Publications on Orphan Drugs

  1. Kerpel-Fronius, S, Baroutsou, V, Becker, S, Carlesi, R, Collia, L, Franke-Bray, B et al.. Development and Use of Gene Therapy Orphan Drugs-Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society. Front Med (Lausanne). 2020;7 :608249. doi: 10.3389/fmed.2020.608249. PubMed PMID:33425952 PubMed Central PMC7785873.
  2. Hirano, M, Berardo, A, Barca, E, Emmanuele, V, Quinzii, C, Simpson, CV et al.. Regulatory environment for novel therapeutic development in mitochondrial diseases. J Inherit Metab Dis. 2020; :. doi: 10.1002/jimd.12353. PubMed PMID:33368420 .
  3. Nuijten, M, Capri, S. Pricing of orphan drugs in oncology and rare diseases. J Mark Access Health Policy. 2020;8 (1):1838191. doi: 10.1080/20016689.2020.1838191. PubMed PMID:33312455 PubMed Central PMC7717868.
  4. Chan, AYL, Chan, VKY, Olsson, S, Fan, M, Jit, M, Gong, M et al.. Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis. Value Health. 2020;23 (12):1580-1591. doi: 10.1016/j.jval.2020.06.020. PubMed PMID:33248513 .
  5. Manning, R. Comment on "Market Exclusivity for Drugs with Multiple Orphan Approvals (1983-2017) and Associated Budget Impact in the US". Pharmacoeconomics. 2020;38 (12):1373-1374. doi: 10.1007/s40273-020-00975-7. PubMed PMID:33164138 .
  6. Padula, WV, Parasrampuria, S, Socal, MP, Conti, RM, Anderson, GF. Reply to Comment on "Market Exclusivity for Drugs with Multiple Orphan Approvals (1983-2017) and Associated Budget Impact in the US". Pharmacoeconomics. 2020;38 (12):1375-1376. doi: 10.1007/s40273-020-00973-9. PubMed PMID:33164137 PubMed Central PMC7721406.
  7. Komarov, IA, Krasilnikova, EI, Zhulev, IA, Zinchenko, RF. [The analysis of detection of rare diseases not included into listings of orphan diseases with preferential medication support]. Probl Sotsialnoi Gig Zdravookhranenniiai Istor Med. 2020;28 (5):893-902. doi: 10.32687/0869-866X-2020-28-5-893-902. PubMed PMID:33161658 .
  8. Jordan, VC. 50th anniversary of the first clinical trial with ICI 46,474 (tamoxifen): then what happened?. Endocr Relat Cancer. 2021;28 (1):R11-R30. doi: 10.1530/ERC-20-0335. PubMed PMID:33151906 PubMed Central PMC7780369.
  9. Socal, MP, Parasrampuria, S, Anderson, GF. Modifying the Criteria for Granting Orphan Drug Market Exclusivity. Value Health. 2020;23 (11):1470-1476. doi: 10.1016/j.jval.2020.08.004. PubMed PMID:33127018 .
  10. Kacetl, J, Marešová, P, Maskuriy, R, Selamat, A. Ethical Questions Linked to Rare Diseases and Orphan Drugs - A Systematic Review. Risk Manag Healthc Policy. 2020;13 :2125-2148. doi: 10.2147/RMHP.S260641. PubMed PMID:33116992 PubMed Central PMC7568613.
  11. Juth, N, Henriksson, M, Gustavsson, E, Sandman, L. Should we accept a higher cost per health improvement for orphan drugs? A review and analysis of egalitarian arguments. Bioethics. 2020; :. doi: 10.1111/bioe.12786. PubMed PMID:33107077 .
  12. Li, X, Lu, Z, Zhang, J, Zhang, X, Zhang, S, Zhou, J et al.. The urgent need to empower rare disease organizations in China: an interview-based study. Orphanet J Rare Dis. 2020;15 (1):282. doi: 10.1186/s13023-020-01568-5. PubMed PMID:33046132 PubMed Central PMC7552513.
  13. Malinowski, KP, Kawalec, P, Trąbka, W, Sowada, C, Petrova, G, Manova, M et al.. Health technology assessment and reimbursement policy for oncology orphan drugs in Central and Eastern Europe. Orphanet J Rare Dis. 2020;15 (1):277. doi: 10.1186/s13023-020-01556-9. PubMed PMID:33032634 PubMed Central PMC7545889.
  14. Chua, KP, Conti, RM. Trends In Orphan Drug Spending And Out-Of-Pocket Spending Among US Children, 2013-18. Health Aff (Millwood). 2020;39 (10):1806-1811. doi: 10.1377/hlthaff.2020.00595. PubMed PMID:33017253 .
  15. Crowell, JL, Burns, TM. Rising Drug Costs for Neurologic Diseases. Continuum (Minneap Minn). 2020;26 (5):1392-1406. doi: 10.1212/CON.0000000000000912. PubMed PMID:33003008 .
  16. Yehia, F, Segal, JB, Anderson, GF. Predictors of orphan drug coverage restrictions in Medicare Part D. Am J Manag Care. 2020;26 (9):e289-e294. doi: 10.37765/ajmc.2020.88494. PubMed PMID:32930558 .
  17. Kinch, MS, Kraft, Z, Schwartz, T. 2019 in review: FDA approvals of new medicines. Drug Discov Today. 2020; :. doi: 10.1016/j.drudis.2020.09.002. PubMed PMID:32927064 .
  18. Daniel, H, Serchen, J, Cooney, TG. Policy Recommendations to Promote Prescription Drug Competition: A Position Paper From the American College of Physicians. Ann Intern Med. 2020;173 (12):1002-1003. doi: 10.7326/M19-3773. PubMed PMID:32926798 .
  19. Balijepalli, C, Gullapalli, L, Druyts, E, Yan, K, Desai, K, Barakat, S et al.. Can Standard Health Technology Assessment Approaches Help Guide the Price of Orphan Drugs in Canada? A Review of Submissions to the Canadian Agency for Drugs and Technologies in Health Common Drug Review. Clinicoecon Outcomes Res. 2020;12 :445-457. doi: 10.2147/CEOR.S264589. PubMed PMID:32922050 PubMed Central PMC7455764.
  20. Berdud, M, Drummond, M, Towse, A. Establishing a reasonable price for an orphan drug. Cost Eff Resour Alloc. 2020;18 :31. doi: 10.1186/s12962-020-00223-x. PubMed PMID:32908456 PubMed Central PMC7472708.
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