Clinical drug development has traditionally been focused on regulatory approval. However, documentation for pricing and reimbursement should be prepared for through the drug development program. Phase II provides a golden opportunity to test the sensitivity of the quality of life and health economical questionnaires. This will in turn make it possible to choose the most sensitive tools for phase III. A valid patient reported outcome instrument is extremely important to prove clinical benefit for the patient and value for money for society.
Example of a patient reported outcome problem solved by Pharma Evidence:
Our client wanted a PRO claim in the labeling, and had, rightfully, used a HRQoL tool in its clinical drug development program.
As it happened, the FDA deemed the PRO tool unvalidated, and on those terms rejected it.
Pharma Evidence used the rapid revision approach, and, based on two cognitive debriefing focus groups and psychometric testing of 52 patients, we managed to adapt the old tool into a new, FDA validated questionnaire, in half the time traditionally required.