ISPOR Europe – a good reason to get out of the office?

As much as you might want a few days away from your office, it can be difficult to get away ­– especially as the pace of work continues to ramp up through to Christmas and the holidays. Well, visit Lumanity at ISPOR Europe in Copenhagen (12–15 November) and your time away from your desk won’t be wasted. Here’s why.

At Lumanity our ongoing research continues to explore how we can best demonstrate the value of innovative technologies to decision makers. Take personalized medicine. Momentum for personalized medicine has led to more therapies being launched in smaller, more targeted indications with implications for the evidence base.

What’s driving value?

Our research explores how we can demonstrate the value of innovative technologies to decision makers in this context. And, the trend of increasingly targeted therapies alongside technological development in data collation and analyses is leading to a rapid increase in using real-world evidence (RWE) to estimate clinical effectiveness.

Take oncology. Within oncology indications a key driver of value is the extent of survival benefit through time – often leading to analysts estimating survival benefit beyond the duration of a clinical trial. At ISPOR Europe our senior analysts (Kearns, Wolff and Kilvert) will present a poster using nivolumab in melanoma to show how extrapolation without external evidence leads to survival estimates that are too low. Their research shows how including external evidence can increase the accuracy of survival extrapolations: ISPOR – Methods for Incorporating External Evidence: A Comparison of Their Impact on Extrapolations and Uncertainty Estimates Using a Melanoma Case-Study.

Advanced methods are enabling us to obtain increasingly accurate estimates of relative survival benefit drawing on methods that attempt to account for unobservable variable bias. Consider for a moment how important RWE is in healthcare decision making today. We’ve worked collaboratively with other agencies and academia to investigate how methods applied to real-world data can be used to reduce selection and confounding bias: ISPOR – Combining Causal Inference and Within-Trial Economic Evaluation Methods to Assess the Cost-Effectiveness of a Mental Health Service Using Real-World Data: The Quasi-Experimental Adapt Study

The patient’s voice counts

When assessing interventions our consultants know how important it is to consider not only clinical outcomes but also what the patient has to say about their health-related quality of life (HRQL). After all, the patient’s opinion and experience is essential – regardless of their age. In a panel discussion about current HTA approaches to incorporating paediatric HRQL into HTA, our Executive Chairman, Ron Akehurst, will debate the issues, and maybe cause a few arguments in the process: ISPOR – Measuring Health Related Quality of Life in Pediatric Life Limiting Progressive Diseases. Whose Perspective Are We Measuring?

We think the paediatric HRQL discussion will be lively and you’ll think your time away from the office has been worth it. But, if you’re still unsure we have more of our work on show at ISPOR for you to consider. First up is an international poster presentation on using clinical outcome assessments in HTA for rare disease indications. Our senior analysts, Hyman, Weijers and Johnson, have reviewed the use of patient engagement and clinical outcomes assessments in HTA reviews conducted by UK, US, and Canadian HTA bodies. They argue that disease specific clinical outcomes assessments have a place in HTA, in addition to the customary use of generic utility health measures: ISPOR – Use of Clinical Outcome Assessments in Health Technology Appraisals for Rare Disease Indications: A Review of UK, US, and Canadian Submissions.

Data has consequences

As analyses trend towards a broader incorporation of data there are consequences for their complexity and the analytic methods we use in health economics and outcomes research (HEOR). So, what is the best approach to applying treatment effect estimates from indirect treatment comparisons (ITCs) within cost-effectiveness (CE) models? Can you apply hazard ratios (HRs) of relative effect to baseline risks estimated using accelerated failure time (AFT) models such as log-logistic and log-normal? (Yes we know, the question that keeps you awake at night!) One of our senior health economists Ollie Hale, teamed up with Professor Nick Latimer of the University of Sheffield and Delta Hat Ltd, to investigate whether the scale of bias varied depending on the AFT model, treatment effect size, or whether censoring was present: ISPOR – The Hazards of Applying Hazard Ratios to Accelerated Failure Time Models: A Simulation Study.

Let’s take you beyond Excel

Integrating a variety of evidence sources to inform better decision making will stretch the capabilities of most health economists’ favoured platform – Excel. But is it a real CE model if it is not built in Microsoft Excel? One of our principle health economists, Rose Hart, is supporting a hands-on introduction to health economics in R which would allow the estimation of treatment benefit, HRQL and the overarching decision model to be programmed in the same platform. Rose has collaborated with academics at Queens University Belfast (Felicity Lamrock), University College London (Gianluca Baio), and University of Bristol (Howard Thom). If you’re interested in finding out more do sign up for the short course session ‘Health Economic Modeling in R: A Hands-on Introduction’ at ISPOR: ISPOR – Health Economic Modeling in R: A Hands-on Introduction. While you’ll need some knowledge of modelling techniques, such as discrete time cohort Markov models and probabilistic sensitivity analysis, we don’t expect you to know R coding. At least, not until you’ve taken this short course.

Stay up to date

At ISPOR Europe you’ll find there is no escaping a common theme running through the conference – ‘HEOR at the Nexus of Policy and Science’ is the conference mantra. Alongside HTA, RWE, patient engagement, you’ll also find plenty of discussion about health policy and digital health technology. We’ve been busy looking at that too. Evidence based health policies are the compass that help us to deliver products and services to safeguard population health. We know that when attempting to estimate the value of new interventions it is essential to not only consider scientific best practice but also the evolving policy context and the analytic preferences of decision makers. Our senior analysts, Guerra Primo, Matthijsse and Westley, studied National Institute for Health and Care Excellence (NICE) 2022 appraisals of single-arm trials, and how they apply external comparator data to ITCs. Their conclusions might surprise you: ISPOR – NICE Technology Appraisals Based on Single-Arm Trials in 2022 – What Can We Learn from Them?

NICE technology appraisals aside and perhaps more notably, we’ve also considered the new German HTA reforms – including the lowering of the orphan revenue cap. It will lead to an increased number of rare conditions being assessed using a framework considered by some to be too inflexible to assess technologies in this context – how should HTA analysts  respond? Our consultants, Matthijsse, Akehurst and Gladwell teamed with the University of Duisburg-Essen, to consider the lessons learned: HTA Reforms in Germany: Implications for Pharmaceutical Pricing and Reimbursement in Germany and the EU

We’re hoping you’ll be at ISPOR to let us know your thoughts. Have we given you enough reasons to tear yourself away from your desk for a few days? Copenhagen is a delightful city by the way. We’re presenting 34 posters, a workshop, a case study, a short course, a breakout session, and an issue panel. We’ll be at Booth C2–005 and hope to see you there.­