The time from target identification to new drug approval is often measured in decades. This can be even more challenging for rare diseases. Indeed, 95% of rare diseases do not have a specific therapy approved. Coordinated efforts to support research along the drug development pipeline can provide long term and comprehensive support to enable scientific breakthroughs for rare diseases. However, this requires coordination across multiple stakeholders. The present article analyzes the funding efforts of four major federal and philanthropic organizations to accelerate the advancement of MEK inhibitors to human clinical trials for NF1-associated tumors.
The time from identifying a drug target to a new drug approval is often measured in decades and can take even longer for therapies to treat rare diseases. In fact, 95% of rare diseases do not have a specific therapy approved at all. Coordinated efforts to augment the drug development pipeline along with long-term and comprehensive support that enable scientific breakthroughs for rare diseases is possible, but it requires integration across multiple stakeholders. With the recently announced granted acceptance from the FDA to file status of the AstraZeneca and MSD (Merck) application for the MEK inhibitor selumetinib to treat plexiform neurofibromas in children, a major milestone towards the first-ever approved treatment for people with Neurofibromatosis Type 1 (NF1) associated tumors is closer than ever. This study analyzes the coordinated funding efforts of four federal and philanthropic organizations to advance drug development for NF1 associated tumors and discusses how these organizations collaborated and evolved practices to optimize funding and research support. This model could be replicated for other rare diseases if funders are able to take an active, coordinated approach to identify gaps, tools, and infrastructures most needed for that specific disease area.
Citation Format: Salvatore La Rosa, Annette C. Bakker, Vidya Browder, Jaishri O. Blakeley, Sharad K. Verma, Ling M. Wong, Jill Morris, Naba Bora. Delivering on the Vision of Bench to Bedside: A Rare Disease Funding Community Collaboration to Develop Effective Therapies for Neurofibromatosis Type 1 Tumors [abstract]. In: Proceedings of the Annual Meeting of the American Association for Cancer Research 2020; 2020 Apr 27-28 and Jun 22-24. Philadelphia (PA): AACR; Cancer Res 2020;80(16 Suppl):Abstract nr 2057.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.