Looking back a few weeks to ISPOR Europe, Will Battershill and Hollie Wheat, Analysts from Delta Hat – Powered by Petauri™, share their key takeaways from the conference, relating to health technology assessment (HTA) decision-making. Will begins with insights on the hot topic of incorporating patient and carer perspectives, whilst Hollie dives into the use of surrogate endpoint evaluation.
Incorporating societal impacts into HTA decision-making
Will Battershill explores a key them from ISPOR Europe: the benefits of incorporating societal impacts into HTA decision-making.
Expanding HTA perspectives beyond the payer
ISPOR Europe featured some thought-provoking discussions on expanding the perspective taken in HTA beyond that of the payer, which typically focuses on costs to the health system and health-related benefits to patients. Speaker panels at the conference explored how incorporating societal impacts, such as bereavement or productivity, could provide a more holistic picture of the disease burden on individuals and their families. These broader perspectives pose practical and conceptual challenges, however, which could complicate decision-making.
Bereavement disutility debate
One hot topic was whether to include bereavement disutility in HTA models. This refers to the negative impact on the health-related quality of life of those close to a deceased patient. The idea is to address the “carer quality-adjusted life year (QALY) trap”, where extending life for a patient with informal carers is considered less valuable than for a patient without carers. Including bereavement disutility introduces a large negative effect after death, which, due to discounting, could shift cost-effectiveness outcomes.
Challenges in incorporating bereavement effects
Becky Pennington (Senior Research Fellow, University of Sheffield) presented evidence from UK household surveys. She showed that both carers and non-carers experience substantial disutility from bereavement, lasting 1–2 years. Dr Tara Lavelle (Assistant Professor of Medicine, Tufts University School of Medicine) presented research demonstrating increased carer productivity after bereavement over time.
Incorporating bereavement effects is not straightforward, however Dr Saskia Knies (Erasmus School of Health Policy & Management), representing the Dutch HTA body, highlighted challenges, such as determining the appropriate time horizon for models. She also pointed out the need to adjust willingness-to-pay thresholds to match a broader perspective. Pennington’s findings suggest that bereavement effects are similar for carers and non-carers. This raises the question of whether these effects should apply universally in survival-related HTAs, potentially diluting their impact on cost-effectiveness outcomes.
Risk of unintended consequences: Productivity gains versus survival
Lavelle’s work also raised an important issue: if post-bereavement productivity gains outweigh caregiving losses, could this unintentionally favour death over survival in societal cost-effectiveness analyses?
Incorporating broader support structures in HTA: A patient perspective
In an educational symposium on adopting a societal perspective for HTA in breast cancer, a patient representative stressed the importance of recognising broader support structures in patients’ lives. The discussions highlighted the complexity of integrating these factors into HTA frameworks, as there is no clear method to do so alongside clinical outcomes.
Complexities of expanding HTA perspectives
Expanding HTA perspectives beyond payer-centric views has prompted insightful research and facilitates a more comprehensive understanding of disease impact. However, these approaches also revealed marked methodological and ethical complexities. I felt that, without clearer frameworks for implementation, broadening HTA perspective might inadvertently introduce ambiguity rather than actionable insights.
Mtech Access presented a poster that looked at this issue as it relates to orphan disease: The Use of Patient and Carer Perspectives and Real-World Evidence in Orphan Disease Health Technology Assessment Submissions.
Surrogate endpoint evaluation in HTA decision-making
Hollie Wheat shares her insights from the session on ‘borrowing information from diverse sources to enhance the evidence base for surrogate endpoint evaluation in HTA decision-making’.
Surrogate endpoints: Filling evidence gaps in HTA
One of the most thought-provoking and applicable sessions was on the use of surrogate endpoint evaluation in HTA decision-making, as led by Sylwia Bujkiewicz (Professor of Biostatistics, University of Leicester), with discussants Anastasios Papanikos (Principal Statistician, GSK), Janharpreet Singh (Professor of Biostatistics, University of Leicester), and Georgios Nikolaidis (Associate Director, IQVIA). Surrogate endpoint analyses help fill evidence gaps in HTA, often relevant where there is uncertainty in short-term data, for example in the context of small clinical trial data set and/or limited numbers of events observed within the period of trial follow-up. The use of surrogate endpoint analysis in HTA is ever increasing – 18 of 47 National Institute for Health and Care Excellence (NICE) technology appraisals between January 2022 and May 2023 included some type of surrogacy analysis.
That being said, existing surrogacy analysis methodology assumes that the relationship between endpoints remains the same for each arm. That is, one surrogate relationship may be applied to more than one treatment group.
Innovations in surrogate endpoint methodology
As surrogacy methodology evolves, however, new models are constantly being developed. These include Bayesian hierarchical models with treatment class as a covariate, mixture models that give more weight to studies with similar information to the study with missing data, and approaches that borrow from real-world evidence to inform surrogate relationships.
Key insights: Reducing uncertainty in surrogate endpoint predictions
One surprising insight from the session was that, with mixture models, sharing information at the progression-free survival level before predicting overall survival reduces uncertainty around the prediction.
Applying surrogate endpoint analysis to my work
I am currently working on a project involving the use of a surrogacy analysis. Since it’s my first experience with these methods, the session helped me gain a deeper understanding of how surrogate endpoints can aid HTA.
Highlights from the Petauri team! From engaging at our stand to presenting posters and sharing laughs over food and drinks – this video captures what made ISPOR Europe special!
Calum Jones (Associate Director – Health Economics, Mtech Access) spoke at a Health Technology Assessment International (HTAi) panel discussion earlier this year (a summary of the key points can be found here), and the Mtech Access team also presented a webinar on overcoming reimbursement and validation challenges using surrogate endpoints.
As the field of HTA continues to evolve, staying informed about the latest methodologies and insights is crucial. With Delta Hat, Mtech Access, and Petauri working together, the opportunities to tap into a broader pool of expertise and collaboration are now greater than ever.
If you’re interested in learning more about surrogate endpoint analysis or other emerging topics in HTA, reach out to us via contact@deltahat.com/info@mtechaccess.co.uk. We’re eager to explore how these developments can impact your work and support your efforts in navigating the challenges of evidence-based decision-making.
