The science of molecular medicine continues to advance, but the key issue in applying the Orphan Drug Act exclusivity protection to new therapies stays the same. Literally.
The Orphan Drug Act prohibits FDA from approving the “same” drug to treat the same orphan-protected population of an already marketed product. That principle is reasonably straightforward in the context of small molecule pharmaceuticals where chemical formulas are simple to interpret
Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.
Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.
Cooperation between health technology assessment bodies across the EU will lead to a better joint clinical assessment process over time, but patients cannot afford a lengthy wait for improvements, speakers at a cell & gene therapy conference said.
The biotech CEO said the Trump Administration’s treatment of FDA employees, including the HHS secretary’s comments about industry capture, are insulting to agency staff, but he is encouraged by FDA Commissioner Martin Makary’s talk of a new conditional approval pathway.
England’s health technology assessment institute explains how its sandbox environment is helping to test new evaluation methods for drugs or indications with which it does not have experience, such as metabolic dysfunction-associated steatohepatitis.
The European Medicines Agency, like its counterpart in the US, is increasingly focusing on the use of alternatives to animal testing.
Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.
