On September 21, 2021, Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. The focus of this webinar was newborn screening in recognition of Newborn Screening Awareness Month, which is recognized during the month of September. The program featured several speakers that addressed a number of subjects related to advocacy and legislation about newborn screening.
About Newborn Screening
Newborn screening is a critical tool that can help save lives and improve outcomes for people that are born with rare genetic conditions. Identifying a rare genetic disease as soon as possible can allow for patients to receive treatment more quickly, and for some illnesses it can truly be a race against the clock before a patient’s life is in danger. Unfortunately, newborn screening varies considerably by state, with some states screening for more conditions than others. The Recommended Uniform Screening Panel (RUSP) provides federal guidelines for screenings, but states are not required to strictly follow them.
Paul Meyer from the Muscular Dystrophy Association provided an update on the Newborn Screening Saves Lives Reauthorization Act. Unfortunately, this bill is currently being bogged down in the Senate. In all likelihood, the legislation only has a realistic chance of passing if it gets tacked onto another, larger package. Paul identifies Senator Richard Burr (R-NC) as the most important person to pressure to provide support.
On the state level, work is continuing to get states aligned with RUSP guidelines. Dylan Simon of the EveryLife Foundation says that Ohio, Georgia, and Arizona have all recently passed RUSP alignment laws, and North Carolina is getting close. These laws aim to speed up the process by which new conditions from RUSP are added to state screening panels.
The MLD Foundation’s Dean Suhr presented guidelines for advocates hoping to convince the Advisory Committee on Heritable Disorders in Newborns and Children to add a condition to the RUSP guidelines. Dean’s main point about speaking with the committee is that their decisions have to be based on rigorous scientific evidence. Therefore, more emotional appeals may be less effective unless you can cite research alongside them.
Randi Clites from the Little Hercules Foundation also provided guidelines for how to interact with state-level newborn screening officials. It’s important to have a clear plan and to prioritize the most achievable and realistic goals. Use past examples of progress to set a timeline to get new diseases added to the newborn screening panel.
Ryan Dalbec of The Assistance Fund also provided some updates on the Advancing Access to Precision Medicine Act and related legislation. With the goal of improving access to various forms of genetic testing, the first version was introduced in 2018. In June 2021, the Ending the Diagnostic Odyssey Act was introduced, which included language from the original bill.
Check out the full webinar here.
