The FDA has taken the unusual step of lobbying the US government legislature to address a recent court decision which could have wide and worrying implications for rare disease R&D.
In October last, a US appeals court overturned a previous FDA court win, saying that they should never have approved a rare disease drug because a previously approved – but more expensive – drug, with the same active ingredient, has Orphan Drug Exclusivity which bars any sponsor from making the same drug for the same disease – even if the sponsor does not rely on the innovator's data.
The FDA has lobbied Congress, expressing alarm and concern at the potential disastrous consequences of this, whereby companies could target even the smallest indication for a specific disease and then automatically receive 7 years of exclusivity for that disease in its entirety.
The FDA predicts that, worryingly, without Congressional action, this ruling will create an incentive for sponsors to take a simple but uncomprehensive route and focus studies on small, easy-to-study populations but not all individuals with the disease. Director of the CDER, Patrizia Cavazzoni has encapsulated the concerns by observing that the decision “... will disproportionately affect children with rare diseases”.
In an effort to rectify the situation, bipartisan legislation called the Retaining Access and Restoring Exclusivity (RARE) Act has been recently introduced to try to ensure that the scope of the orphan exclusivity is clarified to apply only to the same approved use or indication within such rare disease or condition instead of the same disease or condition.
The FDA – and the world of disease research – waits to hear whether or not the Supreme Court will take up the case.
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