Making outcomes measurable: how real-world evidence connects therapy value to cost and utilization

As healthcare stakeholders continue to shift from cost reduction to cost strategy, life sciences organizations are under growing pressure to demonstrate not only how therapies perform, but also how they translate into meaningful real-world outcomes and deliver benefit in routine clinical practice. For life sciences organizations, demonstrating value requires evidence that extends beyond clinical trial efficacy to show how treatments influence patient outcomes, healthcare utilization, and costs across diverse populations and care settings.

Real-world evidence (RWE) and health economics and outcomes research (HEOR) can help provide that evidence by evaluating treatment effectiveness, adherence, and economic impact in routine clinical practice trials. This helps life sciences organizations translate clinical trial evidence into a real-world environment by showing how therapies perform across broader patient populations, care settings, and access conditions.

One example is Carelon Research's collaboration with Sera Prognostics  and Anthem, where real-world evidence was used to evaluate pregnancy risk and help identify pregnant women at increased risk of preterm birth. By combining clinical evidence with real-world payer data, the partnership demonstrated how evidence generation can support earlier intervention, improve outcomes, and inform discussions among healthcare stakeholders.

“Clinical trials tell us how a therapy performs in a controlled setting,” says Emma Richard, MPH, Director, HEOR, Carelon Research. “Real-world evidence helps us understand what that performance looks like in routine care, across broader populations, access conditions, and treatment patterns.”

Payers increasingly consider high-quality real-world evidence alongside clinical trial data to inform coverage, reimbursement, and formulary decisions. A recent survey found that 80% of participating payer stakeholders  currently use real-world data from their own institution to inform treatment coverage decisions. When combined with rigorous analytics and payer-relevant HEOR methodologies, RWE can help life sciences organizations better understand and communicate therapy value.

With the right data and analytic expertise, life sciences organizations can build stronger evidence strategies, support more informed market access conversations, and better understand how treatments contribute to improving outcomes across the care continuum.

Key insights:

• Why measurable outcomes are essential to demonstrating therapy value
• How real-world evidence can connect treatment impact to cost and utilization in routine clinical practice
• Which indicators may matter most to payer and market access stakeholders
• How stronger evidence strategies can support better decision-making across the product life cycle
• Why data quality, analytic rigor, and payer relevance are critical to real-world evidence development
   

Why measurable outcomes matter in pharma value strategies

A therapy's impact is not defined by a single outcome. Healthcare decision-makers evaluate treatments through different lenses based on the decisions they are responsible for making. Providers may focus on clinical effectiveness and quality of care, payers often prioritize healthcare utilization, budget impact, and total cost of care, while patients may place greater importance on access, treatment persistence, symptom improvement, or quality of life. “Value can look different depending on the stakeholder. A provider may be looking at clinical improvement, a payer may be looking at cost of care, and a patient may be looking at whether they can access and stay on therapy,” says Emma Richard.

This is where health economics and outcomes research (HEOR) play an important role. By connecting evidence generation to the measures that matter most to stakeholders, life sciences organizations support more informed access conversations, and better demonstrate how therapies contribute to measurable benefit over time.
 

How real-world evidence extends evidence beyond clinical trials

Randomized controlled trials remain the gold standard for demonstrating safety and efficacy, but they are conducted under carefully controlled conditions and often include narrowly defined patient populations. Routine clinical practice is more complex. Patients differ in their comorbidities, adherence, access to care, provider practices, and treatment pathways, all of which can influence outcomes.

Real-world evidence helps life sciences organizations better understand these variations by showing how treatments are used, continued, switched, or discontinued across broader patient populations. Using data from claims, electronic health records, registries, and other real-world sources, organizations can better understand treatment patterns, persistence, comparative effectiveness, healthcare utilization, and clinical outcomes in everyday practice.
 

Connecting treatment outcomes to economic value

Treatment value is often shaped by more than whether a therapy improves outcomes, but also how those outcomes influence healthcare utilization and costs. HEOR evaluates the relationship between clinical outcomes and economic consequences by examining measures such as hospitalizations, emergency department visits, medication adherence, treatment persistence, clinical complications, and adverse events in relation to total cost of care. Depending on the research objective, analyses may also include comparative effectiveness studies, cost-effectiveness analyses, budget impact models, and patient-reported outcomes. By understanding these broader patterns, organizations can better demonstrate where treatments may create value and how that value is reflected across the care continuum.
 

Generating evidence that supports market access

For evidence to support access conversations, outcomes need to be defined in ways that are meaningful to payer decision-making. Developing payer-relevant evidence requires more than access to data. HEOR-informed RWE can support market access by demonstrating comparative effectiveness and quantifying economic utility. Recent ISPOR research found that among 10 countries analyzed, 80% incorporate cost-effectiveness analysis  in reimbursement decisions, reinforcing the role of economic evidence in access and utilization discussions. By grounding evidence strategies in payer-relevant measures, organizations can support more informed access and utilization discussions and better demonstrate the benefit of treatments across the healthcare system.
 

Building evidence strategies that support decisions across the product life cycle

Evidence generation is not limited to a single stage of product development. A strong real-world evidence strategy and analytics approach can help life sciences organizations generate insights that support decisions before, during, and after launch. In launch planning, real-world evidence can help identify patient populations, treatment patterns, unmet needs, and potential access considerations. This can include understanding which patient populations may benefit most, where gaps in care exist, and what barriers could affect uptake.

As therapies move into market access discussions, evidence strategies can support payer conversations by connecting clinical performance to economic outcomes, utilization, and comparative effectiveness.

The Institute for Clinical and Economic Review (ICER), an independent nonprofit organization that evaluates the clinical effectiveness and economic value of healthcare interventions, produces evidence reports that many payers use during coverage and formulary decision-making. A recent study indicates that approximately 75% of surveyed payers  used ICER Evidence Reports to inform coverage and formulary decisions, with economic data among the top supporting uses. After launch, ongoing evidence generation can help organizations monitor real-world performance, evaluate outcomes over time, and refine strategies as new data, treatment patterns, and stakeholder needs emerge. Post-launch, this can help organizations understand who is taking up a therapy, where gaps remain, and whether payer, patient, or provider barriers may be affecting utilization. “RWE and HEOR can help answer practical questions across the product life cycle: who may benefit most, where gaps in care exist, how access barriers affect uptake, and what the evidence shows after launch,” says Emma Richard.

These insights can also support value-based care strategies by helping stakeholders connect payment, access, and performance to measurable real-world outcomes.
 

Partner with Carelon Research

Carelon Research combines deep payer expertise, robust real-world data, advanced analytics, and rigorous HEOR methodologies to generate evidence that supports market access, reimbursement, and value demonstration. Our multidisciplinary teams partner with life sciences organizations to design and execute evidence strategies that help answer critical clinical, economic, and policy questions across the product life cycle.

Learn how Carelon Research combines real-world evidence and HEOR analytics to help life sciences organizations demonstrate treatment impact, support payer engagement, and inform decisions across the product life cycle: