The overarching goal of this project is to identify key criteria that would make rare diseases research more attractive to industry for research and development. It is expected that some specific (minimum) conditions must be fulfilled to facilitate the uptake by industry and/or to maximize the potential of already existing research. Identification of key criteria that would make industry research and development in rare diseases attractive (in terms of investments of time and resources) is therefore critical to ensure an ongoing pipeline of progress. Funders (both public and private) have scientific programmatic interests in various diseases but the work that they fund can improve the barriers to entry for therapeutic research investments (e.g., via natural history studies, patient registries, epidemiology studies) or to “de-risk” critical aspects at pivotal stages of development. The proposed task force would serve as a critical link between industry, funders, and advocates in accomplishing the stated goals
The objectives of the Chrysalis Project will be threefold:
- Identify key criteria that would make rare diseases research more attractive to industry for research and development.
- Identify gaps in the current funding opportunity landscape to develop the criteria identified in #1.
- Identify other non-financial barriers related to the attractiveness of meeting the criteria identified in #1
Plans and timeline
- Formation of a task force with interested members of the Constituent and Scientific Committees; open call for expression of interests for external experts (Q1 2020).
- Virtual meetings and in-person meetings to develop the process for achieving the stated goals (Q2 2020 – Q2 2021).
- Development and execution of a survey process for CCC Constituent members to identify the key criteria (Q4 2020 – Q2 2021).
- Identification of funding opportunity gaps involving the key criteria (Q1-Q2 2021).