Publications on Orphan Drugs

  1. Mei, L, Xu, L, Wu, S, Wang, Y, Xu, C, Wang, L et al.. Discovery, structural optimization, and anti-tumor bioactivity evaluations of betulinic acid derivatives as a new type of RORγ antagonists. Eur J Med Chem. 2023;257 :115472. doi: 10.1016/j.ejmech.2023.115472. PubMed PMID:37236000 .
  2. Caspersen, E, Guinot, PG, Rozec, B, Oilleau, JF, Fellahi, JL, Gaudard, P et al.. Comparison of landiolol and amiodarone for the treatment of new-onset atrial fibrillation after cardiac surgery (FAAC) trial: study protocol for a randomized controlled trial. Trials. 2023;24 (1):353. doi: 10.1186/s13063-023-07353-6. PubMed PMID:37226174 PubMed Central PMC10210392.
  3. Kinch, MS, Kraft, Z, Schwartz, T. 2022 in review: FDA approvals of new medicines. Drug Discov Today. 2023;28 (8):103622. doi: 10.1016/j.drudis.2023.103622. PubMed PMID:37201782 .
  4. Khera, HK, Venugopal, N, Karur, RT, Mishra, R, Kartha, RV, Rajasimha, HK et al.. Building cross-border collaborations to increase diversity and accelerate rare disease drug development - meeting report from the inaugural IndoUSrare Annual Conference 2021. Ther Adv Rare Dis. 2022;3 :26330040221133124. doi: 10.1177/26330040221133124. PubMed PMID:37180411 PubMed Central PMC10032468.
  5. Michaeli, T, Jürges, H, Michaeli, DT. FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis. BMJ. 2023;381 :e073242. doi: 10.1136/bmj-2022-073242. PubMed PMID:37160306 PubMed Central PMC10167557.
  6. Ross, JS. US Orphan Drug Act. BMJ. 2023;381 :928. doi: 10.1136/bmj.p928. PubMed PMID:37160304 .
  7. Kranz, P, McGauran, N, Banzi, R, Ünal, C, Lotz, F, Kaiser, T et al.. Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases. BMJ. 2023;381 :e072796. doi: 10.1136/bmj-2022-072796. PubMed PMID:37160303 .
  8. Tu, SS, Nagar, S, Kesselheim, AS, Lu, Z, Rome, BN. Five-Year Sales for Newly Marketed Prescription Drugs With and Without Initial Orphan Drug Act Designation. JAMA. 2023;329 (18):1607-1608. doi: 10.1001/jama.2023.3079. PubMed PMID:37159041 PubMed Central PMC10170330.
  9. Ogbole, OO, Akin-Ajani, OD, Ajala, TO, Ogunniyi, QA, Fettke, J, Odeku, OA et al.. Nutritional and pharmacological potentials of orphan legumes: Subfamily faboideae. Heliyon. 2023;9 (4):e15493. doi: 10.1016/j.heliyon.2023.e15493. PubMed PMID:37151618 PubMed Central PMC10161725.
  10. Ciani, O, Meregaglia, M, Battaglia, MA, Brichetto, G, Conte, A, Gasperini, C et al.. Patient-reported outcome measures in drugs for neurological conditions approved by European Medicines Agency 2017-2022. Neurol Sci. 2023; :. doi: 10.1007/s10072-023-06825-6. PubMed PMID:37145229 .
  11. Kim, H, Godman, B, Kwon, HY, Hong, SH. Introduction of managed entry agreements in Korea: Problem, policy, and politics. Front Pharmacol. 2023;14 :999220. doi: 10.3389/fphar.2023.999220. PubMed PMID:37124231 PubMed Central PMC10133550.
  12. Busner, J, Pandina, G, Day, S, Mahableshwarkar, A, Kempf, L, Sheean, M et al.. Patient Centricity: Design and Conduct of Clinical Trials in Orphan Diseases: Third of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development. Innov Clin Neurosci. 2023;20 (1-3):25-31. . PubMed PMID:37122576 PubMed Central PMC10132279.
  13. Okuyama, R. Strengthening the Competitiveness of Japan's Pharmaceutical Industry: Analysis of Country Differences in the Origin of New Drugs and Japan's Highly Productive Firm. Biol Pharm Bull. 2023;46 (5):718-724. doi: 10.1248/bpb.b23-00107. PubMed PMID:37121698 .
  14. Hospodková, P, Gilíková, K, Barták, M, Marušáková, E, Tichopád, A. Opportunities and Threats of the Legally Facilitated Performance-Based Managed Entry Agreements in Slovakia: The Early-Adoption Perspective. Healthcare (Basel). 2023;11 (8):. doi: 10.3390/healthcare11081179. PubMed PMID:37108013 PubMed Central PMC10138524.
  15. Kubota, Y, Narukawa, M. Randomized controlled trial data for successful new drug application for rare diseases in the United States. Orphanet J Rare Dis. 2023;18 (1):89. doi: 10.1186/s13023-023-02702-9. PubMed PMID:37076897 PubMed Central PMC10114466.
  16. Bodó, I, Amine, I, Boban, A, Bumbea, H, Kulagin, A, Lukina, E et al.. Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations. Adv Ther. 2023;40 (6):2752-2772. doi: 10.1007/s12325-023-02510-4. PubMed PMID:37072660 PubMed Central PMC10112829.
  17. Frost, J, Hall, A, Taylor, E, Lines, S, Mandizha, J, Pope, C et al.. How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis. Orphanet J Rare Dis. 2023;18 (1):84. doi: 10.1186/s13023-023-02682-w. PubMed PMID:37069597 PubMed Central PMC10108537.
  18. Miller, KL, Miller Needleman, KI. The effect of the COVID-19 pandemic on US Food and Drug Administration-funded clinical trials and natural history studies for rare diseases. Contemp Clin Trials. 2023;129 :107198. doi: 10.1016/j.cct.2023.107198. PubMed PMID:37068589 PubMed Central PMC10105374.
  19. Hems, S, Taylor, L, Jones, J, Holmes, E. Patient-based evidence: its role in decision making on end-of-life, orphan, and ultra-orphan medicines. Int J Technol Assess Health Care. 2023;39 (1):e19. doi: 10.1017/S026646232300003X. PubMed PMID:37039484 .
  20. Dooms, M. Orphan medical devices have come a long way. Orphanet J Rare Dis. 2023;18 (1):71. doi: 10.1186/s13023-023-02685-7. PubMed PMID:37020310 PubMed Central PMC10077604.
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