Healthcare systems are evolving in how they assess and monitor the value of therapies. With more treatments gaining approval through accelerated regulatory pathways, many enter the market with limited clinical evidence, creating challenges for payers, providers, and patients. To address these gaps, health technology assessment (HTA) agencies are adopting reassessment frameworks that incorporate real-world evidence (RWE) to refine coverage, pricing, and reimbursement decisions over time.
A recent study by Ashley Jaksa, MPH, Patrick J Arena, PhD, and colleagues from Merck & Co., Inc., published in Value in Health, examines how RWE shapes HTA reassessments across six leading HTA agencies. Their findings highlight the increasing reliance on RWE to address clinical uncertainties and the need for structured evidence frameworks to support lifecycle drug evaluations.
Regulatory approval is just the first step in a therapy’s life cycle. While pre-market clinical trials establish efficacy and safety in controlled settings, they do not reflect how treatments perform across diverse, real-world patient populations who often experience complex real-world treatment pathways. Recognizing this gap, HTA agencies are formalizing reassessment frameworks to ensure that therapies continue to provide clinical and economic value after market entry.
Several HTA agencies have already implemented structured reassessment processes:
|
Country (HTA Body) |
HTA Reassessment Approach |
|
United Kingdom (NICE) |
The Cancer Drugs Fund (CDF) and Innovative Medicines Fund (IMF) enable provisional access while additional evidence is collected. |
|
Canada (CADTH)* |
Post-marketing drug evaluations and time-limited reimbursement recommendations guide reassessments. |
|
Germany (IQWiG/G-BA) |
G-BA/IQWiG’s ability to reassess products is limited mainly to orphan drugs as well as drugs with exceptional/conditional approval. |
|
France (HAS) |
Though the landscape is changing, HAS currently has three potential reassessment mechanisms: 1) Post-registration studies, 2) Protocol for temporary use/data collection programs, and 3) Discretionary reassessments. |
|
Netherlands (ZIN) |
Two programs: 1) Conditional Inclusion for orphan drugs and 2) Potential Promising Care for promising but expensive interventions. |
|
Australia (PBAC) |
Managed entry schemes are possible, but almost all are focused on a price discount rather than additional data collection. Post-market reviews (PMRs) also exist, but they are rarely enacted. |
* Now known as CDA-AMC (Canada's Drug Agency - L’Agence des médicaments du Canada). Name change occurred after original submission.
As these approaches highlight, HTA agencies are advancing beyond static approvals, using RWE to guide reimbursement decisions over time.
Although RWE is increasingly used in initial HTA assessments, systematic research on how HTA agencies apply RWE in reassessments remains limited.
To understand RWE’s role in reassessments, we analyzed 40 health technology reassessments (HTARs) across six leading HTA agencies. The analysis identified key trends:
|
Key Finding |
Details |
|
RWE is increasingly used in reassessments |
Over half of the reviewed reassessments incorporated RWE, reinforcing its expanding role in post-market evaluations. However, due to the study design, selection bias likely impacted this finding. |
|
Orphan and oncology therapies are a focal point |
RWE use was particularly high for orphan and oncology therapies. However, due to NICE’s Cancer Drugs Fund, oncology treatments were likely overrepresented. Nevertheless, RWE use was also observed for other therapeutic areas (e.g., cardiovascular disease and autoimmune disease). |
|
RWE is addressing clinical uncertainties |
Historically, RWE has primarily been used in cost-effectiveness models submitted during the initial assessments. In reassessments, however, it often played a direct role in clarifying clinical effectiveness and safety. |
|
Comparative effectiveness studies remain rare |
No de novo RWE comparative effectiveness studies were identified. Short reassessment timelines likely limit RWD accumulation and the ability to implement complex studies, like comparative effectiveness analyses. |
|
RWE is complementing, not replacing, RCTs |
RWE was used alongside clinical trial data as part of a broader evidence package to contextualize trial results. |
|
Sponsors are requesting reassessments |
The sponsor requested reassessments in 20.0% of the HTARs evaluated. Sponsors are requesting reassessments to expand the indication, remove prescribing restrictions, and/or update information that would impact price negotiations. |
As RWE plays a greater role in HTA reassessments, addressing key challenges will ensure it delivers a meaningful impact. Early engagement with HTA agencies, improvements in data quality, and clearer policy frameworks are critical for integrating RWE into decision-making.
Key strategies for improving collaboration include:
Establishing robust RWD infrastructure and transparent reporting practices will enhance the reliability of RWE in reimbursement decisions.
Real-world evidence is becoming central to how health systems monitor the value of therapies after they reach the market. By building RWE into reassessment frameworks, HTA agencies are moving toward a more continuous, dynamic approach to evaluating clinical benefit and economic value.
Early alignment between stakeholders is essential to making this shift effective. When sponsors and agencies coordinate on data expectations and methods, meaningful evidence that answers the right questions can be generated when it matters most.
With the right data infrastructure and scientific rigor, RWE can play a decisive role in shaping pricing, reimbursement, and coverage decisions. As this approach takes hold, patients stand to gain more timely access to therapies that are proven to deliver real-world value.
Get in touch with Aetion® to explore how real-world evidence can enhance your evidence generation strategy.