HEOR, which was once considered a mere support function, is now playing a significant role in pharmaceutical manufacturers’ internal decision-making process. It has become a central component for demonstrating product value, providing critical information for licensing, research, and development as well as pricing and market access strategies.

by Sonam Mehta, MS, Engagement Manager, HEOR & Clinical Operations

Health economics and outcomes research (HEOR) is a proliferating interdisciplinary field that utilizes real-world evidence to understand whether a product or service is efficacious. HEOR can provide valuable information for making healthcare coverage and policy decisions and has gained a lot of attention as healthcare costs have continued to increase despite the fact that higher cost is not associated with better outcomes or quality of life. Thus, HEOR has often been utilized in the attempt to understand the value of a product and its potential in real-world clinical practice.

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"… …pharmaceutical manufacturers need strong evidence to prove that their products deliver superior clinical outcomes and demonstrate economic value in order to meet stringent payer requirements."

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In today’s world of evolving healthcare, pricing pressures, and barriers to market access, pharmaceutical manufacturers need strong evidence to prove that their products deliver superior clinical outcomes and demonstrate economic value in order to meet stringent payer requirements. Currently, several global reimbursement agencies formally ask for HEOR information for their standard assessment process, including the National Institute for Health and Clinical Excellence in the United Kingdom, Spanish health technology assessment (HTA) agencies, the Korean Health Insurance Review Agency, and the Health Intervention and Technology Assessment Program in Thailand.^1-3 Therefore, in order to gain market access and remain competitive, manufacturers must generate evidence to demonstrate clinical and economic benefit to providers, payers, and healthcare decision makers.

Generating the evidence takes profound analytical expertise; a deep understanding of payers, providers, and patients; and actionable insights that will position products for ongoing success. HEOR analysis gives a precise picture of a product’s clinical position in the market, usage patterns, uptake trends, and more. As a consequence, pharmaceutical manufacturers invest heavily in HEOR studies alongside clinical trials in order to collect clinical, economic, and humanistic real-world data throughout the lifecycle of a product.¹ Usually, a value proposition for a product is developed on the basis of findings from phase II research, anticipated significant endpoints from phase III research, and factors considered by clinicians to be the principal treatment and outcome issues. Over the past few decades, HEOR has advanced considerably in methodology and scientific rigor. The various types of HEOR studies include systematic literature reviews (with/without meta-analyses), retrospective database analyses (using payer claims, electronic medical records, or survey data), clinical trial data analyses, economic analyses (burden of illness, cost-effectiveness, and cost-utility), decision-analytic modeling, budget-impact analyses, and global value dossiers.

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"HEOR not only demonstrates the value of an existing product but can also generate evidence that would help position a new product against an established product."

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HEOR not only demonstrates the value of an existing product but can also generate evidence that would help position a new product against an established product. In order to generate additional comparative safety and effectiveness data for new drugs apart from randomized controlled clinical trials, payers can conduct retrospective observational studies using their own routinely collected electronic healthcare data. Administrative claims data is a good source for measuring longitudinal drug and medical service utilization information.⁴ Analyses of these data can facilitate payers in assessing the benefits and risks of new drugs versus existing comparators in the same populations of interest.^5,6 An important benefit of observational HEOR is the ability to identify users of a new drug/device and track their outcomes longitudinally.⁷

In order to describe the current and anticipated future use of HEOR data by healthcare payers and pharmaceutical manufacturers as well as to determine barriers to the use of HEOR data, a study was conducted in 2010 using 2 surveys.⁸ The Managed Care Survey (MCS) was administered to US payers and formulary decision makers that provide health insurance to between tens of thousands and millions of members. The Pharmacy & Therapeutics (P&T) Committee Survey (PTS) was sent to P&T Committees in the United States to explore managed care pharmacists’ perceptions of factors affecting formulary decisions. The results showed that 74% of US respondents to the MCS and 77% of respondents to the PTS indicated that HEOR was currently being used in their decision-making process. Two-thirds of respondents to the MCS (66%) also stated that quality assessment was limited. Additionally, 82% of the respondents to the MCS expected an increase in the use of HEOR data in the future. The most important factors in the P&T Committee decision-making process were safety and efficacy, followed by head-to-head comparisons and cost.

Thus, HEOR, which was once considered a mere support function, is now playing a significant role in the internal decision-making process. It has become a central component for demonstrating product value, which provides useful information for licensing, research and development, as well as pricing and market access strategies. 

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¹ van Nooten F, Holmstrom S, Green J, et al. Health economics and outcomes research within drug development: challenges and opportunities for reimbursement and market access within biopharma research. Drug Discov Today 2012; 17:615-622.
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³ Tantivess S, Teerawattananon Y, Mills A. Strengthening cost-effectiveness analysis in Thailand through the establishment of the health intervention and technology assessment program. Pharmacoeconomics 2009; 27:931-945.
⁴ Schneeweiss S, Avorn J. A review of uses of health care utilization databases for epidemiologic research on therapeutics. J Clin Epidemiol 2005; 58:323-337.
⁵ Schneeweiss S, Gagne JJ, Glynn RJ, et al. Assessing the comparative effectiveness of newly marketed medications: methodological challenges and implications for drug development. Clin Pharmacol Ther 2011; 90:777-790.
⁶ Wahl PM, Gagne JJ, Wasser TE, et al. Early steps in the development of a claims-based targeted healthcare safety monitoring system and application to three empirical examples. Drug Saf 2012; 35:407-416.
⁷ Gagne JJ. You can observe a lot (about medical products) by watching (those who use them). Epidemiology 2013; 24:700-702.
⁸ Holtorf AP, Brixner D, Bellows B, et al. Current and future use of HEOR data in healthcare decision-making in the United States and in emerging markets. Am Health Drug Benefits 2012; 5:428-438.