EV1 DEVELOPMENT OF A GUIDANCE FOR INCLUDING PATIENT-REPORTED OUTCOMES (PROS) IN POST-APPROVAL CLINICAL TRIALS OF ONCOLOGY DRUGS FOR COMPARATIVE EFFECTIVENESS RESEARCH (CER)

Although an FDA Guidance is available to direct the development and inclusion of PROs in preapproval clinical research towards drug approval and labeling, no such guidance exists in the post-market CER context. Because the clinical setting and stakeholders in CER differ from the preapproval space, different methodological and practical considerations are necessary. The aim of this project is to develop specific recommendations for capturing the patient perspective in post-market oncology trials and other studies that fall into the general category termed CER. A semi-structured questionnaire pertaining to the use of PROs in CER was developed based on a review of scientific literature and consultation with PRO study methodologists. In-depth interviews were conducted using these questions with 15 individuals from the clinical research, clinical practice, regulatory, payer, and patient communities. Based on interview responses, a list of potential guidance topics was developed. The Center for Medical Technology Policy convened a multidisciplinary working group of leading medical researchers in PROs, ePRO consultants, and patient advocates to discuss these topics and develop an on outline of best practice recommendations for integrating PROs in CER. A series of recommendations focused on establishing standards for the use of PROs in CER were developed. These recommendations address selection of appropriate measures; inclusion of a ”core minimum item set” across all oncology CER trials; and inclusion of items which assess symptoms, global QOL, perceived value of care, and treatment compliance. The recommendations also provide guidance for how to implement PROs in real-world studies and minimize missing data. The patient perspective is an essential component of CER. PRO methods used in CER differ from the preapproval context. For improved consistency across trials, a guidance is being developed which identifies a standard core item set to be considered for use across all oncology CER studies.