{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,27]],"date-time":"2026-06-27T14:26:03Z","timestamp":1782570363566,"version":"3.54.5"},"reference-count":20,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T00:00:00Z","timestamp":1741046400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Med."],"abstract":"<jats:p>The importance of patient involvement in the therapeutic development ecosystem is being increasingly recognized, however not all stakeholders are fully aware of the unique perspective that patients can bring to these platforms. In this perspective article that is based on a workshop organized at the Retina International World Congress (RIWC) in Dublin in June 2024, we discuss the interpretation of value from patient\u2019s perspective, challenges in the development of innovative medicines such as cell and gene therapies for Inherited Retinal Degenerations, the resources required to bring a drug to market, and the need to incorporate patient voice throughout the drug development pathway from pre-clinical studies to clinical trial designs, regulatory and health technology assessments decisions. We hope that this article will increase awareness among all relevant stakeholders including patients, clinicians, scientists, developers, regulators, decision makers and industry representatives on the importance of involving patients in the developmental lifecycle of novel therapies so that therapies are developed that make a meaningful improvement in patients\u2019 lives.<\/jats:p>","DOI":"10.3389\/fmed.2025.1481670","type":"journal-article","created":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T07:09:11Z","timestamp":1741072151000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":1,"title":["Valuing patients\u2019 high unmet need to drive innovative therapies for inherited retinal degenerations: insights from the RIWC 2024 workshop on value"],"prefix":"10.3389","volume":"12","author":[{"given":"Nabin","family":"Paudel","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Avril","family":"Daly","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2025,3,4]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1186\/s40900-018-0087-1","article-title":"Patient and public involvement in reducing health and care research waste","volume":"4","author":"Minogue","year":"2018","journal-title":"Res Involv Engagem"},{"key":"ref2","doi-asserted-by":"publisher","first-page":"e12454","DOI":"10.5812\/ircmj.12454","article-title":"Patient involvement in health care decision making: a review","volume":"16","author":"Vahdat","year":"2014","journal-title":"Iran Red Crescent Med J"},{"key":"ref3","doi-asserted-by":"publisher","first-page":"929","DOI":"10.1177\/2168479015580384","article-title":"Partnering with patients in the development and lifecycle of medicines: a call for action","volume":"49","author":"Hoos","year":"2015","journal-title":"Ther Innov Regul Sci"},{"key":"ref4","article-title":"RIWC24, hosted by Fighting Blindness","year":"2024"},{"key":"ref5","doi-asserted-by":"publisher","first-page":"101","DOI":"10.2147\/JMDH.S122383","article-title":"Value as the key concept in the health care system: how it has influenced medical practice and clinical decision-making processes","volume":"10","author":"Marzorati","year":"2017","journal-title":"J Multidiscip Healthc"},{"key":"ref6","doi-asserted-by":"publisher","first-page":"286","DOI":"10.1016\/j.jval.2016.11.014","article-title":"Value to whom? 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Seminars in radiation oncology","volume":"33","author":"Narra","year":"2023","journal-title":"WB Saunders"},{"key":"ref14","doi-asserted-by":"publisher","first-page":"a041299","DOI":"10.1101\/cshperspect.a041299","article-title":"Mobility testing and other performance-based assessments of functional vision in patients with inherited retinal disease","volume":"13","author":"Chung","year":"2023","journal-title":"Cold Spring Harb Perspect Med"},{"key":"ref15","doi-asserted-by":"publisher","first-page":"1460","DOI":"10.1016\/j.ophtha.2021.03.031","article-title":"Durability of voretigene neparvovec for biallelic RPE65-mediated inherited retinal disease: phase 3 results at 3 and 4 years","volume":"128","author":"Maguire","year":"2021","journal-title":"Ophthalmology"},{"key":"ref16","article-title":"FDA patient-focused drug development guidance series for enhancing the incorporation of the Patient\u2019s voice in medical product development and regulatory decision making","year":"2024"},{"key":"ref17","article-title":"ICH reflection paper - Proposed ICH guideline work to advance patient focused drug development (PFDD)","year":"2021"},{"key":"ref18","doi-asserted-by":"publisher","first-page":"20","DOI":"10.1016\/j.jhealeco.2016.01.012","article-title":"Innovation in the pharmaceutical industry: new estimates of R&D costs","volume":"47","author":"DiMasi","year":"2016","journal-title":"J Health Econ"},{"key":"ref19","article-title":"EFPIA patients WAIT indicator 2021 survey","author":"Newton","year":"2022"},{"key":"ref20","article-title":"Implementation of the Regulation on health technology assessment","year":"2024"}],"container-title":["Frontiers in Medicine"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fmed.2025.1481670\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T07:09:13Z","timestamp":1741072153000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fmed.2025.1481670\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,3,4]]},"references-count":20,"alternative-id":["10.3389\/fmed.2025.1481670"],"URL":"https:\/\/doi.org\/10.3389\/fmed.2025.1481670","relation":{},"ISSN":["2296-858X"],"issn-type":[{"value":"2296-858X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,3,4]]},"article-number":"1481670"}}