On November 15, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives that are relevant to the treatment and management of rare diseases under the law. In this program, the speakers provided their final updates on 2022 rare disease policy and advocacy efforts and next steps.
Panel speakers for this program included:
- Heather Ferguson, Lymphedema Advocacy Group
- Becky Abbott, National Foundation for Ectodermal Dysplasias
- Lauren Stanford, Parent Project Muscular Dystrophy
- Samantha Miller, Research!America
- Dylan Simon, EveryLife Foundation for Rare Disease
Heather provided an update on the Lymphedema Treatment Act, a proposed bill that would improve insurance coverage for the prescribed medical garments that are needed to manage the disease. This legislation has been 12 years in the making and the bill passed out of committee this summer. The bill was put on the House suspension calendar on November 11th. There are 29 bills on the calendar at the moment, so the next step for the bill is a full House vote.
Becky spoke about the Ensuring Lasting Smiles Act (ELSA), which aims to provide new insurance coverage requirements surrounding specific congenital anomalies, such as:
- Oral defects, such as cleft palate, enamel hypoplasia, and hypodontia
- Hearing defects (microtia)
- Skeletal defects, such as craniosynostosis
- Vision defects, such as aphakia or congenital cataracts
About four percent of children have congenital anomalies, including rare disorders that sometimes cause them. ELSA intends to cover all treatments for these anomalies that are considered medically necessary. This bill passed the House last spring and awaits action in the Senate. The goal is that it will be included in the End-of-Year package or in the Senate H.E.L.P. committee.
Lauren spoke about the BENEFIT Act and next steps for the bill. This is another piece of legislation that has been in the works for a while. The bill aims to require the FDA to incorporate the views of patients (‘patient experience data’) into its decisions, such as drug approvals, as well as require the agency to explain how it does so. There’s a chance it could get included in the EOY package. Otherwise, it will get reintroduced next year.
Samantha gave an overview of FY23 appropriations. The current continuing resolution will fund the government until December 16. The hope for advocates is that the omnibus bill can pass with the current Congress instead of another continuing resolution. Increased funding for research agencies is a top priority, as well as avoiding any future cuts to budgets.
Dylan gave the latest news about the STAT Act and PDUFA. While the PDUFA reauthorization was approved without any direct changes, it did include a commitment letter from the FDA. Some of these commitments included the establishment of the Rare Disease Endpoint Advancement Pilot Program, which intends to assist in the creation of therapy endpoints for drug development.
To learn more information and view the full webinar, click here.
