Cure SMA has created a series of educational modules to address the topics, issues, and questions that are critical as the development of new drugs moves forward.
The organization says a new stage has been reached in the efforts for U.S. FDA-approved treatments for spinal muscular atrophy (SMA), and the SMA community should be committed to making sure families, friends, and supporters have all the necessary information to fully participate in this important time.
Cure SMA is referring mainly to news that AveXis’ gene therapy program received FDA breakthrough designation in July, and that Biogen and Ionis will submit nusinersen for FDA approval. Other ongoing clinical trials are also set to make a difference in SMA treatment.
- New Drug Applications (NDA) and Expanded Access Programs (EAPs): a module that covers what the community can expect as nusinersen and future treatments move up the FDA’s approval process and what provisions can be made for accessing the drug while this process is ongoing.
- Patient-focused Drug Development and Opportunities for Advocacy with the FDA: This module will address the FDA’s requirement to take the patient’s voice into account when considering new drugs for approval as part of the Prescription Drug User Fee Act, and explain to the community what opportunities this patient-focused initiative entails.
- The SMA Community and Industry Relationships: a module that will address how the community and Cure SMA are working with pharmaceutical companies that develop SMA drugs before, during, and after the approval process.
- Insurance and Coverage: This will cover the important topics of insurance for SMA patients and drugs, including how to ensure that, when approved, drugs are broadly covered for all SMA patients.
- FDA Advisory Committees: If the FDA is to approve the first treatment for a rare disease like SMA, most likely it will convene an Advisory Committee (or “ad comm”) as part of their review process. This module will discuss this “ad comm” process, how to participate, and what expectations the community can have at this point.
- Post-Approval Issues: This module covers topics and questions about what happens after drug approval; in this case, the first-ever for SMA. This debate will include the impact on the standard of care, what happens with subsequent drug approvals and combination therapies, and other topics.
According to a press release, all educational modules will include an interactive learning opportunity among webinars, booklets, handouts and other materials that the community can review and refer back to. All webinars will be recorded and posted online for those who won’t be able to participate in the live session.