Session Resources

Session 1: Beyond the Tower of Babel: FDA Leadership on Data Standards - Sept. 8, 2020

Session 1 Slide Deck

Government Resources:

Framework for FDA’s Real-World Evidence Program

FDA’s Technology Modernization Action Plan

https://www.fda.gov/about-fda/reports/fdas-technology-modernization-action-plan

https://www.fda.gov/media/130883/download

Other Resources:

Registries for orphan drugs: generating evidence or marketing tools?

Harnessing the Power of Real-World Evidence (RWE): A Checklist to Ensure Regulatory-GradeData Quality

Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series

Use of real‐world evidence in regulatory decisions for rare diseases in the United States—Current status and future directions

Session 2: Let's Stop Reinventing the Wheel: Scaling Best Practices for Patient Registries - Sept. 15, 2020

Session 2 Slide Deck

Regulatory strategies for rare diseases under current global regulatory statuses: a discussion with stakeholders

Standard Data Structures in Rare Diseases: CDISC User Guide for Duchenne Muscular Dystrophy and Huntington's Disease

Discussion Paper: Use of patient disease registries for regulatory purposes - methodological and operational considerations 

Session 3: Let's Get Real (World Evidence): Applications from the Oncology Community- Sept. 22, 2020

FDA-AACR Real-World Evidence Workshop

Real-world Data for Clinical Evidence Generation in Oncology

Real-world outcomes and management strategies for venetoclax-treated chronic lymphocytic leukemia patients in the United States

Session 4: From Brooklyn to Beijing: Global Rare Disease Registries as Global Learning Platforms - Sept. 29, 2020

COVID-19 Evidence Accelerator