Publications on Orphan Drugs

  1. Gamba, S, Magazzini, L, Pertile, P. R&D and market size: Who benefits from orphan drug legislation?. J Health Econ. 2021;80 :102522. doi: 10.1016/j.jhealeco.2021.102522. PubMed PMID:34530381 .
  2. Lee, B, Bae, EY, Bae, S, Choi, HJ, Son, KB, Lee, YS et al.. How can we improve patients' access to new drugs under uncertainties? : South Korea's experience with risk sharing arrangements. BMC Health Serv Res. 2021;21 (1):967. doi: 10.1186/s12913-021-06919-x. PubMed PMID:34521408 PubMed Central PMC8442279.
  3. Poswar, FO, da Silva, LP, Zambrano, MB, Pedrini, DB, Saute, JAM, Giugliani, R et al.. Clinical trials for genetic diseases in Latin America. Am J Med Genet C Semin Med Genet. 2021; :. doi: 10.1002/ajmg.c.31934. PubMed PMID:34480410 .
  4. Hirai, T, Suzuki, A, Yamori, T, Matsuura, M. Reforms of regulatory pathways for approval of new antineoplastic drugs in Japan from 2004 to 2019 and accompanying changes in pivotal clinical trial designs. Invest New Drugs. 2021; :. doi: 10.1007/s10637-021-01165-8. PubMed PMID:34417913 .
  5. Kamusheva, M, Dimitrova, M, Tachkov, K, Petrova, G, Mitkova, Z. Pharmacotherapeutic Patterns and Patients' Access to Pharmacotherapy for Some Rare Diseases in Bulgaria - A Pilot Comparative Study. Front Pharmacol. 2021;12 :695181. doi: 10.3389/fphar.2021.695181. PubMed PMID:34349654 PubMed Central PMC8326790.
  6. Li, X, Liu, M, Lin, J, Li, B, Zhang, X, Zhang, S et al.. A questionnaire-based study to comprehensively assess the status quo of rare disease patients and care-givers in China. Orphanet J Rare Dis. 2021;16 (1):327. doi: 10.1186/s13023-021-01954-7. PubMed PMID:34294091 PubMed Central PMC8296703.
  7. Pasquini, TLS, Goff, SL, Whitehill, JM. Navigating the U.S. health insurance landscape for children with rare diseases: a qualitative study of parents' experiences. Orphanet J Rare Dis. 2021;16 (1):313. doi: 10.1186/s13023-021-01943-w. PubMed PMID:34266466 PubMed Central PMC8281562.
  8. Angeli, F, Verdecchia, P, Reboldi, G. Aprocitentan, A Dual Endothelin Receptor Antagonist Under Development for the Treatment of Resistant Hypertension. Cardiol Ther. 2021; :. doi: 10.1007/s40119-021-00233-7. PubMed PMID:34251649 .
  9. van den Berg, S, van der Wel, V, de Visser, SJ, Stunnenberg, BC, Timmers, L, van der Ree, MH et al.. Cost-Based Price Calculation of Mexiletine for Nondystrophic Myotonia. Value Health. 2021;24 (7):925-929. doi: 10.1016/j.jval.2021.02.004. PubMed PMID:34243835 .
  10. Johann, P, Lenz, D, Ries, M. The drug development pipeline for glioblastoma-A cross sectional assessment of the FDA Orphan Drug Product designation database. PLoS One. 2021;16 (7):e0252924. doi: 10.1371/journal.pone.0252924. PubMed PMID:34234357 PubMed Central PMC8263276.
  11. Zimmermann, BM, Eichinger, J, Baumgartner, MR. A systematic review of moral reasons on orphan drug reimbursement. Orphanet J Rare Dis. 2021;16 (1):292. doi: 10.1186/s13023-021-01925-y. PubMed PMID:34193232 PubMed Central PMC8247078.
  12. Miller, KL, Fermaglich, LJ, Maynard, J. Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases. Orphanet J Rare Dis. 2021;16 (1):265. doi: 10.1186/s13023-021-01901-6. PubMed PMID:34107994 PubMed Central PMC8191002.
  13. Zhang, L, Wang, J. Incentives to promote the US pharmaceutical innovation: empirical research based on the case of Epogen. Drug Dev Ind Pharm. 2021;47 (5):820-824. doi: 10.1080/03639045.2021.1935997. PubMed PMID:34056986 .
  14. Park, K, Virparia, R, Green, DJ, Epps, C, Wharton, GT, McCune, SK et al.. Inclusion of Infants and Neonates in Pediatric Orphan Product Approvals. Clin Pharmacol Ther. 2021;110 (4):997-1003. doi: 10.1002/cpt.2306. PubMed PMID:34028811 .
  15. Pani, L, Becker, K. New Models for the Evaluation of Specialized Medicinal Products: Beyond Conventional Health Technology Assessment and Pricing. Clin Drug Investig. 2021;41 (6):529-537. doi: 10.1007/s40261-021-01041-6. PubMed PMID:34014509 .
  16. Magelssen, M, Rasmussen, M, Wallace, S, Førde, R. Priority setting at the clinical level: the case of nusinersen and the Norwegian national expert group. BMC Med Ethics. 2021;22 (1):54. doi: 10.1186/s12910-021-00623-5. PubMed PMID:33947377 PubMed Central PMC8094497.
  17. Schmidt, L, Sehic, O, Wild, C. EU FP7 research funding for an orphan drug (Orfadin®) and vaccine (Hep C) development: a success and a failure?. J Pharm Policy Pract. 2021;14 (1):37. doi: 10.1186/s40545-021-00317-8. PubMed PMID:33910624 PubMed Central PMC8080091.
  18. Raghu, VK, Mezoff, EA, Cole, CR, Rudolph, JA, Smith, KJ. Cost-effectiveness of ethanol lock prophylaxis to prevent central line-associated bloodstream infections in children with intestinal failure in the United States. JPEN J Parenter Enteral Nutr. 2021; :. doi: 10.1002/jpen.2130. PubMed PMID:33908050 .
  19. de Andrés-Nogales, F, Cruz, E, Calleja, MÁ, Delgado, O, Gorgas, MQ, Espín, J et al.. A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study). Orphanet J Rare Dis. 2021;16 (1):186. doi: 10.1186/s13023-021-01809-1. PubMed PMID:33902672 PubMed Central PMC8073956.
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