FDA Drafts Guidance for Developing Drugs to Treat ADHD

Regulatory NewsRegulatory News | 03 May 2019 |  By 

The US Food and Drug Administration (FDA) on Friday released draft guidance for sponsors developing stimulant drugs for the treatment of attention deficit hyperactivity disorder (ADHD) in pediatric and adult patients.

FDA explains how ADHD is a common neurobehavioral disorder with onset in childhood. Stimulant drugs (e.g., methylphenidate, amphetamine) are the most commonly prescribed medications for treatment of ADHD, the agency said.

The 7-page draft guidance, which is specifically for methylphenidate and amphetamine products, explains how in certain circumstances, additional clinical trials may not be necessary if a company is using the 505(b)(2) application pathway. The guidance also explains several issues that need to be considered when developing methylphenidate and amphetamine products via this pathway.

“FDA believes it is reasonable to rely on safety information from a listed drug to develop new methylphenidate or amphetamine product via the 505(b)(2) application pathway and, in certain cases, it may be reasonable to rely on the efficacy information. For instance, if a 505(b)(2) applicant can establish a bridge to the relied-upon listed drug by demonstrating either bioequivalence or comparative bioavailability of the proposed drug product with the listed drug, additional clinical trials may not be necessary to support approval of the 505(b)(2) application,” the draft says.

But for new molecular entities, FDA said the pharmacologic properties alone are not sufficient evidence of effectiveness and safety and that a demonstration of safety and effectiveness of stimulant drugs for the treatment of ADHD in 4- to 5-year-old pediatric patients would require at least one randomized, double-blind, placebo-controlled, parallel group study in this population.

“FDA encourages sponsors to discuss the details and timing of the 4- to 5-year-old pediatric patient portion of the drug development programs early, preferably before initiating studies in 6- to 12-year-old pediatric patients,” the draft says.

As far as safety assessments, the draft notes the need to establish, “Long-term safety, including assessment of growth using replicated and standardized measurements of weight (using a calibrated scale) and height (using a stadiometer), for at least 1 year in prepubertal patients.”

The draft guidance also features sections on clinical pharmacology, trial design and pregnancy.

“Women of reproductive potential are sometimes prescribed treatment for ADHD. Therefore, sponsors should consider inclusion of pregnant women in clinical trials when scientifically and ethically justified. If a sponsor excludes pregnant women from trial enrollment, the sponsor should provide a scientific justification,” the draft notes.  

And for postmarketing safety data collection, FDA said sponsors should use existing stimulant drug pregnancy registries (e.g., National Pregnancy Registry for Psychiatric Medications) or establish their own registries.

Attention Deficit Hyperactivity Disorder: Developing Stimulant Drugs for Treatment Guidance for Industry


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