TY - JOUR
T1 - ADVANCE system testing
T2 - Benefit-risk analysis of a marketed vaccine using multi-criteria decision analysis and individual-level state transition modelling
AU - Bollaerts, Kaatje
AU - Ledent, Edouard
AU - de Smedt, Tom
AU - Weibel, Daniel
AU - Emborg, Hanne-Dorthe
AU - Danieli, Giorgia
AU - Duarte-Salles, Talita
AU - Huerta, Consuelo
AU - Martín-Merino, Elisa
AU - Picelli, Gino
AU - Tramontan, Lara
AU - Sturkenboom, Miriam
AU - Bauchau, Vincent
N1 - Funding Information:
The Innovative Medicines Initiative Joint Undertaking funded this project under ADVANCE grant agreement n° 115557 , resources of which were composed of a financial contribution from the European Union's Seventh Framework Programme ( FP7/2007-2013 ) and in kind contributions from EFPIA member companies.
Funding Information:
The authors would like to thank Margaret Haugh, MediCom Consult, Villeurbanne, France for editorial services and Lina Titievsky, Pfizer, USA, for project lead activities. The results described in this publication are from the proof of concept studies conducted as part of the IMI ADVANCE project with the aim of testing the methodological aspects of the design, conduct and reporting of studies for vaccine benefit-risk monitoring activities. The results presented herein relate solely to the testing of these methodologies and are not intended to inform regulatory or clinical decisions on the benefits and risks of the exposures under investigation. This warning should accompany any use of the results from these studies and they should be used accordingly. The views expressed in this article are the personal views of the authors and should not be understood or quoted as being made on behalf of or reflecting the position of the agencies or organisations with which the authors are affiliated. The Innovative Medicines Initiative Joint Undertaking funded this project under ADVANCE grant agreement n? 115557, resources of which were composed of a financial contribution from the European Union's Seventh Framework Programme (FP7/2007-2013) and in kind contributions from EFPIA member companies.
Funding Information:
Tom de Smedt, Hanne-Dorthe Emborg, Giorgia Danieli, Talita Duarte-Salles, Consuelo Huerta, Elisa Martin-Merino, Gino Picelli and Lara Tramontan declared no potential conflicts of interest. Kaatje Bollaerts and Daniel Weibel received consultancy fees from GSK for work unrelated to the submitted work. Miriam Sturkenboom declared that she has received grants from Novartis, CDC and Bill & Melinda Gates Foundation, for work unrelated to the submitted work. Edouard Ledent and Vincent Bauchau declared that he is employed by GSK and holds company shares.
Publisher Copyright:
© 2019 The Authors
PY - 2020/12/22
Y1 - 2020/12/22
N2 - BACKGROUND: The Accelerated Development of VAccine beNefit-risk Collaboration in Europe (ADVANCE) is a public-private collaboration aiming to develop and test a system for rapid benefit-risk (B/R) monitoring of vaccines using electronic health record (eHR) databases in Europe. Proof-of-concept studies were designed to assess the proposed processes and system for generating the required evidence to perform B/R assessment and near-real time monitoring of vaccines. We aimed to test B/R methodologies for vaccines, using the comparison of the B/R profiles of whole-cell (wP) and acellular pertussis (aP) vaccine formulations in children as an example.METHODS: We used multi-criteria decision analysis (MCDA) to structure the B/R assessment combined with individual-level state transition modelling to build the B/R effects table. In the state transition model, we simulated the number of events in two hypothetical cohorts of 1 million children followed from first pertussis dose till pre-school-entry booster (or six years of age, whichever occurred first), with one cohort receiving wP, and the other aP. The benefits were reductions in pertussis incidence and complications. The risks were increased incidences of febrile convulsions, fever, hypotonic-hyporesponsive episodes, injection-site reactions and persistent crying. Most model parameters were informed by estimates (coverage, background incidences, relative risks) from eHR databases from Denmark (SSI), Spain (BIFAP and SIDIAP), Italy (Pedianet) and the UK (RCGP-RSC and THIN). Preferences were elicited from clinical and epidemiological experts.RESULTS: Using state transition modelling to build the B/R effects table facilitated the comparison of different vaccine effects (e.g. immediate vaccine risks vs long-term vaccine benefits). Estimates from eHR databases could be used to inform the simulation model. The model results could be easily combined with preference weights to obtain B/R scores.CONCLUSION: Existing B/R methodology, modelling and estimates from eHR databases can be successfully used for B/R assessment of vaccines.
AB - BACKGROUND: The Accelerated Development of VAccine beNefit-risk Collaboration in Europe (ADVANCE) is a public-private collaboration aiming to develop and test a system for rapid benefit-risk (B/R) monitoring of vaccines using electronic health record (eHR) databases in Europe. Proof-of-concept studies were designed to assess the proposed processes and system for generating the required evidence to perform B/R assessment and near-real time monitoring of vaccines. We aimed to test B/R methodologies for vaccines, using the comparison of the B/R profiles of whole-cell (wP) and acellular pertussis (aP) vaccine formulations in children as an example.METHODS: We used multi-criteria decision analysis (MCDA) to structure the B/R assessment combined with individual-level state transition modelling to build the B/R effects table. In the state transition model, we simulated the number of events in two hypothetical cohorts of 1 million children followed from first pertussis dose till pre-school-entry booster (or six years of age, whichever occurred first), with one cohort receiving wP, and the other aP. The benefits were reductions in pertussis incidence and complications. The risks were increased incidences of febrile convulsions, fever, hypotonic-hyporesponsive episodes, injection-site reactions and persistent crying. Most model parameters were informed by estimates (coverage, background incidences, relative risks) from eHR databases from Denmark (SSI), Spain (BIFAP and SIDIAP), Italy (Pedianet) and the UK (RCGP-RSC and THIN). Preferences were elicited from clinical and epidemiological experts.RESULTS: Using state transition modelling to build the B/R effects table facilitated the comparison of different vaccine effects (e.g. immediate vaccine risks vs long-term vaccine benefits). Estimates from eHR databases could be used to inform the simulation model. The model results could be easily combined with preference weights to obtain B/R scores.CONCLUSION: Existing B/R methodology, modelling and estimates from eHR databases can be successfully used for B/R assessment of vaccines.
KW - Benefit-risk assessment
KW - Electronic health record databases
KW - Europe
KW - Methodological study
KW - Pertussis vaccines
UR - http://www.scopus.com/inward/record.url?scp=85079398459&partnerID=8YFLogxK
U2 - 10.1016/j.vaccine.2019.09.034
DO - 10.1016/j.vaccine.2019.09.034
M3 - Article
C2 - 31677947
SN - 0264-410X
VL - 38 Suppl 2
SP - B65-B75
JO - Vaccine
JF - Vaccine
IS - 2
ER -