In:
Therapeutic Advances in Rare Disease, SAGE Publications, Vol. 4 ( 2023-01)
Abstract:
Plain language summary Making rare disease research attractive to companies The International Rare Diseases Research Consortium (IRDiRC) is an international initiative that aims to speed the diagnosis and treatment of rare diseases through research. The IRDiRC Chrysalis Task Force, comprised of thought leaders from companies, patient advocacy groups, regulatory agencies, and research funders, identified key factors that make rare disease research and development attractive to companies. The Task Force distributed a survey to companies with varied investment portfolios and interests in rare disease research, followed by in-depth interviews based on the survey results. The survey and interview respondents identified both attractive factors and barriers to rare disease research and development. The concept of Return On Investment was used to frame discussion of factors that companies weighed differently, depending on a number of issues that were a function of both the company itself and outside factors. The identified challenges can be addressed by funders, academic researchers, patients and their families, companies, regulators, and payers, which hopefully will lead to significant advances in the research and development of treatments for rare diseases.
Type of Medium:
Online Resource
ISSN:
2633-0040
,
2633-0040
DOI:
10.1177/26330040231188979
Language:
English
Publisher:
SAGE Publications
Publication Date:
2023
detail.hit.zdb_id:
3058592-2
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