The FDA has issued new draft guidance that may make it easier for pharmaceutical companies to develop and market drugs to treat alcoholism. The proposed guidance provides that the FDA may approve drugs that help patients improve their drinking behavior, even if the drugs do not help patients achieve complete sobriety. The draft guidance is being issued consistent with FDA’s “good guidance practices” regulation (21 CFR 10.115) and will, when finalized, “represent the Agency’s current thinking on the development of drugs for the treatment of alcoholism and appropriate endpoints for clinical trials of drugs to treat alcoholism.” The guidance “does not create or confer any rights for or on any person and does not operate to bind FDA or the public.”
For years, abstinence from any alcohol consumption has been the common goal of clinical trials to develop drugs to treat alcoholism. According to the FDA, however, abstinence has been considered an “unattainable threshold” and “a hindrance to clinical development for drugs to treat alcoholism.” The FDA’s new draft guidance, entitled Alcoholism: Developing Drugs for Treatment Guidance for Industry, recognizes that other outcomes besides sobriety (called “surrogate endpoints”) may still be effective at treating alcoholism. Thus, the draft guidance provides that drugs to treat alcoholism may be approved if they are clinically proven to help patients:
- Completely abstain from drinking alcohol; or
- Engage in no heavy drinking days for six months. A heavy drinking day is defined as consuming more than four drinks in a day for men and more than three drinks in a day for women.
Companies may also be able to obtain FDA approval for drugs that help patients reach other surrogate endpoints besides the two listed above. However, companies using other surrogate endpoints would have to submit additional data to the FDA. That data would need to demonstrate that the surrogate endpoint the company selects has a “clinical benefit.” The FDA recommends that companies discuss other potential surrogate endpoints with an FDA division member.
. . . abstinence has been considered an “unattainable threshold” and “a hindrance” . . . .
Before any company submits an application for FDA approval of a drug used to treat alcoholism, the draft guidance recommends that “two adequate and well-controlled trials,” each lasting at least six months, be performed. The draft guidance can be found here. According to the FDA, guidance documents “do not establish legally enforceable responsibilities,” but describe the Agency’s current thinking on a topic and “should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited.”
The FDA’s new draft guidance will be open for public comment until April 13, 2015. To submit an official comment, click here and then click “Submit a Formal Comment” in the upper right corner.