On May 25, 2022, the Rare Disease Legislative Advocates (RDLA) held a briefing for the Rare Disease Congressional Caucus. This virtual event provided an update on various legislative initiatives relevant to the rare disease community on the federal level. The Rare Disease Congressional Caucus, is a bipartisan, bicameral caucus which functions as a platform for engaging and educating members of the legislature on issues relevant to the rare disease community and providing a rare patient voice.
The briefing was moderated by EveryLife Foundation for Rare Diseases Board Member Abbey Hauser. Speakers for the briefing included:
- Remy Brim, Ph.D., BGR Group
- Jamie Sullivan, EveryLife Foundation for Rare Diseases
- Illy Jaffer, Director, Federal Government Relations, American Cancer Society Cancer Action Network
- Ryan Fischer, Parent Project Muscular Dystrophy
Remy gave an update status for the Prescription Drug Fee User Program (PDUFA). This program, which has been in place for decades, must be renewed periodically, and the seventh edition is currently under consideration. This process occurs every five years and includes the commitment letter, which defines the arrangement between the US Food and Drug Administration (FDA) and the pharma industry. Different stakeholders are also allowed to provide input, and the makeup of the letter can alter the law significantly.
This year’s letter is expected to include pilot programs as well as various other improvements and policy changes. Some factors under consideration include chemistry and manufacturing controls, which are important for the gene therapies that will likely play a major role in treating rare genetic disorders. There is also a focus on new trial designs which has implications for the often smaller scale studies used in rare disease. There are also efforts to embrace technological innovations.
Jamie gave an update on the STAT Act and rare disease user fee priorities. The User Fee bill would expand some existing programs as well as authorize new ones. Changes to how the FDA provides drug exclusivity are up for consideration, and there are also proposed reforms to the Accelerated Approval pathway.
Lawmakers have also made changes to the STAT Act that is being floated. The Rare Disease Center of Excellence, for example, is no longer being included. This is a disappointing development for the rare disease community.
This bill also includes a proposal to exchange drug information with insurers earlier in the approval process. Insurance companies are often hesitant to cover rare disease drugs due to lack of information, leading to massive headaches for desperate patients. There are also plans for a voluntary third party payer program.
Ryan talked about patient experience data and the BENEFIT Act. This bill has been a work in progress for several years know and is widely supported by the rare community. A component of this is incorporating patient experience data into the FDA’s decision making (risk-benefit analysis). This data can include:
- Patient-focused drug development meetings
- Qualitative studies
- Observational studies
Hearings on the BENEFIT Act took place in March, and it has been introduced in both chambers.
Illy went into more detail about proposed changes to clinical trials, such as the benefits of decentralized trials. These trial arrangements were field tested when the COVID-19 pandemic began.
Patients are often expected to cover the majority of their own expenses (time off work, food, travel, etc.) to participate in a trial. These barriers compromise the effectiveness and demographic representation of trials and limits who can participate.
Decentralized trials also require access to certain technologies, causing similar issues.