Highlights from ISPOR Europe 2019

ACTUA - 05 november 2019

Sebastian Vermeersch and Marijn Witterzeel are representing Hict at ISPOR Europe 2019. These are their impressions and experiences.

Day 1

Perhaps unsurprisingly, day 1 was mostly about value, in particular in the context of (high cost, curative) cell & gene therapies and (high cost, long-term effect) immuno-oncology products.

Kicking things off during the cell and gene therapies - value and access breakfast session, an excellent presentation by Prof. Drummond outlined the specific challenges faced by curative cell and gene therapies in the context of price and reimbursement and proposed a number of analytic techniques to deal with these challenges. Also of note was a more industry-oriented perspective provided by dr. Arjunji (Senior Director, AveXis), separtating the issues of pricing (reflecting value, impacting budget) and uncertainty (mostly related to long term outcomes), culminating in an overview of some proposed funding models addressing these issues. Slides from this session weren’t made available, unfortunately, but let me know if you want more detail.

Next up was a very interesting debate on alternative value drivers titled fake of novel elements of value. Excellent overview by Prof. Garrison on value elements other than QALY, mostly from the ISPOR taskforce and a highly interesting and practical (formulas warning!) overview of insurance value/value of hope in particular by dr. Jansen. I also really appreciated the sceptical viewpoint presented by Prof. Briggs - I encourage everyone to have a look at his Mickey Mouse “value flower”, retaining only the QALY, cost, equity and severity value elements and reflecting his take on their perceived importance!

The final session I’d like to highlight focused on the hot topic of indication based pricing: Is indication-based pricing feasible and/or beneficial for our society. Great presentation of pros (Prof. Towse) and cons (Prof. Briggs) and a very interesting Belgian take by Francis Arickx (no slides)

Day 2

Day two at ISPOR provided a little more texture to our value-centric day-1 experience.

First off, a topic perhaps not sufficiently considered in the wider context of health economics. Where most analyses heavily focus on balancing the value that investing in a new drug or technology brings, the (rather back-breakingly titled) “Estimating the opportunity cost based cost effectiveness threshold - empirical approaches and the policy challenges for payers” session focused on the (health) outcomes that may be foregone by this investment. It turns out even seemingly straightforward analyses, like looking at the (historic) relationship between health care expenditure and health outcomes delivers surprising results. As Dr. Lomas explains, this link between health care expenditure and health outcomes is heavily influenced by unobserved variables, which hide the impact of the variable inputs we are truly interested in observing. His proposed approach to dealing with these issues requires sufficient data availability. Data availability that may be very different across jurisdictions, making a more generalizable approach difficult.

Taking this concept even further, the presentation by Dr. Henriksson outlines the potential impact setting a cost-opportunity threshold may have on reimbursement decisions based on evidence from the Swedish setting. The policy implications and challenges associated with integrating an opportunity cost approach in practice were further outlined in a very insightful presentation by Danny Palnoch, head of medicines analysis for NHS England.

Perhaps of more direct practical relevance to current reimbursement hot topics, was the educational symposium on curative therapies and risk sharing. In a number of really interesting practical case study presentation, presenters outlined how payers in France, UK, Italy and Germany deal the with the onset of curative (gene) therapy. I’ve been trying to get my hands on the slides (they have not been posted on the conference website) - UPDATE: slides now available.

Finally, for those of you interested in more in-depth statistical knowledge, the advanced workshop on the application of existing and novel network meta-analysis methodologies was a real doozy. And that is not even taking into consideration the most enthusiastic presentation by a statistician I ever personally was privileged to witness. More to point, the different presentations very elegantly explained the shortcomings of traditional hazard-ratio NMAs before taking a deep-dive into more novel approaches like parametric NMA, (mixture and non-mixture) cure models, spline NMA models and NMA models based on fractional polynomials. It even included a handy flowchart. No slides to share again, unfortunately. Though this topic is of sufficient personal interest that I may go dig up the underlying research papers to compile a reconstructed overview.

To close, a special shout-out to Stefaan Vansieleghem (for setting up and facilitating) and all Belgian HEOR colleagues (for being great) present at the Belgian meet-up yesterday. It was really great to chat and dine. I really hope this event sets a standard for future ISPOR events!

Day 3

Our final ISPOR day was short and out-of-the-box.

Shifting from value drivers to novel approaches to financing value and innovation, the morning session "Subscription-based model for drug reimbursement - a fad or the future" provided interesting insights, in particular on the payer's perspective (NHS and Australia). Key take-home: payers are interested in novel approaches, but a strong case with clear added value needs to be presented to them to accept these types of approaches over more conventional risk or budget sharing solutions.

Further out-of-the-box thinking in the "Which is the way forward for health technology assessment of interventions with external benefits? Extended cost-effectiveness analysis vs cost-benefit analysis." session. I was deeply impressed by dr. Sevilla's presentation, which probably contains the best explanation on cost-benefit analyses with welfare functions I've come across to date.

Closing the week's conference program, a brief excursion into a different box altogether, looking at "cost utility analysis or cost-benefit analysis for the economic evaluation of nutrition interventions". Not something I get confronted with on a regular basis. If you're interested, I encourage you to have a look at dr. Freyers presentation on "nutrition economics", providing a good entry point with some telling examples.

Really, ISPOR 2019 has been a great week to catch up on new trends and get new insights in old trends (sometimes in new jackets). Before we sign off on our ISPOR week, there are two more out of-the box points I'd like to draw your attention to. First, on the footsteps of a truly excellent presentation on "diff in diff" methodology, I would invite all of you to have a look at the methodology papers outlined on Dr. Hatfields webpage. These types of methodologic considerations are (and will increasingly be) of huge importance as real-world evidence takes front and center in the health economics field. And finally, a throwback to the very first session we attended on ISPOR, on value and access for cell and gene therapies. In reference to the potential impact mergers and acquisitions may have on drug prices (particularly relevant for many new biotech), Prof. Drummond suggested off the cuff that perhaps a soccer-related model should be considered, where a promising player is often sold under the condition that if the player is re-sold, a cashback to the original club is guaranteed. Out-of-the box innovation - can't get enough of it!

We're not at ISPOR anymore, but should you want to reach out to discuss these, or any other health economics topics of interest, please do so. The full program (and slides) of ISPOR Europe 2019 are available on the ISPOR website.