Development of medicines for rare diseases and inborn errors of metabolism: Toward novel public–private partnerships

Noa Rosenberg, Nina N. Stolwijk, Sibren van den Berg, Joris J. Heus, Vincent van der Wel, Teun van Gelder, Annet M. Bosch, Saco J. de Visser, Carla E. M. Hollak

Research output: Contribution to journalReview articleAcademicpeer-review

3 Citations (Scopus)

Abstract

Medicine development for rare diseases, including inborn errors of metabolism (IEMs) is challenging. Many academic innovations fail to reach the patient, either by stranding in the translational stage or due to suboptimal patient access related to pricing or uncertain effectiveness. Expanding and solidifying the role of the academic in public–private partnerships (PPPs) may present an innovative solution to help overcome these complexities. This narrative review explores the literature on traditional and novel collaborative approaches to medicine development for rare diseases and analyzes examples of PPPs, with a specific focus on IEMs. Several academic institutions have introduced guidelines for socially responsible licensing of innovations for private development. The PPP model offers a more integrative approach toward academic involvement of medicine development. By sharing risks and rewards, failures in the translational stage can be mutually absorbed. If socially responsible terms are not included, however, high pricing can impede patient access. Therefore, we propose a framework for socially responsible PPPs aimed at medicine development for metabolic disorders. This socially responsible PPP framework could stimulate successful and accessible medicine development for IEMs as well as other rare diseases if the establishment of such collaborations includes terms securing joint data ownership and evidence generation, fast access, and socially responsible pricing.

Original languageEnglish
Pages (from-to)806-816
Number of pages11
JournalJournal of Inherited Metabolic Disease
Volume46
Issue number5
Early online date2023
DOIs
Publication statusPublished - Sept 2023

Keywords

  • medicine development
  • orphan medicinal products
  • public–private partnerships
  • rare diseases
  • socially responsible pricing

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