I recently watched the live webcast of the FDA’s meeting where they decided not to approve Bronchitol for Cystic Fibrosis in the US. It was a real eye-opener and I thought it would be worth summarising the decision and providing some commentary from the patient perspective. It made me think about who should be weighing the risks and benefits of new medicines in cases where patients are experts with specialist doctors and treatment options can have major implications on their lives. There were some fantastic presentations and speeches by doctors and other prominent people in the CF community and I hope everyone with a personal or professional interest in CF has the chance to watch them when the footage is eventually put on the FDA’s website.
- deep FDA/medical profession understanding of CF and the real life problems that go with it;
- high-level understanding of the heavy treatment burden and the fact that this makes it difficult to build and maintain a life;
- common intent to develop additional treatment options recognising that people with CF respond differently to particular medications; and
- common intent to develop alternative modes of drug delivery in order to reduce the treatment burden on people with CF.
Here is a link to my follow-up post: Bronchitol: Future Prospects in the US