Publications on Orphan Drugs

  1. 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.
  2. Zhang, L, Wang, J. Incentives to promote the US pharmaceutical innovation: empirical research based on the case of Epogen. Drug Dev Ind Pharm. 2021; :1-5. doi: 10.1080/03639045.2021.1935997. PubMed PMID:34056986 .
  3. 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; :. doi: 10.1002/cpt.2306. PubMed PMID:34028811 .
  4. 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 .
  5. 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.
  6. 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 .
  7. 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.
  8. Li, X, Zhang, X, Zhang, S, Lu, Z, Zhang, J, Zhou, J et al.. Rare disease awareness and perspectives of physicians in China: a questionnaire-based study. Orphanet J Rare Dis. 2021;16 (1):171. doi: 10.1186/s13023-021-01788-3. PubMed PMID:33849615 PubMed Central PMC8042908.
  9. Hagendijk, ME, van der Schans, S, Boersma, C, Postma, MJ, van der Pol, S. Economic evaluation of orphan drug Lutetium-Octreotate vs. Octreotide long-acting release for patients with an advanced midgut neuroendocrine tumour in the Netherlands. Eur J Health Econ. 2021; :. doi: 10.1007/s10198-021-01303-2. PubMed PMID:33829344 .
  10. Liu, I, Colmenares, E, Tak, C, Vest, MH, Clark, H, Oertel, M et al.. Development and validation of a predictive model to predict and manage drug shortages. Am J Health Syst Pharm. 2021; :. doi: 10.1093/ajhp/zxab152. PubMed PMID:33821926 .
  11. Johannesen, S, Huie, JR, Budeus, B, Peters, S, Wirth, AM, Iberl, S et al.. Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis. Front Neurol. 2021;12 :616289. doi: 10.3389/fneur.2021.616289. PubMed PMID:33815246 PubMed Central PMC8012841.
  12. Lee, JH. Pricing and Reimbursement Pathways of New Orphan Drugs in South Korea: A Longitudinal Comparison. Healthcare (Basel). 2021;9 (3):. doi: 10.3390/healthcare9030296. PubMed PMID:33800373 PubMed Central PMC8000795.
  13. Tomeo, F, Mariz, S, Brunetta, AL, Stoyanova-Beninska, V, Penttila, K, Magrelli, A et al.. Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective. Br J Clin Pharmacol. 2021; :. doi: 10.1111/bcp.14838. PubMed PMID:33772837 .
  14. Lochmüller, H, Ramirez, AN, Kakkis, E. Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders. Orphanet J Rare Dis. 2021;16 (1):141. doi: 10.1186/s13023-021-01687-7. PubMed PMID:33743771 PubMed Central PMC7980582.
  15. Nuijten, M, Van Wilder, P. The impact of early phase price agreements on prices of orphan drugs. BMC Health Serv Res. 2021;21 (1):222. doi: 10.1186/s12913-021-06208-7. PubMed PMID:33711994 PubMed Central PMC7953706.
  16. Charlton, V. Does NICE apply the rule of rescue in its approach to highly specialised technologies?. J Med Ethics. 2021; :. doi: 10.1136/medethics-2020-106759. PubMed PMID:33685978 .
  17. Lebecque, P, Thimmesch, M. [Cystic fibrosis prognosis in Europa : chronicle of an announced dilemma]. Rev Med Liege. 2021;76 (3):202-207. . PubMed PMID:33682390 .
  18. Peddapalli, A, Gehani, M, Kalle, AM, Peddapalli, SR, Peter, AE, Sharad, S et al.. Demystifying Excess Immune Response in COVID-19 to Reposition an Orphan Drug for Down-Regulation of NF-κB: A Systematic Review. Viruses. 2021;13 (3):. doi: 10.3390/v13030378. PubMed PMID:33673529 PubMed Central PMC7997247.
  19. Gennet, É. Introducing 'Health Vulnerability'. Towards a Human Right Claim for Innovative Orphan Drugs?. Eur J Health Law. 2020;27 (3):290-307. doi: 10.1163/15718093-BJA10005. PubMed PMID:33652405 .
  20. Chua, KP, Kimmel, LE, Conti, RM. Spending For Orphan Indications Among Top-Selling Orphan Drugs Approved To Treat Common Diseases. Health Aff (Millwood). 2021;40 (3):453-460. doi: 10.1377/hlthaff.2020.01442. PubMed PMID:33646878 PubMed Central PMC8011952.
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