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12: Value-Access [clear filter]
Monday, June 24
 

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
 
Tuesday, June 25
 

8:00am

#219: Personalized Healthcare and Clinical Outcomes: How Real World Endpoints Can Improve Approval and Access to Medicine?
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-584-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will present examples evaluating real world endpoints using electronic health records, current application, and challenges. A panel of experts will provide interpretation, discuss approaches to increase its reliability and acceptability. Join the Study Endpoints Community for a follow up Round Table discussion on Wednesday, June 26, 9:30-10:30AM in the DIA Community Zone 2, Sails Pavilion.

Learning Objectives

Describe opportunities and challenges utilizing real world endpoints in health care; Discuss examples and approaches to increase objectivity and reliability of real world endpoints; Explain implications for regulatory and payer decision-making.

Chair

Natalia Sadetsky, MD, PhD

Speaker

Panelist
Natalia Sadetsky, MD, PhD

Real World Data in 2019: What's New
Michelle Hoiseth

Real World Data Model with Visualization for Value and Access
Sharon Hensley Alford, PhD, MPH

Panelist
Ronald Cantrell, PhD, MPH

Panelist
Gillis Carrigan, PhD, MS



Speakers
avatar for Michelle Hoiseth

Michelle Hoiseth

Chief Data Officer, Parexel
Michelle has been working in the drug and device development industry for 30 years. She has enjoyed a variety of positions that allowed her to create product development plans, run study operations, and support product commercialization objectives. Michelle presently leads PAREXEL’s... Read More →
avatar for Natalia Sadetsky

Natalia Sadetsky

Senior Scientist, Genentech, A Member of the Roche Group
Natalia Sadetsky, is an medical doctor and epidemiologist by training. She has over 18 years of academic and industry experience in oncology. She joined Genentech, a member of a Roche group in 2013 and currently a Senior Data Scientist in Personalized Health Care Data Science, RWD... Read More →
avatar for Sharon Hensley Alford

Sharon Hensley Alford

Offering Management, IBM Watson Health
Sharon Hensley Alford, PhD is Associated Chief Health Officer for Data & Evidence within the Life Science (LS) pillar of IBM Watson Health. Dr. Alford has been working on data and analytic strategy for life science clients since she joined IBM 2+ years ago. Prior to IBM, she worked... Read More →
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Ronald Cantrell

Senior Director and Principal Data Scientist, Verana Health
Ronald Cantrell leads clinical data science at Verana as Senior Director and Principal Data Scientist. He has over a decade of experience working with and analyzing real world data in the life science industry. He previously served as Principal Data Scientist at Genentech, where he... Read More →
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Gillis Carrigan

Director, Epidemiology, Covance Market Access
Dr. Carrigan has over 20 years of experience in observational studies with a focus on study design and methodology. He is experienced with the use of electronic medical records (EMR), administrative claims data, and registry data. He has worked with observational data in a number... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 14B 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 →
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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

4:15pm

#299: Public and Regulatory Response To Drug Pricing Concerns
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-638-L04-P; CME 1.25; IACET 1.25; RN 1.25

Stakeholders agree that generics are an effective approach to lowering drug prices, but approvals are often challenging for sponsors to obtain, and the cheap prices that consumers value make it hard for sponsors to stay on the market. This session will describe the strategies that regulators and payers are using to combat those problems. FDA does not regulate the price of drugs, but the regulatory decisions it makes can nevertheless impact them. For generics, that can mean setting a high bar or a low bar for equivalence and deciding which kinds of products are eligible for accelerated review or additional interactions with the agency. Likewise, payers don’t control the application process, but their decisions go a long way to determining which products are developed. This session will analyze the regulatory strategies that can eventually prove successful, explain how companies and policymakers can best handle the development of complex generics, and discuss new steps payers are making to ensure generic sponsors have a market for the products after a long development process.

Learning Objectives

Identify the steps for successfully developing a complex generic product; Describe new reimbursement approaches being used to encourage a stable generic market; Discuss review pathway selection at the US FDA's Office of Generic Drugs; Recognize product categories where FDA is encouraging generic applications.

Chair

Nielsen Hobbs

Speaker

Panelist
Jan Burkett, MBA, RPh

Panelist
Heather Wall, MBA

Panelist
Adam Kroetsch



Speakers
avatar for Jan Burkett

Jan Burkett

Vice President, Drug Sourcing Officer, Express Scripts
Jan is a pharmacist with diverse experience in pharmacy benefit management, contracting and joint venture strategy. She joined Express Scripts in 1993 and is currently VP, Chief Drug Sourcing Officer, responsible for Express Scripts' drug sourcing procurement and strategy. Jan leads... Read More →
avatar for Gregory Daniel

Gregory Daniel

Deputy Director and Clinical Professor, Duke-Margolis Center For Health Policy
Gregory Daniel, PhD, MPH is the Deputy Director of the Duke-Robert J. Margolis, MD Center for Health Policy and a Clinical Professor in Duke's Fuqua School of Business. He directs the DC-based office of the Center leading its pharmaceutical and medical device policy portfolio, and... Read More →
avatar for Nielsen Hobbs

Nielsen Hobbs

Executive Editor, US Policy and Regulatory, The Pink Sheet/Scrip
Nielsen Hobbs has more than 20 years of experience covering the pharmaceutical industry, and writes frequently about the intersection of business, regulation, science and politics. Before joining the “The Pink Sheet” he worked for Congressional Quarterly. Hobbs enjoys discussing... Read More →
avatar for Heather Wall

Heather Wall

Chief Commercial Officer, Civica Rx
Heather’s expertise spans the healthcare continuum focusing on design, implementation and execution of hospital & health system strategies to improve patient care. Heather has a 20+ year career where she has had accountability for strategy, operations, finance & high-value health... Read More →
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Adam Kroetsch

Research Director, Duke-Margolis Center For Health Policy


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Wednesday, June 26
 

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
SD

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 →
ST

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

4:15pm

#389: Transparency, Expanded Access Navigator, Right to Try: Helping Patients Get Access to Investigational Medicines?
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-708-L04-P; CME 1.25; IACET 1.25; RN 1.25

Numerous steps have been taken to increase access to investigational medicines for patients with serious and life-threatening diseases. This session will explore whether these initiatives have had the desired impact.

Learning Objectives

Describe changes to expanded access programs, including Right to Try; Assess the impact of these changes to the patient; Discuss ways to improve access to investigational medicines for patients.

Chair

Kim Quaintance-Lunn

Speaker

Patient Perspective on Preapproval Access
Melinda Bachini

Innovating Compassionate Use Request Processes with CompAC
Beverly L Harrison

Assessment of Progress
Alison Bateman-House, PhD, MA, MPH



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 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 Kim Quaintance-Lunn

Kim Quaintance-Lunn

Vice President and Head, Regulatory Policy, North American Regulatory Affairs, Bayer
Kim Quaintance-Lunn serves as Vice President and Head, Regulatory Policy, North American Regulatory Affairs, at Bayer. Kim joined Bayer in February 2014 to establish and lead the US regulatory policy function for the organization. She works with colleagues to analyze regulatory policy... Read More →
MB

Melinda Bachini

Advocacy Coordinator, Cholangiocarcinoma Foundation


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA