Fall 2017 Issue of Compass Now Available

The fall 2017 issue of Compass is now available online.

This issue covers Phase II of the SMA Industry Collaboration, as well as highlights and results from the Patient Focused Drug Development Meeting with the FDA.

Cure SMA Launches Phase II of the SMA Industry Collaboration

The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas and data.

In the first phase of the collaboration, the group focused on three major topics of importance to our community: engagement with the FDA and other regulatory agencies, increased education and awareness to parents, families and the medical community to reduce diagnostic delays, and increasing clinical trial site capacity. In phase II, we are actively implementing specific projects in each topic area.

Topic Group Projects:

  • Education – the education topic group will work with stakeholders, families, parents, caregivers and healthcare professionals to improve recognition of SMA, to shorten the time to diagnosis and avoid unnecessary delays in beginning treatment.
  • Clinical trials – the clinical trials topic group will identify and pursue opportunities to increase sites’ ability to conduct new trials and enroll and follow more patients, in addition to optimizing readiness throughout the US.
  • Regulatory – the regulatory topic group will continue to oversee the development of the Voice of the Patient (VoP) Report, in addition to conducting a benefit-risk survey throughout the SMA community to better understand how affected families/individuals make risk assessments when coupled with benefits of a treatment.

Highlights and Results from the Patient Focused Drug Development Meeting with FDA

On April 18, 2017, Cure SMA hosted an externally led Patient-Focused Drug Development (PFDD) meeting to share with the FDA and other stakeholders the perspectives of people living with SMA, its impact on their daily lives, and their expectations and priorities for current and future treatments.

Twenty panelists representing all types, ages and stages of SMA brought their voices and stories to depict the impact of SMA upon their lives. Each round of panelists was followed by polling questions and a period of facilitated discussion with participants at the meeting and from across the US and world, via our live-stream webcast.

In conclusion, the meeting emphasized the urgent need for increased awareness, early diagnosis and available treatments for SMA.

We are grateful to the patients and their representatives, and to the physicians and scientific experts who participated.

For More Information

Check out our news section for more information on the SMA Industry Collaboration and Patient Focused Drug Development Meeting.

To read past issues of Compass, visit our research publications page.

Questions on research? Send us an email at [email protected].

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