Session Description: Economic modeling of Type 2 Diabetes Mellitus (T2DM) occupies a unique place on the cutting edge of health economics. It has a relatively long history (dating back to the seminal NIH model in 1997) and the sophisticated nature of the models themselves has, in part, led to the development of a number of important institutions, self-regulated by the modelers themselves, to address information gaps and quality control (including diabetes-specific modeling guidelines and model review summits). The proposed session intends to capitalize on this unique area and consider the impact and performance of economic modeling in health economic analysis and to identify key lessons for the benefit of future efforts. The session will be chaired by Ulf Persson (The Swedish Institute for Health Economics) and consist of three sections: - Introduction and critical review of the principal T2DM economic models developed since 1997 and their use (public domain) in health technology appraisal (HTA) - Overview and discussion of some important additional decision-making uses that have been discovered for T2DM economic models in the drug development life cycle - Assessment of the impact of the models from different perspectives, including those of modelers themselves, health technology assessment bodies, and the pharmaceutical industry. Recommendations for future improvements will be provided. Abstract #1: Background & History of DM Modeling Rationale/Background: T2DM is common, chronic, and associated with severe and costly micro- and macrovascular complications. Important clinical trials, e.g. the DCCT (for Type 1 DM) and the UKPDS (for Type 2 DM), have demonstrated that intensive treatment with anti-hyperglycemic agents improves patient outcomes by postponing or preventing these complications, with corresponding cost savings. Administering effective new treatments for a chronic lifetime condition such as diabetes is not without cost, however, so HTA has been used extensively in DM to help allocate health care funds wisely (i.e., by guiding drug reimbursement decisions). There are important challenges to providing useful, unbiased health economic data in T2DM. Foremost, T2DM is chronic and progressive, and the long-term outcomes trials needed to weigh the costs of long-term therapy against the benefits and cost savings of avoided future complications are rarely feasible (and if so, do little to assist with decisions that must be made now). Beginning with the seminal NIH model in 1997 (Eastman et al, 1997), most cost-effectiveness evaluations for T2DM have been conducted using economic modeling techniques. A large number of competing economic models of T2DM have been developed and used for HTA applications. Most of these models are complex, quite sophisticated, and almost by necessity opaque (i.e., a full understanding of the model workings is all but impossible for most). Because it is often hard to assess the reliability of modeling results, a number of interesting institutions have evolved to help regulate the modeling of T2DM. In particular, the ADA and others have published DM-specific modeling standards (ADA Consensus Panel, 2004). Moreover, three public summits have been held in which T2DM modelers have been invited in to present, compare, and evaluate the various models (Brown et al, 2000). Objective: Provide a historical summary of important developments in DM modeling as a background for a detailed discussion of what we can learn from 10 years of experience. Abstract # 2: Rationale/Background In addition to the important role that has been allocated to supporting HTA and reimbursement decisions, other important uses of economic models of T2DM have been developed for increasing efficiency in the supply of new drugs. For example, modeling conducted early in the drug discovery and development process can be used to: - Identify earlier those drug prospects that are unlikely to be cost-efficient - Improve clinical trial design (e.g., ensure relevant endpoints, satisfactory study duration, and ensure relevant comparators are included) - Evaluate competing indications- Analyze strategic trade-offs between dosing, efficacy, and tolerability, as well as the consequent impact on patient compliance- Identify which sub-groups are likely to benefit the mostMoreover, economic models can provide information to help assess the value of collecting additional information via clinical testing (i.e., so-called value of information (VOI) analysis). Objectives: To discuss the potential benefits of economic modeling in early-stage decision-making. Abstract # 3: What Can Be Learned From A Decade of T2DM Economic Modeling? Rationale/Background: Economic models of T2DM have been widely used for Cost-Effectiveness analysis and HTA submissions during the past 10 years. To our knowledge, however, no one has evaluated just how useful this economic modeling has actually been. Objective: To spawn a lively discussion, taking into account competing perspectives (e.g., HTA bodies, industry, and the modelers), about what can be learned from the first 10 years of economic modeling in T2DM. Themes relevant to other chronic diseases will be highlighted. Methods: Guided discussion. The speakers will discuss and debate a number of key aspects of T2DM modeling, including the following questions: - Have T2DM economic models been perceived as usable, accurate, reliable, credible/trustworthy, and relevant for making important health care decisions such as reimbursement and usage guidelines? What is the overall degree of acceptance? - How do users of model results (e.g., HTA bodies) deal with the question of transparency? - Have the models been used appropriately in actual practice? - What has been the impact on real-world decision-making? - What are the strengths, weaknesses, and challenges for the future? What should be done in the future to improve model-based HE data? What has, can, and should be done to promote validity and credibility?
