On Wednesday July 1, 2020, the National Academies of Science, Engineering, and Medicine (NASEM) released a report titled “The Clinical Utility of Compounded Bioidentical Hormone Therapy: A Review of Safety, Effectiveness, and Use (2020).” First announced in September 2018, the report was commissioned and funded by the Food and Drug Administration (FDA), and the results align with FDA’s thinking that compounded hormone therapies pose a public health concern. Click here for a summary of NASEM’s recommendations: https://www.pccarx.com/pdf_files/PA_NASEM-cBHT-Recommendations_07-20.pdf

The report contains several deeply concerning recommendations, including that the use of compounded HRT be restricted and all reviewed hormone therapies be considered by FDA to be placed on the agency’s Difficult to Compound List. The report suggests this recommendation is included because compounds are not FDA-approved products and thus lack the population-based bioavailability data and labeling that FDA-approved products have. The recommendations also call for labeling requirements equivalent to FDA boxed warnings, which is counter to federal statute in the Drug Quality Security Act.

• PCCA is concerned that the report overstates the risks and minimizes the benefits of compounded therapies.

• We agree that more trials should be funded to assess the safety, efficacy and bioavailability of compounded hormone preparations, and that the entire scientific community will benefit.

• The committee seems to have ignored the many practitioners who testified about patient monitoring and titration of dosing which produces, in essence, patient-specific bioavailability data, simply because it was not done as part of a large-scale clinical trial. Individual patient bioavailability studies are unrealistic; rather, individual patients are evaluated by their doctors who monitor their lab values, symptoms, quality of life, and other parameters in assessing the value of the therapy.

• The report’s recommendations enter into the practice of medicine. The recommendations attempt to:

  1. Remove the ability of the physician to determine what is best for each individual patient

  2. Minimize the role of the physician in diagnosing, treating and following up with the patient

• The logic employed by NASEM would:

  1. Discourage patients from taking an active role in their own health care

  2. Lead to the end of off label prescribing or the use of any dosage of a drug that is not explicitly described in an approved product’s package insert

• In December 2018, NASEM invited PCCA to be a resource to the NASEM committee.

• Over 18 months, PCCA provided hundreds of literature evaluations to the committee, participated in all public committee meetings, provided testimony, conducted surveys for the committee and presented the results directly to committee staff. Because of PCCA’s involvement in the process, PCCA is specifically named in the final report.

• PCCA communicated these efforts through partnerships with NCPA and IACP (now known as the Alliance for Pharmacy Compounding), as well as presentations at PCCA seminars and webinars, and PCCA’s regular Public Affairs conference calls and newsletters.

• Requests to participate in surveys and provide data to the committees were sent to all PCCA members six separate times. We thank all those who participated in the process.

FDA has not indicated specifically how the agency will use the data in this report, but has provided the following comment: View Report

FDA is reviewing NASEM’s findings to inform our next steps regarding cBHT. 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.

• We are reaching out to medical experts who understand and value the role of compounded bioidentical hormones in individual patient care, so that informed spokespeople can be identified to articulate the numerous concerns with the report.

• We are reaching out to our association partners.

• We plan to develop a shared strategy and action plan.

• Contact PCCA Public Affairs at publicaffairs@pccarx.com to receive continuing updates and more details on next steps.

• Join us for the Public Affairs call on Wednesday, July 15 at 9 a.m. CT to discuss the NASEM report and other timely patient access issues.