PUBLICATIONS

Publications on Orphan Drugs

  1. Abu Esba, LC, Yousef, CC, Khan, MA, Balhareth, S, Al-Omar, HA, Al-Najjar, A et al.. Challenges in evaluation and reimbursement of drugs for rare diseases in Saudi Arabia: a Delphi expert consensus. J Pharm Policy Pract. 2025;18 (1):2575040. doi: 10.1080/20523211.2025.2575040. PubMed PMID:41195237 PubMed Central PMC12584895.
  2. Balakumar, P, Mulukuri, NVLS, Jagadeesh, G. A map of molecular drug targets and therapeutics for the US FDA-approved drugs: The impact of expedited regulatory pathways and first-in-class drug approvals on drug innovation. Pharmacol Ther. 2025; :108945. doi: 10.1016/j.pharmthera.2025.108945. PubMed PMID:41187816 .
  3. Hirano, Y, Yuasa, A, Matsumoto, K, Nakamura, H. Investigation of Differences Between Manufacturers and Public Analyses in Health Technology Assessment in Japan. J Health Econ Outcomes Res. 2025;12 (2):173-182. doi: 10.36469/001c.144530. PubMed PMID:41185668 PubMed Central PMC12579898.
  4. Oany, AR, Upadhyay, S, Tsui, WNT, Hailemariam, A, Latka, S, Landua, JD et al.. Orphan nuclear receptor 4A1 (NR4A1) and NR4A2 are endogenous regulators of CD71 and their ligands induce ferroptosis in breast cancer. Cell Death Dis. 2025;16 (1):776. doi: 10.1038/s41419-025-08143-5. PubMed PMID:41184246 PubMed Central PMC12583510.
  5. Tsitsani, P, Papamitsou, T, Malliarou, M, Soteriades, ES. Exploring Current Health Policies on Rare Diseases in Greece: A Narrative Review. Int J Pediatr. 2025;2025 :5572290. doi: 10.1155/ijpe/5572290. PubMed PMID:41164790 PubMed Central PMC12566958.
  6. Stephenson, AL, Sykes, J, Stanojevic, S, Ma, X, Ostrenga, J, Todd, J et al.. Exploring reduced pulmonary exacerbation rates in cystic fibrosis: a pandemic or modulator effect?. ERJ Open Res. 2025;11 (5):. doi: 10.1183/23120541.00111-2025. PubMed PMID:41158496 PubMed Central PMC12557442.
  7. Gomez-Ferreria, MA, Garcia-Muñoz, V, Zwiers, A. The EMA as a potential driver of competitiveness of orphan medicines in Europe: a focus on regulatory incentives and funding opportunities. Orphanet J Rare Dis. 2025;20 (1):544. doi: 10.1186/s13023-025-04095-3. PubMed PMID:41152943 PubMed Central PMC12570519.
  8. Callenbach, MHE, van den Berg, S, Hulsbosch, A, Hollak, CEM, Leopold, C, Mantel-Teeuwisse, AK et al.. Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix. Orphanet J Rare Dis. 2025;20 (1):540. doi: 10.1186/s13023-025-04020-8. PubMed PMID:41146161 PubMed Central PMC12560585.
  9. Guo, J, Hennessy, G, Young, B, Hill, A. Research and development spending versus revenues after approval for CFTR modulators. J Cyst Fibros. 2025; :. doi: 10.1016/j.jcf.2025.10.008. PubMed PMID:41136304 .
  10. Díez, JJ, Badia, X, Abad, R, Alerany, C, Climente Martí, M, Guiu, JM et al.. Value Contribution of Palopegteriparatide in Adult Patients with Chronic Hypoparathyroidism using Multicriteria Decision Analysis (MCDA). Adv Ther. 2025; :. doi: 10.1007/s12325-025-03396-0. PubMed PMID:41134515 .
  11. Li, J, Li, C, Zhou, Q, Han, W, Fang, M, Xu, Y et al.. Divergent activation patterns of BRS3 revealed by two Chinese herb-derived agonists. Acta Pharm Sin B. 2025;15 (10):5231-5243. doi: 10.1016/j.apsb.2025.06.025. PubMed PMID:41132856 PubMed Central PMC12541581.
  12. Alum, EU, Ekpang Ii, JE, Ekpang, PO, Ainebyoona, C, Nwagu, KE, Nwuruku, OA et al.. Toward an ethical future for orphan drugs: balancing access, affordability, and innovation. J Med Econ. 2025;28 (1):1869-1886. doi: 10.1080/13696998.2025.2577514. PubMed PMID:41117352 .
  13. Zheng, ZZ, Chen, L, Hu, GS, Peng, BL, Huang, QX, Wang, A et al.. Nuclear receptor ESRRA promotes ERα-positive breast cancer through dual action on super enhancers and promoters to regulate gene transcriptional programs. Sci Bull (Beijing). 2025; :. doi: 10.1016/j.scib.2025.09.044. PubMed PMID:41111053 .
  14. Herta, T, Dietz-Fricke, C, Demir, M, Sprinzl, K, Pfeiffer, KH, Grottenthaler, JM et al.. Impact of Handling Perception and Language Barriers on Virologic Response to Daily Subcutaneous Bulevirtide in Hepatitis D. Liver Int. 2025;45 (11):e70404. doi: 10.1111/liv.70404. PubMed PMID:41108575 PubMed Central PMC12535276.
  15. Alipourgivi, F, Lu, T. Celebrating the 40-Year Milestone: NF-ĸB in Oncoimmunity. Cancer Lett. 2025; :218087. doi: 10.1016/j.canlet.2025.218087. PubMed PMID:41106769 .
  16. GBD 2023 Causes of Death Collaborators. Global burden of 292 causes of death in 204 countries and territories and 660 subnational locations, 1990-2023: a systematic analysis for the Global Burden of Disease Study 2023. Lancet. 2025;406 (10513):1811-1872. doi: 10.1016/S0140-6736(25)01917-8. PubMed PMID:41092928 PubMed Central PMC12535838.
  17. GBD 2023 Disease and Injury and Risk Factor Collaborators. Burden of 375 diseases and injuries, risk-attributable burden of 88 risk factors, and healthy life expectancy in 204 countries and territories, including 660 subnational locations, 1990-2023: a systematic analysis for the Global Burden of Disease Study 2023. Lancet. 2025;406 (10513):1873-1922. doi: 10.1016/S0140-6736(25)01637-X. PubMed PMID:41092926 PubMed Central PMC12535840.
  18. Boucheham, A, Franco, JM, Bär, S, MacDonald, E, Zuttion, S, Blagec, L et al.. Galectin-3 Mediated Endocytosis of the Orphan G-Protein-Coupled Receptor GPRC5A. Cells. 2025;14 (19):. doi: 10.3390/cells14191571. PubMed PMID:41090797 PubMed Central PMC12524283.
  19. Godoy, CB, Ramachandran, R, Sapre, P, Ross, JS. Confirmatory evidence supporting single pivotal trial new drug approvals by the Food and Drug Administration, 2015 through 2023. Clin Trials. 2025; :17407745251376620. doi: 10.1177/17407745251376620. PubMed PMID:41090443 .
  20. Griffiths, A, Dunning, L, Facey, K, Jankovic, D, González Malla, C, Low, E et al.. Recommended methods for the collection of clinical expert judgment in rare diseases: Generating evidence to support reimbursement of orphan drugs. Int J Technol Assess Health Care. 2025;41 (1):e76. doi: 10.1017/S0266462325100457. PubMed PMID:41054189 .
Search PubMed