Incorporating Patient Input into the Target Product Profile.

Target product profiles (TPP) are summaries of characteristics which drug developers expect to be necessary for a product to meet patients' needs, receive regulatory and payer approval, and differ from existing treatment options. As the experts on their own disease, patients bring invaluable perspectives to drug development, which cannot be obtained by other means. This communication reports on the development of a systematic guidance framework for a patient-focused, standardized TPP. The guidance was developed in a long-term iterative process, with crucial aspects reviewed and validated with the patient community. Five focus areas of a TPP were identified where patient perspectives are fundamental: target population, unmet need, dosage frequency and route of administration, efficacy endpoints, and acceptability of benefit/risk profile trade-offs. A guiding principle should be to incorporate patient perspectives in a systematic process starting as early as possible. A number of tools are available for obtaining patient perspectives, e.g., desk research, patient advisory boards/patient councils/online bulletin boards, focus groups with patients/caregivers, and/or in-depth interviews. When discussing the proposed process with patient representatives, they identified several key requirements for the interaction between R&D organizations and patient representatives. These include the use of clear language, respect for patients, engagement with patient experts, provision of adequate context and background information. We further discuss the relative importance of integrating patient perspectives into the different focus areas and touch upon the potential benefits to patient organizations from adapting these concepts and processes to enhance their voices in drug development.