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  1. Advancing Regulatory Science

Clinical Use of Drugs Including Bulk Drug Substances Nominated for Use in Compounding by Outsourcing Facilities and Expanded Information Initiative

CERSI Collaborators: Ashlee Mattingly, PharmD, BCPS, James Polli, PhD, Steve Hoag, PhD

FDA Collaborators: Gail Bormel, JD, Gabrielle Cosel, MSc, Charles J Ganley, MD, Elizabeth Hankla, PharmD

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 historical and current clinical use of drugs compounded using each substance.

Project Description and Goals

For this project, the University of Maryland CERSI will be considering how drugs compounded with bulk drug substances nominated for the 503B bulks list have been used historically, and how they are currently used in clinical practice. Researchers will review clinical practice guidelines, published literature, and other sources of information. Researchers also will conduct outreach to medical specialty groups and medical experts in the relevant field(s). When evaluating current and historical uses, researchers will be considering the conditions the substances are used to treat, the length of time the substances have been used, the patient population they have been used to treat, the extent of their use, and whether the substances are typically used to compound drugs that healthcare providers maintain in their offices in advance of identifying the patients who will receive the drugs.

The University of Maryland CERSI will prepare a report summarizing its findings, which will help inform FDA’s regulatory decision-making, specifically its decision about whether to include the substances on the list of bulk drug substances that outsourcing facilities can use in compounding.

Expanded Information Initiative

The University of Maryland CERSI will also engage in a related project to obtain additional information regarding nominations for the 503B bulks list. FDA has observed that in many cases nominators have generally provided FDA with limited information on topics such as what drug products are proposed to be compounded with the bulk drug substance, why starting from a bulk drug substance is necessary and why the compounded drug product is needed. Additional information from nominators is intended to help inform FDA’s assessments as to the clinical need for compounding by outsourcing facilities using nominated bulk drug substances, and generally to help inform agency decision-making, stakeholders, and the public.

Note: Please see the Johns Hopkins University-CERSI project on Bulk Drug Substances Used to Compound Drugs for Patients with Autism Spectrum Disorder (ASD).

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