The FDA has a pilot program called “Project Facilitate” that will handle requests for access to unapproved cancer drugs. They will connect patients and health care providers with contacts at pharmaceutical or biotechnology companies, complete request forms, and identify institutional review boards. Read more about this program here.
Researchers at the University of Pennsylvania recently published a very rare adverse effect in CAR-T therapies. A lentiviral vector bonded to the wrong cell and subsequently, the patient suffered a fatal relapse, with all of the leukemic cells carrying the chimeric antigen receptor. While the risk of similar incidents happening today has decreased, they are [...]
The FDA breakthrough therapy program is designed to expedite approval of medical treatments for serious or life-threatening conditions in cases where preliminary tests have suggested significant improvement compared to existing treatments. Upon review, drugs approved as breakthrough therapies generally have less evidence to support them versus FDA approved non-breakthrough drugs and some do not require [...]
The FDA has been approving expensive drugs at record speed even though there may be limited evidence of their effectiveness. Examples include Nuplazid (hallucinations and delusions associated with Parkinson's disease), Uloric (gout), and Folotyn (peripheral T-cell lymphoma). Learn more about this potential quality issue. Could one of these drugs be the next thalidomide? Read [...]
