The FDA has drawn out a plan on how drug developers targeting rare pediatric diseases can streamline their clinical development programs by teaming up. Officials want drug developers to think about collaborating to test a variety of candidates in single trials, thereby trimming the amount of patients who need to get placebos.
For example, Gaucher disease, the FDA drafted guidance that shows how companies can connect and conduct multi-drug clinical trials. These studies would encourage multiple experimental drugs to be compared against one control group, enhancing the proportion of patients in active arms. Other sections of the guidance propose the use of modeling and simulation to estimate the effect of a treatment in children based on its previous performance in adults. As a whole, the FDA views the proposals streamlining the development of drugs to treat rare pediatric diseases.
“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,” Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research, stated.
The genesis of the document dates back to 2011, when the FDA starting working with the European Medicines Agency (EMA) to initiate a roadmap for the development of Gaucher disease drugs. That resulted in a guidance document that was released for public comment in 2014 and was adopted by the EMA earlier in 2017. It has now spearheaded to the publication of the draft FDA guidance.
Woodcock and her co-workers have duplicated big portions of the Gaucher guidance verbatim but have widened the scope beyond the condition. It’s new form, the FDA guidance proposes extending the principles supporting the Gaucher development model to other diseases.