Scott Gottlieb MD, Commissioner with the Food and Drug Administration (FDA) announced several new FDA guidelines for the development of drugs to treat neurologic diseases, noting that progress across different therapeutic areas has been uneven, including for complex neurological diseases, partly because the complexity of neurologic disease and that the brain, in many respects, is the last organ system for which our understanding of the biologic underpinnings of disease is still unknown. Gottlieb’s statement also notes the difficulty of treating neurologic diseases exists because symptoms and progression of neurologic diseases can also vary significantly with some disorders being present for years before becoming clinically significant, such as Alzheimer’s disease (AD). This delay in recognition of disease makes treatment challenging as significant function is lost before treatment can begin. All of this makes drug development more challenging.
In the statement, the FDA recognized the urgent need for new treatments and a need to become nimbler, more collaborative, and more patient-focused and is making efforts to expand access to safe and effective treatment options and promote innovation by modernizing multiple aspects of drug regulatory programs, including scientific and regulatory guidance for drug development is communicated.
These statements accompanied an announcement of reorganization of the medical review process at the FDA, which the organization says will move them away from siloed and discrete organizational units and instead create a team-based approach within the Center for Drug Evaluation and Research's (CDER) Office of New Drugs. The FDA states that the team-based approach will allow experts across different fields to share best practices and knowledge, integrating people from different disciplines – such as pharmacology and statistics - and across different stages of the life cycle of a product from the pre- and post-market phases who are all working toward a common public health goal.
In particular, the FDA is piloting a new, streamlined process for writing science-based, practical guidance documents more quickly that are concise and free of lengthy narratives without duplicating information available in other documents.
Five separate guidance documents for neurologic disorders were also released regarding drug development for Duchenne muscular dystrophy (DMD) and closely related conditions, migraine, epilepsy, AD and amyotrophic lateral sclerosis (ALS). These guidelines were developed with a greater level of patient input than before, a type of partnership the FDA plans to continue. As one example, the DMD guideline was preceded by a pioneering effort from Parent Project Muscular Dystrophy who submitted a proposed draft guidance in 2014, which the FDA used to inform the final version released today. Similarly, the ALS Association put together a comprehensive proposed draft guidance of their own, funded by the famous "ice bucket challenge."
The new guidance for drug development in AD focuses on supporting early intervention before the onset of dementia and other cognitive or functional symptoms by identifying patients with sensitive cognitive screening, imaging tests, or biomarkers.
The guidance on epilepsy focuses on significant advances in the development of treatments for partial-onset seizures in children, describing certain situations in which independent efficacy trials in pediatric patients are unnecessary and instead endorsing rigorous extrapolation of effectiveness from adult patients to more efficiently transfer gains in treatments in adults to pediatric patients.
Gottlieb also noted that these changes will improve the ability of the FDA to engage with sponsors, patients, and researchers to make the drug development process more scientifically modern and efficient while protecting patients.Next Story