A DELPHI study on aspects of study design to overcome knowledge gaps on the burden of disease caused by serogroup B invasive meningococcal disease

Health and Quality of Life Outcomes

Philipp Henneke¹  Claire Wright²  Christian Dohna-Schwake³  Ole Marten⁴  Wolfgang Greiner⁴  Monika Bullinger⁵  Robert Welte⁶  Markus Kirchner⁶  Florian Koerber⁷  Hannah Christensen⁸  Philippe De Wals⁹  David Bloom^1,10  Andrea L. Monteiro^1,11  Burkhard Lawrenz^1,12  Markus Knuf^1,13  Joseph Patrick Sevilla^1,14  Corinne Buysse^1,15  Nicolas Van de Velde^1,16 

[1] 0000 0000 9428 7911, grid.7708.8, Center for Pediatrics and Adolescent Medicine and Center for Chronic Immunodeficiency, Medical Center - University of Freiburg, 79106, Freiburg, Germany;0000 0000 9642 0149, grid.453944.b, Meningitis Research Foundation, Newminster House, Baldwin Street, BS1 1LT, Bristol, UK;0000 0001 0262 7331, grid.410718.b, Pediatrics I, University Hospital of Essen, Hufelandstr. 55, 45147, Essen, Germany;0000 0001 0944 9128, grid.7491.b, Department of Health Economics and Health Care Management, School of Public Health, Bielefeld University, Universitätsstr. 25, 33615, Bielefeld, Germany;0000 0001 2180 3484, grid.13648.38, Department of Medical Psychology, University Medical Center Hamburg-Eppendorf, Martinistr. 52, 20246, Hamburg, Germany;0000 0004 0609 8483, grid.420105.2, GSK, Prinzregentenplatz 9, 81675, Munich, Germany;0000 0004 0609 8483, grid.420105.2, GSK, Prinzregentenplatz 9, 81675, Munich, Germany;0000 0004 0483 2525, grid.4567.0, Present Address: Institute of Health Economics and Health Care Management, Helmholtz Center Munich, German Research Center for Environmental Health (GmbH), Ingolstädter Landstr. 1, 85764, Neuherberg, Germany;0000 0004 1936 7603, grid.5337.2, Population Health Sciences, Bristol Medical School, University of Bristol, Oakfield House, Oakfield Grove, BS8 2BN, Bristol, UK;0000 0004 1936 8390, grid.23856.3a, Department of Social and Preventive Medicine, Laval University, Avenue de la Médecine 1050, G1V 0A6, Quebec City, Canada;000000041936754X, grid.38142.3c, Department of Global Health and Population, Harvard T.H. Chan School of Public Health, 665 Huntington Avenue, 02115, Boston, Massachusetts, USA;Asas Avaliacoes Economicas Em Saude Ltda ME, Rua Mario Pederneiras, 00055, APT 103 BLC II, Humaitá, 22261-020, Rio de Janeiro, Brazil;0000 0001 2175 0319, grid.185648.6, Present Address: Department of Pharmacy Systems, Outcomes and Policy, College of Pharmacy, University of Illinois at Chicago, Chicago, Illinois, USA;Association of Professional Pediatricians and Adolescent Medicine Specialists in Germany (Berufsverband der Kinder- und Jugendärzte e.V., BVKJ), Mielenforster Str. 2, 51069, Köln, Germany;Private Practice for Pediatrics and Adolescent Medicine, Grafenstr. 80, 59821, Arnsberg, Germany;HELIOS Dr. Horst Schmidt Kliniken, Children‘s Hospital, Ludwig-Erhard-Str. 100, 65199, Wiesbaden, Germany;grid.410607.4, Pediatric Infectious Diseases, University Medicine, 55131, Mainz, Germany;Life Sciences Group, Data for Decisions, LLC, 681 Main Street, Suite 3-37, 02451, Waltham, Massachusetts, USA;grid.416135.4, Department of Pediatrics, Intensive Care Unit, Erasmus Medical Center, Sophia Children’s Hospital, Wytemaweg 80, 3015, Rotterdam, CN, The Netherlands;grid.425090.a, GSK, 20 Avenue Fleming, 1300, Wavre, Belgium;

关键词: Quality of life;    Meningococcal disease;    Neisseria meningitidis;    DELPHI;   

DOI  :  10.1186/s12955-019-1159-0

来源: publisher

【 摘 要 】

BackgroundValue assessment of vaccination programs against serogroup B invasive meningococcal disease (IMD) is on the agenda of public health authorities. Current evidence on the burden due to IMD is unfit for pinning down the nature and magnitude of the full social and economic costs of IMD for two reasons. First, the concepts and components that need to be studied are not agreed, and second, measures of the concepts that have been studied are weak and inconsistent. Thus, the economic evaluation of the available serogroup B meningococcal (MenB) vaccines is difficult. The aims of this DELPHI study are to: (1) agree on the concepts and components determining the burden of MenB diseases that need to be studied; and (2) seek consensus on appropriate methods and study designs to measure quality of life (QoL) associated with MenB induced long-term sequelae in future studies.MethodsWe designed a DELPHI questionnaire based on the findings of a recent systematic review on the QoL associated with IMD-induced long-term sequelae, and iteratively interviewed a panel of international experts, including physicians, health economists, and patient representatives. Experts were provided with a controlled feedback based on the results of the previous round.ResultsExperts reached consensus on all questions after two DELPHI rounds. Major gaps in the literature relate (i) to the classification of sequelae, which allows differentiation of severity levels, (ii) to the choice of QoL measures, and (iii) to appropriate data sources to examine long-term changes and deficits in patients’ QoL.ConclusionsBetter conceptualisation of the structure of IMD-specific sequelae and of how their diverse forms of severity might impact the QoL of survivors of IMD as well as their family network and care-providers is needed to generate relevant, reliable and generalisable data on QoL in the future. The results of this DELPHI panel provide useful guidance on how to choose the study design, target population and appropriate QoL measures for future research and hence, help promote the appropriateness and consistency in study methodology and sample characteristics.

【 授权许可】

CC BY