According to a story from the National Organization for Rare Disorders, patient advocacy organizations are joining forces to send letters to Medicaid directors across the USA to advocate for the importance of Medicaid formulary access.
Formulary access allows for rare disease patients to obtain access to orphan drugs under Medicaid. Orphan drugs are treatments that are typically meant to address one, frequently rare, disease. These drugs may be more expensive compared to others, but they are often the only treatment available in many cases. Across the country, many states are closely monitoring Medicaid costs in order to maintain coverage for the greatest number of people. As a way to cut costs, some states have begun to seek out 1115 waivers, which will allow them to implement formulary restrictions for their respective Medicaid programs. Although this practice is meant to reduce costs for the state, it only does so by directly reducing quality of care and restricting patient access to treatment. Additionally, the use of such waivers will ultimately mean greater health expenses for the state in the long-term.
These restrictions can, in fact, overrule the treatment recommendations made by doctors, which directly inhibits a patient’s ability to get the treatment that they need. The effectiveness of a treatment can be closely linked with how rapidly it can begin being used, so any delays or restrictions mean that rare disease patients will get worse health outcomes and have lowered quality of life. These formulary restrictions are meant to promote the use of cheaper drugs, such as generics, but no orphan drugs have cheaper, generic counterparts. So in instances when these restrictions have been applied to orphan drugs, rare disease patients are left with no viable options.
Some states have also voiced concern over the FDA’s Accelerated Approval program. Accelerated Approval has been subjected to some criticism, with some saying that many therapies earning approval are of lower quality. When it comes to developing rare disease treatments, however, this isn’t usually a problem. Many rare disease treatments are simply impossible to test in the same way as more common disease treatments, because of the small patient population. Surrogate endpoint’ have been developed as a way to measure drug effectiveness instead. These are scientifically verifiable and have been used to test a variety of therapies that are widely considered successful and are commercially available.