A 10-Part Series on ISPOR’s Top HEOR Trends: #1 Real-world evidence

The role of Health Economics and Outcomes Research (HEOR) is expanding, driven by policy shifts, digital health, and rising demand for value-based care.

Inspired by ISPOR’s Top 10 HEOR Trends for 2024/25, we would like to launch a series to explore each trend in depth. We'll share practical insights and examples of how these themes are shaping the future of market access, pricing, and evidence generation.

First up: Real-World Evidence (RWE) in Regulatory and HTA Decision-Making

RWE continues to top ISPOR’s list for the third consecutive report as it becomes increasingly important in healthcare decision-making. 

RWE offers what randomised controlled trials (RCTs) often can’t:

• Insights on real patients in real-world settings

• Inclusion of more diverse populations than those typically in RCTs

• Much faster evidence generation timelines

RWE is great at answering important questions about a product’s safety and effectiveness which speaks to what payers, regulators, and doctors really care about.

What the experts are saying

Regulators and HTA bodies, including the FDA, EMA, NICE, and CADTH, have all recently released detailed guidance focused on:

• Data quality and ensuring data are “fit for purpose”

• Use of registries and external control arms

• Reliable causal inference methods

• Integrating RWE as part of the totality of evidence to support new products or indications

• Increasing transparency in study design and reporting

  
  

Real-world impact

Examples of real-world impact of RWE include:

• FDA approved Ibrance (palbociclib) for male breast cancer based on RWE (https://cancerletter.com/the-cancer-letter/20190419_2/

• EMA’s approval of Abecma (ide-cel) CAR-T therapy for multiple myeloma, where real-world data formed a matched external control arm to support efficacy in a single-arm trial. (https://www.ema.europa.eu/en/documents/presentation/presentation-rwd-derived-external-controls-case-study-abecma-abuzzi-tframke-ema_en.pdf)

• NICE considered real-world data, such as from the Systemic Anti-Cancer Therapy (SACT) dataset, to inform the reimbursement of Osimertinib. (https://www.nice.org.uk/guidance/ta1043/chapter/3-Committee-discussion)

  
  

At Evimed, we’re helping clients use RWE to:

• Fill evidence gaps from observational studies

• Demonstrate long‑term value

• Support complex pricing strategies, including for rare and oncology therapies

  
  

ISPOR HEOR trend_RWE.pdf

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