RDLA Webinar Provides Vital Updates on Legislation Relevant to the Rare Disease Community

On October 15th, 2020, the Rare Disease Legislative Advocates (RDLA) held a webinar discussing some of the latest news regarding ongoing legislation that is most relevant to the rare disease patient community, as well as updates on its plans for next year. Making changes in the law that benefit the rare disease community can be a difficulty and drawn out process, but there are several ongoing developments that could have a significant impact.

Creating Hope Reauthorization Act

Caitlin Van Saint, who works with Representative G.K. Butterfield of North Carolina, discussed the latest on the Creating Hope Reauthorization Act. This bill aims to restore Rare Pediatric Disease designation and the Priority Review voucher programs at the FDA, which are set to expire soon and are currently only active thanks to a temporary authorization.

These programs help incentivize the development of treatments for rare diseases that primarily affect children. In addition, they can allow for drugs to be approved more quickly. Most Priority Review vouchers that have been issued are the direct result of Rare Pediatric Disease designation. Currently, the bill is held up in the Senate, and a decision must be made by December 11th of this year. Learn more about this here.


Melissa Goetz, who lives with familial chylomicronemia syndrome (FCS) and is active with the FCS Foundation, discussed the Helping Experts Accelerate Rare Treatments (HEART) Act, which was partially created as a result of frustrations that the foundation had while attempting to get a treatment approved. The bill calls for greater accountability of the FDA to Congress, more involvement of rare disease experts at hearings, more opportunities for patient feedback and input in the development process, and more. Currently, the HEART Act has been officially introduced in the House. To learn more about the HEART Act, click here.

Proposition 14 – California

Jacqueline Hantgan of Californians for Cures talked about Proposition 14, which is going to be on the ballot in the state of California in the upcoming election. Prop 14 serves as a follow up to Prop 71, which helped establish the California Institute for Regenerative Medicine (CIRM). This organization has a played a vital role in pioneering critical stem cell research that has helped benefit countless patients, including rare disease patients. Prop 14 extends critical funding for CIRM that will allow it to continue to have major impacts and fund critical research. If Prop 14 doesn’t pass, the future of CIRM would be very uncertain. It has been endorsed by more than 80 patient advocacy groups. Learn more about Prop 14 here.

Gabriella Miller Kids First Research Act 2.0

Ellyn Miller joined the webinar to talk about the Gabriella Miller Kids First Research Act 2.0. This bill will help permanently enact the funding and directives implemented by the first Gabriella Kids First Research Act, which will expire in a few years. Gabriella is Ellyn’s daughter, who died as a result of the rare pediatric cancer diffuse intrinsic pontine glioma (DIPG) and testified to Congress in support of the original law. The law will help continue funding for pediatric cancer and birth defect research for the indefinite future. 

State Advocacy

The final panelist was Swapna Kakani, who is an RDLA State Advocacy Fellow. She discussed why working for rare disease policy at the state level is just as important as on the federal, as a lot of medical standards are left up to the states. This means that care for rare disease patients is not consistent across the country. Some states have ballot measures that are relevant to the community and more legislation gets passed at the state level in general. 

The RDLA also announced that Rare Across America would be held from February 22nd to March 5th, 2021 and that Rare Disease Week would be held from July 19th-22nd.

Ready to get involved? Learn more here.

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