On Thursday, February 29th, Rare Disease Day, the House Energy and Commerce Committee will hold a congressional hearing, titled “Legislative Proposals to Support Patients with Rare Diseases” at 10:00 am ET. This hearing will review 18 pieces of legislation intended to support patients living with rare diseases, including the Better Empowerment Now to Enhance and Improve Treatments (BENEFIT) Act. 

A congressional hearing is a critical step in the process of advancing legislation toward becoming law. We thank our congressional champions, Rep. Doris Matsui (D-CA) and Brad Wenstrup (R-OH), and the leaders of the Energy and Commerce Committee Chairwoman Cathy McMorris Rodgers (R-WA), Ranking Member Frank Pallone (D-NJ) and Health Subcommittee Chairman Brett Guthrie (R-KY) and Ranking Member Anna Eshoo (D-CA) for making this bill a priority for the Committee’s consideration.

The BENEFIT Act would require FDA to include in the risk-benefit assessment framework of a new drug application how patient experience data was considered in the review process. The Duchenne community knows how important information from patients and caregivers is to clinical research and that this information must inform FDA decisions. For example, patients and caregivers may prioritize benefits and risks of a drug differently than a clinical researcher or regulator. This information should be weighed by the FDA and they should make public how it was factored into their decision.

Providing this information to the public, and to patient communities making significant investments in developing patient-focused drug development (PFDD) data, increases transparency and will accelerate PFDD strategies more broadly.

You can watch a recording of the hearing here.

Make Your Voice Heard & Support BENEFIT

On March 4th & 5th, PPMD Advocates will head to Capitol Hill to speak with their legislators about supporting the Duchnne community, including asking for support of the BENEFIT Act, which was reintroduced at the beginning of the 118th Congressional session last year.

Please sign up for PPMD’s Action Alerts to take action to support our message from home while PPMD Advocates are on the Hill next week.

If you have any questions please reach out to Lauren Stanford at Lauren@parentprojectmd.org.