Report calls for limits on compounded bioidentical hormone therapy

Regulatory NewsRegulatory News | 02 July 2020 |  By 

Evidence does not support the clinical utility of compounded bioidentical hormone therapies and their use should be limited to patients who cannot use products approved by the US Food and Drug Administration (FDA), according to a consensus study report from the National Academies of Science, Engineering and Medicine (NASEM).
FDA requested the NASEM review due to increasing popularity of compounded bioidentical hormone therapies (cBHT) combined with concerns over healthcare providers’ and patients’ understanding of the uncertain benefits and potential risks associated with the products, according to a statement from the agency.
FDA officials are reviewing the NASEM report. “We will continue to work with compounders, regulators, healthcare professionals and patients as we develop policies that ensure continued access to compounded drugs for patients who need them, while also protecting patients from the risks of receiving a compounded drug when an FDA-approved product is appropriate for their medical care,” according to the statement.
Unlike FDA-approved bioidentical hormone therapies (BHTs), cBHT products are not regulated by the FDA, nor are they subject to the same efficacy, quality and safety testing. Typically used to address age-related symptoms of menopause or male hypogonadism, they are created on a custom basis for each prescription and can be supplied in a variety of vehicles. Suppliers often market their products as natural and superior, although the evidence did not support those claims, according to the NASEM report.
NASEM’s analysis noted that few well-designed clinical trials of cBHT have been conducted; trials that have been conducted included severe methodological limitations. Much of the data supporting the use of cBHT was derived from patient testimonials and anecdotal claims, according to the report.
FDA has been concerned about compounded drugs and cBHTs in particular for several years, commissioning two reports from NASEM as well as establishing research collaborations with Johns Hopkins University and the University of Maryland in September 2018.
Inspections of compounding firms have turned up large numbers of adverse events that were never reported to the FDA, according to an agency statement from September 2019. Officials at the agency’s Center for Drug Evaluation and Research highlighted a particularly significant case in a viewpoint published in JAMA Internal Medicine.
“During a routine inspection unrelated to compounded drugs, [FDA] investigators found records of 4202 adverse events, none of which had ever been reported to the agency,” first author Julie Dohm and colleagues wrote. “The reports, kept in a company-generated data file, concerned the possible association of compounded hormone pellets marketed by BioTE Medical (BioTE) with cancers (including endometrial and prostate cancers), strokes, heart attacks, deep vein thrombosis, cellulitis, and pellet extrusions, as well as adverse events known to be caused by supratherapeutic hormone concentrations.”
“Consumers may be lured by the prospect of personalized medicine and avoiding perceived ‘one-size-fits-all’ treatments,” said Donald Mattison, MD, chief medical officer of Risk Sciences International and chair of the committee that wrote the report. “However, cBHT preparations, across doses and dosage forms, are not supported by a sufficient body of evidence to inform providers and patients of potential benefits and harms. Providers have an obligation to educate patients about the potential risks of using cBHTs and present a clear rationale if they do need to prescribe them.”
NASEM review


© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy