Only 5% of rare diseases have a treatment approved by the Food and Drug Administration (FDA), and for one-third of individuals with a rare disease, it can take between one and five years to receive a proper diagnosis. Half of all patients diagnosed with a rare disease are children, and as many as 3 in 10 children with a rare disease will not live to see their 5th birthday. There are many barriers that patients face to gain access to these orphan therapies across our health care system in addition to often being faced with a patient journey filled with misdiagnosis and lack of treatment options. 

Patients pay copays each month for their rare treatments, the amount set by the plan. For those without coverage, ​​many life sciences companies offer access assistance through patient assistance and free drug programs that reduce the cost to qualifying patients throughout the year. In addition, many charitable organizations offer copay assistance and purchasing support.

Supporters have been unable to explain how PDABs will lower copays, encourage lower premiums, create transparency with associated healthcare costs, or not damage access that patients have with rare disease therapies through their current insurance.

The incentives to develop and bring to market therapies for rare and ultra-rare populations were intended to overcome economic hurdles that shift investment costs to larger, blockbuster therapies. The Inflation Reduction Act (IRA), which was passed in 2022, intended a similar exercise of federal government price settling. Even though an exemption for rare therapies was included in the bill, the implementation is problematic because it eroded the incentives of the seminal Orphan Drug Act. Now we are seeing the closure of rare programs, investment resources fleeing to other therapeutic areas, and market uncertainty caused by federal policymaking.

The Bottom Line

Proponents have shown us no evidence that PDABs will lower copays, encourage lower premiums, create transparency, or not damage access to therapies that patients with rare disease have through their current insurance, and other charitable programs. We urge legislators to navigate this issue with a commitment to ensuring that no patient is left fearful of losing access to the treatments that offer them a chance at a better tomorrow. We believe there are solutions that can enhance affordability, such as copay accumulator and maximizer reform, meaningful PBM transparency, and risk pooling. But proponents of PDAB are focused on an experimental program that has generated zero savings and offers no solution to lower patient costs since the first PDAB was created in 2019.