Wednesday, July 3, 2013

How Does It Make Me Feel?

NEJM has an interesting piece on patient input. They state:

As an oncologist, when I sit with patients to discuss starting a new chemotherapy regimen, their first questions are often “How will it make me feel?” and “How did patients like me feel with this treatment?” Regrettably, this information is generally missing from U.S. drug labels and from published reports of clinical trials — the two information sources most commonly available to people trying to understand the clinical effects of cancer drugs.

 This is a serious issue not only with oncologists. The challenge is to meet the anticipated needs of the patient. Patients respond much better when what they are told matches what happens. Then there are no surprises. They can often deal with a great amount of discomfort much better than lesser amounts of uncertainty.

They continue:

The FDA has taken several recent steps toward encouraging inclusion of the patient perspective in drug development. It issued highly influential guidance on the use of patient-reported outcomes (PROs) in drug development, collaborated with the Critical Path Institute and industry to form the PRO Consortium with the aim of developing robust symptom-measurement tools, and obtained support from Congress in the fifth reauthorization of the Prescription Drug User Fee Act (PDUFA) to expand its internal expertise on the methodology of measuring PROs. (Unfortunately, allocated PDUFA funds have been withheld, which substantially impairs the FDA's ability to implement planned patient-centered programs.)

These FDA efforts are evident in the ruxolitinib label and in the label for abiraterone acetate, approved this year for metastatic prostate cancer, which describes beneficial delays in time to the development of pain and the need for opioid use. Yet in preapproval trials in patients with cancer, symptom or functional-status evaluations that meet the FDA's standards remain rare.

The FDA should clearly expand on this effort. In a sense the patient should have a peer reviewed assessment of what to expect.