Cure SMA Pursues More Education and Better Clinical Trial Access, Capacity

Cure SMA announced the start of Phase 2 of its SMA Industry Collaboration, aiming to advance the development of specific goals.

“The SMA Industry Collaboration is a multifaceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data,” according to a news release from Cure SMA.

“The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that are critical to advancing drug development in SMA and will benefit the broader SMA community,” the release states. “The members of the SMA Industry Collaboration are Astellas, AveXis, Biogen, Genentech/Roche, Cytokinetics, and Ionis.”

This collaboration has two parts. The first part consisted of discussing three major topics, such as the engagement with the U.S. Food and Drug Administration (FDA) and other regulatory agencies, educating physicians to improve patient diagnosis, and increasing clinical trial site capacity. The first part of the collaboration culminated in a Patient-Focused Drug Development Meeting with the FDA to address these matters.

“Your voice, which we heard today loud and clear and in great detail, helps FDA as we perform our public health mission and as we evaluate and approve new drug applications,” Jonathan Goldsmith, MD, associate director for rare diseases in the Office of New Drugs at the FDA’s Center for Drug Evaluation and Research, said in a press release released soon after the April 2017 meeting.

The second part (Phase 2) of the SMA Industry Collaboration aims to produce a Voice of the Patient Report based on the Patient Focused Drug Development Meeting and plan new forms of engagement with the FDA. It will also focus the development of groups looking at education and clinical trials.

The education group will look to increase SMA awareness among physicians, families and caregivers, and promote a reduction in diagnosis delays.

“Diagnostic delays can put significant financial, logistical and emotional strain on families,” reads the release by Cure SMA. “More importantly, they can also reduce the opportunity for early treatment. The education topic group will work with stakeholders to improve recognition of SMA, to shorten the time to diagnosis and avoid unnecessary delays in beginning treatment.”

The group dedicated to clinical trials will fight for increased site capacity and availability across the country.

“The goal is to improve our community’s access to clinical trial sites and increase capacity,” Cure SMA said. “In this phase, the topic group will focus both on assessing and improving existing clinical trial networks, as well as reaching out to potential new SMA clinical trial sites.”