May 3, 2019
“The FDA is committed to providing drug developers with advice for specific diseases and conditions to stimulate development of new treatment options,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “Today we’ve issued the FDA’s first-ever draft guidance on developing stimulant drug treatments for attention deficit hyperactivity disorder, or ADHD. While many of the recommendations we’re providing in this draft guidance are already in practice – and we’ve approved products using an approach that is consistent with these recommendations – we want to make sure that future drug developers have up-to-date, clear information about our scientific expectations, such as clinical trial design, particularly in pediatric patients. This may help drug developers save time and resources, and ultimately, bring safe and effective new medicines to patients more efficiently. This type of disease-specific drug development guidance is an example of the strategies we’re implementing to modernize the agency’s new drug program.”
Today the U.S. Food and Drug Administration published the draft guidance for industry, Attention Deficit Hyperactivity Disorder: Developing Stimulant Drugs for Treatment, to provide general recommendations to companies developing stimulant drugs for treatment of ADHD in pediatric and adult patients. ADHD is a common disorder that begins in childhood. Symptoms include difficulty staying focused and paying attention, difficulty controlling behavior, and very high levels of activity. ADHD can impact a person’s family, social, academic and work life. Stimulant drugs (e.g., methylphenidate and amphetamine) are the most commonly prescribed medications for the treatment of ADHD.
The draft guidance explains that, because ADHD is a disorder that begins in childhood, a new drug application for any drug intended to treat ADHD should include data from adequate and well-controlled studies in pediatric patients. Additionally, the draft guidance provides information on clinical trial designs and describes the types of data that would be expected to demonstrate safety and effectiveness under different types of FDA approval pathways.
The recommendations in the draft guidance are intended to identify ways to streamline the development of stimulants for treatment of ADHD and promote innovation. The draft guidance also provides the following advice for methylphenidate and amphetamine 505(b)(2) development programs:
- Identifies ways in which drug companies can extrapolate findings from available safety and efficacy studies to reduce unnecessary burden and exposure of subjects to active drug or placebo;
- Recommends use of sparse pharmacokinetic (PK) sampling and PK modeling from adult data to simulate anticipated PK profiles in pediatric patients;
- Recommends specific criteria to determine when extrapolation is appropriate and when clinical trials are necessary to characterize the benefit-risk profile for specific patient populations (i.e., younger pediatric patients age 4 to 12 years, including 4 and 5-year-old preschoolers; adolescents 13 to 17 years of age; and adults).
The draft guidance also identifies safety monitoring parameters to track concentration-dependent adverse reactions of special interest in stimulant drug trials, such as increased blood pressure and heart rate, insomnia, appetite suppression and delayed growth.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.