PROTECT Rare Act – Providing Realistic Opportunity To Equal and Comparable Treatment for Rare Act
Over 7,000 rare diseases affect more than 30 million people in the United States. CRPS is considered a rare disease as less than 200,000 people in the US are affected. There is a lack of clinical research for the diagnosis and treatment of rare diseases because there are too few patients for drug companies be willing or able to sponsor clinical trials. Rare disease patients are all too often left fighting for off-label access to a treatment. This creates an even larger problem for patients relying on Medicare, as Part D plans are prohibited from including off-label uses of medications in the formulary. For these patients, there is not even an appeal or reconsideration mechanism available to appeal.
The PROTECT Rare Act (HB 6094) is designed to ensure parity in coverage for Medicare and Medicaid beneficiaries with rare diseases by including peer-reviewed literature and clinical guidelines and outcomes as “medically accepted use”. There is precedent for using medications “off-book” as certain medications were deemed “medically acceptable” for cancer treatment in the 1970’s. For many people with rare disease, off-label use of certain medications are the only treatment available.
This bill is supported by more than 60 rare disease organizations, including the RSDSA.
We encourage you to contact your federal representatives and Senators and ask them to support the PROTECT Rare Act.