Update on SMA-FDA Interactions

The SMA drug pipeline has grown dramatically in just over a decade. Of the 17 programs in the pipeline, seven of these are in clinical trials, and two of those are in Phase 3 clinical trials.

Reaching and achieving FDA approval is a major hurdle in our path to a treatment and cure. And, consistent with our strategy of pursuing many different therapeutic avenues at once, we may face this hurdle many times.

When the FDA evaluates a drug for approval, they must weigh many different factors, including the quantity and quality of evidence of safety and effectiveness, potential benefits of a treatment versus the potential risks, the impact the treatment will have on the patient community, and more. This is why it is so crucial that we build strong relationships with the FDA and other regulatory bodies. We want to be sure the voice of our patient community is heard when the FDA considers these drugs for approval.

The Cure SMA FDA Engagement Initiative

Earlier this year, families and representatives from the Cure SMA community met with the FDA as part of our ongoing FDA Engagement Initiative. The goal of these most recent meetings was to further expand the FDA’s understanding of the priorities of our patient community in drug development.

During the meetings, patients voiced opinions on their assessment the most important/burdensome aspects of SMA on patients and their families, what would be meaningful change in SMA patients’ everyday lives, and the overall impact that an approved treatment would have for our patient community.


Every five years, Congress must renew Prescription Drug User Fee Act (PDUFA), which is the law that allows FDA to collect fees from drug manufacturers to fund the new drug approval process and other related activities. When PDUFA was reauthorized in 2012, known as PDUFA V, a stronger emphasis was placed on patient input in the drug development process. This resulted in the creation of the Patient-Focused Drug Development (PFDD) Initiative, to more systematically gather patients’ perspectives on their condition and available therapies to treat their condition.

As part of this commitment, FDA is holding at least 20 public meetings over the course of PDUFA V, each focused on a specific disease area, as well as offering participation in other community-wide meetings. In late 2014, Cure SMA submitted a formal request to be granted one of these meetings. If selected, a PFDD meeting for SMA would be one way to provide our patient community with an opportunity to impact the future of SMA drug development.

Past and Future Activities

Prior steps in our FDA Engagement Initiative have included:

  • The SO-SMART Workshop. During the 2014 Annual SMA Conference in Washington DC, the National Institutes of Health (NIH) hosted a forum to discuss the appropriate outcome measures for clinical trials, using SMA as an example population. The NIH asked Cure SMA to organize and lead the panel discussion on patient and caregiver perspectives on clinical trial design. In addition, the workshop participants included scientists, patients, caregivers/parents, and representatives from Cure SMA, the SMA Foundation, the Food and Drug Administration (FDA), NINDS, and private industry.
  • Focus groups with families and organizations within the SMA community to learn more about the patient point of view on drug development. A total of nearly 100 people participated, including individuals with SMA, parents, and clinical experts. The results showed the difficulties of living with SMA, from the long process of finding a diagnosis to the wide-ranging psychosocial effects of coping with SMA. The results also showed that even very small improvements in functional ability could have enormous benefit, while small losses in functional ability could translate to devastating effects on the family. Our thanks to our co-funders of the focus groups: SMA Foundation, Isis Pharmaceuticals, and Biogen.
  • Two comprehensive surveys: one of parents whose children have participated in a clinical trial, and one of parents whose children have not. The participator survey focused on motivations, hopes, and communication when parents have children with SMA participating in clinical trials. 53 parents of children with SMA who participated in a clinical trial in the last ten years completed the survey. A total of 222 parents whose children have never participated in a trial took the second survey. This survey looked at the reasons why parents of children with SMA have not involved their children in clinical trials. Our thanks to Parent Project Muscular Dystrophy (PPMD) for partnering with us on the two parent surveys.
  • A keynote session with Richard Moscicki, MD, at our 2014 Annual SMA Conference. Dr. Moscicki is Deputy Director for Science Operations at the FDA’s Center for Drug Evaluation and Research.

Cure SMA has also been working with other government and regulatory bodies through events like our Congressional Dinner and SMA Day on the Hill. Rep. Fred Upton, who chairs the Energy and Commerce Committee, was one of the congressional guests at our 2014 Hope on the Hill Congressional Dinner. Chairman Upton leads the 21st Century Cures Initiative, which was created to investigate how the government can help “accelerate the discovery, development, and delivery of promising new treatments and cures for patients.” At a hearing to discuss the release of the most recent discussion draft, Chairman Upton cited the SMA community—including sisters Brooke and Brielle Kennedy, from his home state of Michigan, who have SMA type II—as part of the inspiration for his support of 21st Century Cures.

2015 Annual SMA Conference

At the upcoming 2015 Annual SMA Conference, we will be showing more of the initial results from the above activities. In addition, future plans for our FDA Engagement Initiative will be announced. Also at the conference, our moderated panel on SMA clinical trials will include John Whyte, MD, MPH, Director of Professional Affairs and Stakeholder Engagement, Center for Drug Evaluation and Research, FDA, as well as panelists from Ohio State University, Nemours Children’s Hospital, Genzyme Corporation, The Hastings Center, Cytokinetics, Nationwide Children’s Hospital, Biogen, F. Hoffmann-La, Isis Pharmaceuticals, and Novartis.

Our thanks to the many families, organizations, and staff at the FDA that have been our partners in this effort. Their willingness to share their time, energy, and experiences is crucial to our success.

Do you like what you're reading?

Help make a difference in the lives of people affected by spinal muscular atrophy.

Leave a Comment

Your email address will not be published. Required fields are marked *

Scroll to Top