An online decision tree for vaccine efficacy trial design during infectious disease epidemics: The InterVax-Tool

Steven E. Bellan, Rosalind M. Eggo, Pierre Stéphane Gsell, Adam J. Kucharski, Natalie E. Dean, Richard Donohue, Matt Zook, W. John Edmunds, Frank Odhiambo, Ira M. Longini, Marc Brisson, Barbara E. Mahon, Ana Maria Henao-Restrepo

Research output: Contribution to journalArticlepeer-review

6 Scopus citations

Abstract

Background: Licensed vaccines are urgently needed for emerging infectious diseases, but the nature of these epidemics causes challenges for the design of phase III trials to evaluate vaccine efficacy. Designing and executing rigorous, fast, and ethical, vaccine efficacy trials is difficult, and the decisions and limitations in the design of these trials encompass epidemiological, logistical, regulatory, statistical, and ethical dimensions. Results: Trial design decisions are complex and interrelated, but current guidance documents do not lend themselves to efficient decision-making. We created InterVax-Tool (http://vaxeval.com), an online, interactive decision-support tool, to help diverse stakeholders navigate the decisions in the design of phase III vaccine trials. InterVax-Tool offers high-level visual and interactive assistance through a set of four decision trees, guiding users through selection of the: (1) Primary Endpoint, (2) Target Population, (3) Randomization Scheme, and, (4) Comparator. We provide guidance on how key considerations – grouped as Epidemiological, Vaccine-related, Infrastructural, or Sociocultural – inform each decision in the trial design process. Conclusions: InterVax-Tool facilitates structured, transparent, and collaborative discussion of trial design, while recording the decision-making process. Users can save and share their decisions, which is useful both for comparing proposed trial designs, and for justifying particular design choices. Here, we describe the goals and features of InterVax-Tool as well as its application to the design of a Zika vaccine efficacy trial.

Original languageEnglish
Pages (from-to)4376-4381
Number of pages6
JournalVaccine
Volume37
Issue number31
DOIs
StatePublished - Jul 18 2019

Bibliographical note

Funding Information:
SEB was supported by National Institute of Health ( NIH ) National Institute of Allergy and Infectious Diseases grant K01AI125830 . RME acknowledges funding from the National Institute for Health Research through the Health Protection Research Unit in Immunisation at the London School of Hygiene & Tropical Medicine in partnership with Public Health England, from an HDR UK Innovation Fellowship (grant MR/S003975/1 ), and from the Innovative Medicines Initiative 2 (IMI2) Joint Undertaking under grant agreement EBOVAC1 (grant 115854 ). The IMI2 is supported by the European Union Horizon 2020 Research and Innovation Programme and the European Federation of Pharmaceutical Industries and Associations. AJK was supported by a Sir Henry Dale Fellowship jointly funded by the Wellcome Trust and the Royal Society (grant 206250/Z/17/Z ). NED and IML were supported by National Institutes of Health (NIH) grants U54 GM111274 and R37-AI032042 , and WHO funding. WJE acknowledges VEEPED (Vaccine Efficacy Evaluation for Priority Emerging Diseases) for funding. VEEPED is funded by the National Institute for Health Research using Official Development Assistance (ODA) funding.

Funding Information:
SEB was supported by National Institute of Health (NIH) National Institute of Allergy and Infectious Diseases grant K01AI125830. RME acknowledges funding from the National Institute for Health Research through the Health Protection Research Unit in Immunisation at the London School of Hygiene & Tropical Medicine in partnership with Public Health England, from an HDR UK Innovation Fellowship (grant MR/S003975/1), and from the Innovative Medicines Initiative 2 (IMI2) Joint Undertaking under grant agreement EBOVAC1 (grant 115854). The IMI2 is supported by the European Union Horizon 2020 Research and Innovation Programme and the European Federation of Pharmaceutical Industries and Associations. AJK was supported by a Sir Henry Dale Fellowship jointly funded by the Wellcome Trust and the Royal Society (grant 206250/Z/17/Z). NED and IML were supported by National Institutes of Health (NIH) grants U54 GM111274 and R37-AI032042, and WHO funding. WJE acknowledges VEEPED (Vaccine Efficacy Evaluation for Priority Emerging Diseases) for funding. VEEPED is funded by the National Institute for Health Research using Official Development Assistance (ODA) funding. We gratefully acknowledge input from Peter Dull, Laura Rodrigues, Peter Smith, and Conall Watson in addition to the rest of the WHO R&D Blueprint Working Group on Vaccine Study Design and the attendees of the WHO Consultation on Zika Vaccine Trial Design who piloted the tool. SEB conceived the idea of a vaccine efficacy trial design decision tree. SEB, RME, PG, and AJK devised the interactive nature of the decision support tool, the hierarchical structure of the decision trees, and developed the guidance content and organization of the tool. AMHR convened the WHO Blueprint working groups for vaccine evaluation in four occasions since March 2016. AMHR, FO, MB, BEM, IL WJE, and NED contributed to the guidance content and structure. All authors contributed to the iterative development of the tool, reviewed the guidance content within the tool, and reviewed the final manuscript.

Publisher Copyright:
© 2019 The Authors

Keywords

  • Decision support system
  • Emerging infectious diseases
  • Epidemics
  • Outbreaks
  • Phase III trial
  • Public Health Emergency
  • Scientific communication
  • Vaccine trial design
  • Vaccines

ASJC Scopus subject areas

  • Molecular Medicine
  • Immunology and Microbiology (all)
  • Veterinary (all)
  • Public Health, Environmental and Occupational Health
  • Infectious Diseases

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