From February 28 to March 2, rare disease advocates met in Washington, DC for the 12th Annual Rare Disease Week on Capitol Hill. Organized by Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, this was the first time since 2020 that the program was able to be held in-person. Patient Worthy had the privilege to attend this year’s event and provide media coverage. 

The program began with a screening of a rare disease focused documentary. The film, Special Blood, focused on several different families as they learned to live with hereditary angioedema, a rare genetic disorder that causes sudden episodes of swelling. The screening featured a panel discussion with several members of the cast.

The next day consisted of the legislative conference, in which advocates learned about what it takes to meet with members of Congress and advocate for the rare community. Participants received general information about the state of Congress following the most recent round of mid-term elections which took place at the end of last year. In addition, we were given in-depth information on the four legislative asks that we would be asking our Congressional representatives and senators to support.

The asks that we were advocating for this year included:

1. Calling on members of Congress to sign on to a Congressional letter addressed to the US Food and Drug Administration (FDA). This letter calls on the agency to create an internal task force that would be used to review the agency’s activities related to rare disease. 
2. Support rare disease appropriations priorities. This includes increased funding for the rare disease activities of the FDA and NIH, as well as support of the National Academy of Medicine in a process of creating a legal definition for ultra-rare diseases, as well as investigating the possible implications and opportunities that would accompany it. 
3. Sign on as a cosponsor to the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act. The BENEFIT Act has been a long-standing goal of the rare disease community that would require the FDA to disclose how patient-focused drug development (PFDD) and patient experience data was considered during the evaluation process of approved treatments. It would also require such data to be considered as part of risk-benefit assessments. 
4. Join the Rare Disease Caucus, a bicameral, bipartisan caucus that aims to raise awareness and advocate for rare disease policy initiatives. 

The next day, the meetings with Congress members were held. In most cases, advocates met with Congressional staffers, not the members themselves. Nearly 600 advocates participated in Rare Disease Week, with 300 meetings held.

The final event of the week was the Rare Artist Reception, in which the recipients of the Rare Artist Award were able to display their artwork and talk about what inspired them. 

Were you unable to attend? You can watch some recordings from Rare Disease Week here.