FDA Issues New Draft Guidance On Individualized Drug Products

Federal regulators have issued a new draft guidance which seeks to provide guidelines for the creation and approval of individualized drugs developed specifically for patients with unique genetic diseases.

In a Federal Register Notice posted on January 5, the FDA outlined how manufacturers should seek to gain approval for individualized antisense oligonucleotide (ASO) drugs, which have become increasingly popular with advances in science, and are often submitted by parties other than the usual drug manufacturers.

“Developing these products – also referred to as “n of 1” therapies by some because they are designed for a patient population of one person – brings a set of challenges and considerations not seen with the typical drug development process,” according to an FDA press release announcing the draft guidance. “These investigators may be less familiar with FDA’s regulations, policies and practices, and less experienced in interacting with the FDA.”

According to the agency, this technology is most advanced in the development of ASO products, and the FDA hopes the new guidance will bring clarity to the approval of these types of drugs, as well as other individualized drug products.

The FDA press release states the agency is aware of a number of ethical and societal issues it will need to address involving the use of these individualized drug treatments. The FDA warns that individuals and family members need to be kept aware of how effectiveness will be measured, and that they understand the agency’s efforts to manage the development of these investigational drug products “before emotions influence decisions,” and that these patients and their families recognize that some of the products will fail or how they could lead to unforeseen side effects.

The guidance covers how to obtain feedback from the FDA; the process and expectations of submissions to the FDA for drug approval, how informed consent should be obtained, and includes recommendations on Institutional Review Board requirements and protocols.

The FDA is taking public comment on the draft guidance until March 8, 2021. Comments can be submitted electronically to https://www.regulations.gov, or through written/paper submissions sent to: Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.