Past Event

Rare Disease Treatments: Regulatory and Policy Reform


Scientific advances are making new treatments possible for some of the more than 7,000 rare diseases that currently impact one in ten Americans.

Yet, some advocates say current regulatory measures are holding back further progress and lack engagement with rare disease patients and experts. Efforts to change the status quo are ongoing on Capitol Hill. Legislation introduced in Congress seeks to improve protocols for rare diseases therapies, drug review processes and patient access to treatment. 

On World Rare Disease Day, The Hill will be joined by policymakers, healthcare professionals, researchers, clinicians and patient advocates for a comprehensive discussion about the nuances of regulatory reforms and policy implications for the 30 million Americans living with rare diseases. 


Monday, February 28, 2022
12:00PM ET/9:00AM PT



  • Rep. Brett Guthrie (R-KY), Member, Energy & Commerce Committee
  • Sarah Bacon, Rare Disease Patient Advocate
  • Freda Lewis-Hall, Former CMO and Executive Vice President, Pfizer
  • Kelly Maynard, President & Founder, Little Hercules Foundation
  • Frank McCormack, MD, Professor of Medicine; Director, Division of Pulmonary, Critical Care, & Sleep Medicine, University of Cincinnati
  • Mark Skinner, President & CEO, Institute for Policy Advancement 
  • Dr. Wen-Hann Tan, Attending Physician, Division of Genetics and Genomics, Boston Children’s Hospital & Associate Professor of Pediatrics, Harvard Medical School
  • Dr. Janet Woodcock, Deputy Principal Commissioner, FDA

Sponsor Perspective:

  • Del Lebel, Head of US Government Affairs and Policy, Alexion, AstraZeneca Rare Disease
  • Mike Page, Vice President, Global Regulatory Affairs Development Strategy, Alexion, AstraZeneca Rare Disease


  • Steve Clemons, Editor-at-Large, The Hill


Join the conversation! Tweet us: @TheHillEvents and #TheHillRareDiseases






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