Publications on Orphan Drugs

  1. Rajueni, K, Chakraborty Choudhury, M. Assessment of the availability of repurposed orphan drugs in India. PLOS Glob Public Health. 2023;3 (9):e0001498. doi: 10.1371/journal.pgph.0001498. PubMed PMID:37756282 PubMed Central PMC10529578.
  2. Dane, A, Klein Gebbink, AS, Brugma, JD, Degrassat-Théas, A, Hug, MJ, Houlind, MB et al.. Prices of Orphan Drugs in Four Western European Countries Before and After Market Exclusivity Expiry: A Cross-Country Comparison of List Prices and Purchase Prices. Appl Health Econ Health Policy. 2023; :. doi: 10.1007/s40258-023-00832-6. PubMed PMID:37751107 .
  3. Wang, P, Chow, SC. Innovative thinking of clinical investigation for rare disease drug development. Orphanet J Rare Dis. 2023;18 (1):299. doi: 10.1186/s13023-023-02909-w. PubMed PMID:37740206 PubMed Central PMC10517458.
  4. Vallano, A, Pontes, C, Agustí, A. The challenges of access to innovative medicines with limited evidence in the European Union. Front Pharmacol. 2023;14 :1215431. doi: 10.3389/fphar.2023.1215431. PubMed PMID:37719853 PubMed Central PMC10500193.
  5. Kochar, N, Ip, S, Vardanega, V, Sireau, NT, Fineberg, NA. A cost-of-illness analysis of the economic burden of obsessive-compulsive disorder in the United Kingdom. Compr Psychiatry. 2023;127 :152422. doi: 10.1016/j.comppsych.2023.152422. PubMed PMID:37713953 .
  6. Sharma, R. Innovative Genoceuticals in Human Gene Therapy Solutions: Challenges and Safe Clinical Trials of Orphan Gene Therapy Products. Curr Gene Ther. 2023; :. doi: 10.2174/1566523223666230911120922. PubMed PMID:37702177 .
  7. Mayer-Hamblett, N, Clancy, JP, Jain, R, Donaldson, SH, Fajac, I, Goss, CH et al.. Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective. Lancet Respir Med. 2023; :. doi: 10.1016/S2213-2600(23)00297-7. PubMed PMID:37699421 .
  8. Lo Cicero, S, Castelli, G, Blaconà, G, Bruno, SM, Sette, G, Pigliucci, R et al.. L1077P CFTR pathogenic variant function rescue by Elexacaftor-Tezacaftor-Ivacaftor in cystic fibrosis patient-derived air-liquid interface (ALI) cultures and organoids: in vitro guided personalized therapy of non-F508del patients. Respir Res. 2023;24 (1):217. doi: 10.1186/s12931-023-02516-0. PubMed PMID:37674160 PubMed Central PMC10483775.
  9. Zhi, W, Liu, M, Yang, D, Zhang, S, Lu, Y, Han, J et al.. Analysis of marketed orphan drugs in China. Intractable Rare Dis Res. 2023;12 (3):132-140. doi: 10.5582/irdr.2023.01030. PubMed PMID:37662620 PubMed Central PMC10468412.
  10. Meregaglia, M, Malandrini, F, Angelini, S, Ciani, O. The Assessment of Patient-Reported Outcomes for the Authorisation of Medicines in Europe: A Review of European Public Assessment Reports from 2017 to 2022. Appl Health Econ Health Policy. 2023; :. doi: 10.1007/s40258-023-00827-3. PubMed PMID:37659000 .
  11. Hu, H, Lin, G, He, F, Liu, J, Jia, R, Li, K et al.. Design, synthesis, and biological evaluation of carbonyl-hydrazine-1-carboxamide derivatives as anti-hepatic fibrosis agents targeting Nur77. Bioorg Chem. 2023;140 :106795. doi: 10.1016/j.bioorg.2023.106795. PubMed PMID:37657195 .
  12. Arabloo, J, Azari, S, Gorji, HA, Rezapour, A, Alipour, V, Ehsanzadeh, SJ et al.. Cost-effectiveness of brentuximab vedotin in Hodgkin lymphoma: a systematic review. Eur J Clin Pharmacol. 2023; :. doi: 10.1007/s00228-023-03557-6. PubMed PMID:37656182 .
  13. Fishman, J, Wilson, K, Drzewiecka, A, Pochopień, M, Dingli, D. The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA. J Comp Eff Res. 2023;12 (10):e230055. doi: 10.57264/cer-2023-0055. PubMed PMID:37655691 .
  14. Luo, S, Xie, C, Lin, N, Lin, D, Gu, D, Lin, S et al.. Cost-effectiveness analysis of an orphan drug-tebentafusp in patients with metastatic uveal melanoma and a call for value-based pricing. Melanoma Res. 2023; :. doi: 10.1097/CMR.0000000000000919. PubMed PMID:37650713 .
  15. Enya, K, Lim, Y, Sengoku, S, Kodama, K. Increasing orphan drug loss in Japan: Trends and R&D strategy for rare diseases. Drug Discov Today. 2023;28 (10):103755. doi: 10.1016/j.drudis.2023.103755. PubMed PMID:37648019 .
  16. Balkhi, B, Almuaither, A, Alqahtani, S. Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union. Saudi Pharm J. 2023;31 (9):101738. doi: 10.1016/j.jsps.2023.101738. PubMed PMID:37638213 PubMed Central PMC10458326.
  17. Palcau, AC, Brandi, R, Mehterov, NH, Botti, C, Blandino, G, Pulito, C et al.. Exploiting Long Non-Coding RNAs and Circular RNAs as Pharmacological Targets in Triple-Negative Breast Cancer Treatment. Cancers (Basel). 2023;15 (16):. doi: 10.3390/cancers15164181. PubMed PMID:37627209 PubMed Central PMC10453179.
  18. Getaneh, Y, Getnet, F, Ning, F, Rashid, A, Liao, L, Yi, F et al.. HIV-1 Disease Progression and Drug Resistance Mutations among Children on First-Line Antiretroviral Therapy in Ethiopia. Biomedicines. 2023;11 (8):. doi: 10.3390/biomedicines11082293. PubMed PMID:37626789 PubMed Central PMC10452141.
  19. Vannucchi, AM, Guglielmelli, P. Acute Myocardial Infarction and Chronic Myeloproliferative Neoplasms: Friend and Enemy, Depending on Circumstances. JACC CardioOncol. 2023;5 (4):469-471. doi: 10.1016/j.jaccao.2023.07.001. PubMed PMID:37614589 PubMed Central PMC10443195.
  20. Wang, Y. Catalyst Pharms., Inc. v. Becerra: When the Food and Drug Administration Repeatedly Ignores the Plain Language of the Orphan Drug Act (ODA). J Law Health. 2023;36 (2):139-158. . PubMed PMID:37585550 .
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