FDA takes steps to advance the development of novel therapies for stimulant use disorders.

 FDA takes steps to advance the development of novel therapies for stimulant use disorders.

Today, the U.S. Food and Drug Administration has released a fresh draft guidance to aid sponsors in the advancement of remedies for stimulant use disorders. This guidance, titled "Stimulant Use Disorders: Developing Drugs for Treatment," will be the pioneering document to encompass the FDA's up-to-date perspective on the comprehensive development program and clinical trial structure for the creation of medications and biologics that bolster the treatment of moderate to severe cocaine use disorder, methamphetamine use disorder, and prescription stimulant use disorder.

Currently, there is a lack of FDA-approved medication for the treatment of stimulant use disorder. When completed, it is anticipated that the guidance will provide valuable support in the advancement of innovative therapies that are urgently required to bridge the gaps in treatment," stated Marta Sokolowska, Ph.D., deputy center director for Substance Use and Behavioral Health in the FDA's Center for Drug Evaluation and Research. "This guidance represents one of the measures outlined in the agency's Overdose Prevention Framework, aimed at promoting the appropriate prescription of stimulants and the development of evidence-based treatments for stimulant use disorder.

The draft guidance includes recommendations concerning clinical trial design for assessing treatments for stimulant use disorder. Designing clinical studies to evaluate the safety and effectiveness of these treatments presents unique challenges. These challenges range from selecting suitable study populations to determining the most appropriate clinical endpoints, all of which necessitate careful examination. However, there are opportunities for enhancing clinical trial design and developing person-centered trials, which could potentially improve the sensitivity in detecting treatment effects and link them to meaningful long-term outcomes.

Moreover, the draft guidance outlines essential considerations throughout the drug development process. These considerations encompass trial conduct, data collection, methods for assessing treatment response, subject safety, and new drug application requirements. For instance, the guidance integrates valuable insights gained from experience, highlighting approaches that are unlikely to succeed while reflecting current recommendations for treating stimulant use disorders and evaluating treatment response.

Stimulant use disorder refers to a range of symptoms associated with the consumption of stimulant drugs such as methamphetamine, cocaine, and amphetamines. However, it does not include caffeine or nicotine. A diagnosis of stimulant use disorder is established when a healthcare professional identifies a pattern of amphetamine-type substance use, cocaine use, or other stimulant use that results in significant impairment or distress. These indicators include an inability to reduce or control stimulant consumption, cravings for stimulant use, continued stimulant use despite negative consequences, and the need for increased amounts of stimulant to achieve the desired effect.

To promote the safe use and appropriate prescription of prescription stimulants, the agency has implemented measures. These measures include requiring sponsors to update and standardize prescribing information for medications used in the treatment of attention deficit/hyperactivity disorder (ADHD) and other disorders where stimulants are prescribed. Additionally, the FDA has awarded a grant to the National Academies of Sciences, Engineering, and Medicine to hold a workshop on the diagnosis and treatment of ADHD in adults. Furthermore, the FDA has funded multiple research projects to provide insights into the prevention of prescription stimulant misuse, addiction, and overdose.

The FDA continues to encourage the development of treatments for stimulant use disorder and innovative trial designs. In recent years, the agency has engaged in workshops and public meetings with patients, patient advocates, researchers, industry representatives, and other stakeholders. These interactions aim to enhance the FDA's understanding of the stimulant use disorder landscape, facilitate comprehension of the clinical context for drug review, and inform regulatory decision-making.

Comments on the draft guidance are welcomed within a 60-day period to ensure that the agency takes them into consideration.

 The FDA, an agency under the U.S. Department of Health and Human Services, safeguards public health by ensuring the safety, efficacy, and integrity of human and veterinary medications, vaccines, and other biological products for human use, as well as medical devices. It is also responsible for preserving the safety and security of our nation's food supply, cosmetics, dietary supplements, radiation-emitting electronic devices, and the regulations pertaining to tobacco products.