The FDA has outlined how developers of drugs targeting rare pediatric diseases can streamline their clinical development programs by collaborating. Officials want drug developers to consider multicompany clinical trials to test multiple candidates in single trials, thereby cutting the number of patients who need to receive placebos.
Using Gaucher disease as an example, the FDA’s draft guidance (PDF) shows how companies can team up and run multidrug clinical trials. These studies would enable multiple experimental candidates to be compared against a single control group, increasing the proportion of patients in active arms.
Other sections of the guidance propose the use of modeling and simulation to predict the effect of a drug in children based on prior performance in adults. Collectively, the FDA sees 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, said in a statement.
The genesis of the document dates back to 2011, when the FDA began working with the European Medicines Agency (EMA) to create a roadmap for the development of Gaucher disease drugs. That resulted in a guidance document that was released for public comment in 2014 and adopted by the EMA earlier this year. It has now led to the publication of the draft FDA guidance.
Woodcock and her colleagues have copied large sections of the Gaucher guidance word for word but have extended its scope beyond the condition. In its new form, the FDA guidance proposes extending the principles underpinning the Gaucher development model to other diseases.
The guidance is still in a draft form and is open for comment for 60 days.