Accepted for/Published in: JMIR Formative Research
Date Submitted: Jun 30, 2019
Open Peer Review Period: Jul 5, 2019 - Aug 30, 2019
Date Accepted: Nov 11, 2020
(closed for review but you can still tweet)
Warning: This is an author submission that is not peer-reviewed or edited. Preprints - unless they show as "accepted" - should not be relied on to guide clinical practice or health-related behavior and should not be reported in news media as established information.
The REDUCE-RISK trial: a systematic review of the economic literature to provide input to the research protocol from a health economic point of view
ABSTRACT
Background:
The REDUCE-RISK trial (ClinicalTrials.gov Identifier: NCT02852694) is set up to compare the effectiveness of weekly subcutaneously administered methotrexate with daily oral azathioprine/6-mercaptopurine in low risk Crohn’s disease (CD) or subcutaneously administered adalimumab in high risk CD in a paediatric population (age 6-17).
Objective:
A systematic economic literature review was performed to provide input to the research protocol in order to gather the necessary information to allow the performance of an evidence-based economic evaluation when the trial is finished.
Methods:
A) Data sources: The Centre for Reviews and Dissemination (CRD) Health Technology Assessment (HTA) database, websites of HTA institutes, CRD’s National Health Service Economic Evaluation Database (NHS EED), Medline (OVID), and EMBASE databases were consulted to retrieve (reviews of) relevant economic evaluations. B) Eligibility criteria: Studies were eligible if they included a paediatric or adult population with inflammatory bowel diseases (Crohn’s disease (CD) and ulcerative colitis (UC)) treated with adalimumab (Humira®). There were no restrictions for the comparator. Only economic evaluations expressing outcomes in life-years gained or quality-adjusted life years gained were selected.
Results:
Twelve primary studies were identified. None of these studies included a paediatric population due to a lack of supporting trials. The economic evaluations identified in our systematic review indicate adalimumab is an appropriate intervention for inclusion in such a trial. From a health economic point of view, it is important to make an incremental analysis comparing such an intervention with standard care, and not immediately versus another (expensive) biological treatment. Information on the impact of children’s school attendance and parents’ productivity is currently lacking in the economic evaluations and none of the underlying trials measured QoL with a generic utility instrument.
Conclusions:
The review of the economic literature of adalimumab for the treatment of patients with CD supports the performance of a trial with biologicals in paediatric patients, including making a distinction according to severity of disease. Carrying out an economic literature review has allowed us to decide which variables should be added to the research protocol from an economic point of view. Measurements for children’s and parents’ quality of life (EQ-5D questionnaires), children’s school attendance and parents’ productivity (WPAI-CD-CG questionnaire) were added to the research protocol. This will provide support for the calculation of the cost-effectiveness of the interventions evaluated in the REDUCE-RISK trial. Clinical Trial: ClinicalTrials.gov Identifier: NCT02852694
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