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. 


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.


William Smith, Ph.D.

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Marni Cartelli

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Patient Correspondent: Lillian Isabella

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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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