Does The Orphan Drug Act Need A Tune-Up?
A better description of the US FDA’s regulatory flexibility may be necessary, but tinkering with the foundational law for rare disease drug development could open the door for more substantial and unwanted changes.
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Agency officials don’t think a new definition or incentives will fix the issues related to development in rare diseases with only a handful of patients, as advocates continue pushing the idea.
India has exempted imports of top-selling oncology drug Merck’s Keytruda from customs duty as also rare disease drugs listed in the National Policy For Rare Diseases. However, patient advocacy groups feel more fiscal measures and manufacturing incentives are needed to improve access
FDA, NIH, sponsors and others use different figures, but some want everyone will settle on 10,000 for policy discussions, advocacy and research.