US FDA Outlines Streamlined Development Path For Targeted Therapies

The US FDA has laid out a more definitive pathway for molecularly-targeted drugs seeking indications in multiple patient subsets, but still left open the possibility that additional clinical evidence could be ordered or indications adjusted later should contrary evidence arise.

The new draft guidance outlining development standards for targeted therapies in low-frequency molecular subsets of a disease allows sponsors a potentially streamlined pathway to approval for multiple molecular groupings...