Publications on Orphan Drugs

  1. 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.
  2. 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.
  3. 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 .
  4. 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 .
  5. 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 .
  6. 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 .
  7. 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.
  8. 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.
  9. Hollak, CEM, Sirrs, S, van den Berg, S, van der Wel, V, Langeveld, M, Dekker, H et al.. Registries for orphan drugs: generating evidence or marketing tools?. Orphanet J Rare Dis. 2020;15 (1):235. doi: 10.1186/s13023-020-01519-0. PubMed PMID:32883346 PubMed Central PMC7469301.
  10. Miller, KL, Mueller, C, Liu, G, Miller Needleman, KI, Maynard, J. FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development. Orphanet J Rare Dis. 2020;15 (1):234. doi: 10.1186/s13023-020-01514-5. PubMed PMID:32883327 PubMed Central PMC7469294.
  11. Badia, X, Vico, T, Shepherd, J, Gil, A, Poveda-Andrés, JL, Hernández, C et al.. Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019. Orphanet J Rare Dis. 2020;15 (1):224. doi: 10.1186/s13023-020-01507-4. PubMed PMID:32859228 PubMed Central PMC7456380.
  12. Chua, KP, Conti, RM. Policy Implications of the Orphan Drug Designation for Remdesivir to Treat COVID-19. JAMA Intern Med. 2020;180 (10):1273-1274. doi: 10.1001/jamainternmed.2020.2759. PubMed PMID:32804187 .
  13. Coyle, D, Durand-Zaleski, I, Farrington, J, Garrison, L, Graf von der Schulenburg, JM, Greiner, W et al.. HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies. Eur J Health Econ. 2020; :. doi: 10.1007/s10198-020-01212-w. PubMed PMID:32794011 .
  14. Toumi, M, Millier, A, Cristeau, O, Thokagevistk-Desroziers, K, Dorey, J, Aballéa, S et al.. Social Preferences for Orphan Drugs: A Discrete Choice Experiment Among the French General Population. Front Med (Lausanne). 2020;7 :323. doi: 10.3389/fmed.2020.00323. PubMed PMID:32766260 PubMed Central PMC7379418.
  15. Bannenberg, WJ, 't Hoen, EFM. [Orphan drugs and drug pirates]. Ned Tijdschr Geneeskd. 2020;164 :. . PubMed PMID:32749801 .
  16. Marselis, D, Hordijk, L. From blockbuster to "nichebuster": how a flawed legislation helped create a new profit model for the drug industry. BMJ. 2020;370 :m2983. doi: 10.1136/bmj.m2983. PubMed PMID:32727745 .
  17. Burns, TM, Crowell, JL, Smith, AG. A crisis in US drug pricing: Consequences for patients with neuromuscular diseases, physicians, and society, part 2. Muscle Nerve. 2020; :. doi: 10.1002/mus.27018. PubMed PMID:32725709 .
  18. Burns, TM, Crowell, JL, Smith, AG. A crisis in US drug pricing: Consequences for patients with neuromuscular diseases, physicians and society, part 1. Muscle Nerve. 2020; :. doi: 10.1002/mus.27015. PubMed PMID:32725634 .
  19. Mullin, AP, Corey, D, Turner, EC, Liwski, R, Olson, D, Burton, J et al.. Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington's Disease. Clin Transl Sci. 2020; :. doi: 10.1111/cts.12845. PubMed PMID:32702147 .
  20. Bayoumy, AB, de Boer, NKH, Ansari, AR, Crouwel, F, Mulder, CJJ. Unrealized potential of drug repositioning in europe during COVID-19 and beyond: a physcian's perspective. J Pharm Policy Pract. 2020;13 :45. doi: 10.1186/s40545-020-00249-9. PubMed PMID:32695427 PubMed Central PMC7366879.
Search PubMed