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Monday, June 24 • 3:30pm - 4:30pm
#170: Making Early Access for Patients Happen

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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.


Monika Schneider, PhD


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

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 →
avatar for Monika Schneider

Monika Schneider

Managing Associate, Duke-Margolis Center For Health Policy
Monika Schneider is a Managing Associate within the biomedical innovation team at the Duke-Margolis Center for Health Policy. Her work focuses incentive mechanisms and payment reform models that could encourage development in areas of medical unmet need. Monika's portfolio includes... 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 PDT
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  12: Value-Access, Forum |   05: Patient Engagement, Forum