Regulatory Focus™ > News Articles > FDA's 'Temperance Movement' Guidance Focuses on Alcoholism Endpoints

FDA's 'Temperance Movement' Guidance Focuses on Alcoholism Endpoints

Posted 11 February 2015 | By Alexander Gaffney, RAC

FDA's 'Temperance Movement' Guidance Focuses on Alcoholism Endpoints

A new draft policy issued by the US Food and Drug Administration (FDA) aims to make it easier for drug companies to develop new treatments for alcoholism by allowing them to focus on patients who give up "heavy" drinking, and not drinking entirely.


The policy, Alcoholism: Developing Drugs for Treatment, is a draft guidance document intended to address some of the clinical development hurdles which might otherwise complicate the process of developing a drug and obtaining marketing approval from FDA.

In general, FDA demands evidence from companies to show that a drug is both safe and effective. What those terms means, however, is ambiguous, and depends upon both the condition being treated and the characteristics of the treatment.

For example, a cancer drug might be shown to be effective if it manages to delay the growth of a tumor for several months, even if a patient isn't cured of the cancer. For other conditions, FDA might require proof that a drug is better than the current standard of treatment (i.e. the best treatment out there), or cure an infection within a set period of time.

The Guidance

FDA's guidance on developing drugs to treat alcoholism is meant to clear up some of the potential ambiguity around showing that a drug is safe and effective by setting definitional standards about how to diagnose a patient as actually havingalcoholism, and establishing which endpoints are appropriate in a testing environment.

At the outset of the guidance, FDA makes an interesting point: Depending on the benefits offered by the drug, FDA might be willing to accept that a drug is "effective" even if the company can't show that it causes abstinence from drinking—a traditional and obvious endpoint for more alcoholism studies.

Instead, companies might be able to use so-called surrogate endpoints—essentially intermediate signals that show that a person is progressing toward a positive outcome.

Such endpoints are commonly used to assess cancer drugs, where a shrinking tumor might indicate an extended lifespan for a patient. While the use of surrogates can make for a shorter (and therefore less expensive) trial, they can also hide long-term side effects or other negative effects that might be discovered during the course of a longer trial.

A Focus on Heavy Drinking

Still, FDA said it will be willing to accept the surrogate endpoint of a drug's effect on a patient's "drinking behavior" as long as a company can show the behavior "can be reasonably predictive of clinical benefit."

For example, FDA said it would accept a surrogate endpoint of "no heavy drinking days," which the National Institute of Health (NIH) defines as a day with fewer than either four drinks (men) or three drinks (women). As long as patients can attain and sustain this no-heavy-drinking pattern for six months after treatment, FDA said it will be willing to accept the endpoint.

"Patients who attain and sustain complete abstinence from alcohol may be assumed to accrue clinical benefit" as well, FDA wrote, citing an NIH study which found that "patients who never exceeded the heavy drinking limits had minimal alcohol-related consequences and were much less likely to have relapsed at follow-up."

A company should, in general, conduct "two adequate and well-controlled trials" to support a drug application, FDA added. Trials should be double-blind, placebo-controlled, superiority trials.


Alcoholism: Developing Drugs for Treatment (FR)

Categories: Regulatory News

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