HTA Frameworks for RA Therapies Need Improvement, Researchers Say

Cost-effectiveness models for the treatment of rheumatoid arthritis (RA) first emerged in the early 2000s when highly effective but also high-cost biologic therapies began to reach patients. However, these health technology assessment (HTA) models need improvement, a recent paper says.
Kelly Davio
May 13, 2019
Cost-effectiveness models for the treatment of rheumatoid arthritis (RA) first emerged in the early 2000s when highly effective but also high-cost biologic therapies began to reach patients. However, these models need improvement, a recent paper says.

According to the paper, which appeared in Plos One, current health technology assessments (HTAs) for RA rely on disease activity measures that are not aligned to guideline-recommended target measures of remission, and they may even be biased in favor of certain therapies insofar as they privilege measures like C-reactive protein reduction (an outcome particularly achieved with interleukin-6 inhibitors).

Furthermore, disease progression tends to be measured using the Health Assessment Questionnaire (HAQ), which is more sensitive to changes in patients with recent-onset disease. Most models also do not report cost effectiveness according to important subgroups, extra-articular manifestations may not be appropriately considered, and current models do not allow for data in which patients require dose escalation or de-escalation, or reinitiation of biologic therapy after discontinuation and subsequent disease flares.

As a response to these concerns, the authors of the paper developed a conceptual model based on the International Society of Pharamacoeconomic and Outcomes Research-Society of Medical Decision Making Modeling Good Research Practices Task Force-2 guidelines. They conducted a systematic literature review of existing models as well as an analysis of an RA registry. Then, they convened an expert panel for review.

The resulting model proposes using at least 2 composite measures of disease activity, with 1 used in sensitivity analyses, to evaluate treatment response and disease progression until an objective measure of disease activity is established in RA and used in routine clinical practice.

It also proposes utility mapping based on disease activity and the consideration of subgroups based on prognostic factors and potential treatment-effect modifiers. Stratification of cost-effectiveness by subgroups could have implications for targeting specific treatments or combinations to improve overall clinical outcomes and costs, and could allow for more personalized medicine in RA, the investigators write.

The model also calls for the incorporation of realistic treatment patterns based on clinical practice and registry datasets, as well as the incorporation of extra-articular outcomes and mortality based on disease activity.

Next steps for developing this model, write the authors, include building a model prototype and evaluating its feasibility, as well as considering other registries, including those that focus on treat-to-target approaches. More work will also be needed to specifically address cost effectiveness from the societal perspective, they add.

Alemao E, Al MJ, Boonen AA, et al. Conceptual model for the health technology assessment of current and novel interventions in rheumatoid arthritis [published online October 5, 2018]. Plos One. doi: 10.1371/journal.pone.0205013.


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