[9/26/2018] FDA is today announcing an agreement with the National Academies of Science, Engineering & Medicine (NASEM), and expanded agreements with the University of Maryland and Johns Hopkins University Centers for Regulatory Science and Innovation (CERSIs). These institutions will conduct research to help inform the public and the agency’s policies regarding compounded drugs.
NASEM will conduct two studies. The first will examine the clinical utility of treating patients with compounded bioidentical hormone replacement therapy (BHRT) products. Compounded BHRT products, such as progesterone and testosterone, are used at times instead of FDA-approved drug products for hormone replacement therapy. Some compounders market BHRT products as superior to FDA-approved drugs by making assertions that they are more natural or safer or better for patients than FDA-approved drug products. However, compounded BHRT products are not FDA-approved, which means these products have not undergone an FDA assessment of quality, safety, effectiveness and bioavailability (the extent and rate at which the drug enters the body). NASEM will provide a report evaluating the available scientific evidence relating to the safety and effectiveness of these products.
NASEM’s second study will examine available evidence of the safety and effectiveness of multi-ingredient compounded topical pain creams. These creams typically contain multiple active pharmaceutical ingredients (APIs). Often at least one of the ingredients is also found in an FDA-approved topical pain cream, such as lidocaine. However, the remaining ingredients may be active ingredients in drugs approved by FDA for non-topical administration and for non-pain-related indications. FDA is aware of adverse events, ranging from skin reactions to death, in patients who have applied compounded topical pain creams. NASEM will provide recommendations regarding the treatment of patients with compounded topical pain creams based on its research.
The agency also expanded two previously announced research collaborations with UMD and JHU CERSIs to gather and analyze important information for developing the list ofbulk drug substances, or APIs, that may be used in compounding by outsourcing facilities.
The UMD CERSI will expand its research to 225 bulk drug substances over the next three years. UMD CERSI’s work will focus on direct engagement with medical specialists through interviews, focus groups and surveys to better understand how compounded drugs that contain these substances are used in patient care.
The JHU CERSI completed its exploratory research and will initiate a one-year research project to evaluate available evidence regarding the safety and effectiveness of six bulk drug substances – inositol, methylcobalamin, glutathione, 2,3-dimercapto-1-propanesulfonic acid sodium, oxytocin and melatonin – used in compounded drugs for use in patients with autism spectrum disorder. The project will also include research into how compounded drug products containing these ingredients are used in clinical practice.
FDA believes the results of these research projects will provide important information that will inform the agency’s policies and increase public understanding regarding these compounded products.