risks of collecting real world data in an single-patient requests (SPR)/named-patient program (NPP) and its potential implications on regulatory and access strategies.

Chair

Beverly L Harrison

Speaker

Pre-Approval Access and Real World Evidence Landscape Set-up
Sasha Richardson, MBA, MSc

Panelist
Arnaud Foucher, MBA, MS

Panelist
Alison Bateman-House, PhD, MA, MPH

Panelist
Michael Fine, MD



Speakers
avatar for Alison Bateman-House

Alison Bateman-House

Assistant Professor, Division of Medical Ethics, NYU Langone Health
Alison Bateman-House, PhD, MPH, MA, is an assistant professor in the Division of Medical Ethics at NYU Langone Health's School of Medicine. She is co-chair of the Working Group on Compassionate Use and Preapproval Access (CUPA), an academic group that studies ethical issues concerning... Read More →
avatar for Michael Fine

Michael Fine

Medical Director, Health Net
Dr. Michael Fine is a physician executive with over forty years of experience in all aspects of health care delivery. He is board certified in Internal Medicine and was among the first group of physicians who received additional certification in Geriatric Medicine. Doctor Fine began... Read More →
avatar for Arnaud Foucher

Arnaud Foucher

EMEA Medical Program Director, Medical Affairs, Europe, Middle-East and Africa, Janssen
Arnaud Foucher is EMEA Medical Program Director at Janssen, where he is responsible for the Company Regional Evidence Generation & Pre-Approval Access Programs, leading the EMEA Medical Program Management team. With a 20 years career in the pharmaceutical industry, Arnaud is focused... Read More →
avatar for Beverly Harrison

Beverly Harrison

Head, Patient Support, Janssen Pharmaceutical Companies of Johnson & Johnson
Leading the Patient Support Group @ Janssen R&D (a J&J Co.), reporting directly to the Chief Medical Officer, Beverly focuses on developing strategies for patients’ needs by working with internal/external stakeholders & engaging patient advocacy organizations. She is a R&D & nonprofit... Read More →
avatar for Sasha Richardson

Sasha Richardson

Managing Director, EY
Sasha is an Executive Director in EY's Commercial Life Science Advisory practice and brings over 20 years of global experience in the healthcare / life science sector. Prior to EY, she headed up the US (SF/NY based) and EU (London based) Commercial & Market Access Consulting Practice... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#334: Understanding and Exploring Elements of a Patient-Focused Product Launch
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-667-L04-P; CME 1.00; IACET 1.00; RN 1.00

A true patient-focused drug development (PFDD) approach requires implementation throughout the entirety of a product’s lifecycle, including pre-clinical, development, product launch and post-market approval. While the role of patients in medicine development has been expanding and evolving within the last decade, most notably since 2012 when the U.S. FDA Safety and Innovation Act was signed into law, much of this work has been focused on incorporating the patient’s voice into research, development and clinical trial operations. With progress to date, the present day provides an opportunity to understand how sponsors develop PFDD when preparing for a launch and incorporating patient engagement principles into their commercialization plans.

Learning Objectives

Discuss how and when Patient Advocacy and Patient Engagement functions within a sponsor company partner with the Commercial and Market Access functions; Discuss the role of patient input into product-specific marketing and disease education materials; Identify communications strategies that include messaging and content for patients and caregivers; Describe regulatory and compliance considerations for regional launches; Identify opportunities for improved collaboration moving forward.

Chair

Jennifer Helfer, PhD

Speaker

Panelist
Michele Rhee, MBA, MPH

Panelist
Nicole Boice

Panelist
Kari Luther Rosbeck



Speakers
avatar for Jennifer Helfer

Jennifer Helfer

Patient Advocacy, bluebird bio, Inc.
Jennifer Helfer joined bluebird bio in December of 2015. As part of the Patient Advocacy team, Jen serves as a conduit to patient organizations and advocates. Patient Advocacy is responsible for building and sustaining trusted collaborations with stakeholders to address patient and... Read More →
avatar for Michele Rhee

Michele Rhee

Head of Patient Affairs, Enzyvant
As a member of the rare disease patient community for over a decade, Michele has dedicated her career to getting treatments to the patients who need them. Michele is a founding board member of Costs of Care and volunteers as a SAMbassador for SAMFund. She received her MBA from the... Read More →
avatar for Kari Rosbeck

Kari Rosbeck

President and Chief Executive Officer, Tuberous Sclerosis Alliance
Kari joined the TS Alliance in June 2001 and became President and CEO in November 2007. Kari has been involved in nonprofit fundraising and volunteer management for nearly 30 years. During Kari’s tenure as President and CEO, the TS Alliance established a comprehensive research program... Read More →
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Nicole Boice

Founder, Global Genes Project


Wednesday June 26, 2019 10:30am - 11:30am
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#335: Highlights of the Patient Engagement Preparedness, Capabilities, Experience, and Impact (PEPCEI) Study
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-736-L04-P; CME 1.00; IACET 1.00; RN 1.00

Pharmaceutical and biotechnology companies are piloting and adopting a variety of patient centric initiatives across their R&D functions – including development planning, protocol design, study conduct, benefit/risk assessment, regulatory submissions, medical writing and disclosure & transparency. Overall approach and level of adoption in functional areas varies widely within and between companies. DIA, Tufts CSDD, and 20 participating biopharmaceutical companies collaborated to develop a comprehensive process for assessing the adoption and integration of patient centric initiatives within participating company operating areas. Following development and validation of an assessment tool and process, an independent team led by DIA and the Tufts CSDD conducted assessments within participating companies. Data from recently completed assessments will be used to identify leadership areas, to recommend areas where patient centric practices can be strengthened; and to discuss areas where patient-focus in drug development can be expected to drive change in the future. Aggregate measures will be derived from the data and published as benchmarking data for the broader biopharmaceutical community. In this session, experiences and lessons learned with the collaborative development and use of the assessment tool will be discussed by members of the study team and collaborating companies.

Learning Objectives

Describe the role and possible contributions of multiple functions in supporting patient focus in biopharma R&D; Discuss the benefits of cross-functional collaboration in advancing patient involvement in &D; Explain the importance of understanding the internal landscape of patient-centric efforts within one’s company.

Chair

Kenneth A. Getz, MBA

Speaker

Panelist
Jane E. Myles, MS

Panelist
Debra Michaels, MS

Panelist
Patrick Cauntay



Speakers
avatar for Kenneth Getz

Kenneth Getz

Deputy Director, Research Professor (PHCM); Founder and Chairmen, TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT; CISCRP
Kenneth A. Getz is the Director of Sponsored Research and an associate professor at the Tufts Center for the Study of Drug Development, Tufts University School of Medicine, where he directs research programs on drug development management strategies and tactics, outsourcing, and global... Read More →
avatar for Debra Michaels

Debra Michaels

Associate Director, Scientific Programs & Patient Engagement Lead, DIA
In her content-focused role at DIA, Debra Michaels provides guidance on educational content strategy and development across the DIA Americas portfolio of educational programs as well as subject matter expertise in selected areas. She is the lead for DIA’s Patient Engagement thought... Read More →
PC

Patrick Cauntay

Assistant Director, Patient Advocacy and Engagement, Ionis Pharmaceuticals Inc
Patrick Cauntay is Assistant Director at Ionis Pharmaceuticals. He is responsible for researching and developing local and global advocacy relationships and innovative partnerships that provide a deeper understanding of various diseases from the patient perspective. Bridging antisense... Read More →
avatar for Jane Myles

Jane Myles

Head, Operational Intelligence and Innovation, Genentech, A Member of the Roche Group
Jane is the Head, Operational Intelligence and Innovation for Roche, working at Genentech. Her passion is driving innovation into the way we design and execute trials to get medicines to patients faster. She's worked at Genentech for 17 years in many roles, including line manager... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

10:30am

#344: Meaningful Patient-Focused Drug Development for Rare Disease and Personalized Medicine
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-672-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will provide a platform to discuss measurement challenges and practical considerations for selection of fit-for-purpose clinical outcome assessments (COAs) to support endpoints in rare disease drug development programs.

Learning Objectives

Discuss practical considerations for utilizing clinical outcome assessments (COAs) to support clinical trial endpoint(s) in drug development; Identify the measurement challenges and recommendations for the selection of COAs for rare disease drug development.

Chair

Michelle Campbell, PhD

Speaker

Generalized Pairwise Comparisons for Benefit/Risk Assessment in Personalized Medicine
Marc E. Buyse, DrSc

Endpoint Selection and Use of Clinical Outcome Assessments (COAs) in Rare Disease Drug Development: A Regulatory Perspective
Lili Garrard, PhD

Selection of Fit-for-Purpose COAs to Support Endpoints in Rare Disease
Jean Paty, PhD



Speakers
avatar for Marc Buyse

Marc Buyse

Chief Scientific Officer, International Drug Development Institute (IDDI)
Marc Buyse holds master’s degrees from Brussels University (Belgium), the Cranfield School of Management (UK) and a doctorate degree from the Harvard School of Public Health (USA). He had worked for 12 years at the EORTC in Brussels and for 2 years at the Dana Farber Cancer Institute... Read More →
MC

Michelle Campbell

Division of Neurology Products, Office of New Drugs, CDER, FDA
Michelle Campbell is the Sr. Clinical Analyst for Stakeholder Engagement and Clinical Outcomes for the Division of Neurology Products, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Previously, Dr. Campbell was a... Read More →
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Lili Garrard

Mathematical Statistician, Office of Biostatistics, OTS, CDER, FDA
Lili Garrard is a statistical reviewer on the Clinical Outcome Assessment (COA) Statistical Support Team at the Division of Biometrics III, Office of Biostatistics (OB), Office of Translational Sciences (OTS), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration... Read More →
avatar for Jean Paty

Jean Paty

Vice President, Patient-Centered Endpoints, IQVIA
Dr. Paty is an acknowledged leader in the effective strategies and practices of capturing patient perspective data for use in the clinical development of new medical products. He has published extensively in the areas of Patient Reported Outcomes (PRO) and electronic PRO (ePRO), as... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#374 EE: Enabling Patient-Centric Clinical Studies: TransCelerate’s Patient Engagement Tools
Component Type: Workshop
Level: Intermediate

TransCelerate BioPharma is a collaboration of 19 of the world’s leading pharmaceutical organizations that has undertaken a Patient Experience initiative to enable greater patient engagement and partnership between patient and sponsors.

Learning Objectives

Describe TransCelerate’s Patient Experience Initiative and the toolkits; Recognize the value of patient engagement in the development of clinical study protocols; Identify the clinical study participant experience as a valuable outcome measure; Appreciate the opportunity for embedding these practices into the development process.

Chair

Mary Elmer, BSN, MSN, RN

Speaker

Facilitator
Lani Hashimoto

Facilitator
Anne Marie L. Inglis, PhD

Facilitator
Christina Cantrell, MSc



Speakers
avatar for Mary Elmer

Mary Elmer

Executive Director, Patient, Caregiver and Consumer Experience, Patient Engagement Operations, Merck
Mary Elmer is the Executive Director, Patient, Caregiver, and Consumer Experience, Patient Engagement Operations at Merck. This group seeks to create an experience that engages consumers and patients in optimizing their health, wellbeing, and healthcare outcomes.She also leads a team... Read More →
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Christina Cantrell

Senior Patient Insights Leader, Genentech, A Member of the Roche Group
Christina has worked for over 15 years in clinical research, always with a focus on the patient’s experience and primarily in outcomes research and insights gathering. She has worked at both large and community hospitals, health technology vendors, and she currently works at Roche... Read More →
LH

Lani Hashimoto

Clinical Trial Intelligence Manager, Novartis Pharmaceuticals Corporation
LH

Lani Hashimoto

Clinical Program Benchmark Manager, Novartis Pharmaceuticals Corporation
avatar for Anne Marie Inglis

Anne Marie Inglis

Director, US Clinical Operations, Development Clinical Operations, Pharma R&D, GlaxoSmithKline
Dr. Inglis has over 20+ years of experience in clinical research, spanning all phases of research, specializing in clinical operations. Most recently, she was Head of US Pharma Clinical Operations for GSK, where she was responsible for operational delivery of US Clinical Trials. Currently... Read More →


Wednesday June 26, 2019 2:00pm - 3:00pm
E and E Area Exh Hall San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#358: Improving the Trial Experience for Rare Disease Patients: Identifying and Overcoming Obstacles
Component Type: Workshop
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-697-L04-P; CME 1.25; IACET 1.25; RN 1.25

Participants will engage in an “overcoming obstacles” activity designed to encourage collaboration among rare disease patients, trial sponsors, and advocates. You will identify obstacles to trial participation and work to develop creative solutions.

Learning Objectives

Describe valuable and detailed input from patients on clinical trial design; Design patient interactions that encourage collaboration among patients, sponsors, and advocacy group; Explain how to optimize clinical trials to ensure they meet the needs of rare disease patients.

Chair

Kelly Franchetti, RN

Speaker

Patient Perspective
Kim Stephens, PhD, MA

Industry Perspective
Kelly Franchetti, RN

Academic Perspective
Ellyn Getz



Speakers
avatar for Kelly Franchetti

Kelly Franchetti

RN, CCRN, CEN Vice President, Global Head Patient Insights & Engagement, ICON plc.
Kelly has a diverse background comprised of nursing, advocacy & clinical research. She is also the mother of a child with a rare disease. This gives her a unique 360 degree view of patients’ & caregiver’s perspectives in the realm of disease management, treatment and burden. As... Read More →
avatar for Ellyn Getz

Ellyn Getz

Associate Director, Development and Community Engagement, CISCRP
Ellyn Getz is the Associate Director of Development and Community Engagement at CISCRP. She is responsible for directing CISCRP’s educational and outreach initiatives, including AWARE for All: Clinical Research Education Days, MT Pharmacy and Journey to Better Health mobile experiential... Read More →
avatar for Kim Stephens

Kim Stephens

President, Project Alive - Hunter Syndrome
Dr. Kim Stephens is the President of Project Alive, a nonprofit organization whose mission is to cure Hunter Syndrome/Mucopolysaccharidosis II through research and advocacy. Having a son with Hunter Syndrome herself, this is also a personal mission for Dr. Stephens. Dr. Kim Stephens... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 9 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#359: Training for the Electronic Capture of PRO Data in Clinical Trials: Views from ePRO Vendors, Sponsors, Sites, and Patients
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-698-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum presents the ePRO Consortium’s best practices recommendations for training subjects and sites on technology use in clinical trials. Sponsor, ePRO vendor, site, and patient perspectives will be shared on training challenges and solutions.

Learning Objectives

Discuss best practice recommendations from the Consortium for training on the use of technology to collect PRO data in clinical trials; Identify challenges associated with training subjects and study sites from a vendor, sponsor, site, and patient perspective.

Chair

Serge Bodart, MS

Speaker

Panelist
Jenny Ly, PhD

Panelist
Patricia DeLong, MS

Panelist
T.J. Sharpe, PMP

Panelist
Jessica Branning



Speakers
avatar for Serge Bodart

Serge Bodart

Senior Advisor, Outcomes Science, CRF Bracket
Serge has extensive experience working in patient outcomes and new technologies. With Dr Bruno Pornel, he founded SYMFO, a European based ePRO provider in 2000. After he sold his company, he acted as the eCOA Subject Matter Expert at Biomedical Systems. Serge is currently Senior Advisor... Read More →
avatar for Patricia DeLong

Patricia DeLong

Manager, Patient-Reported Outcomes, Janssen Global Services, LLC
Patricia (Trish) Shepherd Delong has worked in the PRO and eCOA fields for more than ten years in both contract research and pharmaceutical organizations. She currently holds the role of PRO Process expert within the PRO team at Janssen. In this role Trish represents the PRO functional... Read More →
avatar for Jenny Ly

Jenny Ly

Clinical Science Advisor, ERT
Jenny Ly is currently a Clinical Science Advisor at ERT. She is a neuropsychologist with over 10 years of experience in the management and design of clinical trials. She has extensive experience developing training content for site staff, study participants, and caregivers that align... Read More →
avatar for T.J. Sharpe

T.J. Sharpe

Patient Advocate, Starfish Harbor LLC
T.J. Sharpe is a Stage IV melanoma patient who shares his journey through cancer in the Patient #1 Blog.  He was diagnosed in August 2012 with melanoma tumors in multiple organs, only four weeks after his second child was born. Since then, he has undergone six surgeries and four... Read More →
avatar for Jessica Branning

Jessica Branning

Owner and Co-Founder, ClinCloud
Jessica Branning is the Co-Founder and Owner of ClinCloud, LLC, a Florida based clinical research site. She has nearly 5 years of clinical research experience in multiple therapeutic areas including Alzheimer's disease, Parkinson's disease, migraine, osteoarthritis, diabetes, and... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 10 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#362: Patient Engagement Quality Guidance: Results and Learnings from Global Multistakeholder Pilots
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-686-L04-P; CME 1.25; IACET 1.25; RN 1.25

This panel session will present actual projects that are testing the Patient Engagement Quality Guidance in practice in various scope and context to determine how the guidance could have increased the impact or improve the results/outcomes in the projects.

The expert panel will share their interim feedback and learnings as well as challenges and how they’ve overcome them, focusing on the practical implementation of the PE Quality Guidance in these different situations. They will explore common themes and identify variations to provide a holistic overview of the usage of the PE Quality Guidance in different settings. The panel will also provide insights from different stakeholder perspectives on the use and value of the PE Quality Guidance and how it can facilitate an improved and systematic PE across the medicines’ development lifecycle. Speakers will include: representatives piloting/with practical experience of the Quality Guidance from patient/patient groups; academia and industry. Finally, the audience will get access to these resources to start their own PE journey.

Learning Objectives

Describe the Patient Engagement Quality Guidance and the seven quality criteria that it presents; Discuss actual projects from various organizations; Identify how to operationalize the guidance and move from paper to action; Describe a patient engagement platform that connects and interlinks people, organizations, initiatives, resources, and tools; Recognize the co-created resources.

Chair

Nicholas Brooke, MBA

Speaker

Panelist
Jennifer Martin

Panelist
Kay Jane Warner

Panelist
Thomas Gegeny, MS



Speakers
avatar for Nicholas Brooke

Nicholas Brooke

Executive Director, Patient Focused Medicines Development (PFMD), The Synergist.org
Nicholas Brooke is Founder of The Synergist, a collaboration platform incubator that brings key players together with the express aim of solving significant societal problems through collective action. Under Nicholas’ leadership, The Synergist acts as a backbone, providing vision... Read More →
TG

Thomas Gegeny

Senior Scientific Director, Envision Pharma Group
Tom Gegeny, MS, ELS, MWC joined Envision Pharma Group in 2006 and is currently a Senior Medical Director. He has over 20 years of experience in medical communications, including previous roles as Executive Director and Senior Editor at The Center for AIDS Information and Advocacy... Read More →
avatar for Jennifer Martin

Jennifer Martin

Vice President, Program Development, National Kidney Foundation
As Vice President for Program Development for NKF, Jennifer Martin is responsible for developing and implementing an overall strategy for NKF programs and services throughout the country, working in close collaboration and partnership with kidney patients, families, and living donors... Read More →
KW

Kay Warner

Patient Engagement Lead, Patients in Partnership, GlaxoSmithKline
Kay Warner is a Director within the Patients in Partnership team at GlaxoSmithKline Research and Development. Kay moved to her current role as Patient Engagement Lead in January 2017 and is patient engagement subject matter expert at GSK having interacted with many individual patients... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#372: Advancing Value and Access With Technology
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-695-L04-P; CME 1.25; IACET 1.25; RN 1.25

The purpose of this session is to describe advanced technologies being used to advance value and access through specific, real case examples, in terms of their use for contracting and value assessment. Speakers will describe the technology and use cases associated with blockchain, distributed networks and use of artificial intelligence in outcomes-based contracting among other technological advances.

Learning Objectives

Describe the use of technology to advance value and access; Discuss the use of specific technologies, including blockchain, artificial intelligence, and distributed networks.

Chair

Richard Gliklich, MD

Speaker

The Power of the New Healthcare Data Intelligence
Ian Rentsch, JD

Advancing Outcomes-Based Contracts with AI
Robert A. DiCicco, PharmD



Speakers
avatar for Richard Gliklich

Richard Gliklich

Chief Executive Officer, OM1
Dr. Richard Gliklich is the CEO of OM1, Inc., an outcomes and technology company focused on real world evidence and measuring and predicting outcomes. Previously, he was founder and CEO of Outcome, which he led from inception through its acquisition by Quintiles. A graduate of Yale... Read More →
avatar for Ian Rentsch

Ian Rentsch

Chief Executive Officer, Clinerion
Ian Rentsch has over 23 years’ experience in Multinational Corporate Affairs, Outsourcing Management & Clinical Research Development working in large multinational companies including large biopharma and Contract Research Organizations Globally and in Emerging Markets. Most recent... Read More →
avatar for Robert DiCicco

Robert DiCicco

Deputy Chief Health Officer, IBM Watson Health
Rob DiCicco is a Deputy Chief Health Officer for IBM Watson Health where he focuses on Life Sciences. Rob joined IBM in 2018 after a long career in Pharmaceutical R&D spanning nearly 30 years. His area of expertise includes clinical pharmacology and experimental medicine, clinical... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#378.1 SB: On the Soapbox: Good for People and Good for Research - Individuals as Research Partners
Component Type: Forum
Level: Basic
CE: ACPE 0.50 Knowledge UAN: 0286-0000-19-737-L04-P; CME 0.50; IACET 0.50; RN 0.50

The intersection of data privacy and an acknowledgement that research data lacks depth, diversity, and real-world information are driving new health database models. The benefits of promoting people from subjects to research partners will be covered.

Learning Objectives

Identify new models in patient engagement and data stewardship towards richer research; Analyze how patient privacy and data connectivity can co-exist; Explore the benefits of patients as partners in research versus research subjects.

Chair

Dawn Barry, MBA


Speakers
avatar for Dawn Barry

Dawn Barry

President and Co-Founder, LunaPBC
Dawn Barry is President and Co-founder of Public Benefit Corporation, LunaPBC. LunaPBC launched LunaDNA, the first people-powered health data sharing platform owned by its community of data donors. LunaDNA members share in the value created by the platform through the offering of... Read More →


Wednesday June 26, 2019 3:30pm - 4:00pm
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

4:15pm

#380: Incorporating Patient Input into the Design and Conduct of Clinical Trials
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-712-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will outline efforts, best practices, and key considerations to enhance the incorporation of patient input into the design and conduct of clinical trials.

Learning Objectives

Outline current efforts and opportunities to incorporate patient input into the design and conduct of clinical trials (CT); Discuss approaches, best practices, and key considerations to facilitate patient recruitment, enrollment and retention, and minimize the burden of patient participation in CTs; Identify opportunities to enhance patient engagement and ensure patient input informs CTs.

Chair

Meghana Chalasani, MHA

Speaker

Panelist
Alexis Reisin Miller, JD

Panelist
Michelle Tarver, MD, PhD

Panelist
Marilena Flouri, PhD

Panelist
Mary Elmer, BSN, MSN, RN

Panelist
T.J. Sharpe, PMP



Speakers
avatar for Meghana Chalasani

Meghana Chalasani

Senior Research Analyst, Office of the Center Director, CDER, FDA
Meghana Chalasani currently serves as a senior research analyst for the Patient-Focused Drug Development (PFDD) Program in FDA’s Center for Drug Evaluation and Research (CDER). She works closely on CDER’s various PFDD initiatives and provides strategic, regulatory, program, and... Read More →
AM

Alexis Miller

Senior Director, Regulatory Science and Policy, Sanofi
Alexis is a Senior Director for Sanofi Global Regulatory Science & Policy, helping to shape the regulatory and policy environment on topics like patient-focused drug development, digital health technology regulation, and various PDUFA initiatives and FDA program implementation. Prior... Read More →
avatar for Michelle Tarver

Michelle Tarver

Director of Patient Science and Engagement, OST, CDRH, FDA
The Patient Science and Engagement Program fosters innovative approaches to collecting, analyzing and integrating the patient perspective in the development, evaluation and surveillance of medical devices, including digital health technologies. Under her leadership, CDRH is making... Read More →
avatar for Mary Elmer

Mary Elmer

Executive Director, Patient, Caregiver and Consumer Experience, Patient Engagement Operations, Merck
Mary Elmer is the Executive Director, Patient, Caregiver, and Consumer Experience, Patient Engagement Operations at Merck. This group seeks to create an experience that engages consumers and patients in optimizing their health, wellbeing, and healthcare outcomes.She also leads a team... Read More →
MF

Marilena Flouri

Mathematical Statistician, OTS, CDER, FDA
Marilena is a statistical reviewer supporting the Division of Dermatology and Dental Products (DDDP) in the Center for Drug Evaluation and Research (CDER) at the FDA. Marilena joined the FDA in 2016 immediately after receiving her Ph.D. in Statistics from the University of Maryland... Read More →
avatar for T.J. Sharpe

T.J. Sharpe

Patient Advocate, Starfish Harbor LLC
T.J. Sharpe is a Stage IV melanoma patient who shares his journey through cancer in the Patient #1 Blog.  He was diagnosed in August 2012 with melanoma tumors in multiple organs, only four weeks after his second child was born. Since then, he has undergone six surgeries and four... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 9 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#384: Patient Preferences in Decision Making and the PREFER Project: Past, Present, and Future
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-703-L04-P; CME 1.25; IACET 1.25; RN 1.25

Speakers will present an historical overview of decision making, including PREFER’s place in that evolving landscape; the results from the ongoing PREFER project; and how patients and their representatives provide valuable insight into the project.

Learning Objectives

Discuss the evolution of decision making and the expanding role of patient preferences; Describe the aims and results of the PREFER project regarding when and how to integrate patient preferences into medical product decision making from diverse perspectives: industry, regulator, and patient; Identify the value gained by including patient representatives in study development.

Chair

Kristin E Bullok, PhD

Speaker

Benefit-Risk Evaluation with the Patient’s Perspective: Past, Present, and Future
Juhaeri Juhaeri, PhD

Patient Preference Studies: Why, When, and How
Rachael L. DiSantostefano

Patient Involvement in Preference Study Design: A Case Study
Jenny Sharpe, PhD



Speakers
avatar for Kristin Bullok

Kristin Bullok

Benefit-Risk Management Scientist, Global Patient Safety, Eli Lilly and Company
Kristin Bullok, PhD, is a research scientist in benefit-risk management at Eli Lilly and Company, Global Patient Safety. Since joining Lilly, she has years of combined experience in conducting structured benefit-risk assessments, consulting on patient preference trade-off studies... Read More →
avatar for Rachael DiSantostefano

Rachael DiSantostefano

Senior Director, Benefit Risk, Epidemiology, Janssen Research & Development, LLC
Rachael L. DiSantostefano, MS PhD, is a Senior Director of Benefit-Risk in the Epidemiology Department within Janssen Pharmaceuticals, R&D, LLC. She has 25 years of pharmaceutical research experience across the quantitative disciplines of epidemiology, biostatistics, and health outcomes... Read More →
avatar for Juhaeri Juhaeri

Juhaeri Juhaeri

Vice President and Head, Epidemiology and Benefit-Risk Evaluation, Sanofi
Juhaeri Juhaeri, Ph.D., is Vice President and Head of Epidemiology and Benefit-Risk at Sanofi. Juhaeri is an Epidemiologist and Statistician with over 20 year experience in epidemiologic research in academia and in pharmaceutical industry. He joined Sanofi in 2001 where he established... Read More →
avatar for Jenny Sharpe

Jenny Sharpe

Research Communications Manager, Muscular Dystrophy UK
Jenny is Research Communications Manager for Muscular Dystrophy UK, a charity bringing together over 60 rare and ultra-rare muscle weakening and wasting conditions. She is primarily responsible for overseeing the charity’s communications about research. This involves translating... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Thursday, June 27
 

9:00am

#407: A Patient Engagement Wrap Up: Lessons Learned from DIA 2019 and Where Do We Go from Here
Component Type: Workshop
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-721-L04-P; CME 1.25; IACET 1.25; RN 1.25

The final day of DIA 2019 presents a unique opportunity for the multidisciplinary stakeholders interested in the continued growth and evolving trajectory of patient engagement and patient-focused drug development to convene and collectively consider opportunities to carry on this important work beyond the annual meeting. Members of DIA’s Annual Meeting Planning Committee who represent the Patient Engagement track will host an interactive forum to discuss key take-home messages discussed throughout the four days of DIA 2019 and implications for the future of patient engagement.

Learning Objectives

Identify themes and emerging trends relevant to partnering with patients and expanding how we incorporate the patient’s voice into drug development; Describe practical solutions for applying learnings from DIA 2019; Identify opportunities to communicate and share findings across stakeholder groups, foster adoption of the learnings, and continue to advance our shared understanding of outcomes of existing and emerging patient engagement work; Discuss implications for the future.

Chair

Kristin Voorhees, MA

Speaker

Facilitator
K. Kimberly McCleary

Facilitator
Jennifer Helfer, PhD

Facilitator
Deborah E. Collyar

Facilitator
Steven L. Roberds, PhD

Facilitator
Kristin Voorhees, MA



Speakers
avatar for Deborah Collyar

Deborah Collyar

President, Patient Advocates In Research (PAIR)
Deborah Collyar has applied a wealth of leadership skills from her successful IT business career to help create patient-centered medical research and healthcare, after her first cancer diagnosis at the age of 32. She founded Patient Advocates In Research (PAIR) in 1996, “where research... Read More →
avatar for Jennifer Helfer

Jennifer Helfer

Patient Advocacy, bluebird bio, Inc.
Jennifer Helfer joined bluebird bio in December of 2015. As part of the Patient Advocacy team, Jen serves as a conduit to patient organizations and advocates. Patient Advocacy is responsible for building and sustaining trusted collaborations with stakeholders to address patient and... Read More →
avatar for K. Kimberly McCleary

K. Kimberly McCleary

Founder and Chief Executive Officer, The Kith Collective, LLC
Kim McCleary has been at the forefront of patient engagement for nearly 30 years. She is a nationally regarded subject matter expert on patient-focused medical product development, patient-centered benefit-risk assessment, and organizational change. McCleary serves on numerous advisory... Read More →
avatar for Steven Roberds

Steven Roberds

Chief Scientific Officer, Tuberous Sclerosis Alliance
Steve leads the development and execution of the TS Alliance's scientific strategy through partnerships and conversations with stakeholders including individuals and families affected by TSC, basic and clinical researchers, healthcare providers, government agencies, and other non-profit... Read More →
avatar for Kristin Voorhees

Kristin Voorhees

Senior Manager, Patient Advocacy, Ultragenyx Pharmaceutical
As Senior Manager of Patient Advocacy at Ultragenyx Pharmaceutical, Kristin supports, educates, and empowers the rare disease community and strives to make a positive difference in the lives of patients, caregivers and families. She is trained in health communications and has led... Read More →


Thursday June 27, 2019 9:00am - 10:15am
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA