FDA Uses New Pilot Plan for the Development of Guidance on Neurological Treatments

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FDA Commissioner Scott Gottlieb issued a statement that FDA will be modernizing regulatory programs in order to advance novel treatments.

In a Feb. 15, 2018 statement, FDA Commissioner Scott Gottlieb, MD, announced agency efforts to modernize its drug regulatory programs as part of its commitment to help advance novel therapies. Specifically, the agency plans to enhance its communication of scientific and regulatory guidance regarding drug development.

According to Gottlieb, the agency will be reorganizing from a “siloed structure” to a team-based approach in the Center for Drug Evaluation and Research’s Office of New Drugs. Experts from different fields and disciplines will share knowledge on diseases to better integrate expertise through the different stages of drug development and commercialization.

As part of this effort, the agency will be creating a new process for writing science-based guidance documents. A pilot program will develop concise documents that will not duplicate information already available. Five guidance documents have been developed using this new approach. Gottlieb stated that this new modernization of guidance development includes an enhanced focus on patient input. 

“While the agency engages with patients on many fronts, this set of guidances showcases unique involvement from patient communities in collaborating with the FDA to make sure patient voices are heard. We will continue this type of partnership,” Gottlieb said.


The new guidance documents developed under this new approach include information on the development of drugs for the treatment of neurological diseases such as Duchenne muscular dystrophy, amyotrophic lateral sclerosis (ALS), Alzheimer’s, migraines, and partial onset seizures.

“The guidance documents we’ve issued today are an important step in facilitating efficient development of treatments for patients with serious neurological conditions. 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 quickly to an environment where the science is changing at a breathtaking speed across many disease areas,” Gottlieb stated.  

Source: FDA