Conceptual model for the health technology assessment of current and novel interventions in rheumatoid arthritis

Alemao, E.; Al, Maiwenn; Boonen, Annelies; Stevenson, M; Verstappen, Suzanne; Michaud, Kaleb; Weinblatt, Michael E.; Mölken, Maureen P.M.H. Rutten-van

doi:10.1371/journal.pone.0205013

Cited by 10 publications

(7 citation statements)

References 87 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The likelihood that the available data to inform sequencing effects may improve over time also supports developing a model that is easily updated. This is consistent with recent calls for the use of disease-specific reference models [ 155 ], pre-verified modules [ 156 ], and open-source models [ 157 ] to improve the accuracy of economic evaluations. Our review identified some good examples where a model was further developed over time to address multiple reimbursement decisions (e.g.…”

Section: Discussionsupporting

confidence: 88%

Quantitative Evidence Synthesis Methods for the Assessment of the Effectiveness of Treatment Sequences for Clinical and Economic Decision Making: A Review and Taxonomy of Simplifying Assumptions

et al. 2020

View full text Add to dashboard Cite

Sequential use of alternative treatments for chronic conditions represents a complex intervention pathway; previous treatment and patient characteristics affect both the choice and effectiveness of subsequent treatments. This paper critically explores the methods for quantitative evidence synthesis of the effectiveness of sequential treatment options within a health technology assessment (HTA) or similar process. It covers methods for developing summary estimates of clinical effectiveness or the clinical inputs for the cost-effectiveness assessment and can encompass any disease condition. A comprehensive review of current approaches is presented, which considers meta-analytic methods for assessing the clinical effectiveness of treatment sequences and decision-analytic modelling approaches used to evaluate the effectiveness of treatment sequences. Estimating the effectiveness of a sequence of treatments is not straightforward or trivial and is severely hampered by the limitations of the evidence base. Randomised controlled trials (RCTs) of sequences were often absent or very limited. In the absence of sufficient RCTs of whole sequences, there is no single best way to evaluate treatment sequences; however, some approaches could be re-used or adapted, sharing ideas across different disease conditions. Each has advantages and disadvantages, and is influenced by the evidence available, extent of treatment sequences (number of treatment lines or permutations), and complexity of the decision problem. Due to the scarcity of data, modelling studies applied simplifying assumptions to data on discrete treatments. A taxonomy for all possible assumptions was developed, providing a unique resource to aid the critique of existing decision-analytic models.

show abstract

Section: Discussionsupporting

confidence: 88%

Quantitative Evidence Synthesis Methods for the Assessment of the Effectiveness of Treatment Sequences for Clinical and Economic Decision Making: A Review and Taxonomy of Simplifying Assumptions

et al. 2020

View full text Add to dashboard Cite

show abstract

“…In addition to the above specific limitations relating to the implementation of the modeling framework, more recent proposals for alternative cost-effectiveness evaluation methodologies in RA should be noted. Alemao et al recently presented an RA conceptual model proposing, among other things, use of composite measures of disease activity to evaluate treatment response as well as disease progression, utility mapping and mortality assessment based on disease activity, and incorporation of extra-articular outcomes and of subgroups based on prognostic factors [39]. While the baricitinib modeling framework is consistent with the overarching model structure of the conceptual model suggested by Alemao et al, the consensus suggestions highlight areas for future extension of the baricitinib model.…”

Section: Discussionmentioning

confidence: 99%

A modeling framework for the economic evaluation of baricitinib in moderate-to-severe rheumatoid arthritis

Schlueter

Rouse

Pitcher

et al. 2020

Expert Review of Pharmacoeconomics & Outcomes Research

View full text Add to dashboard Cite

Objectives: The approval in more than 50 countries of baricitinib, an oral Janus Kinase inhibitor for the treatment of Rheumatoid Arthritis (RA), warrants a framework for corresponding economic evaluations. To develop a comprehensive economic model assessing the cost-effectiveness of baricitinib for the treatment of moderately-to-severely active RA patients in comparison to other relevant treatments, considering the natural history of the disease, real world treatment patterns, and clinical evidence from the baricitinib trials. Methods: A systematic literature review of previously developed models in RA was conducted to inform the model structure, key modeling assumptions and data inputs. Consultations with rheumatologists were undertaken to validate the modeling approach and underlying assumptions. Results: A discrete event simulation model was developed to international best practices with flexibility to assess the cost-effectiveness of baricitinib over a lifetime in a variety of markets. The model incorporates treatment sequencing to adequately reflect treatment pathways in clinical practice. Outcomes assessed include cost and quality-adjusted life years, allowing for a full incremental analysis of cost-effectiveness of competing treatments and treatment sequences. Conclusion:The economic model developed provides a robust framework for future analyses assessing the cost-effectiveness of baricitinib for the treatment of RA in specific country settings.

show abstract

“…Since the literature of cost-effectiveness simulation Markov models has grown exponentially over the past years, we will not review more models here, but rather refer the reader to the studies in [103,102,111,56,52,120,54,14,44,51,65,3] and the references therein. However, in Section 5, we will return to these types of models and discuss their importance on personalising RA treatments for different patients in different countries, which might impact also the deterministic mathematical models used to understand the biological mechanisms behind the evolution of the disease.…”

Section: Probabilistic Cost-effectiveness Models For Ra Treatment Strmentioning

confidence: 99%

Quantitative Predictive Modelling Approaches to Understanding Rheumatoid Arthritis: A Brief Review

Macfarlane

Chaplain

Eftimie

2019

Cells

View full text Add to dashboard Cite

Rheumatoid arthritis is a chronic autoimmune disease that is a major public health challenge. The disease is characterised by inflammation of synovial joints and cartilage erosion, which lead to chronic pain, poor life quality and, in some cases, mortality. Understanding the biological mechanisms behind the progression of the disease, as well as developing new methods for quantitative predictions of disease progression in the presence/absence of various therapies is important for the success of therapeutic approaches. The aim of this study is to review various quantitative predictive modelling approaches for understanding rheumatoid arthritis. To this end, we start by briefly discussing the biology of this disease and some current treatment approaches, as well as emphasising some of the open problems in the field. Then, we review various mathematical mechanistic models derived to address some of these open problems. We discuss models that investigate the biological mechanisms behind the progression of the disease, as well as pharmacokinetic and pharmacodynamic models for various drug therapies. Furthermore, we highlight models aimed at optimising the costs of the treatments while taking into consideration the evolution of the disease and potential complications.

show abstract

Conceptual model for the health technology assessment of current and novel interventions in rheumatoid arthritis

Cited by 10 publications

References 87 publications

Quantitative Evidence Synthesis Methods for the Assessment of the Effectiveness of Treatment Sequences for Clinical and Economic Decision Making: A Review and Taxonomy of Simplifying Assumptions

Quantitative Evidence Synthesis Methods for the Assessment of the Effectiveness of Treatment Sequences for Clinical and Economic Decision Making: A Review and Taxonomy of Simplifying Assumptions

A modeling framework for the economic evaluation of baricitinib in moderate-to-severe rheumatoid arthritis

Quantitative Predictive Modelling Approaches to Understanding Rheumatoid Arthritis: A Brief Review

Contact Info

Product

Resources

About