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Cytel at ISPOR Europe: Two Workshops

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Cytel will be represented at over 60 presentations at ISPOR Europe 2022, with more issue panels and workshops than any other participating organization. For more information on these upcoming workshops, see below. To download the full Cytel program, click here:


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Using Target Trial Emulation for Estimation of Treatment Effects Using Real-World Data

Cytel presenter: Grace Hsu


Health technology assessment (HTA) bodies are increasingly considering real-world data (RWD) for informing decision-making. The target trial emulation (TTE) framework has emerged as a promising approach to estimate treatment effects while minimizing common biases and methodological pitfalls in non-randomized studies. By applying design principles from randomized controlled trials, TTE explicitly ties the design and analysis of the non-randomized study to the target trial, facilitating the interpretation and communication of its underlying assumptions and study findings. In addition, TTE provides a structured approach for identifying potential limitations of RWD and the extent to which these affect the HTA decision.

The purpose of this workshop is to illustrate how the use of TTE can estimate per-protocol treatment effects when evidence comes primarily from a single-arm trial and an external (real-world) control arm. We will use three advanced non-small-cell lung cancer (aNSCLC) trials for illustrating the advantages of the TTE framework as well as major challenges.


Participants will be introduced to a broad overview of the target trial framework and the adoption of TTE for HTA. The workshop then will review design considerations and an application in a subset of aNSCLC trials.

Grace Hsu will chair the session. Manuel Gomes will provide a general introduction to TTE. Sreeram Ramagopalan will illustrate how these methods have been applied to aNSCLC. Stephen Duffield will outline the potential value of TTE from a HTA perspective.

Audience participation will include engaging in a poll-driven question-and-answer period to identify potential issues and solutions for TTE scenarios. This workshop would be valuable to researchers, industry analysts, and HTA representatives who are interested in trial emulation and analysis of RWD.



Surrogacy Analysis in HTA Clinical and Cost Effectiveness Analyses; How to Statistically Validate Your Models

Cytel presenter: Bart Heeg



HTA bodies are expected to make decisions on the clinical efficacy and/or cost effectiveness, by examining the potential value in improving final patient relevant outcomes. For conditions with high disease burden and unmet treatment needs, data collection on final outcomes may delay urgent patient access to new treatments. In these circumstances, surrogate outcomes may substitute for and predict a relevant final patient outcome. However, their use can result in decision uncertainty and inaccurate technologies’ assessment.

Several frameworks have been proposed to increase transparency in the use of surrogate outcomes. However, most of these frameworks have not been extended to cover the impact of surrogacy use to the modelling assumptions in economic evaluations supporting HTA submissions.


The workshop will start with an introduction to the concept of surrogacy analysis and the importance to consider the totality of clinical and statistical evidence on the surrogacy relationship with final outcomes (Marc Buyse). Then, a newly developed web-based decision tool to support HTA agencies and payers when faced with surrogate endpoint evidence and address areas of uncertainties will be presented (Oriana Ciani). The workshop will continue with presenting the main statistical methodologies (risk equations, multi-state models, and partition survival models) to accommodate for the use of surrogate outcomes in the economic modelling, the pros and challenges for each method and their implementation in HTAs (Bart Heeg). Finally, Mario Ouwens will provide a summary of discussants’ recommendations on when and how surrogate outcomes can be reliably supporting HTA submissions. The session will conclude with an interactive discussion including the use of real time polling questions by presenting three hypothetical cases of HTA submissions using surrogate outcomes and potential impact on final decision making. This workshop will benefit statisticians, payers, and industry intending to use surrogate methodologies in submissions.

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ISPOR Europe, the leading global conference for health economics and outcomes research (HEOR) and real-world evidence (RWE), will welcome thought leaders from industry, academia, healthcare, and government for panel discussions, workshops, poster sessions, and more.

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