Currently submitted to: JMIR Formative Research
Date Submitted: Jun 30, 2019
Open Peer Review Period: Jul 5, 2019 - Aug 30, 2019
(closed for review but you can still tweet)
NOTE: This is an unreviewed Preprint
Warning: This is a unreviewed preprint (What is a preprint?). Readers are warned that the document has not been peer-reviewed by expert/patient reviewers or an academic editor, may contain misleading claims, and is likely to undergo changes before final publication, if accepted, or may have been rejected/withdrawn (a note “no longer under consideration” will appear above).
Peer-review me: Readers with interest and expertise are encouraged to sign up as peer-reviewer, if the paper is within an open peer-review period (in this case, a “Peer-Review Me” button to sign up as reviewer is displayed above). All preprints currently open for review are listed here. Outside of the formal open peer-review period we encourage you to tweet about the preprint.
Citation: Please cite this preprint only for review purposes or for grant applications and CVs (if you are the author).
Final version: If our system detects a final peer-reviewed “version of record” (VoR) published in any journal, a link to that VoR will appear below. Readers are then encourage to cite the VoR instead of this preprint.
Settings: If you are the author, you can login and change the preprint display settings, but the preprint URL/DOI is supposed to be stable and citable, so it should not be removed once posted.
Submit: To post your own preprint, simply submit to any JMIR journal, and choose the appropriate settings to expose your submitted version as preprint.
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
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).
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.
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.
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.
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
Request queued. Please wait while the file is being generated. It may take some time.
© The authors. All rights reserved. This is a privileged document currently under peer-review/community review (or an accepted/rejected manuscript). Authors have provided JMIR Publications with an exclusive license to publish this preprint on it's website for review and ahead-of-print citation purposes only. While the final peer-reviewed paper may be licensed under a cc-by license on publication, at this stage authors and publisher expressively prohibit redistribution of this draft paper other than for review purposes.