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Major Regulatory Challenges Enabling Decision Making for Early Patient Access: Regulatory Tools and Sources of RWE
Session Chair(s)
Karl Broich, DrMed
President
Federal Institute for Drugs and Medical Devices (BfArM), Germany
Joseph Scheeren, PharmD
Founder
Scheeren HealthCare LLC, United States
The commitment for enabling early patient access to new innovative medicines is one of the major challenges in regulatory science. From a regulator’s perspective, early access tools are in place in the EU and the US in order to support early approval. In addition, real world evidence (RWE) presents a significant opportunity to improve regulatory decision making. Technological advances permit collection of real world data (RWD) from an increasing number of sources like patient registries and electronic patient records etc., while new artificial Intelligence approaches permit to translate these data into RWE. This cross functional DIAmond session will discuss opportunities and challenges from the regulator (EU and US), industry and patient perspective.
Learning Objective : • Understand the challenges of establishing a patient registry in an ultra-orphan disease area • Understand steps that can be taken to create larger and richer registries • Develop a perspective on how best to work with industry partners who want to support registry efforts
Speaker(s)
Early Access to Medicines in the EU - Regulatory Tools and Challenges in the Context of Other Regions
Tomas Salmonson, DrMed, PhD
Critical Path Institute, United States
Partner
Current Status and Challenges of Early Access Tools and the Opportunities of Real-World Evidence (RWE) for Regulatory Decision Making in the US
Sandra Kweder, MD
Greenleaf Health/Elilquent, United States
Principal, Drug and Biological Products
Major Challenges for Industry and Opportunities for Real World Data Generation Including Patient Registry to Enable Regulatory Decision Making
Badri Rengarajan, MD
Jazz Pharmaceuticals, United States
Life Sciences
Panel Discussion
Elizabeth Vroom
Duchenne Parent Project, Netherlands
President
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