Last week, the FDA released a draft guidance document aimed at improving the treatment situation for pediatric patients suffering from rare diseases. The guidance, titled “Pediatric Rare Diseases—A Collaborative Approach for Drug Development Using Gaucher Disease as a Model”, lays out a new approach for pediatric drug development that could enhance the efficiency of clinical trial efforts and bring important, new drugs to market quicker.
The 14-page document was developed in collaboration with the European Medicines Agency (EMA) and uses Gaucher disease as a disease model. According to the FDA, although the guidance focuses on Gaucher’s, the document is intended to be applicable to other rare pediatric disorders.
There are a number of strategies proposed in the document, including the development of multi-arm, multi-company clinical trials. Such trials would allow companies to combine drug development efforts and test multiple investigational drugs in the same clinical trial. This would result in one single arm control group acting as a comparison for multiple drugs, which would limit the number of patients receiving a placebo product.
The guidance document also encourages companies to use data from trials conducted in adults and older pediatric patients to predict how a drug could work in young pediatric patients. According to the guidance, using modelling and simulation strategies, extrapolated efficacy data could be used when the course of the disease and the predicted response to the drug is sufficiently similar in the pediatric and adult populations.
Additionally, the FDA has recognized that, in pediatric drug development, it is often useful to develop and employ age-specific endpoints. The guidance document advises that, if such endpoints are necessary, they should be identified and brought to the FDA for review as early as possible.
The FDA will be accepting comments on the document for two months, until February 5, 2018. After this date, the agency will release its final determinations. To find out more, click here to access the full FDA guidance document.