FDA advisory panel votes against approval of DMD drugIn the words of the U.S. Food and Drug Administration (FDA), pharmacy compounding is “a practice in which a licensed pharmacist, a licensed physician, or, in the case of an outsourcing facility, a person under the supervision of a licensed pharmacist, combines, mixes, or alters ingredients of a drug to create a medication tailored to the needs of an individual patient.” This personalized approach to the preparation of prescription drugs has been hailed by some researchers as an alternative to expensive medications, and it also can be of benefit for certain patients (i.e., in the case of an individual allergic to an additive in the medication, or a patient with difficulty swallowing who needs the meds in a liquid form, or even a child for whom a needed drug is only commercially-available in an adult strength).

Potential case-by-case benefits aside, earlier this month the FDA released two new draft guidance documents explaining how it will place restrictions on compounded medications that are, in essence, replications of commercially available non-compounded drugs. The documents apply to pharmacies, physicians, federal facilities and outsourcing facilities. The FDA says that because compounded drugs are not evaluated for safety, quality and efficacy (as drugs bearing the agency’s stamp of approval are), the compounded medications come with a greater risk to patients.

Medications outside of CGMP: the FDA says they’re riskier

According to the FDA, its Current Good Manufacturing Practice (CGMP) regulations “contain minimum requirements for the methods, facilities, and controls used in manufacturing, processing, and packing of a drug product. The regulations make sure that a product is safe for use, and that it has the ingredients and strength it claims to have.” With compounded medications existing outside the FDA’s purview, the issue arises of some compounding pharmacies not being held to the CGMP stipulations. The FDA warns that taking low-quality or improperly prepared compounded medications can have dire consequences. And we don’t need to reach too far back into the past to find an example—a widespread outbreak of fungal meningitis in 2012 was ultimately traced back to a Massachusetts compounding pharmacy and led to more than 60 deaths and 750 infections.

Avoiding unnecessary risk is the rationale buttressing the FDA’s proposed restriction on compounded medications that are in essence replicating drugs already commercially accessible to patients.

Outsourcing facilities: a new category of compounders

The recently-released draft guidance documents relate to “Compounded Drug Products That Are Essentially Copies of Approved Drug Products” under Section 503A and 503B of the Federal Food, Drug, and Cosmetic Act (FD&C Act).

Section 503A was added to the FD&C Act by the Food and Drug Administration Modernization Act in 1997 and was amended by the Drug Quality and Security Act (DQSA) in 2013. It addresses the criteria that must be met for compounded drugs (prepared by a licensed pharmacist in a federal facility or in a pharmacy licensed by the state) to be exempt from CGMP regulations, labels that include directions for use, and the approval of drugs under new drug applications (NDAs) or abbreviated new drug applications (ANDAs). Further, the DQSA drafted a new section 503B that discusses outsourcing facilities (which represent a new category of compounders in the FD&C Act). Under 503B, outsourcing facilities must meet the same criteria as compounding pharmacists operating in a state-licensed or federal facility in order to qualify for the aforementioned labeling, NDAs and ANDAs exemptions (but not the CGMP).

Defining “Drug Products That Are Essentially Copies of Approved Drug Products”

Regarding the specifics around defining what is “essentially” a copy of an approved drug, section 503A of the draft guidance specifies that a compounded medication will fall under that category if:

  • The compounded drug contains the same active pharmaceutical ingredient(s) (API) as the FDA-approved, commercially-available drug;
  • The API(s) of the compounded drug and the commercially-available drug have the same strength (or the strengths are approximate or can be substituted with another dosage strength);
  • The FDA-approved drug available commercially can be administered in the same prescribed manner as the compounded medication (however, an exception is noted in the case of a prescriber’s determination that an individual patient would experience a significant difference in taking the approved drug).

In addition to expressing concern over patients potentially putting themselves at risk by taking a compounded drug that essentially can be replaced with FDA-approved medicine, the FDA goes on to address the reality of pharmaceutical competition that could impede funding of certain drugs:

“Sponsors may be less likely to invest in and seek approval of innovative, life-saving medications if a compounder could, after a drug is approved, compound ‘substitutes’ that have not had to demonstrate safety and effectiveness and are not produced in accordance with CGMP requirements or labeled with adequate directions for use. Sponsors might also be less likely to seek approval of an ANDA for a generic drug if compounders were permitted to compound drugs that are essentially copies of commercially available drugs without going through the ANDA process.”


For more information/questions regarding any legal matters, please email info@nelsonhardiman.com or call 310.203.2800.