An opinion expressed by Sheila Frame, President of Americas at Amryt Pharma, was published recently in the Pittsburgh Post-Gazette. Ms. Frame, who serves on the board of the Rare Disease Company Coalition, believes that the 118th US Congress, which convenes this month, represents an opportunity to provide more research, develop more drugs, and find more cures for the 15 million children who are plagued by rare diseases.
Potential Bipartisanship
It is estimated that thirty million people in the U.S. are living with a rare, debilitating, and sometimes fatal disease. Approximately 50% of these cases are children.
An opportunity lies in a possible compromise on some of the issues such as advancing cures or facilitating access to patient care.
A New Law That Provides A Source of Funding
The Prescription Drug User Fee Act of 1992 (PDUFA) authorized the FDA to charge a fee from companies that produce biological products and human drugs.
The fees represent a source of funding so that the FDA may continue to maintain an efficient review process for biological products and drugs.
In September 2022 the President signed into law the sixth reauthorization of the PDUFA. The new law provides assurance that the fees will continue to flow to the FDA from 2023 through 2027.
A Rare Disease Endpoint Advancement Pilot Program was included in the September 2022 reauthorization. An endpoint associated with a rare disease is established (usually in clinical trials) to give evidence of a drug’s effectiveness in treating rare diseases.
Drug development programs for rare diseases will be advanced through improved collaboration between the FDA and trial sponsors. New endpoints will be identified that measure the effectiveness of a medicine. The goal is to develop improved therapies for rare diseases while expediting drugs to patients.
Ms. Frame notes that 90% of rare diseases do not have an FDA-approved treatment. The new program is clear evidence of the potential progress that is possible with the collaboration of the biopharmaceutical community and policymakers.
The first step in finding a cure is diagnosing the disease. The Newborn Screening Saves Lives Act which was passed in the House in June of 2021 is still pending. The bill would expand and expedite the screening of newborns with inherited diseases.
Approximately four million babies in the U.S. are screened each year. Newborn screening finds that one out of 178 children screened has a potentially fatal condition. Ms. Frame and her organization are asking the Senate to act and reauthorize the Newborn Screening Act to expand early diagnosis for these children.
The BENEFIT ACT (Better Empowerment Now to Enhance Framework and Improve Treatment) is proposed legislation that would give families an expanded role in accelerating cures and new treatments.
A United Community
Ms. Frame reminds us that the impact of a public policy can ensure innovation and access in bringing medicine to patients with no other options.
Democrats and Republicans are being asked to build on past endeavors and reach across the aisle in this new Congress. They are asked to stand with these children and their families in fighting rare diseases.
