{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T13:20:25Z","timestamp":1790169625612,"version":"4.1.0"},"reference-count":30,"publisher":"Springer Science and Business Media LLC","issue":"4","license":[{"start":{"date-parts":[[2025,4,25]],"date-time":"2025-04-25T00:00:00Z","timestamp":1745539200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,4,25]],"date-time":"2025-04-25T00:00:00Z","timestamp":1745539200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ther Innov Regul Sci"],"published-print":{"date-parts":[[2025,7]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Target product profiles (TPP) are summaries of characteristics which drug developers expect to be necessary for a product to meet patients\u2019 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&amp;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.<\/jats:p>","DOI":"10.1007\/s43441-025-00783-1","type":"journal-article","created":{"date-parts":[[2025,4,25]],"date-time":"2025-04-25T11:37:15Z","timestamp":1745581035000},"page":"659-667","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Incorporating Patient Input into the Target Product Profile"],"prefix":"10.1007","volume":"59","author":[{"given":"Jean-Noel","family":"Talabardon","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janet E.","family":"Church","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masanori","family":"Okuse","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michaela","family":"Dinboeck","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sheila","family":"Dickinson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon","family":"Messner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Boisclair","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Pierre","family":"Malkowski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,4,25]]},"reference":[{"key":"783_CR1","doi-asserted-by":"publisher","first-page":"10026","DOI":"10.3390\/ijerph181910026","volume":"18","author":"S Flessa","year":"2021","unstructured":"Flessa S, Huebner C. Innovations in Health Care\u2014a conceptual framework. Int J Environ Res Public Health. 2021;18:10026.","journal-title":"Int J Environ Res Public Health"},{"key":"783_CR2","unstructured":"European Medicines Agency. Engagement framework: EMA and patients, consumers and their organisations [Internet]. 2022. Available from: https:\/\/www.ema.europa.eu\/documents\/other\/engagement-framework-european-medicines-agency-patients-consumers-their-organisations_en.pdf"},{"key":"783_CR3","unstructured":"US Food and Drug Administration. FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient\u2019s Voice in Medical Product Development and Regulatory Decision Making. FDA [Internet]. 2023 [cited 2023 Oct 30]; Available from: https:\/\/www.fda.gov\/drugs\/development-approval-process-drugs\/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical"},{"key":"783_CR4","unstructured":"CIOMS Working Group XI on Patient involvement in the development, regulation and safe use of medicines. Patient involvement in the development, regulation and safe use of medicines [Internet]. Council for International Organizations of Medical Sciences (CIOMS); 2022. Available from: https:\/\/cioms.ch\/publications\/product\/patient-involvement\/"},{"key":"783_CR5","doi-asserted-by":"crossref","unstructured":"Bandyopadhyay A. Target Product Profile: A Planning Tool for the Drug Development. MOJ Bioequivalence Bioavailab [Internet]. 2017 [cited 2024 Feb 10];3. Available from: https:\/\/medcraveonline.com\/MOJBB\/target-product-profile-a-planning-tool-for-the-drug-development.html","DOI":"10.15406\/mojbb.2017.03.00044"},{"key":"783_CR6","unstructured":"World Health Organisation. Target product profiles [Internet]. [cited 2024 Apr 17]. Available from: https:\/\/www.who.int\/observatories\/global-observatory-on-health-research-and-development\/analyses-and-syntheses\/target-product-profile\/who-target-product-profiles"},{"key":"783_CR7","doi-asserted-by":"publisher","first-page":"922","DOI":"10.1016\/j.xphs.2023.01.029","volume":"112","author":"M Algorri","year":"2023","unstructured":"Algorri M, Cauchon NS, Christian T, O\u2019Connell C, Vaidya P. Patient-centric product development: a summary of select regulatory CMC and device considerations. J Pharm Sci. 2023;112:922\u201336.","journal-title":"J Pharm Sci"},{"key":"783_CR8","unstructured":"Patient Engagement for Medicines Development. The PFMD Book of Good Practices [Internet]. 2020 [cited 2025 Feb 26]. Available from: https:\/\/patientfocusedmedicine.org\/bogp\/2020\/the-book-of-good-practices.pdf"},{"key":"783_CR9","unstructured":"Dinboeck M, Noopur N, Krause A, Kaur S. Patient Involvement in Regulatory and Health Technology Assessment Processes: A Call for Enhanced Alignment. Value Outcomes Spotlight. 2024;27\u201333."},{"key":"783_CR10","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2022.948161","volume":"13","author":"T Wang","year":"2022","unstructured":"Wang T, McAuslane N, Goettsch WG, Leufkens HGM, De Bruin ML. Challenges and opportunities for companies to build HTA\/payer perspectives into drug development through the use of a dynamic target product profile. Front Pharmacol. 2022;13: 948161.","journal-title":"Front Pharmacol"},{"key":"783_CR11","unstructured":"Novartis AG. Novartis commitment to patients and caregivers 2025 factsheet [Internet]. 2025 [cited 2025 Mar 1]. 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JEC and MO report that their affiliated organizations have received support from Novartis, but that they did not receive payment for their contribution and participation in this publication. The views expressed in this work are those of the authors and do not necessarily represent those of their affiliated organizations.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}