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Every year I look forward to the release of ISPOR’s list of top 10 HEOR trends. As a Scientific Director of Health Economics and Outcomes Research, some of the most tangible trends I’ve noted are the growth of real-world evidence generation and a push towards value-driven healthcare and shared decision making.

Real-World Evidence

Real-world evidence offers many benefits when compared to traditional controlled trials – for example, more flexible inclusion criteria leading to more representative study populations and the availability of large sample sizes. At the same time, care must be taken to avoid biases due to lack of randomization and missing data. Choosing a methodological approach that best fits study parameters is therefore important.

HealthCore recently conducted a comparative effectiveness analysis across several antihyperglycemic agents for type 2 diabetes through a combination of exact and propensity score matching to reduce selection bias [1]. In another study, researchers used decomposition methods to evaluate heterogeneous treatment effects in a group of patients initiating antihyperglycemic injectables [2].

As real-world evidence is increasingly used to inform healthcare decision making, including regulatory decisions, robust methodology is becoming ever more critical.

Value-Driven Healthcare and Decision Making

The focus and structure of healthcare decision making is also changing. Payor insights have the potential to exponentially increase the value of HEOR studies for life science partners as a collaborative approach can allow both stakeholder groups to align objectives across partners and look for opportunities to directly improve health outcomes.

In one collaborative study, HealthCore used a randomized multi-arm approach to evaluate diabetes education materials across commercially insured populations combining both prospective and retrospective elements [3]. Working in collaboration with a payor allowed researchers to improve the study design and socialize findings directly to the relevant stakeholders.

Another important input into payor decision making is cost-effectiveness analysis, which compares the additional costs, or savings, of a new treatment to the additional improvement in outcomes. All or at least some of these components can often be estimated directly from claims databases, enabling generation of evidence from a population that is highly relevant from a payor perspective. HealthCore recently conducted such an analysis to evaluate the impact of a new lipid test on costs and risk of heart disease [4].

The demand for such analyses – and value assessments more broadly – with a U.S. focus is expected to continue increasing. We are excited about these developments and the potential improvements to health outcomes decision making.

[1] Mody R, Huang Q, Yu M, Zhao R, Patel H, Grabner M, Landó LF. Adherence, persistence, glycaemic control and costs among patients with type 2 diabetes initiating dulaglutide compared with liraglutide or exenatide once weekly at 12‐month follow‐up in a real‐world setting in the United States. Diabetes Obes Metab. 2019 Apr; 21(4):920-929.

[2] Brekke L, Buysman E, Grabner M, Ke X, Xie L, Baser O, Wei W. The Use of Decomposition Methods in Real-World Treatment Benefits Evaluation for Patients with Type 2 Diabetes Initiating Different Injectable Therapies: Findings from the INITIATOR Study. Value Health. 2017 Dec;20(10):1252-1259.

[3] Bieszk N, Grabner M, Wei W, Barron J, Quimbo R, Yan T, Biel B, Chu JW. Personalized Care and the Role of Insulin as a Vehicle to Optimizing Treatments in Diabetes Care. J Manag Care Spec Pharm. 2017 Nov;23(11):1160-1168.

[4] Grabner M, Winegar D, Punekar RS, Quimbo RA, Cziraky MJ, Cromwell WC. Cost Effectiveness of Achieving Targets of Low-Density Lipoprotein Particle Number Versus Low-Density Lipoprotein Cholesterol Level. Am J Cardiol. 2017 Feb 1;119(3):404-409.