Brief · Use case · Market Access

Real-World Evidence Payer Assessment.

LiveAssessment

Judge whether real-world evidence can carry a managed entry agreement for a high-cost therapy, how strong each support is and what would overturn the view.

PositionReal-world evidence alone is sufficient

SettingA managed entry agreement for a high-cost therapy

ExpertHEOR Evidence Lead

Not askedA recommended action

LengthComprehensive

Real-World Evidence Payer Assessment is a ready-made use case for Market Access. It ships under the Assessment template.

For market access and HEOR leads preparing a payer discussion. You get a position with its confidence, each supporting finding graded for certainty with the reason, the strongest payer objection answered and the weaknesses ranked. Claims are cited or marked as gaps.

Add it to your library to reuse from New → From template. Edit the saved copy in your library on the service form.

The decision

How well supported is the position that real-world evidence alone is sufficient to support a managed entry agreement for a high-cost specialty therapy with a single pivotal trial?

What the assessment examines

The position makes two claims: that real-world evidence can carry the evidentiary load, and that it is enough for an agreement. The assessment examines each.

Part of the position	What the assessment examines	Where it looks
What real-world evidence can do	The NICE framework identifies when real-world data can reduce uncertainty, for example long-term outcomes, while treating randomized trials as the reference standard for causal effects and asking developers to justify non-randomized evidence.^{1National Institute for Health and Care Excellence. NICE real-world evidence framework (ECD9). Published 23 June 2022.}	The NICE real-world evidence framework, published 23 June 2022.
How the study is built	Whether the real-world study is specified so that its risk of bias can be judged and its analysis repeated.^{2Wang, S. V. et al. HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: a good practices report of a joint ISPE/ISPOR task force. Value in Health, 2022;25(10):1663–1672.}	The HARPER protocol template from ISPE and ISPOR.
What an agreement needs	The costs of collecting evidence, negotiating, monitoring and evaluating an agreement, and the need to match the study design to the uncertainty being addressed.^{3Garrison, L. P. Jr, Towse, A., Briggs, A. et al. Performance-based risk-sharing arrangements: good practices for design, implementation, and evaluation. Value in Health, 2013;16(5):703–719.}	The ISPOR good practices for performance-based risk-sharing arrangements.
Where agreements are used	How performance-based agreements work in OECD and EU countries and what improvements are proposed.^{4Wenzl, M. and Chapman, S. Performance-based managed entry agreements for new medicines in OECD countries and EU member states. OECD Health Working Paper No. 115, 2019.}	The OECD review of performance-based managed entry agreements, 2019.

What the assessment does

What the evidence is for. The position says real-world evidence is sufficient, so the assessment asks what uncertainty the agreement is meant to resolve and whether real-world data can resolve it. NICE’s framework treats real-world data as able to reduce specific uncertainties and does not set a minimum quality bar.^{1National Institute for Health and Care Excellence. NICE real-world evidence framework (ECD9). Published 23 June 2022.}

The objection from confounding. Without randomization, differences between patients can look like treatment effects. The assessment restates this objection fairly and answers it from the study design, naming the methods that address it and what they leave open.^{1National Institute for Health and Care Excellence. NICE real-world evidence framework (ECD9). Published 23 June 2022.,2Wang, S. V. et al. HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: a good practices report of a joint ISPE/ISPOR task force. Value in Health, 2022;25(10):1663–1672.}

An agreement has costs. The ISPOR task force found that the costs of evidence, negotiation, monitoring and evaluation can make an agreement hard to justify, and that good governance matters. The assessment weighs the position against those points.^{3Garrison, L. P. Jr, Towse, A., Briggs, A. et al. Performance-based risk-sharing arrangements: good practices for design, implementation, and evaluation. Value in Health, 2013;16(5):703–719.}

Where it is weak. The weakest point is usually the single pivotal trial: real-world evidence cannot replace missing randomized evidence on the primary effect. The assessment says which uncertainty the real-world data would address and which they would not.

Recommended length

Comprehensive

The position will be challenged by people who hold another view. Comprehensive adds the alternatives considered and the reasoning behind the support, which is what a challenger asks for.

You can pick another length on the form. The other two stay available.

Assumptions the assessment checks

The assessment states the assumptions the position rests on and says which one would hurt most if it failed. Examples for this position:

The uncertainty the agreement addresses is one that real-world data can resolve.
The real-world source has the outcomes and confounders the analysis needs.
Both parties accept the study design before data are collected.

Standards, guidance and sources

Real-world evidence

NICE real-world evidence framework (ECD9), 23 June 2022.^{1National Institute for Health and Care Excellence. NICE real-world evidence framework (ECD9). Published 23 June 2022.}
HARPER, the harmonized protocol template for real-world evidence studies of treatment effects (ISPE and ISPOR).^{2Wang, S. V. et al. HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: a good practices report of a joint ISPE/ISPOR task force. Value in Health, 2022;25(10):1663–1672.}

Managed entry agreements

ISPOR good practices for performance-based risk-sharing arrangements.^{3Garrison, L. P. Jr, Towse, A., Briggs, A. et al. Performance-based risk-sharing arrangements: good practices for design, implementation, and evaluation. Value in Health, 2013;16(5):703–719.}
OECD, performance-based managed entry agreements for new medicines in OECD countries and EU member states, 2019.^{4Wenzl, M. and Chapman, S. Performance-based managed entry agreements for new medicines in OECD countries and EU member states. OECD Health Working Paper No. 115, 2019.}

The decision practice behind the memo itself is on the template page.

What gets flagged

Any claim that real-world data can establish a causal effect without a stated method, any uncertainty the data cannot address, and any agreement cost the position leaves out.

What arrives

The position
The basis
Why the evidence supports it
The strongest objection, answered
Other views considered
Where it is weak

What it will not do

It does not recommend an action. A Decision Memo takes the assessment as its starting point.

It does not predict whether a payer will agree.

It does not recommend an action. An assessment says how well a position is supported; a Decision Memo recommends what to do.

Certainty ratings are analyst judgment from an AI specialist, not measurements, and the reason for each is on the page so you can disagree with it.

It does not settle a question the evidence leaves open. Where the evidence is thin, the assessment says so and says what would settle it.

References

[1] National Institute for Health and Care Excellence. NICE real-world evidence framework (ECD9). Published 23 June 2022. Known from NICE’s page listing and search excerpts; the framework was not read in full.
[2] Wang, S. V. et al. HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: a good practices report of a joint ISPE/ISPOR task force. Value in Health, 2022;25(10):1663–1672. Summarized from ISPOR’s page and search excerpts; the full text is behind a paywall.
[3] Garrison, L. P. Jr, Towse, A., Briggs, A. et al. Performance-based risk-sharing arrangements: good practices for design, implementation, and evaluation. Value in Health, 2013;16(5):703–719. Read from the abstract on ISPOR’s page.
[4] Wenzl, M. and Chapman, S. Performance-based managed entry agreements for new medicines in OECD countries and EU member states. OECD Health Working Paper No. 115, 2019. Known from the OECD publication page and search excerpts; not read in full.

Links checked 9 October 2026

These are the sources the template drew on. None of their authors or issuers has reviewed it.

About the Brief service

A Brief is a written answer from one specialist, at the depth you asked for. It arrives with its sources, and it closes by saying plainly what would change the conclusion. Nothing is hidden behind a summary, and anything the expert could not source is marked as a gap rather than filled in.

To edit a saved template, open New → Brief → From template, or the library rail on the service form.