October 2023 - Congressman Dunn (R-FL), Congresswoman Doris Matsui (D-CA) and Reps. Mike Thompson (D-CA), Mike Kelly (R-PA), and Markwayne Mullin (R-OK) introduced the The Providing Realistic Opportunity to Equal and Comparable Treatment for Rare (“PROTECT Rare”) Act, legislation to support patients with rare and ultra-rare diseases in getting evidence-based, medically necessary care covered by their health insurance. The bill permits Medicare and Medicaid to use clinical guidelines and peer-reviewed literature to allow for coverage of rare disease treatments.
Problem. There are often so few patients with a very rare condition for whom drug companies will not, or often cannot, do a clinical trial. Rare disease patients are all too often left fighting for off-label access to a treatment. For patients relying on Medicare Part D, access to off-label treatments within the standard of care can be particularly problematic since Part D plans are prohibited from including off-label uses not listed in compendia in the Part D benefit. For these patients, there is not even an appeal or reconsideration mechanism available to overcome the “fact” that the prescribed use is outside of the Part D benefit.
Additionally, these treatments are often used in combination with other FDA approved treatments, making the FDA approved treatment more effective or supporting the effectiveness of the FDA approved treatment. Managing all of this can be quite challenging for a patient attempting to live as best they can with a rare disease and navigate the complexity of the healthcare system.
Solution. Ensure parity in coverage for Medicare and Medicaid beneficiaries with low-prevalence conditions by aligning the statutory definition of “medically accepted use” for low-prevalence conditions with sources likely to include the standard of care, i.e., not just FDA label and compendia, but peer-reviewed literature, and clinical guidelines.
Example - Cutaneous lymphoma -- Off-Label use of interferon, topical steroids and other drugs used to treat skin conditions is frequent and quite helpful in managing the disease. The increase in prices for these drugs or lack of access to them because an insurer will not cover it due to being off-label, creates limitations for effective treatment for some patients as there may be no alternatives suitable to managing their form of the disease.
“We look forward to the enactment of this important legislation, and for the work the sponsors have put into this so far. We are confident that this will make a significant difference in the lives of the rare disease patients we represent and work with every day,” said Haystack Project CEO, Chevese Turner.
This bill is supported by more than 60 rare disease organizations.
To learn more about the PROJECT Rare Act and how you can get involved, visit: https://haystackproject.org/protectact