Cure SMA Advocacy Activities for the 2017 Year-to-Date

The first half of 2017 has been an active six months for Cure SMA and SMA advocates. Cure SMA and members of our community have been working with stakeholders groups across multiple settings to ensure our patients’ voices are heard. Below is a summary of the key issues and audiences we’ve targeted in our engagement so far this year.

Federal Funding for Newborn Screening and Biomedical Research

Since January, Cure SMA has been communicating directly with Members of Congress about the funding levels needed to support key federal government programs that make a difference for the SMA community, such as the newborn screening (NBS) programs at the Health Resources and Services Administration (HRSA) and the Centers for Disease Control and Prevention (CDC), which provide recommendations and assistance to states on adoption of new conditions for NBS. Cure SMA also has called 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 NBS pilot studies for SMA. In support of these important federal investments, we have submitted testimonies and other correspondence to Congress and have continually engaged with Congressional offices by way of meetings, letters, and emails. (Click to read our House testimony and Senate testimony.) Additionally, we have welcomed the active participation of our community members who have reached out to these offices to express the significance of these programs and urge support.

[Picture: Senator Chris Van Hollen (D-MD) with Dr. Thomas Crawford, Professor of Neurology and Pediatrics and Co-Director, Muscular Dystrophy Association Clinic at Johns Hopkins]

“Repeal and Replace” of the Patient Protection and Affordable Care Act (ACA)

Cure SMA continues to monitor and provide updates to our community on Congressional efforts to pass legislation that would repeal key parts of the ACA. In May, the House of Representatives passed the American Health Care Act (AHCA), which could have an adverse impact on SMA patients’ access to health care services by allowing states to opt out of certain patient protections, such as preexisting condition safeguards, and also by reducing funding for Medicaid programs, which provides health care coverage to millions of low-income people and individuals with disabilities.

While the AHCA passed in April, Senate Republicans have decided they will not take up the bill, but instead will draft their own bill, which has not yet been released. As we actively await the Senate’s legislation, we are on the ready to alert our community on opportunities to engage so that the voices of SMA patients and families are heard throughout the consideration of health care reform legislation.

SMA Care and Treatment Coverage and Payment Policy (CPP) Project

Following FDA’s approval of SPINRAZA™ on December 23, 2016, Cure SMA sprang into 2017 launching the CPP Project, which has had two distinct but complementary tracks: outreach to private and public health insurers on behalf of our community; and outreach to our community about coverage and access. As part of our external outreach, we have engaged with public and private insurers, regulators, and other stakeholders, to advocate for prompt, comprehensive coverage for SMA treatments and care.

At the same time, we have been working to provide patients and their families with the tools and information they need to successfully navigate this often complex area of health insurance access, coverage, and care. While the project covers the full breadth of treatment and care for SMA, it is specifically focused on obtaining timely coverage and adequate access and reimbursement for new treatments as they are approved by the FDA.

To date, we have sent 125 letters of written testimony to governors and state Medicaid directors, regarding the importance of timely and broad coverage for Spinraza, as well as more than 25 similar letters to commercial payers. This written testimony is supplemented by in-person meetings and other activities as detailed in our most recent CPP update.

Patient Focused Drug Development (PFDD) Meeting with FDA

Cure SMA’s PFDD meeting – held in April 2017 just outside of Washington, D.C. near the FDA headquarters – was designed to provide the FDA with an overview of the impact SMA has on individuals and families, and of the expectations and priorities for current and future treatments. Twenty panelists, representing patients and caregivers of all types, ages, and stages of SMA, testified about their diagnostic and care journeys and highlighted the meaningfulness of the types of clinical gains that would have on their quality of life and the lives of their loved ones. Each round of panelist testimony was followed by a period of facilitated discussion and polling questions. More than 400 individuals from the SMA community, FDA, and other stakeholders participated, including in-person and webcast audiences. Cure SMA is currently preparing a Voice of the Patient Report summarizing key themes from the very successful meeting.

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