Chrysalis Project Task Force

Art by Davide Bonazzi www.davidebonazzi.com

Introduction

This project will identify key criteria that would make rare disease research more attractive to industry for research and development.
In order for industry to initiate or continue research into rare diseases, it must overcome barriers specific to the nature of rare disease research. We expect that certain minimum conditions must be fulfilled and that these will be both financial and non-financial.
Funders of rare disease research have a double effect of meeting their own goals as well as reducing the barriers to further research. This may be via natural history studies, patient registries, epidemiology studies or by reducing the risks of investment.
The Chrysalis project seeks to explore these barriers, as well as the criteria for investment into rare disease research and development, by serving as a link between industry, funders and advocates.

Objectives

The objectives of the Chrysalis Project will be threefold:

1. Identify the key criteria that determine the attractiveness of rare disease
   research to industry.
2. Identify gaps in the current funding opportunity landscape to develop the
   criteria identified in #1.
3. Identify other non-financial barriers related to the attractiveness of
   meeting the criteria identified in #1

Plans and timeline

• Formation of 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).
• Survey analysis followed by design and execution of interviews (Q2-Q3 2021)
• Identification of funding opportunity gaps involving the key criteria (Q3 2021).
• Publication and dissemination of results (Q4 2021)

Members

• Adam Hartman (Chair) – National institute of neurological disorders and stroke
  (NINDS) National Institute for Health, USA
• Katherine Beaverson (Chair) – Pfizer, USA
• Eileen Li – CORD, China
• Diego Ardigo Chiesi Farmaceutici S.p.A., Italy
• Annemieke Aartsma-Rus Dutch Center for RNA Therapeutics, Leiden
  UniversityMedical Center, Leiden, Netherlands
• Annamaria Merico Fondazione Telethon, Italy
• Marjon Pasmooij Medicines Evaluation Board of the Netherlands,
  Netherlands
• Cristina de Min SOBI
• Annalisa Trama Istituto Nazionale dei Tumori (National Cancer Institute),
  Milan, Italy
• Prof Juan Bueren, PhD Research Centre for Energy, Environment and
  Technology (CIEMAT), Madrid, Spain
• Vinciane Pirard M.D. Orchard Therapeutics; Brussels
• Rita Magenheim PAREXEL Interntional ePAG representative at European
  Reference Network GENTURIS ; Germany
• Michihiko Wada Clinical & Translational Research Center, Keio University
• Misako Hamamura Head of Takeda’s Rare Disease Business Unit
• Simon Frost Tiber Capital Group, LLC; US
• Jane Larkindale Duchenne Regulatory Science Consortium, Rare Disease
  Cures Accelerator- Data and Analytics Platform”
• Gregory LaRosa Pfizer, USA
• Jayden Rodgers Linear Clinical Research Ltd
• Nicholas Ekow Thomford University of Cape Coast, Department of Medical
  Biochemistry, School of Medical Sciences
• Jennifer Austin International Alliance of Dermatology Patient
  Organizations (aka Globalskin)