On August 2nd, members of the spinal muscular atrophy community kicked off SMA Awareness Month by meeting with a dozen Senate and House congressional offices to advocate for issues that impact our community. The teams conducting these visits included families affected by SMA, and Cure SMA staff.

These Senators and Representatives serve on committees important to our community’s goals, including the House Energy & Commerce Committee; Senate Health, Education, Labor and Pensions (HELP) Committee; the House and Senate Appropriations Committees; and the Labor, Health and Human Services, Education and Related Agencies Appropriations Subcommittee.

One primary goal of these meetings was to advocate for necessary federal funding for newborn screening. Though implementation of newborn screening happens at the state level, the federal government also plays a key role. This role includes funding newborn screening programs, and overseeing the Recommended Uniform Screening Panel (RUSP), which advises states on which conditions should be screened for. Currently, SMA is undergoing a nine month evidence review process for inclusion on the RUSP.

In June, the President proposed a budget that would eliminate all the funding for the Health Resources and Services Administration (HRSA) Heritable Disorders Program, which supports the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). The ACHDNC is the group that reviews conditions for inclusion on the RUSP.

The budget would also reduce funding for the Centers for Disease Control and Prevention’s (CDC) program that supports states in their efforts to implement newborn screening, particularly initiatives that focus on the roll-out of new conditions as they are added to the RUSP.

The House is currently considering a budget that would reject these cuts, and maintain funding for these vital programs. In our visits with House offices, we thanked them for maintaining this funding, and encouraged them to ensure that this funding is protected through the entire FY18 budget process. In our visits with Senate offices, we encouraged them to follow the House’s lead in maintaining this funding. We also updated both House and Senate offices on SMA’s current progress in the RUSP process.

During the visits, we also advocated for increased investment in the biomedical research programs at the National Institutes of Health (NIH), which provide vital support for the research and development of new SMA therapies and have made significant contributions to the implementation of newborn screening pilot studies for SMA.

Finally, we used these visits to advocate for continued protection of Medicaid and Medicare , and for robust reimbursement of orphan drugs, including Spinraza. We used information collected at April’s Patient-Focused Drug Development Meeting with the FDA to educate the offices on the impact of SMA, the treatments and therapies that many SMA families rely on, and the importance of protection for these programs.

On August 3rd, Cure SMA staff attended the ACHDNC meeting. During the meeting, the committee provided an update on the evidence review process for SMA, as well as a timeline for their final decision, which is set to be announced at their February 2018 meeting. Cure SMA staff also provided public comment in support of the SMA nomination.

Thank You

Thank you to Kelly and Jeff Eakin; Brandi and Michael Akins; and Jonathan, Kristen and Max Lasko for participating in these visits, and for their generosity with their time and their stories.

Our thanks to the Senate and House offices for taking the time to meet with our community, and for the support they have already offered on these important issues.