1. Before the Study even began, FDA provided one-sided information to NASEM on compounded medications, particularly hormone medications, that profoundly shaped the NASEM Committee’s position on cBHRT;
2. As the Study began to materialize, FDA played a role in determining who should serve on the NASEM Committee and who NASEM should rely on as cBHRT subject-matter “experts”;
   1. [None of these professionals on the NASEM Committee treated women in menopause with cBHRT as part of their professional endeavors]
   2. [there are specific conflicts of interest in those advising the Board]
3. During the course of the Study, FDA and the NASEM Committee consistently collaborated over substantive aspects of the Study; and
4. Finally, FDA was given the opportunity to review and comment on the Report before it was published—a Report that was supposed to be advice independent from FDA.

1. The NASEM study committee was populated by esteemed healthcare professionals, but there was not a pharmacist with patient-facing experience, much less a pharmacy compounder, in their number. Neither was there a physician with substantive experience in bioidentical hormone therapy. Likewise, the peer reviewers for the study included few compounders – but did include one former FDA employee (and current FDA contractor) who is a long-time, well-known opponent of pharmacy compounding.
2. Although compounded drugs are exempt from the new drug approval process because they are prepared to meet individual patient needs, the foundation of NASEM’s analysis seems to focus on the absence of new-drug-caliber studies of compounded meds. The report makes an illogical leap by effectively deeming cBHT unsafe unless it can mirror drug manufacturing in terms of safety and effectiveness data, labeling, AE reporting, pharmacokinetic data, and scale of clinical trials.
3. NASEM said it based its recommendations in large part on a review of literature, but identifies only 13 studies as having, in the judgment of the committee, suitable “rigor and relevance” – these, out of literally hundreds of studies out there, not to mention abundant patient outcomes data that could and should have been considered and weighed.
4. The report calls for restricting the use of cBHT to patients with allergies to FDA-approved drugs, despite the fact that many FDA-approved therapies may not meet the dosing needs of patients – a point not addressed by the report’s recommendations.
5. The recommendations seem to suggest that a prescriber’s medical judgment and a patient’s preferences should play no role in determining a proper course of treatment – a stunning assertion that is applied to no other aspect of medicine or pharmacy care. Women’s needs are much more complex than the cookie-cutter approach offered by current FDA-approved drugs. Individualized therapy is the result of a balanced approach between prescriber, patient, and pharmacist assessing each patient’s unique needs.
6. The NASEM study seems to suggest that states that do not adopt USP <795> and <797> chapters in whole, without question or amendment, are somehow under-regulating compounders, yet it provides no evidence of patient harm that has resulted in states that adhere to different standards.
7. The report cites concerns over bioavailability. However, many compounders or prescribers perform saliva or serum testing to confirm cBHT is appropriate therapy. Yet how many prescribers routinely check levels of FDA-approved products?
8. The report sends mixed messages. Individualized therapy is a part of the FDA’s newly established goal of creating personalized therapies to target each patient. Factors such as age, weight, genetics, past medical history all contribute to appropriate treatment and dosage selection. Yet these NASEM recommendations seem to assert that FDA-approved drugs are always superior to compounded therapies.

• “The Panel Put Policy-Making Before Patient Need: An Independent Analysis of the FDA-Commissioned NASEM Report…”

Introduction

In the United States, the Food and Drug Administration (“FDA”) oversees the approval, marketing, and labeling of prescription drugs. However, it is the right and the duty of the physician to prescribe what he or she thinks is best for the patient. In its Code of Medical Ethics, the American Medical Association (AMA) states:

“The practice of medicine, and its embodiment in the clinical encounter between a patient and a physician, is fundamentally a moral activity that arises from the imperative to care for patients and to alleviate suffering. The relationship between a patient and a physician is based on trust, which gives rise to physicians’ ethical responsibility to place patients’ welfare above the physicians’ own self-interest or obligations to others, to use sound medical judgment on patients’ behalf, and to advocate for their patients’ welfare.”

Consistent with this principle, U.S. law prohibits the invasion of the FDA into the purview of the physician, as the FDA is not purposed to regulate the practice of medicine or to otherwise interfere with a doctor’s care of a patient. It is well-recognized that flexibility and respect for physicians’ autonomy in caring for patients is “crucial for (doctors] to be able to judge what is best for each individual patient and to provide effective medical care of the highest quality.” Indeed, as recently as November 2020, the AMA “confirm[ed] its strong support for the autonomous clinical decision-making authority of a physician,” and reaffirmed that it “strongly opposes the FDA’s intrusion into the practice of medicine by making decisions for individual care.”

Despite its mandate to respect prescribers’ autonomy, the FDA recently commissioned a report setting forth recommendations that constitute an intrusion into a physician’s ability to practice medicine and to meet his or her patient’s needs. On July 1, 2020, the National Academies of Sciences, Engineering, and Medicine (“NASEM”) published The Clinical Utility of Compounded Bioidentical Hormone Therapy: A Review of Safety, Efficacy, and Use (the “NASEM Report” or the “Report”), wherein NASEM received a directive from the FDA to “assess the clinical utility of treating patients with compounded bioidentical hormone therapy (“CBHT”). Specifically, the FDA commissioned

Conclusion

As detailed throughout this white paper, cBHT is used for a variety of medically necessary reasons by patients across the U.S. Continued access to CBHT is critical to patient health and well-being.

While there are opportunities for improving BHT compounding practices, such as establishing clearer guidelines for patient monitoring, the NASEM Report does not provide an accurate or complete lens through which these opportunities can be evaluated. Rather, the NASEM Report offers a biased perspective of CBHT, promulgated by a Committee with practically no patient-facing experience with hormone therapy and guided by a regulatory body with a clear interest in either bringing compounding under its purview or effectively prohibiting the practice of CBHT altogether.

Moreover, as a practical matter, it is unlikely that the concerns of the FDA and those in opposition to CBHT will be addressed through the implementation of the Committee’s recommendations. Most physicians and pharmacists-like any other professional-understand that it is in their own best interest to offer the best possible care to patients as a practical matter. To do otherwise would result in both financial and reputational loss, as it would be in violation of their professional oaths and could have significant ramifications (e.g., the loss of their professional licensure). Laws at the state and federal level mitigate the risk of improper or dangerous business practices. State boards of medicine and pharmacy establish requirements and guidelines for safely treating patients and compounding treatments, respectively. While it may be tempting to seek to regulate every aspect of the health care continuum, it is important to recognize that there will always be bad actors. Try as it might, the FDA cannot prevent individuals from committing a crime or unintentionally causing harm. However, if the Committee’s recommendations are implemented, the FDA will adversely affect the safe practice of medicine and the well-being of CBHT patients nationwide.

In sum, NASEM’s recommendations seek to severely restrict-or eliminate entirely-the ability of the physician to prescribe cBHT to his or her patients. In turn, the restriction on the physician’s ability to prescribe cBHT would limit the patient’s choices for treatment. When a patient’s choices are limited, so too is his or her path to wellness.

We therefore recommend that patient need must take priority over policy-making.

We recommend that FDA not rely on or consider the NASEM Report and instead work to ensure that prescribers, patients, and pharmacists may continue to collaborate on treatment options that work best for the individual patient in a manner that is safe and effective. More specifically, we recommend that:

1. FDA should work collaboratively with medical and pharmacy compounding industries to address concerns around labeling and adverse event reporting for compounded therapies.
   • Developing a system in which patients are consistently provided with information specific to their prescription medication should be encouraged, including developing product monographs for ingredients used in compounded treatments. The system should ensure that patients receive up-to-date, accurate, and consistent information about their prescription medications.
   • The current adverse event reporting system and process should be evaluated for improvements to ensure compounded therapies are being addressed. Improvements to this system, if necessary, should broadly address concerns with CBHT and other compounded therapies.
2. FDA should sponsor or encourage sponsorship of educational campaigns directed to medical providers, pharmacists and/or patients focusing on the benefits and risks of CBHT compared to commercially available hormone therapies. Such educational programs should provide fair-balanced benefit and risk information in a manner that is respectful of the doctor-patient relationship.
3. FDA should develop and/or improve upon systems to encourage adherence to Code of Ethics for prescribers and pharmacists. Appropriate use of CBHT should be encouraged. Patients are not served if cBHT is not being appropriately used (i.e., overuse and/or underuse).
4. Compounding pharmacies should be expected to comply with USP Standards related to the preparation of compounded prescriptions. State Boards of Pharmacy should assess their current requirements and monitoring and enforcement efforts around compliance with USP Standards.