Gathering Structured Patient Insight to Drive the PRO Strategy in COPD: Patient-Centric Drug Development from Theory to Practice.

We illustrate our experience of gathering patient insights on the most patient-relevant symptoms in chronic obstructive pulmonary disease (COPD) via a structured and systematic approach towards 'patient-centric' drug development, leveraging recent advances in digital technologies using online platforms. The four-step approach comprised the following: literature search, social media listening (SML) study, online bulletin board (OBB) exercise, and design of an online patient preference study (PPS). The initial online studies (SML and OBB) revealed that, besides dyspnoea and exacerbations, patients perceive cough and mucus production as equally important aspects of disease management for COPD. To further build and quantify patients' understanding of the importance of these symptoms, an online patient preference survey is underway. Based on these findings, we have elected to include the Cough and Sputum Assessment Questionnaire or CASA-Q, a validated instrument to collect patient-reported outcomes (PRO), besides the use of the COPD assessment test or CAT to assess the severity and impact of COPD in drug development studies for COPD. Additionally, to capture movement and sleep disturbance, we consider the inclusion of actigraphy as a digital evidence-capture end point. Lastly, in a phase II trial, a survey questionnaire on incontinence will be administered to evaluate the importance of this issue among patients. We believe that integrating insights derived from "online" studies (SML, OBB, and PPS) into drug development offers an opportunity to truly listen to patients' voices in early product design ensuring relevance of end points selected for the clinical trial program. This approach also has the potential to complement conventional qualitative and quantitative data collection requirements for PRO instrument development. While awaiting final guidance from the US Food and Drug Administration, or FDA, the recently released draft documents on collecting representative patients' input reference social media as a tool to collect qualitative patient preference data and these developments suggest that patient preference data can influence future clinical trial design, end point selection, and regulatory reviews.Funding: Novartis Pharma AG, Basel.