From FDA News:
The FDA is taking steps toward getting more patient input during the drug development process, a move that puts the agency more in line with its EU counterparts, agency officials said at the DIA annual meeting.
The FDA has met with patients from over 20 different disease areas to hear their perspectives on how their treatments are working and how their diseases impact their lives as part of the PDUFA reauthorization process. The agency wants to glean information they don’t get from clinical trials, said Robert Temple, deputy director for clinical science in CDER, during a session at the DIA annual meeting in Washington, D.C.
Temple said patients’ chief complaints aren’t being factored into drug development or clinical trials, and they should be. Trial designs for new drugs should aim to identify the three most irritating symptoms patients with a particular disease have, so that drugmakers can focus on solving those issues. “Any endpoint that affects a patient is an endpoint that needs to be included in a trial,” he said.
Theresa Mullin, director of CDER’s Office of Strategic Programs, echoed that statement, saying the motivator for the FDA to include more patient input is the need to develop better endpoints. The agency can’t properly assess medicines without fully understanding the value, benefit and effect on patients, she said.
In the EU, patient participation is grounded in legislation, with patients having input at all levels of a clinical trial, said Enrica Alteria, head of human medicines evaluation at the European Medicines Agency. The EMA holds meetings with patient advisory and advocacy groups four times a year, and patients view labeling information and leaflets, she added.
As part of that patient discussion, Alteria said the agency consults with health technology assessment bodies early in the drug development process to gather additional patient evidence. In this respect, the EMA takes a broader lifespan approach than the FDA, she said.
The FDA is precluded by law from taking cost into account when it decides to approve or disapprove a drug. Temple said the agency wants people to have choices. “We try hard not to think about the economics.”
