On September 28, 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 updates on the VALID Act, State RUSP alignment, the Newborn Screening Saves Lives Reauthorization Act, and the Precision Medicine Answers for Kids Today Act.
Speakers for the program included:
- Dylan Simon, EveryLife Foundation for Rare Diseases
- Claire Ellis, EveryLife Foundation for Rare Diseases
- Debbie Jessup, Office of Rep. Lucille Roybal-Allard
- Ryan Dalbec, The Assistance Fund
Dylan provided an update on the VALID Act and its current progress in Congress. This legislation aims to update the regulation of in-vitro diagnostics (IVDs) and lab developed tests, which are considered a type of in-vitro diagnostic under current law. Currently, the regulation of these tests is the responsibility of the US Food and Drug Administration (FDA). The VALID Act would create a new type of IVD called in-vitro clinical tests (IVCTs) that would also be the responsibility of the FDA. However, their regulation would fall along new risk-based pathways. It has been introduced in both chambers.
Claire talked about updates related to state adherence to the Recommended Uniform Screening Panel (RUSP), which are federal guidelines for newborn screening. Mucopolysaccharidosis II (MPS II) was the latest disease that was added to RUSP, with Krabbe disease also being under consideration. The goal of RUSP alignment legislation is to speed up the time it takes for states to add new diseases to their screening panels. Legislative models being considered include auto inclusion (in which a state must add any newly recommended diseases within a given time frame) and vote for inclusion, in which a state commission or other body votes to decide if a new disease should be added.
Debbie Jessup, a staffer for rep. Lucille Roybal-Allard (D-CA), talked about the Newborn Screening Saves Lives Reauthorization Act. The latest version has passed the House but has been stalled in the Senate by Rand Paul, who has complained about provisions such as mandatory informed consent as violations of privacy. The bill would ideally be pegged onto another piece of larger “must pass” legislation.
Ryan gave an update on the Precision Medicine Answers for Kids Today Act, a bill that aims to help streamline and expand access to specialized forms of genetic testing, such as gene panels, whole exome sequencing, and whole genome sequencing. These tests can be vital for successfully diagnosing rare genetic disorders; delayed diagnosis is a major issue in many rare diseases. When patients are diagnosed earlier, they are able to begin treatment earlier as well. In a progressive genetic disorder, prompt intervention can be critical.
Unfortunately, this bill is still early in its life cycle and the main focus for advocates is to continue lobbying and developing a base of support in Congress.
Check out a recording of the full program here.
