By: Sheila Frame, President, Americas at Amryt Pharma and serves as a Board Member of the Rare Disease Company Coalition. She has spent more than 20 years in the biopharmaceutical industry, including in commercial leadership roles at Novartis and Bristol Myers Squibb.
Originally Published at RealClearScience.com (January 18, 2023)
The United States Congress is officially a house divided with the two chambers controlled by rival parties. The split will significantly affect either party’s ability to pass significant legislation through Congress, possibly resulting in two years of partisan deadlock that may remain unresolved until the next election cycle in 2024. Political polarization has become our new normal. Yet, I believe there are some areas where Americans can and do stand united. One of those areas: supporting health care for our kids.
In the US today, approximately 30 million people live with a rare disease, about half of whom are children. While many of these diseases are inherited at birth, they are also chronic, can worsen over time, and are often life-threatening. As the 118th Congress begins in January and works to identify areas of bipartisan compromise – accelerating diagnosis, advancing cures and supporting access to patient care for these 15 million kids can be a bipartisan victory.
It is important to recognize achievements from the 117th Congress that will disproportionately benefit kids with rare disease. In particular, the reauthorization of the Prescription Drug User Fee Act included the establishment of a Rare Disease Endpoint Advancement (RDEA) Pilot Program. This new program seeks to advance rare disease drug development programs by providing new ways for clinical trial sponsors to collaborate with the U.S. Food & Drug Administration (FDA). This will support identification of new endpoints to measure a medicine’s effectiveness, hopefully leading to more effective rare disease therapies while reaching patients sooner. This program is a noteworthy example of the progress that can be accomplished when policymakers and the innovative biopharmaceutical community collaborate with patients’ best interests in mind. Yet, with over 90% of rare diseases without an FDA-treatment, there is so much work left to do.
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