REFERENCES
1. Ng QX, Ong C, Chan KE, et al. Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review. Health Econ Rev 2024;14:42.
2. Austin CP, Cutillo CM, Lau LPL, et al. Future of rare diseases research 2017-2027: an IRDiRC perspective. Clin Transl Sci 2018;11:21-7.
3. Monaco L, Zanello G, Baynam G, et al. Research on rare diseases: ten years of progress and challenges at IRDiRC. Nat Rev Drug Discov 2022;21:319-20.
4. Beaverson KL, Julkowska D, Letinturier MCV, et al. The IRDiRC chrysalis task force: making rare disease research attractive to companies. Ther Adv Rare Dis 2023;4:26330040231188979.
5. Levine JA, Stemitsiotis C. De-risking rare disease acquisitions: a win-win-win for patients, biotech and investors. Nat Rev Drug Discov 2024;23:10-1.
6. Jekunen A. Decision-making in product portfolios of pharmaceutical research and development-managing streams of innovation in highly regulated markets. Drug Des Devel Ther 2014;2014:2009-16.
7. Koleva-Kolarova R, Buchanan J, Vellekoop H, et al. Financing and reimbursement models for personalised medicine: a systematic review to identify current models and future options. Appl Health Econ Health Policy 2022;20:501-24.
8. Postma MJ, Noone D, Rozenbaum MH, et al. Assessing the value of orphan drugs using conventional cost-effectiveness analysis: is it fit for purpose? Orphanet J Rare Dis 2022;17:157.
9. Khachatryan A, Read SH, Madison T. External control arms for rare diseases: building a body of supporting evidence. J Pharmacokinet Pharmacodyn 2023;50:501-6.
10. Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis 2011;6:42.
14. Au service de la science. Agence nationale de la recherche. Accessed May 16, 2024. Available from: https://anr.fr/ [Last accessed on 2 Sep 2024].
15. Amselem S, Gueguen S, Weinbach J, Clement A, Landais P. RaDiCo Program. RaDiCo, the French national research program on rare disease cohorts. Orphanet J Rare Dis 2021;16:454.
18. Patterson AM, O'Boyle M, VanNoy GE, Dies KA. Emerging roles and opportunities for rare disease patient advocacy groups. Ther Adv Rare Dis 2023;4:26330040231164425.
20. Rennane S, Baker L, Mulcahy A. Estimating the cost of industry investment in drug research and development: a review of methods and results. Inquiry 2021;58:469580211059731.
21. López JC, Suojanen C. Harnessing venture philanthropy to accelerate medical progress. Nat Rev Drug Discov 2019;18:809-10.