Methods applied in cost-effectiveness models for treatment strategies in type 2 diabetes mellitus and their use in Health Technology Assessments: a systematic review of the literature from 2008 to 2013

Abstract

Objective:

To identify and compare health-economic models that were developed to evaluate the cost-effectiveness of treatments for type 2 diabetes mellitus (T2DM), and their use within Health Technology Assessments (HTAs).

Methods:

In total, six commonly used databases were searched for articles published between October 2008 and January 2013, using a protocolized search strategy and inclusion criteria. The websites of HTA organizations in nine countries, and proceedings from five relevant conferences, were also reviewed. The identified new health-economic models were qualitatively assessed using six criteria that were developed based on technical components, and characteristics related to the disease or the treatments being assessed. Finally, the number of times the models were applied within HTA reports, published literature, and/or major conferences was determined.

Results:

Thirteen new models were identified and reviewed in depth. Most of these were based on identical key data sources, and applied a similar model structure, either using Markov modeling or microsimulation techniques. The UKPDS equations and panel regressions were frequently used to estimate the occurrence of diabetes-related complications and the probability of developing risk factors in the long term. The qualitative assessment demonstrated that the CARDIFF, Sheffield T2DM and ECHO T2DM models seem technically equipped to appropriately assess the long-term health-economic consequences of chronic treatments for patients with T2DM. It was observed that the CORE model is the most widely described in literature and conferences, and the most often applied model within HTA submissions, followed by the CARDIFF and UKPDS models.

Conclusion:

This research provides an overview of T2DM models that were developed between 2008 and January 2013. The outcomes of the qualitative assessments, combined with frequent use in local reimbursement decisions, prove the applicability of the CORE, CARDIFF and UKPDS models to address decision problems related to the long-term clinical and economic consequences of new and existing T2DM treatments.

Keywords: :

• Diabetes
• Health economics
• Model
• Systematic literature review
• Qualitative assessment

Transparency

Declaration of funding

Pharmerit International was funded by Bristol-Myers Squibb and AstraZeneca to undertake this research and develop the manuscript.

Author contributions: It is confirmed that all authors a) contributed to the conception and design, or analysis and interpretation of the data; b) contributed to the drafting of the article or revising it critically for important intellectual content; c) helped with the final approval of the version to be published; and d) agreed to be accountable for all aspects of the work ensuring that questions related to the accuracy or the integrity of the work are appropriately investigated and resolved.

Declaration of financial/other relationships

M.C was an employee of Pharmerit International at the time of the research and is now employee of UCB Biopharma SPRL and B.G.V. have disclosed that they are employees of Pharmerit International, which received funding for this research. F.J.S., R.T., and M.R. have disclosed that they are employees of at the time of the research Bristol-Myers Squibb and AstraZeneca that contributed to the research and manuscript development. P.M., professor at the Centre for Health Economics, Swansea University reviewed and approved the manuscript giving his technical expertise.

CMRO peer reviewer 1 has disclosed that he has been a consultant to Novartis, BMS, Allergan, AstraZeneca, Shire, Takeda, Bayer, Roche, Bausch and Lomb, Lundbeck, BioMarin, BI, Baxter, Sanofi, Ferring, Nycomed, Archimedes, Galderma UK, and Chugai Pharma. CMRO peer reviewer 2 has disclosed that he has been a consultant to Avita Medical Ltd, Bayer plc, Daiichi-Sankyo Europe, EUSA Pharma, GlaxoSmithKline, Ipsen Pharma, Janssen EMEA, Janssen UK, Menarini Pharma UK and Norgine Ltd. CMRO peer reviewer 3 has no relevant financial or other relationships to disclose.

Acknowledgments

The authors have disclosed that they had no outside editorial assistance in preparing this manuscript. The sponsors reviewed the article. The authors thank the editors of CMRO and the three anonymous reviewers for their valuable comments.

Data from this paper were presented previously in poster form at ISPOR 12th Annual European Congress 2013: PRM90.