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REVIEW article

Front. Pharmacol.
Sec. Experimental Pharmacology and Drug Discovery
Volume 15 - 2024 | doi: 10.3389/fphar.2024.1441807
This article is part of the Research Topic What does Experimental Pharmacology and Drug Discovery look like in 2035? View all 6 articles

Unlocking the full potential of rare disease drug development: exploring the Not-for-Profit sector's contributions to drug development and access

Provisionally accepted
Stefano Vavassori Stefano Vavassori 1*Sean Russel Sean Russel 2,3Celeste Scotti Celeste Scotti 1Stefano Benvenuti Stefano Benvenuti 1*
  • 1 Telethon Foundation, Rome, Italy
  • 2 Fondazione Telethon ETS, Milan, Italy
  • 3 PrimeRA Pharma Partners, LLP, Nottingham, United Kingdom

The final, formatted version of the article will be published soon.

    This commentary provides a comprehensive overview of the challenges and opportunities in the field of drug development for rare diseases and especially of gene therapy products for ultra-rare diseases. It discusses the limited market size, reimbursement and scientific complexities that deter pharmaceutical investment in this field. Highlighting the pivotal role of charitable organizations like Fondazione Telethon, it showcases their efforts in funding research and ensuring access to innovative therapies. This commentary also addresses the challenges in therapy distribution, particularly regarding sustainability and global access. It outlines Fondazione Telethon's operational model to try to address these challenges. Finally, it appeals to governments and regulatory bodies to implement policies and incentives aimed at further fostering innovation and accessibility in rare disease drug development and access.

    Keywords: drug developement, NGO = nongovernmental organization, Rare disease (RD), Drug access, Authorities, Gene therapy (GT)

    Received: 31 May 2024; Accepted: 29 Jul 2024.

    Copyright: © 2024 Vavassori, Russel, Scotti and Benvenuti. 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:
    Stefano Vavassori, Telethon Foundation, Rome, Italy
    Stefano Benvenuti, Telethon Foundation, Rome, Italy

    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.