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.
Rare disease advocates are wondering whether the Orphan Drug Act should be amended to better describe regulatory flexibility, a concept that is well-known in the abstract, but more difficult for the US Food and Drug Administration to define and apply.
Often the traditional evidentiary standard that conventional products must meet is not possible for rare diseases with small patient populations
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.
