Engaging companies in rare disease research: New findings from the IRDiRC Chrysalis Task Force
The International Rare Diseases Research Consortium (IRDiRC)’s Chrysalis Task Force has published recent findings elaborating the different financial and nonfinancial factors which make rare disease research attractive to companies. By better understanding these factors, stakeholders such as funders, researchers, and patients can tailor their research activities to have the most significant impact possible.
IRDiRC is a global collaborative initiative which seeks to advance the understanding and impact of rare disease diagnosis and treatment on a global scale. Its Task Forces and Working Groups work in service of this goal, by identifying gaps and key issues in rare disease research, and producing guidelines, recommendations and resources to help overcome them. Formed in 2020, the Chrysalis Task Force was conceived as a link between industry, funders and advocates, with the ultimate goal of overcoming barriers specific to rare disease research through an understanding of the criteria for investment in this area.
The new findings draw on survey responses from various-sized companies with interests in rare disease research, as well as a series of targeted interviews. Using these methods, the Task Force was able to identify a number of factors which make rare disease research and development either more or less attractive. Some of the main factors identified which would encourage the initiation of new projects include the opportunity to be first in market, incentives such as accelerated marketing authorisation or market exclusivity, and the existence of patient registries to facilitate participant recruitment. On the other hand, factors such as the absence of advocacy organisations and challenges in collaborating effectively with academia were identified as barriers.
Top factors which would encourage companies to initiate new rare disease projects, with number of respondents (adapted from Beaverson et al, 2023)
These overall factors, in combination with additional findings on specific aspects of R&D such as drug development and market research, give insight into the steps rare disease stakeholders can take to make research more attractive to companies. They also shed light on how regulators can leverage incentives such as accelerated pathways and market exclusivity to encourage innovation. By identifying these barriers and facilitators to engaging companies in rare disease research, the Task Force is providing the rare disease community with an opportunity to more easily seek solutions for the ongoing high levels of unmet medical need.
