Publications on Orphan Drugs

  1. Abuasal, B, Ahmed, MA, Patel, P, Albusaysi, S, Sabarinath, S, Uppoor, R et al.. Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities. Clin Pharmacol Ther. 2021; :. doi: 10.1002/cpt.2501. PubMed PMID:34860361 .
  2. Abdallah, K, De Boeck, K, Dooms, M, Simoens, S. A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe. Front Pharmacol. 2021;12 :746710. doi: 10.3389/fphar.2021.746710. PubMed PMID:34858177 PubMed Central PMC8630624.
  3. Schmidinger, M. Changing the Course of an Orphan Disease. N Engl J Med. 2021;385 (22):2090-2091. doi: 10.1056/NEJMe2114846. PubMed PMID:34818485 .
  4. Magalhaes, M. Should rare diseases get special treatment?. J Med Ethics. 2021; :. doi: 10.1136/medethics-2021-107691. PubMed PMID:34815319 .
  5. van den Berg, S, de Visser, S, Leufkens, HGM, Hollak, CEM. Drug Repurposing for Rare Diseases: A Role for Academia. Front Pharmacol. 2021;12 :746987. doi: 10.3389/fphar.2021.746987. PubMed PMID:34744726 PubMed Central PMC8564285.
  6. Chambers, JD, Margaretos, NM, Enright, DE, Wang, R, Ye, X. Is an Orphan Drug's Cost-Effectiveness Associated with US Health Plan Coverage Restrictiveness?. Pharmacoeconomics. 2021; :. doi: 10.1007/s40273-021-01096-5. PubMed PMID:34697718 .
  7. Jommi, C, Listorti, E, Villa, F, Ghislandi, S, Genazzani, A, Cangini, A et al.. Variables affecting pricing of orphan drugs: the Italian case. Orphanet J Rare Dis. 2021;16 (1):439. doi: 10.1186/s13023-021-02022-w. PubMed PMID:34666819 PubMed Central PMC8527608.
  8. Malozowski, S. Growth Hormone and Insulin-Like Growth Factor Dysregulation in Pediatric Chronic Kidney Disease. Horm Res Paediatr. 2021; :. doi: 10.1159/000520186. PubMed PMID:34644711 .
  9. Varghese, MS, Liu, CL, Kazi, DS. The Price of Progress: Cost, Access, and Adoption of Novel Cardiovascular Drugs in Clinical Practice. Curr Cardiol Rep. 2021;23 (11):163. doi: 10.1007/s11886-021-01598-w. PubMed PMID:34599393 PubMed Central PMC8486158.
  10. 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 .
  11. 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.
  12. 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;187 (3):381-387. doi: 10.1002/ajmg.c.31934. PubMed PMID:34480410 .
  13. 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 .
  14. 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.
  15. 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.
  16. 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.
  17. Angeli, F, Verdecchia, P, Reboldi, G. Aprocitentan, A Dual Endothelin Receptor Antagonist Under Development for the Treatment of Resistant Hypertension. Cardiol Ther. 2021;10 (2):397-406. doi: 10.1007/s40119-021-00233-7. PubMed PMID:34251649 PubMed Central PMC8555037.
  18. 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 .
  19. 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.
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