Publications on Orphan Drugs

  1. Miller, KL, Kraft, S, Ipe, A, Fermaglich, L. Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies. Expert Opin Orphan Drugs. 2022;9 (11-12):265-272. doi: 10.1080/21678707.2021.2047021. PubMed PMID:35548705 PubMed Central PMC9087212.
  2. Costa, E, Girotti, S, Pauro, F, Leufkens, HGM, Cipolli, M. The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis. Orphanet J Rare Dis. 2022;17 (1):188. doi: 10.1186/s13023-022-02350-5. PubMed PMID:35525974 PubMed Central PMC9078013.
  3. Jandhyala, R. The effect of adding real-world evidence to regulatory submissions on the breadth of population indicated for rare disease medicine treatment by the European Medicines Agency. J Pharm Policy Pract. 2022;15 (1):36. doi: 10.1186/s40545-022-00433-z. PubMed PMID:35509059 PubMed Central PMC9069778.
  4. Kinch, MS, Kraft, Z, Schwartz, T. 2021 in review: FDA approvals of new medicines. Drug Discov Today. 2022; :. doi: 10.1016/j.drudis.2022.04.010. PubMed PMID:35439613 .
  5. Nguyen, CQ, Alba-Concepcion, K, Palmer, EE, Scully, JL, Millis, N, Farrar, MA et al.. The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review. Orphanet J Rare Dis. 2022;17 (1):167. doi: 10.1186/s13023-022-02317-6. PubMed PMID:35436886 PubMed Central PMC9014615.
  6. Panzer, AD, Margaretos, NM, Bridger, N, Osani, MC, Lai, RC, Chambers, JD et al.. Patients' access to 2018 FDA-approved drugs 1 year post approval. Am J Manag Care. 2022;28 (4):e153-e156. doi: 10.37765/ajmc.2022.88869. PubMed PMID:35420754 .
  7. Postma, MJ, Noone, D, Rozenbaum, MH, Carter, JA, Botteman, MF, Fenwick, E et al.. Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?. Orphanet J Rare Dis. 2022;17 (1):157. doi: 10.1186/s13023-022-02283-z. PubMed PMID:35382853 PubMed Central PMC8981887.
  8. Cho, I, Han, E. Drug Lag and Associated Factors for Approved Drugs in Korea Compared with the United States. Int J Environ Res Public Health. 2022;19 (5):. doi: 10.3390/ijerph19052857. PubMed PMID:35270550 PubMed Central PMC8910054.
  9. Huang, YJ, Chao, WY, Wang, CC, Chang, LC. Orphan drug development: The impact of regulatory and reimbursement frameworks. Drug Discov Today. 2022;27 (6):1724-1732. doi: 10.1016/j.drudis.2022.03.002. PubMed PMID:35257859 .
  10. Amaral, MD. Precision medicine for rare diseases: The times they are A-Changin'. Curr Opin Pharmacol. 2022;63 :102201. doi: 10.1016/j.coph.2022.102201. PubMed PMID:35255452 .
  11. Villa, F, Di Filippo, A, Pierantozzi, A, Genazzani, A, Addis, A, Trifirò, G et al.. Orphan Drug Prices and Epidemiology of Rare Diseases: A Cross-Sectional Study in Italy in the Years 2014-2019. Front Med (Lausanne). 2022;9 :820757. doi: 10.3389/fmed.2022.820757. PubMed PMID:35252257 PubMed Central PMC8891228.
  12. Ward, LM, Chambers, A, Mechichi, E, Wong-Rieger, D, Campbell, C. An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries. Orphanet J Rare Dis. 2022;17 (1):113. doi: 10.1186/s13023-022-02260-6. PubMed PMID:35246200 PubMed Central PMC8895096.
  13. Serrano, P, Yuen, HW, Akdemir, J, Hartmann, M, Reinholz, T, Peltier, S et al.. Real-world data in drug development strategies for orphan drugs: Tafasitamab in B-cell lymphoma, a case study for an approval based on a single-arm combination trial. Drug Discov Today. 2022;27 (6):1706-1715. doi: 10.1016/j.drudis.2022.02.017. PubMed PMID:35218926 .
  14. Bai, H, Zhang, SQ, Lei, H, Wang, F, Ma, M, Xin, M et al.. Menin-MLL protein-protein interaction inhibitors: a patent review (2014-2021). Expert Opin Ther Pat. 2022;32 (5):507-522. doi: 10.1080/13543776.2022.2045947. PubMed PMID:35202550 .
  15. Gozzo, L, Romano, GL, Brancati, S, Cicciù, M, Fiorillo, L, Longo, L et al.. Access to Innovative Neurological Drugs in Europe: Alignment of Health Technology Assessments Among Three European Countries. Front Pharmacol. 2021;12 :823199. doi: 10.3389/fphar.2021.823199. PubMed PMID:35185551 PubMed Central PMC8854989.
  16. Song, H, Pei, X, Liu, Z, Shen, C, Sun, J, Liu, Y et al.. Pharmacovigilance in China: Evolution and future challenges. Br J Clin Pharmacol. 2022; :. doi: 10.1111/bcp.15277. PubMed PMID:35165914 .
  17. Blonda, A, Barcina Lacosta, T, Toumi, M, Simoens, S. Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries. Front Pharmacol. 2021;12 :750742. doi: 10.3389/fphar.2021.750742. PubMed PMID:35126102 PubMed Central PMC8814578.
  18. Luzzatto, L, Makani, J. Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet. Front Pharmacol. 2021;12 :770640. doi: 10.3389/fphar.2021.770640. PubMed PMID:35082665 PubMed Central PMC8784510.
  19. O'Connor, D. An interview on rare diseases and orphan medicines with Dr Daniel O'Connor, medical assessor at the Medicines and Healthcare products Regulatory Agency (MHRA), UK. Curr Med Res Opin. 2022;38 (2):159-160. doi: 10.1080/03007995.2021.2023394. PubMed PMID:35045782 .
  20. Stephenson, D, Ollivier, C, Brinton, R, Barrett, J. Can Innovative Trial Designs in Orphan Diseases Drive Advancement of Treatments for Common Neurological Diseases?. Clin Pharmacol Ther. 2022;111 (4):799-806. doi: 10.1002/cpt.2528. PubMed PMID:35034352 .
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