Publications on Orphan Drugs

  1. 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 .
  2. 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 .
  3. 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.
  4. 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.
  5. 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 .
  6. 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.
  7. 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.
  8. 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 .
  9. 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 .
  10. 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.
  11. 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 .
  12. 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.
  13. Aranda-Reneo, I, Rodríguez-Sánchez, B, Peña-Longobardo, LM, Oliva-Moreno, J, López-Bastida, J. Can the Consideration of Societal Costs Change the Recommendation of Economic Evaluations in the Field of Rare Diseases? An Empirical Analysis. Value Health. 2021;24 (3):431-442. doi: 10.1016/j.jval.2020.10.014. PubMed PMID:33641778 .
  14. Brown, DG, Wobst, HJ. A Decade of FDA-Approved Drugs (2010-2019): Trends and Future Directions. J Med Chem. 2021;64 (5):2312-2338. doi: 10.1021/acs.jmedchem.0c01516. PubMed PMID:33617254 .
  15. Schulz, S, Passon, AM, Perleth, M, Kulig, M, Paschke, N, Matthias, K et al.. The Evaluation of Orphan Drugs by the German Joint Federal Committee-An Eight-Year Review. Dtsch Arztebl Int. 2020;117 (50):868-869. doi: 10.3238/arztebl.2020.0868. PubMed PMID:33612156 PubMed Central PMC8025936.
  16. Radhakrishna, S, Dahiya, M, Pipalava, P, Jose, V. Regulatory Approval Scenario of Biosimilars in Pediatric Patients in the United States and European Union. Pharmaceut Med. 2021;35 (2):93-112. doi: 10.1007/s40290-020-00377-2. PubMed PMID:33595810 .
  17. Lopata, E, Terrone, C, Gopalan, A, Ladikos, N, Richardson, T. Meeting the affordability challenges posed by orphan drugs: a survey of payers, providers, and employers. J Manag Care Spec Pharm. 2021; :1-8. doi: 10.18553/jmcp.2021.20553. PubMed PMID:33586514 .
  18. Horgan, D, Moss, B, Boccia, S, Genuardi, M, Gajewski, M, Capurso, G et al.. Time for Change? The Why, What and How of Promoting Innovation to Tackle Rare Diseases - Is It Time to Update the EU's Orphan Regulation? And if so, What Should be Changed?. Biomed Hub. ;5 (2):1-11. doi: 10.1159/000509272. PubMed PMID:33564657 PubMed Central PMC7841717.
  19. Koçkaya, G, Atalay, S, Oğuzhan, G, Kurnaz, M, Ökçün, S, Sar Gedik, Ç et al.. Analysis of patient access to orphan drugs in Turkey. Orphanet J Rare Dis. 2021;16 (1):68. doi: 10.1186/s13023-021-01718-3. PubMed PMID:33549137 PubMed Central PMC7868010.
  20. Grimm, SE, Pouwels, X, Ramaekers, BLT, Wijnen, B, Knies, S, Grutters, J et al.. Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to "TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases"). Orphanet J Rare Dis. 2021;16 (1):62. doi: 10.1186/s13023-020-01666-4. PubMed PMID:33522936 PubMed Central PMC7849113.
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