The Food and Drug Administration (FDA) has released a new guidance on clinical trials for following neurological disorders: Alzheimer’s disease (AD), amyotrophic lateral sclerosis, Duchenne muscular dystrophy, pediatric seizures, and migraine. The purpose of this guideline is to provide sponsors with more specific directives so that to save time and resources.
Instead of publishing any detailed diagnostic criteria for early AD, the FDA claims that criteria should reflect existing understanding of AD pathophysiology and progression. The FDA suggested to concentrate on assessing AD potential treatments in the earliest stages of AD, while diagnostic criteria for early AD must be reliable enough. Moreover, the FDA said that evaluating co-primary endpoints such as functional and cognitive improvement is not clinically significant.
As for amyotrophic lateral sclerosis there has not been many changes apart from pointing out that studies should demonstrate “clinically meaningful benefits in symptoms, function, or survival”. However, to prove drug safety it is necessary to enroll enough patients with different amyotrophic lateral sclerosis variants.
Due to a lack of DMD treatments, the FDA has claimed that they may allow clinical studies on “less than usual” nonclinical testing “if justified”. They have also added that investigating clinical pharmacology “is likely not needed to support a new drug’s approval”.
There has been one major change for clinical trials on pediatric seizures. The FDA has said that it was demonstrated that dose-response results in adults and children are comparable. Therefore, there is no need to conduct trials with children to prove drug efficacy as it is possible to extrapolate the results to children over the age of four years.
With regards to migraine, it is no longer necessary to demonstrate potential drug efficacy on four co-primary endpoints — pain, nausea, photophobia, and phonophobia. Now it will be necessary to show efficacy using only two primary endpoints: pain reduction and effect on a patient’s “most bothersome symptom”.