5 Neurological Treatment Guidances Out

FDA has published five guidances as part of a broader focus on advancing treatments for neurological disorders that are not adequately addressed by available therapies. Commissioner Scott Gottlieb says (statement includes links to guidances) the documents “provide details on how researchers can best approach drug development for certain neurological conditions — Duchenne muscular dystrophy and closely related conditions, migraine, epilepsy, Alzheimer’s disease, and amyotrophic lateral sclerosis (ALS).

Also, Gottlieb says, the five documents are part of a new, streamlined process being piloted for writing science-based, practical guidance documents and issuing them more quickly. “Guidances in the pilot are intended to be concise and free of lengthy narratives that didn’t help advance the goals of providing clear scientific feedback,” he says. “These new guidances should not duplicate information already available in other documents.”

The guidances, he says, “are each a culmination of thoughtful scientific collaboration within the agency and incorporate input from patients, researchers, and advocates. We hope that providing up-to-date, clear information about our scientific expectations, such as clinical trial design and ways to measure effectiveness, will save companies time and ultimately bring effective new medicines to patients more efficiently.”

The Duchenne muscular dystrophy guidance was preceded by a pioneering effort from Parent Project Muscular Dystrophy that in 2014 submitted an independent proposed draft guidance that Gottlieb says provided important scientific and patient input and helped inform FDA development of its draft and final guidances.

The ALS Association provided a comprehensive proposed draft guidance on developing new treatments for ALS that gave the agency scientific advice and insight into the disease that helped advance its draft guidance.

Gottlieb says FDA has been working closely with patients and the scientific community to gain the knowledge to support intervention in very early Alzheimer’s disease in ways that have the potential to stop the disease before it causes clinical problems. The guidance describes innovative strategies for trials incorporating patients with Alzheimer’s who haven’t experienced any visible impairment in the form of cognitive or functional deficits, but who may be identified through use of sensitive cognitive screening, imaging tests, or biomarkers.

The migraine guidance finalizes a 2014 draft that describes innovative approaches to clinical trial design for assessing effectiveness in treating acute migraine.

And the epilepsy guidance provides what Gottlieb says is a significant advance in development of treatments for partial onset seizures in children, describing certain situations in which independent efficacy trials in pediatric patients are unnecessary and endorsing a rigorous approach based on extrapolation of effectiveness demonstrated in adult patients.

“The guidance documents we’ve issued today are an important step in facilitating efficient development of treatments for patients with serious neurological conditions,” Gottlieb concludes. “But they’re also just one part of our ongoing efforts to modernize the drug review process and foster beneficial new innovation. These changes, both outward facing and internal, will improve our ability to engage with sponsors, patients, and researchers, and adapt more quickly to an environment where the science is changing at a breathtaking speed across many disease areas.”