Publications on Orphan Drugs

  1. Gu, D, Janetos, TM. Ophthalmic Drug Discovery in the United States over the past Two Decades. Ophthalmic Epidemiol. 2020; :1-6. doi: 10.1080/09286586.2020.1786591. PubMed PMID:32597285 .
  2. Shah, KK, Kogut, S, Slitt, A. Challenges in Evaluating Safety and Efficacy in Drug Development for Rare Diseases: A Review for Pharmacists. J Pharm Pract. 2020; :897190020930972. doi: 10.1177/0897190020930972. PubMed PMID:32583733 .
  3. Padula, WV, Parasrampuria, S, Socal, MP, Conti, RM, Anderson, GF. Market Exclusivity for Drugs with Multiple Orphan Approvals (1983-2017) and Associated Budget Impact in the US. Pharmacoeconomics. 2020; :. doi: 10.1007/s40273-020-00934-2. PubMed PMID:32533523 .
  4. Darrow, JJ, Najafzadeh, M, Stefanini, K, Kesselheim, AS. Regulatory approval characteristics of antimicrobial versus non-antimicrobial products, 1984-2018: an evaluation of Food and Drug Administration flexibilities. Lancet Infect Dis. 2020;20 (7):e159-e164. doi: 10.1016/S1473-3099(20)30197-3. PubMed PMID:32502431 .
  5. Lanar, S, Acquadro, C, Seaton, J, Savre, I, Arnould, B. To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases. Orphanet J Rare Dis. 2020;15 (1):134. doi: 10.1186/s13023-020-01400-0. PubMed PMID:32493385 PubMed Central PMC7268539.
  6. Schey, C, Postma, MJ, Krabbe, PFM, Topachevskyi, O, Volovyk, A, Connolly, M et al.. Assessing the Preferences for Criteria in Multi-Criteria Decision Analysis in Treatments for Rare Diseases. Front Public Health. 2020;8 :162. doi: 10.3389/fpubh.2020.00162. PubMed PMID:32457865 PubMed Central PMC7225315.
  7. Gandjour, A, Schüßler, S, Hammerschmidt, T, Dintsios, CM. Predictors of negotiated prices for new drugs in Germany. Eur J Health Econ. 2020; :. doi: 10.1007/s10198-020-01201-z. PubMed PMID:32451745 .
  8. Tanaudommongkon, I, John Miyagi, S, Green, DJ, Burnham, JM, van den Anker, JN, Park, K et al.. Combined Pediatric and Adult Trials Submitted to the US Food and Drug Administration 2012-2018. Clin. Pharmacol. Ther. 2020; :. doi: 10.1002/cpt.1886. PubMed PMID:32394430 .
  9. Lee, SH, Yoo, SL, Bang, JS, Lee, JH. Patient Accessibility and Budget Impact of Orphan Drugs in South Korea: Long-Term and Real-World Data Analysis (2007-2019). Int J Environ Res Public Health. 2020;17 (9):. doi: 10.3390/ijerph17092991. PubMed PMID:32357397 PubMed Central PMC7246809.
  10. Aschenbrenner, DS. First Treatment for Rare Soft Tissue Cancer. Am J Nurs. 2020;120 (5):22. doi: 10.1097/01.NAJ.0000662784.12004.66. PubMed PMID:32332358 .
  11. Kim, S, Cho, H, Kim, J, Lee, K, Lee, JH. The current state of patient access to new drugs in South Korea under the positive list system: evaluation of the changes since the new review pathways. Expert Rev Pharmacoecon Outcomes Res. 2020; :1-7. doi: 10.1080/14737167.2020.1758559. PubMed PMID:32308058 .
  12. Chambers, JD, Silver, MC, Berklein, FC, Cohen, JT, Neumann, PJ. Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs. J Gen Intern Med. 2020; :. doi: 10.1007/s11606-020-05805-2. PubMed PMID:32291711 .
  13. Garbade, SF, Zielonka, M, Mechler, K, Kölker, S, Hoffmann, GF, Staufner, C et al.. FDA orphan drug designations for lysosomal storage disorders - a cross-sectional analysis. PLoS ONE. 2020;15 (4):e0230898. doi: 10.1371/journal.pone.0230898. PubMed PMID:32267884 PubMed Central PMC7141691.
  14. Brenna, E, Polistena, B, Spandonaro, F. The implementation of health technology assessment principles in public decisions concerning orphan drugs. Eur. J. Clin. Pharmacol. 2020;76 (6):755-764. doi: 10.1007/s00228-020-02855-7. PubMed PMID:32219539 .
  15. Franzen, N, Retèl, VP, Schats, W, van Harten, WH. Evidence Underlying Policy Proposals for Sustainable Anticancer Drug Prices: A Systematic Review. JAMA Oncol. 2020; :. doi: 10.1001/jamaoncol.2019.6846. PubMed PMID:32215592 .
  16. Kimmel, L, Conti, RM, Volerman, A, Chua, KP. Pediatric Orphan Drug Indications: 2010-2018. Pediatrics. 2020;145 (4):. doi: 10.1542/peds.2019-3128. PubMed PMID:32127360 PubMed Central PMC7111489.
  17. Wouters, OJ, McKee, M, Luyten, J. Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018. JAMA. 2020;323 (9):844-853. doi: 10.1001/jama.2020.1166. PubMed PMID:32125404 PubMed Central PMC7054832.
  18. Rawson, NSB. Alignment of health technology assessments and price negotiations for new drugs for rare disorders in Canada: Does it lead to improved patient access?. J Popul Ther Clin Pharmacol. 2020;27 (1):e48-e64. doi: 10.15586/jptcp.v27i1.658. PubMed PMID:32124580 .
  19. Cherla, A, Howard, N, Mossialos, E. The 'Netflix plus model': can subscription financing improve access to medicines in low- and middle-income countries?. Health Econ Policy Law. 2020; :1-11. doi: 10.1017/S1744133120000031. PubMed PMID:32122423 .
  20. Czech, M, Baran-Kooiker, A, Atikeler, K, Demirtshyan, M, Gaitova, K, Holownia-Voloskova, M et al.. A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries. Front Public Health. 2019;7 :416. doi: 10.3389/fpubh.2019.00416. PubMed PMID:32117845 PubMed Central PMC6997877.
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