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  1. Advancing Regulatory Science

Bulk Drug Substances Used to Compound Drugs for Patients with Autism Spectrum Disorder (ASD)

CERSI Collaborators: Karen A. Robinson, MSc, PhD (Principal Investigator), G. Caleb Alexander, MD, MS, Vi Gilmore, PharmD, BCPS, Christine Ladd-Acosta, PhD, Roma Vasa, MD, Heather Volk, PhD

FDA Collaborators: Julie Dohm JD, PhD, Ruey Ju, PharmD, JD, Sara Rothman, MPH Gabrielle Cosel, MSc, Charles J Ganley, MD

Project Start Date: June 1, 2018

Regulatory Science Challenge

Compounding is generally the process by which drug ingredients are combined, mixed, or altered to create a medication tailored to the needs of an individual patient. Compounding may be practiced by a licensed pharmacist, a licensed physician, or, in the case of another type of compounder called an outsourcing facility, a person under direct supervision of a licensed pharmacist.

Drugs compounded by outsourcing facilities are exempt from certain sections of the Federal Food, Drug, and Cosmetic Act (FD&C Act or Act), including requirements for FDA approval of a drug and labeling with adequate directions for use, if they meet certain conditions described under section 503B of the Act. One of these conditions is that if the outsourcing facility compounds drugs using bulk drug substances (active pharmaceutical ingredients), the substances must be used to compound drugs in shortage or appear on a list established by FDA identifying bulk drug substances for which there is a clinical need (503B bulks list).

To determine whether to include substances nominated by the public on the 503B bulks list, FDA intends to evaluate several factors, including, in certain cases, the safety, any evidence of effectiveness, and the historical and current clinical use of drugs compounded using each substance.

Project Description and Goals

Several of the bulk drug substances have been nominated for use in compounding drugs for patients with autism spectrum disorder (ASD). For this project, the Johns Hopkins University CERSI researchers will systematically study available safety and effectiveness information on certain bulk drug substances nominated or identified for use in compounding drugs for patients with ASD. FDA intends to consider this information as it evaluates these substances for use in compounding.

Note: Please see the University of Maryland CERSI project on Clinical Use of Drugs Including Bulk Drug Substances Nominated for Use in Compounding by Outsourcing Facilities.