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.

Impact of cough and mucus on COPD patients: primary insights from an exploratory study with an Online Patient Community.

Background: Qualitative research provides real-life information on patients' condition and facilitates informed design of future clinical studies. Objective: We used Online Communities as a qualitative research tool to evaluate the effect of cough and mucus on COPD patients. Methods: Two 2-week Online Communities were run in parallel in the UK and in the USA, including COPD patients with persistent cough and excessive mucus. Patients anonymously posted their responses to pre-assigned tasks, supervised and guided by a trained moderator. Five themes around the impact of cough and mucus were explored with new questions posted every 2-3 days. On the final day, high-level conclusions were shared with patients for feedback. Data were analyzed following the principles of grounded theory. Results: Twenty COPD patients (UK, n=10; USA, n=10) participated in the Online Communities. We found that cough and mucus disrupted COPD patients' lives at functional, emotional, social and economic levels. Patients created daily rituals and adjusted their lifestyle to cope with the impact of these symptoms. Patients identified themselves with our conclusions and saw the Online Community as an effective forum to share their experiences. Conclusion: Findings of our study add to the body of evidence on the negative impact of COPD symptoms and unmet needs of these patients.