PUBLICATIONS

Publications on Orphan Drugs

  1. Jiao, Y, Yuan, F, Liu, T, Han, J, Sun, J, Chu, S et al.. Patent Analysis Perspectives on China's Orphan Drug R&D: Status and Technology Characteristics. Ther Innov Regul Sci. 2026; :. doi: 10.1007/s43441-026-00927-x. PubMed PMID:41697637 .
  2. van der Zanden, TM, Mooij, MG, de Hoog, M, de Wildt, SN. Beyond the label: Rethinking off-label drug use in paediatrics. Towards a scientifically grounded and safer future for paediatric pharmacotherapy. Br J Clin Pharmacol. 2026; :. doi: 10.1002/bcp.70461. PubMed PMID:41693205 .
  3. Nakai, K. Japan's Pharmaceutical Regulatory Reform to Overcome Drug Loss Issue. Ther Innov Regul Sci. 2026; :. doi: 10.1007/s43441-026-00928-w. PubMed PMID:41691137 .
  4. Bouwman, L, Leufkens, H, Sepodes, B, Torre, C. Safety population size and duration of exposure prior to approval of new medicines: A database analysis of medicines centralised approved in the European Union between 2011 and 2023. PLoS One. 2026;21 (2):e0342698. doi: 10.1371/journal.pone.0342698. PubMed PMID:41662378 PubMed Central PMC12885265.
  5. Manders, EA, van Der Wel, V, Schlingemann, R, Hollak, CEM, de Visser, SJ. Repurposing biosimilars, rethinking costs: a framework for sustainable drug pricing for repurposed bevacizumab for intravitreal injections. Eur J Health Econ. 2026; :. doi: 10.1007/s10198-025-01891-3. PubMed PMID:41653377 .
  6. Walter, E, Ghinelli, F, Goyer, I, Leone, M, Pirracchio, R. Cost-effectiveness of vasopressin in the treatment of septic shock: insights from a European societal perspective. J Med Econ. 2026;29 (1):319-333. doi: 10.1080/13696998.2026.2622854. PubMed PMID:41637478 .
  7. Papanicolas, I, Wouters, OJ, Sawaya, T, Han, J, Wei, J, Naci, H et al.. Review Times For New Drugs And Submission Delays Among The FDA And 4 International Regulators, 2014-22. Health Aff (Millwood). 2026;45 (2):184-192. doi: 10.1377/hlthaff.2025.00595. PubMed PMID:41628382 .
  8. Cabello, JF, Salinas, R. Global Access to IMD Drugs: Bridging the Gap Between High-Income Countries and Latin America. J Inherit Metab Dis. 2026;49 (2):e70152. doi: 10.1002/jimd.70152. PubMed PMID:41621834 .
  9. Akodad, S, Haon, B, Rochaix, L, Stevens, H. Orphan medical devices: addressing the regulatory and access gaps in the EU and US. Front Public Health. 2025;13 :1729821. doi: 10.3389/fpubh.2025.1729821. PubMed PMID:41613088 PubMed Central PMC12847439.
  10. Kaló, Z, Petrova, G, Németh, B, Bonča, PD, Delnoij, D, Bennett, A et al.. Transferability of Real World Evidence to Support HTA Recommendations in Lower Income European Countries. Health Sci Rep. 2026;9 (2):e71534. doi: 10.1002/hsr2.71534. PubMed PMID:41608382 PubMed Central PMC12836872.
  11. Li, Y, Zhang, B, Zhang, Z, Yan, W, Wang, H, Xu, X et al.. Inhibition of the RORC/GPX4 mediated ferroptosis regulatory axis suppresses tumor growth and alleviates enzalutamide resistance in prostate cancer. Cell Mol Biol Lett. 2026;31 (1):11. doi: 10.1186/s11658-025-00846-z. PubMed PMID:41580593 PubMed Central PMC12837284.
  12. Alperin, C, Nguyen, N, Duffey, M, Clark, EH. Priced Out of Treatment: The Exorbitant Cost of Antiparasitic Drugs in the United States. Open Forum Infect Dis. 2026;13 (1):ofaf784. doi: 10.1093/ofid/ofaf784. PubMed PMID:41567261 PubMed Central PMC12817971.
  13. Camilleri, M, Parkman, HP. Review Article: Drug Approval for Gastroparesis-Suggestions for Improving the Process for Positive Results. Aliment Pharmacol Ther. 2026; :. doi: 10.1111/apt.70544. PubMed PMID:41566871 .
  14. Martin, P, Ding, J, Hanchard, M. Why is the pharmaceutical industry investing in targeted therapies? The emergence of "premium pharma". Soc Sci Med. 2026;392 :118888. doi: 10.1016/j.socscimed.2025.118888. PubMed PMID:41548522 .
  15. Hong, EH, Hyeong, J, Ahn, JH, Han, Y, Kim, S, Kim, SM et al.. Limosilactobacillus reuteri alleviates psoriasis via aryl hydrocarbon receptor-mediated regulation of Interkeukin-17A. Int Immunopharmacol. 2026;172 :116194. doi: 10.1016/j.intimp.2026.116194. PubMed PMID:41548440 .
  16. Minder, AE, Barman-Aksözen, J. From darkness to light: Case report on afamelanotide-treatment in a 9-year-old child with erythropoietic protoporphyria. JAAD Case Rep. 2026;68 :6-8. doi: 10.1016/j.jdcr.2025.11.022. PubMed PMID:41542313 PubMed Central PMC12800419.
  17. Abdallah, K, Blonda, A, Godman, B, Huys, I, Claes, K, Denier, Y et al.. Navigating market access after conditional reimbursement: a communication roadmap for disinvesting orphan drugs. Int J Technol Assess Health Care. 2026;42 (1):e15. doi: 10.1017/S0266462326103444. PubMed PMID:41540590 .
  18. Havlik, J, Isaac, S, Radovan, C, Ostacher, MJ, Smith, D, Rhee, TG et al.. Innovation in Psychiatric Drug Development: A Quantitative Analysis of FDA-Approved Psychiatric Drugs, 2012-2024. J Clin Psychiatry. 2026;87 (1):. doi: 10.4088/JCP.25m16063. PubMed PMID:41532845 .
  19. Avxentyev, NA, Makarov, AS, Germanenko, OY, Vlodavets, DV, Kuzenkova, LM. [Pharmacoeconomic evaluation of spinal muscular atrophy therapy in patients with four SMN2 copies diagnosed through newborn screening]. Zh Nevrol Psikhiatr Im S S Korsakova. 2025;125 (12):188-199. doi: 10.17116/jnevro2025125121188. PubMed PMID:41524367 .
  20. Khalifa, A, Malden, DE, Ratmann, O, Chen, Y, Grabowski, MK, Chang, LW et al.. Incidence and prevalence of orphanhood in Rakai, Uganda: a population-based cohort study, 1995-2022. Lancet Glob Health. 2026;14 (2):e251-e260. doi: 10.1016/S2214-109X(25)00440-1. PubMed PMID:41519154 .
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