Comment from Briah Baker

Thank you for the opportunity to comment on the FDA’s new public docket for reviewing bulk drug substances used in compounding under Section 503A. This is an important step toward improving how the agency collects and evaluates information about the active ingredients that may be allowed in compounded medications, which may be essential for patients who need customized treatment. Compounded drugs are essential in the U.S. healthcare system, and we are seeing a major increase in compounded medications as the need to meet GLP-1 demands increases. However, compounded medications are exempt from many federal quality and safety standards, including premarket approval requirements, which highlights a critical need for the FDA to ensure active ingredients that are being used in compounded drugs are supported by scientific evidence. This docket allows the FDA to collect all necessary information to make informed decisions that maintain regulatory compliance while ensuring patient access remains. I strongly support the FDA’s requirement for supporting information for each substance outlined in the docket. As the FDA noted, many substances are nominated for the 503A bulks list while lacking essential data that makes evaluation nearly impossible. Approximately 390 substances were submitted without sufficient evidence to determine their safety, historical use, r whether they are appropriate for compounding. Without these data points, it is not possible for the FDA to responsibly determine whether the substance can be permitted for use. This is especially true when considering the risks associated without compounded drugs which have been implicated in multiple safety incidents, such as the 2012 fungal meningitis outbreak linked to contaminated compounded steroid injections. I also support the decision to encourage nominators to submit this information using an editable Excel file which will allow the FDA’s review and determination to be streamlined and will reduce duplicative nominations. This standardized approach will improve consistency, transparency, and efficiency. Finally, I strongly support the FDA’s reminder that nominators must not submit confidential or personally identifiable information since these electronic submissions become a part of public dockets. The option to submit confidential information via paper submissions is appropriate and consistent with existing procedures. Therefore, the FDA’s requirement for nominators to submit characterization information (both physical and chemical), historical usage patterns, pharmacological activity, safety data, and justification for compounding over using an FDA-approved alternative is entirely justified. These requirements are in line with the statutory criteria under Section 503A(c)(2) and reflect best practices for ensuring that scientific evidence informs regulatory decisions. However, while I support the framework overall, I recommend the FDA further clarify two areas that would benefit nominators, stakeholders, and the public. The first is differentiating between the roles of historical use and evidence of effectiveness. These concepts can vary widely across medical contexts, so the FDA should make a point to clearly define what constitutes each. Without clearly defining these parameters, nominators may overstate unsupported uses in ways that could bias evaluations. The second recommendation is to clarify what is considered appropriate justification for nominators to submit when FDA-approved alternatives exist. For example, is a temporary shortage adequate? Is possible allergies to exipients adequate? Developing clear guidance on this requirement would reduce subjective interpretations and ensure consistency across nominations. In summary, the FDA’s establishment of this docket represents a critical step toward ensuring the safe, appropriate use of bulk drugs to compound medications, especially as the demand for compounded medications continues to grow. By requiring submissions that are complete and guided in science, the FDA is promoting transparency, reducing risks associated with compounded drugs, and ensuring that only substances with adequate evidence of safety and necessity are considered for the 503A bulks list. I encourage the FDA to continue refining its guidance for clarity, consistency, and strong scientific standards throughout the nomination and review process. Thank you again for the opportunity to comment.