PFMD have taken time to respond to the recent Food and Drug Administration (FDA) open consultancy and subsequent update on its Benefit-Risk Assessment in Drug Regulatory Decision-Making.

We believe the document offers clarity, is comprehensive and provides tactical guidance on the practical approaches and methods to collect, analyse, report and utilise robust and meaningful input from patients and caregivers. We also believe, however, that the qualitative aspects of engaging with patients to collect comprehensive and representative patient input require additional attention and that the guidance document would benefit from inclusion of these aspects. Thus we have proposed that the FDA documents be enhanced by addition of complementary guidance on the qualitative perspective of engaging with patients.