The International Rare Diseases Research Consortium (IRDiRC) has created a Guidebook to facilitate drug development for rare diseases by organizing available tools into a standardized framework.
Coens, C.; Pe, M.; Dueck, A.C.; Sloan, J.; Basch, E.; Calvert, M.; ... ; Setting Int Stand Analyzing Patien 2020
Patient-reported outcomes (PROs), such as symptoms, function, and other health-related quality-of-life aspects, are increasingly evaluated in cancer randomised controlled trials (RCTs) to provide... Show morePatient-reported outcomes (PROs), such as symptoms, function, and other health-related quality-of-life aspects, are increasingly evaluated in cancer randomised controlled trials (RCTs) to provide information about treatment risks, benefits, and tolerability. However, expert opinion and critical review of the literature showed no consensus on optimal methods of PRO analysis in cancer RCTs, hindering interpretation of results. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium was formed to establish PRO analysis recommendations. Four issues were prioritised: developing a taxonomy of research objectives that can be matched with appropriate statistical methods, identifying appropriate statistical methods for PRO analysis, standardising statistical terminology related to missing data, and determining appropriate ways to manage missing data. This Policy Review presents recommendations for PRO analysis developed through critical literature reviews and a structured collaborative process with diverse international stakeholders, which provides a foundation for endorsement; ongoing developments of these recommendations are also discussed. Show less
Bottomley, A.; Reijneveld, J.C.; Koller, M.; Flechtner, H.; Tomaszewski, K.A.; Greimel, E.; ... ; 5th EORTC Quality Life Canc 2019