DIA 2019 Global Annual Meeting

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

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

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

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

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

Component Type: Forum



Chair

Mary Stober Murray, MBA

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

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

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

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

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

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

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

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

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

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

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

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

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

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