Analysis of indirect treatment comparisons in national health technology assessments and requirements for industry submissions

Es-Skali, Ischa J; Spoors, John

doi:10.2217/cer-2017-0092

Cited by 11 publications

(6 citation statements)

References 4 publications

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“…An alternative approach is to consider the results of indirect treatment comparisons (ITCs) that examine the comparative efficacy of each treatment for selected key endpoints in cohorts of patients with the same well-defined selected clinical and inflammatory phenotypes. Such ITCs can provide useful comparative evidence [27], which is required by some policymakers [28].…”

Section: Introductionmentioning

confidence: 99%

Pairwise indirect treatment comparison of dupilumab versus other biologics in patients with uncontrolled persistent asthma

Bateman

Khan

et al. 2022

Respiratory Medicine

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Section: Introductionmentioning

confidence: 99%

Pairwise indirect treatment comparison of dupilumab versus other biologics in patients with uncontrolled persistent asthma

Bateman

Khan

et al. 2022

Respiratory Medicine

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“…ITCs in particular are commonly used in HTAs to provide comparative evidence, especially in the absence of direct evidence from head-to-head trials; ITCs are also important in providing robust estimates that combine direct and indirect evidence when head-to-head data are available. 29,30 However, comparative effectiveness research is often challenging to conduct and therefore unavailable, particularly in rare diseases such as BD, and in the clinical trial setting. 48 We performed a comparative SLR and similarity assessment to determine the feasibility of an ITC of the EULAR-recommended treatments for BD-associated oral ulcers.…”

Section: Discussionmentioning

confidence: 99%

“…Open makers with a summary of information related to the use of a health technology. [28][29][30] The SLR underlying the EULAR recommendations for mucocutaneous manifestations found heterogeneously reported outcomes for BD-associated oral ulcers (eg, number, duration, frequency, severity), discrepancies in statistical significance of efficacy outcomes, a dearth of adverse events reporting, and lack of head-to-head comparisons. 21 The authors noted that the limitations observed in the literature hindered a comparison of the efficacy of treatments and that a formal comparative effectiveness analysis would be valuable.…”

Section: Dovepressmentioning

confidence: 99%

Comparability of European League Against Rheumatology-Recommended Pharmacological Treatments of Oral Ulcers Associated with Behçet’s Disease: A Systematic Literature Review of Randomized Controlled Trials

Nazareth

Hart

Ronnebaum

et al. 2020

OARRR

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Objective Oral ulcers are the cardinal manifestation in Behçet’s disease (BD). The 2018 European League Against Rheumatism (EULAR) recommendations describe treatments for BD-associated oral ulcers with mucocutaneous involvement; however, little comparative effectiveness information for these agents is available. In the absence of head-to-head trials, an indirect treatment comparison (ITC) could provide useful evidence regarding comparative effectiveness of BD treatments. The purpose of this study was to conduct a comparative systematic literature review (SLR) and similarity assessment of randomized controlled trials (RCTs) investigating the oral ulcer-related efficacy outcomes of EULAR-recommended treatments for BD-associated oral ulcers to determine the feasibility of an ITC. Methods An SLR was performed to identify relevant RCTs indexed in MEDLINE/Embase before May 29, 2019. RCT similarities for the ITC were assessed based on a step-wise process recommended by the International Society for Pharmacoeconomics and Outcomes Research. Results In total, 317 articles were identified, of which 14 RCTs, reflecting 11 EULAR-recommended treatments, were evaluated in a similarity assessment. Number of oral ulcers, resolution of oral ulcers, and healing time for oral ulcers were identified as the possible oral ulcer-related outcomes. After completing the similarity assessment of these outcomes, it was determined that a robust ITC was infeasible for the three oral ulcer-related outcomes due to heterogeneity in outcomes reporting, study design, and/or patient characteristics. More broadly, the results underscore the need for and consistent use of standardized measures for oral ulcer outcomes to facilitate comparative research. Conclusion In the absence of head-to-head RCTs and infeasibility of quantitative ITC, comparative assessments for BD-associated oral ulcers are limited, including comparative effectiveness and cost-effectiveness evaluations. Healthcare decision-makers must continue to base treatment decisions on the extent and strength of available evidence (eg, robust RCTs), clinical guidelines, real-world experience, and patient considerations.

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“…On top of that, this review focused on direct comparison studies only. Indirect comparisons or meta-analyses may also have potential value to inform healthcare decision-making, although currently they are not routinely accepted by regulatory agencies except when they demonstrate noninferiority [71].…”

Section: Discussionmentioning

confidence: 99%

Comparative and combined effectiveness of innovative therapies in cancer: a literature review

et al. 2019

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To achieve therapeutic innovation in oncology, already expensive novel medicines are often concomitantly combined to potentially enhance effectiveness. While this aggravates the pricing problem, comparing effectiveness of novel yet expensive (concomitant) treatments is much needed for healthcare decisionmaking to deliver effective but affordable treatments. This study reviewed published clinical trials and real-world studies of targeted and immune therapies. In total, 48 studies compared and/or combined multiple novel products on breast, colorectal, lung and melanoma cancers. To a great extent, products evaluated in each study were owned by one manufacturer. However, cross-manufacturer assessments are also needed. Next to costs and intensive market competition, the absence of a regulatory framework enforcing real-world multiproduct studies prevents these from being conducted. Trusted third parties could facilitate such real-world studies, for which appropriate and efficient data access is needed.

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Analysis of indirect treatment comparisons in national health technology assessments and requirements for industry submissions

Cited by 11 publications

References 4 publications

Pairwise indirect treatment comparison of dupilumab versus other biologics in patients with uncontrolled persistent asthma

Pairwise indirect treatment comparison of dupilumab versus other biologics in patients with uncontrolled persistent asthma

Comparability of European League Against Rheumatology-Recommended Pharmacological Treatments of Oral Ulcers Associated with Behçet’s Disease: A Systematic Literature Review of Randomized Controlled Trials

Comparative and combined effectiveness of innovative therapies in cancer: a literature review

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