Funding Models to Support the Spectrum of Rare Disease Research and Development


The successful development of therapies for any disease requires support from early stages (basic/fundamental research), through more mature preclinical, translational, or early clinical stages, then more mature clinical stages, and finally, post-marketing studies. How different funders decide when to fund at a given stage in a treatment’s development is fairly opaque. Knowledge of the factors that contribute to this process might help other funders understand better how to facilitate the development of these treatments.  

The IRDiRC Chrysalis Task Force addressed some of these questions for companies but led to the realization that this knowledge was lacking for other types of funders. Furthermore, it is unclear how the decisions of one type of investor might impact the decisions of other funders or the funding ecosystem for rare diseases. A better understanding of the landscape (and even a recognition of the variability) would likely ultimately benefit those living with rare diseases. Both successes and failures might be very informative. 


  1.  Identify key motivating factors for different types of funders of rare disease research – why, when, etc. This includes questions such as, what can funders do to ensure that projects they fund will continue their development at stages where they do not provide support? 

  1. Identify how different types of funders decide at which point in a research study’s lifecycle they will provide support. This might include discussions about different types of funding instruments (and perhaps identify novel multi-modal mechanisms that could be tested in the future). 

  1. Identify the key influencing factors for effective public-private partnerships at different stages of a treatment’s life cycle.  

  1. Identify models of public-private partnerships, including means of sharing information (with attention to tech transfer issues and regulatory requirements). 


  • Recruitment and Assembly of the Task Force Members (Q1 2023). 
  • Literature review and key opinion leader (KOL) interviews to identify factors in the objectives (Q2-Q3 2023). 
  • Task force research into, and description of, model systems that best identify continua of funding and programmatic support for rare disease research programs (Q3 2023). 
  • Publication (Q4 2023). 

Members (19)

  • Adam Hartman – National Institute of Neurological Disorders and Stroke (NINDS) at NIH, USA (Co-Chair)
  • Lucia Monaco – Former Fondazione Telethon, Italy (Co-Chair)
  • Naohika Aketa – Keio University Hospital, Japan
  • Dimitrios Athanassiou – World Duchenne Organization, Greece
  • Jida el Hajjar – Canadian Organization of Rare Disorders (CORD), Canada
  • Simon Frost – Tiber Capital Group, USA
  • Alicia Granados – Sanofi, Spain
  • Anthony Haight – Sparx Biopharmaceuticals Corp., USA
  • Anneliene Jonker – University of Twente & Duchenne Parent Project, The Netherlands
  • Susan Kahn – SRK Consulting, USA
  • Sukhun Kang – University of California Santa Barbara, USA
  • Persefoni Kritikou – HCD Economics, Greece
  • Christina Kyriakopoulou – European Commission, Belgium
  • Eileen Li – RareStone Group and Chinese Organization for Rare Disorders, China
  • Christopher McMaster – Canadian Institutes for Health Research (CHIR), Canada
  • Samantha Parker – InnoSkel, France
  • Daniel Scherman – Foundation for Rare Diseases, France
  • Nivedita Valence – Pharmanovia, UK
  • Samuel Wiafe – Rare Disease Ghana Initiative, Ghana