Publications on Orphan Drugs

  1. Brasil, S, Allocca, M, Magrinho, SCM, Santos, I, Raposo, M, Francisco, R et al.. Systematic Review: Drug Repositioning for Congenital Disorders of Glycosylation (CDG). Int J Mol Sci. 2022;23 (15):. doi: 10.3390/ijms23158725. PubMed PMID:35955863 .
  2. Pearson, C, Schapiro, L, Pearson, SD. The next generation of rare disease drug policy: ensuring both innovation and affordability. J Comp Eff Res. 2022; :. doi: 10.2217/cer-2022-0120. PubMed PMID:35946484 .
  3. Blonda, A, Denier, Y, Huys, I, Kawalec, P, Simoens, S. How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries. Front Pharmacol. 2022;13 :902150. doi: 10.3389/fphar.2022.902150. PubMed PMID:35928274 PubMed Central PMC9343828.
  4. Seki, K, Suzuki, H, Abe, S, Saotome, C. Lifecycle management of orphan drugs approved in Japan. Orphanet J Rare Dis. 2022;17 (1):299. doi: 10.1186/s13023-022-02456-w. PubMed PMID:35906686 PubMed Central PMC9336109.
  5. Abozaid, GM, Kerr, K, McKnight, A, Al-Omar, HA. Criteria to define rare diseases and orphan drugs: a systematic review protocol. BMJ Open. 2022;12 (7):e062126. doi: 10.1136/bmjopen-2022-062126. PubMed PMID:35906057 .
  6. Xiang, Z, Jiang, W, Yan, B, Jiang, J, Zheng, H. Current status and trend of clinical development of orphan drugs in China. Orphanet J Rare Dis. 2022;17 (1):294. doi: 10.1186/s13023-022-02440-4. PubMed PMID:35897012 PubMed Central PMC9327367.
  7. Mazzucato, M, Minichiello, C, Visonà Dalla Pozza, L, Vianello, A, Toto, E, De Lorenzi, M et al.. [The impact of the use of treatments not included in the reimbursement classes in the care of rare patients: a real world study.]. Recenti Prog Med. ;113 (7):440-450. doi: 10.1701/3850.38339. PubMed PMID:35852080 .
  8. Casali, PG, Provenzano, S, Trama, A. [The problem of rare cancers.]. Recenti Prog Med. ;113 (7):430-433. doi: 10.1701/3850.38337. PubMed PMID:35852078 .
  9. Crisafulli, S, Trifirò, G. [The role of real-world evidence for the study of rare diseases epidemiology and the post-marketing evaluation of orphan drugs.]. Recenti Prog Med. ;113 (7):425-429. doi: 10.1701/3850.38336. PubMed PMID:35852077 .
  10. Costa, E, Grimaldi, G, Del Grosso, V, Isgrò, A, Genazzani, A. [Access to medicines for rare diseases: regulatory aspects and public health priorities.]. Recenti Prog Med. ;113 (7):415-424. doi: 10.1701/3850.38335. PubMed PMID:35852076 .
  11. Costa, E, Magrini, N. [Harmonizing development, approval and access of drugs for rare diseases in Europe.]. Recenti Prog Med. ;113 (7):407-410. doi: 10.1701/3850.38333. PubMed PMID:35852074 .
  12. Wu, X, Xu, W, Yu, M, Zhang, F, Wang, H. Clinical trials of orphan drugs in China over the decade 2012-2022: Opportunities and challenges. Pharmacol Res. 2022;182 :106349. doi: 10.1016/j.phrs.2022.106349. PubMed PMID:35835367 .
  13. Stafinski, T, Glennie, J, Young, A, Menon, D. HTA decision-making for drugs for rare diseases: comparison of processes across countries. Orphanet J Rare Dis. 2022;17 (1):258. doi: 10.1186/s13023-022-02397-4. PubMed PMID:35804398 PubMed Central PMC9264608.
  14. Zhou, N, Ji, H, Li, Z, Hu, J, Xie, JH, Feng, YH et al.. Influencing Factors of Health Technology Assessment to Orphan Drugs: Empirical Evidence in England, Scotland, Canada, and Australia. Front Public Health. 2022;10 :861067. doi: 10.3389/fpubh.2022.861067. PubMed PMID:35784205 PubMed Central PMC9247336.
  15. Gabison, EE, Guindolet, D. Neurotrophic Keratitis: A rare disease that requires proactive screening and orphan drug treatments. Ocul Surf. 2022;25 :154. doi: 10.1016/j.jtos.2022.06.008. PubMed PMID:35781074 .
  16. Partin, C. Rarely mentioned: how we arrived at the quantitative definition of a rare disease. Proc (Bayl Univ Med Cent). 2022;35 (4):498-504. doi: 10.1080/08998280.2022.2048613. PubMed PMID:35754591 PubMed Central PMC9196794.
  17. Mohammadshahi, M, Olyaeemanesh, A, Ehsani-Chimeh, E, Mobinizadeh, M, Fakoorfard, Z, Akbari Sari, A et al.. Methods and criteria for the assessment of orphan drugs: a scoping review. Int J Technol Assess Health Care. 2022;38 (1):e59. doi: 10.1017/S0266462322000393. PubMed PMID:35730573 .
  18. Fontrier, AM. Market access for medicines treating rare diseases: Association between specialised processes for orphan medicines and funding recommendations. Soc Sci Med. 2022;306 :115119. doi: 10.1016/j.socscimed.2022.115119. PubMed PMID:35700552 .
  19. Rawson, NSB. Health technology assessment and price negotiation alignment for rare disorder drugs in Canada: Who benefits?. Orphanet J Rare Dis. 2022;17 (1):218. doi: 10.1186/s13023-022-02390-x. PubMed PMID:35698235 PubMed Central PMC9190095.
  20. Gandini, E, Marcou, G, Bonachera, F, Varnek, A, Pieraccini, S, Sironi, M et al.. Molecular Similarity Perception Based on Machine-Learning Models. Int J Mol Sci. 2022;23 (11):. doi: 10.3390/ijms23116114. PubMed PMID:35682792 PubMed Central PMC9181189.
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