Beaverson Katherine L, Julkowska Daria, Letinturier Mary Catherine V, Aartsma-Rus Annemieke, Austin Jennifer, Bueren Juan, Frost Simon, Hamamura Misako, Larkindale Jane, LaRosa Greg, Magenheim Rita, Merico Annamaria, Pasmooij Anna Maria Gerdina, Pirard Vinciane, Ekow Thomford Nicholas, Wada Michihiko, Wong-Rieger Durhane, Hartman Adam L
Pfizer, Inc, Cambridge, MA, USA.
IRDiRC Scientific Secretariat, French National Institute of Health and Medical Research (Inserm), Paris, France.
Ther Adv Rare Dis. 2023 Jul 29;4:26330040231188979. doi: 10.1177/26330040231188979. eCollection 2023 Jan-Dec.
The International Rare Diseases Research Consortium (IRDiRC) is an international initiative that aims to use research to facilitate rapid diagnosis and treatment of rare diseases.
IRDiRC launched the Chrysalis Task Force to identify key financial and nonfinancial factors that make rare disease research and development attractive to companies.
The Chrysalis Task Force was comprised of thought leaders from companies, patient advocacy groups, regulatory agencies, and research funders. The Task Force created a survey that was distributed to companies of different sizes with varied investment portfolios and interests in rare disease research. Based on the survey results, the Task Force then conducted targeted interviews.
The survey and interview respondents identified several factors that make rare disease research and development attractive (e.g. a good understanding of the underlying biology) as well as barriers (e.g. absence of an advocacy organization representing the affected community's needs). The concept of Return On Investment allowed the exploration of factors that were weighed differently by survey and interview respondents, depending on a number of intrinsic and extrinsic issues.
The Chrysalis Task Force identified factors attributable to rare disease research and development that may be of interest to and actionable by funders, academic researchers, patients and their families, companies, regulators, and payers in the medium term to short term. By addressing the identified challenges, involved parties may seek solutions to significantly advance the research and development of treatments for rare diseases.
国际罕见病研究联盟（IRDiRC）是一项国际倡议，旨在通过研究促进罕见病的快速诊断和治疗。
IRDiRC发起了蝶变特别工作组，以确定使罕见病研发对公司具有吸引力的关键财务和非财务因素。
蝶变特别工作组由来自公司、患者权益倡导组织、监管机构和研究资助者的思想领袖组成。该特别工作组创建了一项调查，并将其分发给不同规模、对罕见病研究有不同投资组合和兴趣的公司。根据调查结果，特别工作组随后进行了有针对性的访谈。
调查和访谈受访者确定了若干使罕见病研发具有吸引力的因素（例如对基础生物学的充分理解）以及障碍（例如缺乏代表受影响群体需求的倡导组织）。投资回报率的概念使得能够探索因一些内在和外在问题而在调查和访谈受访者中被不同权衡的因素。
蝶变特别工作组确定了罕见病研发相关的因素，这些因素在中期到短期内可能会引起资助者、学术研究人员、患者及其家属、公司、监管机构和支付方的兴趣并可供他们采取行动。通过应对已确定的挑战，相关各方可以寻求解决方案，以显著推进罕见病治疗的研发。
