October 2, 2014
Identifying sets of symptoms common to all patients affected by one or several rare diseases has proven useful to clinical researchers and drug developers in order to address patient needs and substantiate drug labelling. In an article published in the Journal of General Internal Medicine, Basch and Bennett support the use of patient-reported outcomes (PRO) to measure patient reactions and progress throughout clinical trials. While regulatory agencies, standards organizations and international societies have issued a number of guidance documents on PROs, many trials on rare diseases still do not include PROs in clinical data. The authors suggest that PROs should be conducted by experienced investigators and tailored to patient populations and specific symptoms, such as the NIH’s Patient Reported Outcomes Measurement Information System (PROMIS).