FDA outlines “multiarm, multicompany” collaborations

Teaming Up for Clinical Trials

In an effort to reduce the number of patients needing to receive placebos in clinical trials and to lower overall costs, the U.S. Food & Drug Administration (FDA) has come up with a draft proposal that shows how drug developers targeting rare pediatric diseases can form collaborations to “streamline their clinical development programs.” According to an article by Nick Paul Taylor in Fierce Biotech, FDA officials would like for drug developers to think about joining forces to run tests on multiple candidates in single trials.

The FDA has provided guidance in a draft form, “Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model: Guidance for Industry,” using Gaucher disease as an example. Open for comment for 60 days, the document explains how companies can join together in running multidrug clinical trials. If carried out the way the FDA anticipates, the studies could allow for the comparison of multiple experimental candidates against a single control group, thus increasing the proportion of patients in active parts of the trial.

The document also suggests that developers use modeling and simulation as prognosticators of the effects of drugs in children based on their previous performance in adults. The objective of the proposals in the document is to streamline the development of pharmaceuticals used to treat rare pediatric diseases.

According to Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research, “The FDA has drafted an approach to pediatric rare disease drug development that could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would receive a placebo instead of a potentially helpful drug.” She and her associates have used the Gaucher guidance as a model for doing clinical trials for other conditions. In its new form, the FDA guidance “proposes extending the principles underpinning the Gaucher development model to other diseases,” Taylor’s article said.  

The document has roots dating back to 2011, when the FDA began collaborating with the European Medicines Agency (EMA) to develop a roadmap for the approval process of drugs for Gaucher disease. In turn, a guidance document was disseminated for public comment in 2014, adopted by the EMA several months ago and published as a draft FDA guidance document that will, when finalized, “represent the current thinking of the Food and Drug Administration (FDA or Agency) on this topic,” the document says. The draft guidance “does not establish any rights for any person and is not binding on FDA or the public,” meaning that it is acceptable for drug developers to “use an alternative approach if it satisfies the requirements of the applicable statutes and regulations.”

In its introduction, the guidance document provides a caveat: “In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances 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 use of the word ‘should’ in Agency guidances means that something is suggested or recommended, but not required.”