Barriers to Rare Disease Treatments
Play • 58 min

Rare Disease Day is February 28. New and exciting medical innovations and drug therapies for rare disease abound, but accessing those treatments is another challenge. Discriminatory healthcare metrics like quality-adjusted-life-years (QALYs) devalue rare disease patient lives, signaling that a patient’s life isn’t worth covering treatments and therapies. 

Dr. Bill Smith explains how QALYs are used and how it prevents patients from getting the life-saving care they need. Plus, Patients Rising Field Correspondent Kate speaks with Marni Cartelli, a rare disease patient living with complex regional pain syndrome, and Patient Correspondent Lillian Isabella celebrates Rare Disease Day by sharing her story of being diagnosed with PKU. 

Guest:

William Smith, Ph.D. 

Visiting Fellow in the Life Sciences, The Pioneer Institute

Dr. William S. Smith is The Pioneer Institute’s Visiting Fellow in the Life Sciences. Smith has 25 years of experience in government and in corporate roles, including senior staff positions on Capitol Hill, the White House Office of National Drug Control Policy, and the Massachusetts Governor’s office where he served under Governors Weld and Cellucci. 

He spent ten years at Pfizer Inc as Vice President of Public Affairs and Policy where he was responsible for Pfizer’s corporate strategies for the U.S. policy environment. He later served as a consultant to major pharmaceutical, biotechnology and medical device companies. Dr. Smith earned his Ph.D. in political science with distinction at The Catholic University of America.

Links:

William Smith, Ph.D.

The Biden administration needs to look beyond ICER for evaluating drug therapies

Inside Xavier Becerra’s quest for health care for immigrants

Maryland Senate overrides Hogan veto of bill that funds nation’s first drug affordability board

Senate Democrats push for public option as Biden weighs health-care reform plans

Marni Cartelli

She Started A Nonprofit Around Her Rare Disease. Here's Why.

Rare Disease Patients, You Must Make Yourselves Heard

Swapna, Alabama, and the Spotlight on Rare Disease

Patients Rising Concierge 

Patient Correspondent: Lillian Isabella

Need help?

The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands. Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at PatientsRisingConcierge.org

Have a question or comment about the show, want to suggest a show topic or share your story as a patient correspondent?

Drop us a line: podcast@patientsrising.org

The views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising.

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