PUBLICATIONS

Publications on Orphan Drugs

  1. Stevens, H, Siotis, G, Castanheira, M, Dewatripont, M, Akodad, S, Daems, S et al.. Rebalancing innovation, affordability, and access for orphan drugs in the European Union. Lancet Reg Health Eur. 2026;68 :101778. doi: 10.1016/j.lanepe.2026.101778. PubMed PMID:42571386 PubMed Central PMC13452220.
  2. Quan, G, Zhou, Y, Liu, Y, Wu, Y, Yan, Y. Reconceiving orphan drug market exclusivity as a conditional public-law entitlement: a sustainable regulatory governance model with comparative lessons for China. Front Med (Lausanne). 2026;13 :1896433. doi: 10.3389/fmed.2026.1896433. PubMed PMID:42558582 PubMed Central PMC13437677.
  3. Maxwell, CR, Turner, TJ, Wallstroem, E, Patel, S, Pappas, SC, Lewis, JH et al.. Tolebrutinib liver safety: A detailed overview. Mult Scler Relat Disord. 2026;113 :107404. doi: 10.1016/j.msard.2026.107404. PubMed PMID:42546561 .
  4. Chen, JC, Madsen, C, Kaltenboeck, A. The Economics Of Orphan Blockbuster Drug Development. Health Aff (Millwood). 2026;45 (8):838-845. doi: 10.1377/hlthaff.2026.00209. PubMed PMID:42546238 .
  5. Quinn, J, Amersdorffer, J, Boysen, M, Duffy-Roger, O, Hewitt, C, Jain, M et al.. Reframing the value of reserve antibiotics: how ultra-orphan reimbursement pathways could offer fair and equitable access to new antibiotics. Health Policy Open. 2026;11 :100177. doi: 10.1016/j.hpopen.2026.100177. PubMed PMID:42541082 PubMed Central PMC13427455.
  6. GBD 2023 LDL Cholesterol Collaborators, Razo, C, DeCleene, NK, Johnson, CO, Stark, BA, LeGrand, K et al.. Global Burden of Elevated LDL-C: Findings From the Global Burden of Disease Study 2023. JAMA. 2026; :. doi: 10.1001/jama.2026.8628. PubMed PMID:42525403 PubMed Central PMC13420188.
  7. Fang, S, Chen, S. Regulatory Alignment on Surrogate Endpoints for Slowly Progressive Ultra-Rare Neurological Diseases: Bridging the Gap Between Biomarker Science and Global Regulatory Acceptance. Ther Innov Regul Sci. 2026; :. doi: 10.1007/s43441-026-01026-7. PubMed PMID:42525369 .
  8. Yuan, J, Li, M, Jiang, B, Lu, ZK, Zhao, J, Jiang, X et al.. Clinical benefit, price and epidemiology of orphan drugs approved in China: a cross-sectional analysis of orphan drug policy (ODP) in China. BMJ Open. 2026;16 (7):e108748. doi: 10.1136/bmjopen-2025-108748. PubMed PMID:42521314 PubMed Central PMC13422930.
  9. Siebert, M, McGonigle, R, Gutiérrez-Sacristán, A, Naci, H, Wagner, AK, Bourgeois, FT et al.. Completion and outcomes of postmarketing requirements and commitments issued by the US Food and Drug Administration for new drugs, 2016-24: cross sectional evaluation of postmarketing studies. BMJ Med. 2026;5 (1):e002307. doi: 10.1136/bmjmed-2025-002307. PubMed PMID:42516723 PubMed Central PMC13405232.
  10. Khaidarov, S, Moldakaryzova, A, Dautov, D, Sagatbayeva, N, Yeszhan, B, Nurgaliyeva, B et al.. Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy: A Critical Synthesis of Approved and Emerging Strategies Through the Lens of an Underrepresented Population. Genes (Basel). 2026;17 (7):. doi: 10.3390/genes17070826. PubMed PMID:42510866 PubMed Central PMC13410084.
  11. Guimaron, S, Mercier, J, Hautbergue, T, Ruffenach, G, Amsallem, M, Savale, L et al.. Right ventricular remodeling and circulating metabolic profiles of patients with chronic thromboembolic pulmonary hypertension before and after pulmonary endarterectomy. JHLT Open. 2026;13 :100618. doi: 10.1016/j.jhlto.2026.100618. PubMed PMID:42502728 PubMed Central PMC13400446.
  12. Fu, M, Wallach, JD, So, AD, Doernberg, H, Li, X, Guan, X et al.. Drug and country characteristics associated with global approval and launch of FDA-approved novel therapeutics. Health Aff Sch. 2026;4 (7):qxag181. doi: 10.1093/haschl/qxag181. PubMed PMID:42482967 PubMed Central PMC13387065.
  13. Shahzad, M, Elsallab, M, Siebert, M, Bourgeois, FT. Assessment of United States Food and Drug Administration Approval of Cell and Gene Therapies. Clin Pharmacol Ther. 2026; :. doi: 10.1002/cpt.70396. PubMed PMID:42482162 PubMed Central PMC13388474.
  14. Casilli, G, Galeone, C, De Nigris, M, Genazzani, AA, Popoli, P, Marcellusi, A et al.. Closing the first wave of drug innovativeness in italy: final evidence from 2017 to 2025. Eur J Health Econ. 2026; :. doi: 10.1007/s10198-026-01956-x. PubMed PMID:42479353 .
  15. Monroy, S, González-Zamora, JF. Acute hepatic porphyrias in Mexico: A tale of challenges and achievements facing rare diseases. Mol Genet Metab. 2026;149 (1-2):110206. doi: 10.1016/j.ymgme.2026.110206. PubMed PMID:42468372 .
  16. Reinisch, W, Stepek, D, Kempinski, R, Danese, S, Sands, BE, Ratiu-Duma, B et al.. Efficacy and safety of duvakitug in patients with ulcerative colitis (RELIEVE UCCD): a phase 2b, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2026;11 (9):775-785. doi: 10.1016/S2468-1253(26)00120-2. PubMed PMID:42462750 .
  17. Jairath, V, Kierkuś, J, Duvall, GA, Danese, S, Sands, BE, Ratiu-Duma, B et al.. Efficacy and safety of duvakitug in patients with Crohn's disease (RELIEVE UCCD): a phase 2b, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2026;11 (9):786-796. doi: 10.1016/S2468-1253(26)00119-6. PubMed PMID:42462749 .
  18. Wani, S, Alsabti, H, Allamki, S, Almandhari, A, Alhajji, S, Alrashdi, I et al.. Establishing multiple cost-effectiveness thresholds in Oman: a stakeholder-driven approach. Health Econ Rev. 2026; :. doi: 10.1186/s13561-026-00817-w. PubMed PMID:42455234 .
  19. Yao, S, Yang, X, Ren, M, Zhang, H, Yu, S, Chen, T et al.. AI-powered therapeutic aptamer drug discovery: Targeting the CT-domain of CTGF for duchenne muscular dystrophy. Acta Pharm Sin B. 2026;16 (7):4552-4574. doi: 10.1016/j.apsb.2026.02.016. PubMed PMID:42453419 PubMed Central PMC13366227.
  20. Czarniak, P, Moorin, R, Hughes, J, Gruber, K, Youens, D. Medications That Regulate Pregnane X Receptor: A Systematic Review of Current Evidence. Pharmacol Res Perspect. 2026;14 (4):e70288. doi: 10.1002/prp2.70288. PubMed PMID:42444583 PubMed Central PMC13366386.
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