SAFETY AND EFFICACY OF COMPOUNDED TOPICAL FORMULATIONS

To encourage pharmacists to take a leadership role in developing processes that would ensure quality, safety, and effectiveness of compounded topical formulations; further,

To advocate that ASHP expand its repository of evidence-based formulations that could serve as a resource for compounding topical formulations; further,

To advocate that public and private payers and healthcare providers collaborate to create standardized and efficient methods for authorizing payment for medically necessary compounded topical formulations; further,

To encourage hospitals and health systems to develop policies and procedures to guide clinicians in making informed decisions regarding the prescribing and use of compounded topical formulations; further,

To encourage pharmacists to take a leadership role in developing and providing education on the safety and efficacy of compounded topical formulations to providers and consumers.

Rationale

Compounded topical formulations are meant to be customized for individuals whose needs cannot be met by commercially available drugs. Unlike the drugs made by conventional manufacturers that require Food and Drug Administration (FDA) approval, compounded drugs such as various topical formulations are not evaluated by the FDA for safety, effectiveness, or quality, and many are exempt from the new-drug approval process, current good manufacturing practice, and other FDA requirements. In addition, quality standards for compounded drugs are generally lower than those for FDA-approved drugs; therefore, compounded drugs can pose increased safety risks (e.g., being contaminated or having the wrong potency) or lack efficacy.

Because some drugs do have FDA approval for topical application, clinicians and patients may not be aware of potential safety risks or potential lack of effectiveness associated with certain ingredients and combinations of ingredients in compounded topical pain creams. When these agents are compounded, at least one of the ingredients is an active ingredient in an FDA-approved topical pain cream (e.g., lidocaine), while the remaining ingredients may be active ingredients in drugs approved by the FDA for non-topical administration to treat non-pain-related indications (e.g., antidepressants, anticonvulsants, antivirals, narcotics). In addition, the literature supporting the use of the additional agents outside their normal vehicle of administration is often not well designed or sufficiently powered to demonstrate efficacy. A study published by the U.S. Department of Defense found that these combination-compounded pain creams were no better than placebo creams and, given their higher costs, which had escalated to cost of $6 million per day, should no longer be used.

Issues of fraud are also well known with compounded topical formulations. In August 2018, the Department of Health and Human Services Office of Inspector General (OIG) found that from 2006 to 2015, spending for these drugs increased 625%, and spending for compounded topical drugs—such as creams, gels, and ointments—grew at an even faster pace. Medicare Part D sponsors cover these drugs under certain circumstances. The OIG also found that Part D spending for compounded topical drugs increased 2353% from 2010 to 2016, rising from $13.2 million to $323.5 million. Much of this growth occurred from 2014 to 2016, when spending increased by more than $200 million and raised concerns that the drugs that were billed to Part D were not always dispensed or medically necessary. Upon investigation, the OIG found that many of the parties charging Part D were in a handful of cities, with thousands of prescriptions written by a single provider and filled by a limited number of pharmacies. This led HHS to conclude that the prescribers may not have had legitimate doctor-patient relationships with the beneficiaries.

Given these challenges, pharmacists will need to assume a leadership role in developing processes to ensure the quality, safety, and effectiveness of compounded topical formulations, including developing and providing education on compounded topical formulations for providers and consumers, and expanding the ASHP repository of evidence-based formulations. Public and private payers and healthcare providers will need to collaborate to create standardized and efficient methods for authorizing payment for medically necessary compounded topical formulations, and hospitals and health systems will need to develop policies and procedures to guide clinicians in making informed decisions regarding prescribing and use of compounded topical formulations.

Finally, with the updates to USP 795, which outlines provisions on non-sterile topical compounding, issues with training, competence, and compounding topicals during times of drug shortages, ASHP should continue to be a leader in this area for its members.