94% of researchers rate our articles as excellent or good
Learn more about the work of our research integrity team to safeguard the quality of each article we publish.
Find out more
PERSPECTIVE article
Front. Med.
Sec. Regulatory Science
Volume 12 - 2025 | doi: 10.3389/fmed.2025.1481670
Valuing patients' high unmet need to drive innovative solutions for inherited retinal degenerations: Insights from the RIWC 2024 workshop on Value
Provisionally accepted
Nabin Paudel *
Avril Daly - Retina International, Dublin, Ireland
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's 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' lives.
Keywords: inherited retinal degeneration (IRD), value, innovation, Patient voice, regulation and health technology assessment
Received: 16 Aug 2024; Accepted: 17 Feb 2025.
Copyright: © 2025 Paudel and Daly. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Nabin Paudel, Retina International, Dublin, Ireland
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.