By allowing compounding pharmacies to produce certain unapproved peptides, the Department of Health and Human Services could, in the short term, put patients in harm’s way. Longer term, the policy shift threatens the industry that brings new medicines to market.
Last week, the Department of Health and Human Services took the bold step of allowing the compounding of six unapproved peptide products. There are several reasons why this is a bad idea.
As a pharmacist, I strongly support compounding as an essential part of pharmacy practice. It serves patients whose needs cannot be met by commercially available medicines, including those with ingredient allergies or specific dosage requirements and those affected by legitimate shortages. I have defended that role in multiple comment letters to the FDA. But expanding commercial access to experimental peptides without adequate clinical evidence could put patients at risk, undermine confidence in legitimate compounding, and weaken incentives for rigorous drug development.
Nevertheless, the FDA and HHS have in recent months been moving toward broader access to compounded experimental peptides.
In July, FDA’s Pharmacy Compounding Advisory Committee (PCAC) recommended including six of seven peptides on the list of bulk substances permitted for traditional pharmacy compounding. On October 14, FDA submitted an interim peptide-access policy for White House review, and last week, the HHS released an interim enforcement discretion policy that, for all intents and purposes, allows the six unapproved peptides to be compounded and sold to consumers. Specifically, HHS said it does not intend to take enforcement action against state-licensed pharmacies, federal facilities, or licensed physicians compounding these peptides while FDA considers its final position.
But the PCAC panel that voted in favor of allowing production of these peptides was stacked with professionals working in the peptide field, as overseen by Health Secretary Robert F. Kennedy Jr., who has championed broader access to peptides. Meanwhile, the FDA’s scientific reviewers recommended against all seven peptides, raising substantive concerns.
For BPC-157, they found insufficient evidence to establish effectiveness for ulcerative colitis. For KPV, they found no human clinical data to support its proposed uses. They also raised concerns about the substances’ identity and purity and their potential to trigger immune reactions. HHS now cites additional ingredient-testing data, historical use, and human-exposure information to justify its decision, but those materials should be evaluated against the specific evidence gaps the FDA staff scientists identified.
Moreover, peptide medicines already have an established path to FDA approval. Published reviews identify multiple FDA-approved peptide-based drugs, demonstrating that this pathway can bring these treatments to patients. That makes a transparent explanation of departures from the agency scientists’ conclusions especially important.
Indeed, the risks of unapproved peptides are drawing bipartisan scrutiny. In an Oct. 14 letter, a group of senators from both sides of the aisle urged the FDA to strengthen enforcement against online vendors selling unapproved drugs, including Eli Lilly’s next-gen obesity candidate retatrutide, under “research use only” labels, citing risks of contamination, mislabeling, and misuse.
Developing medicines, including peptide drugs, requires capital long before a product generates revenue. Investors finance studies that may fail, and developers must establish whether a treatment’s benefits justify its risks. Studies that find a treatment ineffective or reveal unacceptable risks protect patients even when no marketable medicine results.
Compounding rules should not create a competing route that makes those investments less worthwhile. If businesses can sell experimental peptides broadly without conducting comparable research, the incentive to fund that research could weaken. A policy that rewards early commercialization over evidence generation risks directing investment away from rigorous development and toward marketing. Patients could bear the consequences through fewer well-studied treatment options and persistent uncertainty.
The risk extends beyond the peptides under discussion. If HHS and FDA apply similar policies to other drug classes or disease areas because available treatments are inadequate, developers could face the same uncertainty across a much wider range of research programs. A company considering clinical development must assess whether it can sustain years of testing while competing with sellers already collecting revenue without the same obligations.
Widespread commercial access outside a trial could also make recruitment harder. Patients may choose a marketed product instead of participating in a study with eligibility rules, monitoring and a control group. That could further delay the evidence that responsible prescribing requires.
It is important that patients with serious or life-threatening illnesses not face avoidable delays in accessing investigational medicines. Clinical trials can provide eligible patients access to investigational treatments. When trial participation isn’t possible, and no satisfactory alternative exists, the FDA’s expanded access program, often called compassionate use, may permit treatment before approval. FDA, manufacturers, and research centers should make these pathways easier to navigate while protecting patients and preserving clinical research.
A policy that rewards early commercialization over evidence generation risks directing investment away from rigorous development.
Beyond the risks to patients and to the established research paradigm, broader compounding access also creates opportunities for abuse. Traditional compounding pharmacies are regulated, primarily by states, but their products do not undergo FDA premarket approval. Although qualifying compounded drug products are exempt from federal current good manufacturing practice requirements, HHS policy requires the original bulk-ingredient manufacturer to comply. Compounders must also test ingredients, obtain patient-specific prescriptions, and report serious adverse events and product quality issues to the FDA and relevant states.
HHS argues that this approach offers patients a more controlled alternative to illicit online sellers. Licensed pharmacies may offer better quality control, but quality testing and adverse-event reporting cannot replace clinical studies that establish whether a peptide works and whether its benefits outweigh its risks. In addition, permission to compound a substance should not be marketed as FDA approval or proof of safety and effectiveness.
HHS and FDA should explain how expanded access will preserve incentives for clinical research. Regulators should verify compliance with the policy’s sourcing, testing, and reporting conditions and ensure patients receive clear disclosures about the products’ unapproved status. FDA and state regulators must enforce the distinction between legitimate compounding and businesses using the classification to evade manufacturing or approval requirements. That includes action against exaggerated therapeutic claims and operations that disguise routine commercial production as individualized care.
I have consistently argued that consumer demand and business preference cannot substitute for clinical need. The same principle applies here. Compounding should remain available for patients who need it while public policy supports the research that can turn promising molecules into treatments patients can trust.
