255
Views
0
CrossRef citations to date
0
Altmetric
Original Research

Accelerate vaccine development using seamless phase 2/3 trial designs

, &
Pages 523-534 | Received 14 Dec 2023, Accepted 24 Apr 2024, Published online: 07 May 2024

References

  • Mostafavi E, Eybpoosh S, Karamouzian M, et al. Efficacy and safety of a protein-based SARS-CoV-2 vaccine: a randomized clinical trial. JAMA Netw Open. 2023;6(5):e2310302–e2310302. doi: 10.1001/jamanetworkopen.2023.10302
  • Chalkias S, Whatley JL, Eder F, et al. Original SARS-CoV-2 monovalent and omicron BA. 4/BA. 5 bivalent COVID-19 mRNA vaccines: phase 2/3 trial interim results. Nature Med. 2023;29(9):2325–2333. doi: 10.1038/s41591-023-02517-y
  • Polack FP, Thomas SJ, Kitchin N, et al. Safety and efficacy of the BNT162b2 mRNA COVID-19 vaccine. N Engl J Med. 2020;383(27):2603–2615. doi: 10.1056/NEJMoa2034577
  • Jiang L, Yuan Y. Seamless phase II/III design: a useful strategy to reduce the sample size for dose optimization. JNCI. 2023;115(9):1092–1098. doi: 10.1093/jnci/djad103
  • U.S. Food & Drug Administration. Adaptive design clinical trials for drugs and biologics guidance for industry [updated 2019 Nov 29; cited 2023 Sep 12]. Available from: https://www.fda.gov/media/78495/download
  • U.S. Food & Drug Administration. Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics guidance for industry [updated 2022 Mar 1; cited 2023 Sep 13]. Available from: https://www.fda.gov/media/120721/download
  • Ellenberg S, Eisenberger M. An efficient design for phase III studies of combination chemotherapies. Cancer Treat Rep. 1985;69(10):1147–1152.
  • Thall P, Simon R, Ellenberg S. Two-stage selection and testing designs for comparative clinical trials. Biometrika. 1988;75(2):303–310. doi: 10.1093/biomet/75.2.303
  • Schaid DJ, Wieand S, Therneau TM. Optimal two-stage screening designs for survival comparisons. Biometrika. 1990;77(3):507–513. doi: 10.1093/biomet/77.3.507
  • Bauer P, Kieser M. Combining different phases in the development of medical treatments within a single trial. Statist Med. 1999;18(14):1833–1848. doi: 10.1002/(SICI)1097-0258(19990730)18:14<1833:AID-SIM221>3.0.CO;2-3
  • Stallard N, Todd S. Sequential designs for phase III clinical trials incorporating treatment selection. Stat Med. 2003;22(5):689–703. doi: 10.1002/sim.1362
  • Liu Q, Pledger GW. Phase 2 and 3 combination designs to accelerate drug development. J Am Stat Assoc. 2005;100(470):493–502. doi: 10.1198/016214504000001790
  • Schmidli H, Bretz F, Racine A, et al. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: applications and practical considerations. Biom J. 2006;48(4):635–643. doi: 10.1002/bimj.200510231
  • U.S. Food & Drug Administration. Multiple endpoints in clinical trials guidance for industry [updated 2022 Oct 20; cited 2023 Sep 15]. Available from: https://www.fda.gov/media/162416/download
  • Europen Medicines Agency. Guideline on multiplicity issues in clinical trials [updated 2016 Dec 15; cited 2023 Sep 15]. Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-multiplicity-issues-clinical-trials_en.pdf
  • Castellsagué X, Giuliano AR, Goldstone S, et al. Immunogenicity and safety of the 9-valent HPV vaccine in men. Vaccine. 2015;33(48):6892–6901. doi: 10.1016/j.vaccine.2015.06.088
  • Demets DL, Lan KG. Interim analysis: the alpha spending function approach. Stat Med. 1994;13(13‐14):1341–1352. doi: 10.1002/sim.4780131308
  • O’Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics. 1979;35(3):549–556. doi: 10.2307/2530245
  • Lan KKG, Demets DL. Discrete sequential boundaries for clinical-trials. Biometrika. 1983;70(3):659–663. doi: 10.2307/2336502
  • Cecco S, Muraro E, Giacomin E, et al. Cancer vaccines in phase II/III clinical trials: state of the art and future perspectives. Curr Cancer Drug Targets. 2011;11(1):85–102. doi: 10.2174/156800911793743664
  • Rustomjee R, McClain B, Brennan MJ, et al. Designing an adaptive phase II/III trial to evaluate efficacy, safety and immune correlates of new TB vaccines in young adults and adolescents. Tuberculosis. 2013;93(2):136–142. doi: 10.1016/j.tube.2012.11.005
  • Zhu FC, Chen W, Hu YM, et al. Efficacy, immunogenicity and safety of the HPV-16/18 AS04-adjuvanted vaccine in healthy Chinese women aged 18–25 years: results from a randomized controlled trial. Int J Cancer. 2014;135(11):2612–2622. doi: 10.1002/ijc.28897
  • Duong TN, Thiem VD, Anh DD, et al. A phase 2/3 double blinded, randomized, placebo-controlled study in healthy adult participants in Vietnam to examine the safety and immunogenicity of an inactivated whole virion, alum adjuvanted, A(H5N1) influenza vaccine (IVACFLU-A/H5N1). Vaccine. 2020;38(6):1541–1550. doi: 10.1016/j.vaccine.2019.11.059
  • Zhu FC, Hu SY, Hong Y, et al. Efficacy, immunogenicity and safety of the AS04‐HPV‐16/18 vaccine in Chinese women aged 18‐25 years: end‐of‐study results from a phase II/III, randomised, controlled trial. Cancer Med. 2019;8(14):6195–6211. doi: 10.1002/cam4.2399
  • Offen W, Chuang-Stein C, Dmitrienko A, et al. Multiple co-primary endpoints: medical and statistical solutions: a report from the multiple endpoints expert team of the pharmaceutical research and manufacturers of America. Drug Inf J. 2007;41(1):31–46. doi: 10.1177/009286150704100105
  • Sozu T, Sugimoto T, Hamasaki T, et al. Sample size determination in clinical trials with multiple endpoints. New York (NY): Springer; 2015.
  • Xiong C, Yu K, Gao F, et al. Power and sample size for clinical trials when efficacy is required in multiple endpoints: application to an Alzheimer’s treatment trial. Clin Trial. 2005;2(5):387–393. doi: 10.1191/1740774505cn112oa
  • U.S. Food & Drug Administration. Guidance for industry for the evaluation of combination vaccines for preventable diseases: production, testing and clinical studies [updated 1997 Apr 1; cited 2023 Sep 20]. Available from: https://www.fda.gov/media/77191/download
  • Europen Medicines Agency. Guideline on influenza vaccines: non-clinical and clinical module [updated 2016 Jul 21; cited 2024 Mar 20]. Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/influenza-vaccines-non-clinical-and-clinical-module_en.pdf
  • Cerqueira FP, Jesus AMC, Cotrim MD. Adaptive design: a review of the technical, statistical, and regulatory aspects of implementation in a clinical trial. Ther Innov Regul Sci. 2019;54(1):246–254. doi: 10.1007/s43441-019-00052-y
  • Chang M. Adaptive design theory and implementation using SAS and R. Boca Raton, Florida: Chapman and Hall/CRC; 2014.
  • Asakura K, Hamasaki T, Evans SR. Interim evaluation of efficacy or futility in group-sequential trials with multiple co-primary endpoints. Biom J. 2017;59(4):703–731. doi: 10.1002/bimj.201600026
  • Maca J, Dragalin V, Gallo P. Adaptive clinical trials: overview of phase III designs and challenges. Ther Innov Regul Sci. 2014;48(1):31–40. doi: 10.1177/2168479013507436
  • Gouglas D, Christodoulou M, Hatchett R. The 100 days mission—2022 global pandemic preparedness summit. Emerg Infect Dis. 2023;29(3):e221142. doi: 10.3201/eid2903.221142