Many rare disease patients, upon a diagnosis, believe that because they have coverage (commercial, Medicaid or Medicare) that they will be able to utilize the medicine or technology that was developed for their rare disease.
Unfortunately, this is not always the case. The reality can often be much different. After rounds of prior authorizations and appeals, patients are left with uncertainty.
Many advances have been made, with significant advances in rare disease treatment on the horizon. However, the health care system in the United States has been slow in preparing and even slower in making those advances available to patients. We must not only continue to advance therapies through discovery and development, but we must also make certain that patients ultimately have access to these therapies post-approval.
The Rare Access Action Project (RAAP) began in 2017 as an ad hoc coalition of life sciences and patient stakeholders focused on advocating for solutions to rare disease access to health care. The organization has evolved and is now a non-profit that is engaged in ongoing initiatives on both the Federal and State levels.
RAAP is committed to exploring policy solutions to address structural issues in access and coverage. RAAP will engage with research, public education, issue advocacy, and lobbying activities to shine a light on the challenges facing rare disease patients; and offer common sense solutions to them.
While rare diseases individually affect small numbers of patients, collectively they affect as many as 25 million Americans – making this a serious public health concern.
An orphan disease is defined as a condition that affects fewer than 200,000 people nationwide. Some diseases have patient populations of fewer than a hundred.
There are over 7,000 known rare diseases and over 50 percent of rare diseases affect children.
During the first 25 years of the Orphan Drug Act (passed in 1983), 326 new drugs were approved by the FDA and brought to market for all rare disease patients combined.
Rare diseases account for 35% of children’s deaths in the first year of life.
RAAP is working to forge a partnership with State policy stakeholders to create exciting new forums for educating state legislators on how state and local programs impact rare disease patients, as well as, keeping pace with new science to ensure continued patient access to care. More information will be forthcoming on this exciting initiative.
Medicare Part D was designed in the early 2000s before the revolution in oral, outpatient medicines occurred. Policymakers, to keep programs costs down, implemented a 5% catastrophic cost share for patients. However, with those advances, the catastrophic copayment has become a significant barrier to patients who believe they have both coverage and access to their medicines.
In 2017, RAAP developed a solution by proposing changes to help rare disease beneficiaries. Our proposal would amend an existing law (the coverage gap discount program statute) to require capping the patient cost sharing for orphan medicines in catastrophic coverage.
This is an important protection because patients with orphan diseases face high out of pocket costs, and any patient who enters catastrophic coverage must already have spent roughly $3,700 out-of-pocket on drugs covered under Part D. Medicare patients with the very rare diseases are especially vulnerable to high cost sharing, as they are mainly on fixed incomes with limited additional resources.
UPDATE: Congress and the Administration have expressed willingness to address the 5 percent catastrophic copayment in Medicare Part D. We appreciate their willingness to address this issue, which is critical for patients with rare diseases.
UPDATE: Click HERE to read an OpEd by RAAP President Mike Eging.
However, rare disease patients are threatened by the drug reform plan, currently under consideration by Congress that restricts the development of life-saving therapies. Proposed changes to Medicare Part D drug coverage could severely limit the discovery of new treatments and cures. To protect the health of rare disease patients, we call on Congress to pass Medicare Part D reforms that evenly distribute liability among all manufacturers — ensuring access to future innovation.
With a high proportion of patients covered through the program, Medicaid has become an increasingly important link between patients and the care they need. RAAP has already begun engaging state and federal stakeholders to identify programmatic challenges and to offer solutions.