FDA issues new draft guidance for Orphan Drug development

The U.S. Food and Drug Administration (FDA) published its recommendation for a new approach to drug development for rare pediatric diseases. The new approach supports the FDA’s commitment to increase the efficiency and speed of developing drugs, as well as supporting the sponsors of the research. The draft guidance, ‘Pediatric Rare Diseases: Collaborative Approach for Drug Development Using Gaucher Disease as Model’, will help companies navigate a new approach for partnering and evaluating multiple drugs in the same clinical trials, as reported in RareDR. This would reduce the number of patients necessary to be treated with placebo in these trials. Through the proposed controlled, multi-arm, multi-company clinical trials, several products can be tested in a more time-efficient manner. (December 6, 2017)


  • The FDA has stated that they devised this draft guidance working alongside officials from the European Medicines Agency (EMA), which would suggest that the EMA may well consider a similar approach and new guidance recommendation for pharmaceutical companies in Europe
  • The aim to reduce the number of patients receiving the placebo in the planned multi-company trials could lead to more patients having positive outcomes from the drug in development
  • This guidance comes after the Senate passed the Republican tax reform bill that could lead to major reduction in the Orphan Drug Tax Credit. It is thought that the proposed reduction to the credit could result in a significant drop in the number of orphan drugs being developed (a 2015 report estimated a one-third drop if the tax credit were completely repealed)
  • This new guidance comes at an important time, when accelerating the approval process could become absolutely vital given the potential negative impact of tax reform for rare disease patients. A decrease in the time taken to develop a drug would go some way to assuaging the reduction in the number of drugs in development
  • Closer alignment between pharmaceutical companies and government agencies has long been known to be of vital importance to deliver effective therapies to patients. This is another example of how regulators are moving towards a more collaborative system

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