HTA Rare disease

Rare Diseases: A Conversation on Value Assessments

Check out an event this Friday, December 10, at 10am EST/7am PST on at The Hill on Rare Diseases: A Conversation on Value Assessments. A summary of the topic and the speaker list are below.

One in 10 Americans suffers from a rare disease—that’s 30 million Americans who need access to treatment. According o the NIH, a rare disease is one that affects fewer than 200,000 people in the United States at any given time. Since the passage of the Orphan Drug Act in 1983, progress has been made in researching and developing drugs for the 7,000 rare diseases currently identified by scientists, but many diseases still lack adequate therapies, preventing patients from accessing new innovations. 

What are some of the unique roadblocks drug manufacturers face when developing orphan drugs? How can we encourage new breakthrough treatments at prices patients can afford? What can policymakers do to increase innovation and access to orphan drugs for all patients?

The Hill will bring together researchers, advocates, and more for a comprehensive discussion on orphan drugs.

Friday, December 10, 2021
10:00AM ET/7:00AM PT


  • Rep. Larry Bucshon M.D. (R-IN), Member, Energy and Commerce Subcommittee on Health
  • Rep. Anna Eshoo (D-CA), Chair, Energy and Commerce Subcommittee on Health
  • Dr. Ge Bai, Professor, Healthcare Policy & Management, Johns Hopkins Bloomberg School of Public Health
  • Tammy Boyd, JD MPH, Chief Policy Officer and Counsel, Rare Disease Diversity Coalition
  • Annie Kennedy, Chief of Policy, Advocacy, & Patient Engagement, EveryLife Foundation for Rare Diseases
  • Kelly Maynard, President, Little Hercules Foundation
  • Dr. Brett McQueen, Director, Center for Pharmaceutical Value, University of Colorado
  • Debra Regier, MD, Medical Director, Rare Disease Institute, Children’s National Hospital
  • Dr. Jason Shafrin, Founder, Editor, and Chief Writer, Healthcare Economist; Senior Managing Director, Center for Healthcare Economics and Policy, FTI Consulting

Register here.

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