PRO65 Engaging Patients in Drug Development for Regulatory and HTA Decisions: A Case Study in Patients with Hidradenitis Suppurativa

Abstract

Regulatory agencies and Health Technology Assessment (HTA) bodies are increasingly recognizing the importance of the patient’s voice in new drug development and reimbursement decisions. However, inconsistencies remain on the best practices to describe patients’ perspectives living with disease. This case study provides an example of a systematic qualitative study in Hidradenitis Suppurativa (HS) to demonstrate impact of disease, unmet need, and important aspects of treatment to patients with HS. This information is critical to assess patient benefit for informed regulatory and reimbursement decisions. A total of 36 qualitative interviews were conducted in patients with moderate to severe HS. Patients were asked open-ended questions regarding living with HS including specific symptoms of HS, impact of HS on their functioning, well-being, daily activities, and other specific treatment outcomes meaningful to patients. Impact on health-related quality of life (HRQOL) was assessed by the Dermatology Life Quality Index (DLQI) and 5-level EuroQoL-5D (EQ-5D-5L). In general, subjects reported living with HS as painful, difficult, and embarrassing. Commonly reported (>60%) symptoms of HS included: pain, drainage, itching, swelling, odor, tenderness, heat, and lesion-related pressure. Pain was reported as the most bothersome and difficult to manage symptom. Individuals with HS also reported being negatively impacted in terms of social/emotional functioning, and ability to perform daily activities with a DLQI mean total score of 14 ± 7, indicating a large effect on a patient’s life. General health status was significantly impaired with mean EQ-5D VAS score of 67 ± 18. As an effective treatment, patients indicated ≥10% improvement in pain as meaningful. Insights gathered from patients with HS help describe the patients’ experience. Developing a validated PRO instrument to evaluate HS symptoms and impact on HRQOL will be critical to demonstrate patient benefit with a novel therapy and help inform regulatory and HTA decisions.