PUBLICATIONS

Publications on Orphan Drugs

  1. GBD 2021 Europe Life Expectancy Collaborators. Changing life expectancy in European countries 1990-2021: a subanalysis of causes and risk factors from the Global Burden of Disease Study 2021. Lancet Public Health. 2025; :. doi: 10.1016/S2468-2667(25)00009-X. PubMed PMID:39983748 .
  2. Windecker, S, Fraser, AG, Szymanski, P, Gilard, M, Lüscher, TF, Abid, L et al.. Priorities for medical device regulatory approval: a report from the European Society of Cardiology Cardiovascular Round Table. Eur Heart J. 2025; :. doi: 10.1093/eurheartj/ehaf069. PubMed PMID:39977258 .
  3. Topouzis, S, Papapetropoulos, A, Alexander, SPH, Cortese-Krott, M, Kendall, DA, Martemyanov, K et al.. Novel drugs approved by the EMA, the FDA and the MHRA in 2024: A year in review. Br J Pharmacol. 2025; :. doi: 10.1111/bph.17458. PubMed PMID:39971274 .
  4. Cacoub, E, Lefebvre, NB, Milunov, D, Sarkar, M, Saha, S. Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics. Front Public Health. 2025;13 :1520467. doi: 10.3389/fpubh.2025.1520467. PubMed PMID:39963479 PubMed Central PMC11830808.
  5. Yuanyuan, AN, Wang, L, Yanjie, LI, Yanchun, W, Xiaobin, R, Hongbing, HE et al.. Effect and mechanism of Sanqi in treating periodontitis. J Tradit Chin Med. 2025;45 (1):66-75. doi: 10.19852/j.cnki.jtcm.20241111.001. PubMed PMID:39957160 PubMed Central PMC11764944.
  6. Louie, AD, Huntington, KE, Lee, Y, Mompoint, J, Wu, LJ, Lee, S et al.. TRAIL receptor agonist TLY012 in combination with PD-1 inhibition promotes tumor regression in an immune-competent mouse model of pancreatic ductal adenocarcinoma. Am J Cancer Res. 2025;15 (1):286-298. doi: 10.62347/ROAT5658. PubMed PMID:39949937 PubMed Central PMC11815385.
  7. Kummar, S, Bui, N, Messersmith, WA, Whiting, J, Portnoy, M, Lim, A et al.. Nirogacestat-the pathway to approval of the first treatment for desmoid tumors, a rare disease. Ther Adv Rare Dis. 2025;6 :26330040251317546. doi: 10.1177/26330040251317546. PubMed PMID:39944188 PubMed Central PMC11815814.
  8. Enya, K, Kageyama, I, Kobayashi, Y, Lim, Y, Sengoku, S, Kodama, K et al.. Evolving Research and Development Landscape for Rare Diseases: Growing Concerns Over Orphan Drug Lag in Japan. Clin Pharmacol Ther. 2025; :. doi: 10.1002/cpt.3553. PubMed PMID:39903453 .
  9. E Abdel Aziz, S, El-Nakib, HE, Schaletzky, J, Ahmed, NS. Analytical Methodologies for Anti-Infective Orphan Drugs: A Comprehensive Review of FDA Approvals (2013-2023) Part 1. Crit Rev Anal Chem. 2025; :1-26. doi: 10.1080/10408347.2025.2459721. PubMed PMID:39899335 .
  10. Yoon, CH, Ross, MI, Gastman, BR, Luke, JJ, Ascierto, PA, Long, GV et al.. Adjuvant Pembrolizumab in Stage II Melanoma: Outcomes by Primary Tumor Location in the Randomized, Double-Blind, Phase III KEYNOTE-716 Trial. Ann Surg Oncol. 2025; :. doi: 10.1245/s10434-024-16642-6. PubMed PMID:39893343 .
  11. Grilley, BJ, Hume, A, Galipeau, J. Sickle cell disease gene therapy drug expenses and reimbursement: a litmus test for commercial pricing strategy and patient access for curative therapies. Cytotherapy. 2025; :. doi: 10.1016/j.jcyt.2025.01.008. PubMed PMID:39891631 .
  12. Li, J, Li, L, Hou, C, Tian, Z, Zhou, Y, Zhang, J et al.. Discovery of the first potent ROR1 degrader for the treatment of non-small cell lung cancer. Eur J Med Chem. 2025;286 :117325. doi: 10.1016/j.ejmech.2025.117325. PubMed PMID:39889450 .
  13. Mattingly, TJ 2nd, Happe, LE, Cranston, L. Emerging trends in managed care pharmacy: A mixed-method study. J Manag Care Spec Pharm. 2025;31 (2-a Suppl):S2-S10. doi: 10.18553/jmcp.2025.31.2-a.s2. PubMed PMID:39888855 PubMed Central PMC11785360.
  14. Gentilini, A, Neez, E, Wong-Rieger, D. Rare Disease Policy in High-Income Countries: An Overview of Achievements, Challenges, and Solutions. Value Health. 2025; :. doi: 10.1016/j.jval.2024.12.009. PubMed PMID:39880194 .
  15. Mattingly, TJ 2nd, Happe, LE, Cranston, L. Emerging trends in managed care pharmacy: A mixed-method study. J Manag Care Spec Pharm. 2025;31 (1-b Suppl):S2-S10. doi: 10.18553/jmcp.2025.31.1-b.s2. PubMed PMID:39873561 PubMed Central PMC11780272.
  16. Abozaid, GM, Kerr, K, Alomary, H, Al-Omar, HA, McKnight, A. Global insight into rare disease and orphan drug definitions: a systematic literature review. BMJ Open. 2025;15 (1):e086527. doi: 10.1136/bmjopen-2024-086527. PubMed PMID:39863413 PubMed Central PMC11784410.
  17. Campbell, K, Neul, JL, Lieberman, DN, Berry-Kravis, E, Benke, TA, Fu, C et al.. A randomized, placebo-controlled, cross-over trial of ketamine in Rett syndrome. J Neurodev Disord. 2025;17 (1):4. doi: 10.1186/s11689-025-09591-y. PubMed PMID:39856538 PubMed Central PMC11761732.
  18. Ursino, MG, Milano, A, De Angelis, FV, Alessi, E, Trotta, F. Pricing for Multi-Indication Drugs in the Italian Regulatory Context. Pharmacoecon Open. 2025; :. doi: 10.1007/s41669-024-00555-x. PubMed PMID:39847272 .
  19. Ka, K. Orphan drugs in epilepsy treatment- a review. Epilepsy Behav. 2025;164 :110238. doi: 10.1016/j.yebeh.2024.110238. PubMed PMID:39826182 .
  20. Hong, J, Bae, EY, Cha, S, Lee, J. The value-for-money assessment and funding arrangements for high-priced drugs in an era of uncertainty: a comparative analysis of national health technology assessment agencies in South Korea, England, Australia, and Canada. BMC Health Serv Res. 2025;25 (1):74. doi: 10.1186/s12913-025-12207-9. PubMed PMID:39810177 PubMed Central PMC11731375.
Search PubMed