Patient-Reported Outcomes vs Empirical Evidence in Clinical Trials

An article in The Scientist discusses the FDA’s decision to require patient-reported outcomes from some pharmaceutical companies during the drug development process. Over the last few years, the FDA has moved to require patient-reported outcomes such as results of validated patient questionnaires from more drug developers. In 2006, the FDA issued a 32-page draft guidance for designing questionnaires, capturing patient feedback, and analyzing data.

Patient-reported outcomes are measurements of responses during the clinical trial that cannot be obtained through other types of clinical testing or questioning, such as reduction of pain or fatigue and quality-of-life improvement. While the FDA director for the study of end points and label development states that a patient-reported outcome can be more reliable and less variable than a clinician’s assessment, others argue that the questionnaires are subjective and a perception of “pain” can vary widely from patient to patient. In addition, when questionnaires are completed on behalf of children or elderly patients, how can validity of the results be guaranteed? Who would monitor the investigator and ensure that he/she does not complete questionnaires on “behalf” of patients, thereby potentially skewing the results of the study?

The potential burden on the drug developers to add patient-reported outcomes if requested by the FDA may add a year or more to the drug development process. It is also an additional financial consideration, as development and validation of a questionnaire can cost $500,000 or more. On the other hand, there is a strong advantage to having validated data with positive patient outcomes in the drug’s product labeling. The drug developer has a valuable opportunity to use the patient-outcomes data for promotional purposes in promotional and educational materials and also in reimbursement negotiations with managed-care payers or government agencies.

Source: Silverman E. Whether drug companies like it or not, the FDA is pushing patient-reoprted outcomes in trials. So what are they good for? The Scientist. 2007;21:64. Available at: http://www.the-scientist.com/article/print/53617.