Meeting round-up: ISPOR Europe 2018 (part 2)

Have you missed ISPOR Europe 2018 but are eager to know all about it? Time to continue reading! In yesterday’s post, I wrote about ISPOR’s outstanding short-course on causal inference and the superb sessions I had attended on day 1. This blog post is about day 2, Tuesday 13th, which was another big day.

The second plenary session was on fairness in pharmaceutical pricing. It was moderated by Sarah Garner, with presentations by many key stakeholders. The thought-provoking discussion highlighted the importance of pharmaceutical pricing policy and the large role that HTA can have in shaping it.

Communicating cost-effectiveness analysis was the next session, where myself, together with Rob Hettle, Gabriel Rogers and Mike Drummond, discussed the pitfalls and approaches to explaining cost-effectiveness models to non-health economists. This was a hugely popular session! We were delighted by the incredibly positive feedback we received, which reassured us that we are clearly not alone in finding it difficult to communicate cost-effectiveness analysis to a lay audience. We certainly feel incentivised to continue working on this topic. The slides are available here, and for the audience’s feedback, search on twitter #communicateCEA.

The lunch was followed by the open meeting of ISPOR Women in HEOR Initiative with Shelby Reed, Olivia Wu and Louise Timlin. It is really encouraging to see ISPOR taking a proactive stance to gender balance!

The most popular session in the afternoon was Valuing a cure: Are new approaches needed, with Steve Pearson, Jens Grueger, Sarah Garner and Mark Sculpher. The panel showed the various perspectives on the pricing of curative therapies. Payers call for a sustainable pricing model, whilst pharma warns that pricing policy is necessarily linked to the incentives for investment in research. I agree with Mark in that these challenges are not unique to curative therapies. As pharmaceutical therapies have greater health benefits but at large costs, it is pressing that cost-effectiveness assessments are also able to consider the opportunity cost of funding more costly treatments. See here for a roundup of the estimates already available.

I then attended the excellent session on Drug disinvestment: is it needed and how could it work, moderated by Richard Macaulay. Andrew Walker explained that HTA agencies’ advice does not always go down well with local payers, highlighting this with an amusing imaginary dialogue between NICE and a hospital. Detlev Parow argued that payers find that prices are often unaffordable, hence payment schemes should consider other options, such as treatment success, risk-sharing agreements and payment by instalments. Bettina Ryll made an impressive case from the patients’ perspective, for whom these decisions have a real impact.

The conference continued late into the evening and, I suspect, long into the early hours of Wednesday, with the ever-popular conference dinner. Wednesday was another day full of fascinating sessions. The plenary was titled Budget Impact and Expenditure Caps: Potential or Pitfall, moderated by Guillem López-Casasnovas. It was followed by inspiring sessions that explored a wide range of topics, presented by the top experts in the relevant fields. These really delved into the nitty-gritty on subjects, such as using R to build decision models, the value of diagnostic information, and expert elicitation, just to name a few.

I don’t think I’m just speaking personally when I say that ISPOR Barcelona was an absolutely brilliant conference! I’ve mentioned here a few of the most outstanding sessions, but there were many, many more. There were so many sessions at the same time that it was physically impossible to attend all of those with a direct relevance to my research. But fortunately, we can access all the presentations by downloading them from the ISPOR website. I’ll leave the suggestion to ISPOR here, that they should think about filming some of the key sessions and broadcasting them as webinars after the conference. This could create a further key resource for our sector.

As in previous editions, ISPOR Barcelona truly confirms ISPOR Europe in the top HTA conferences in Europe, if not the world. It expertly combines cutting-edge methodological research with outstanding applied work, all with the view to better inform decision making. As I’m sure you can guess, I’m already looking forward to the next ISPOR Europe in Copenhagen on the 2nd-6th November 2019, and the amazing sessions which will indubitably be featured!


Meeting round-up: ISPOR Europe 2018 (part 1)

ISPOR Europe 2018, which took place in Barcelona on the 10th-14th November, was an exceptional conference. It had a jam-packed programme on the latest developments and most pressing challenges in health technology assessment (HTA), economic evaluation and outcomes research. In two blog posts, I’ll tell you about the outstanding sessions and thought-provoking discussions in this always superb conference.

For me, proceedings started on Sunday, with the excellent short-course Adjusting for Time-Dependent Confounding and Treatment Switching Bias in Observational Studies and Clinical Trials: Purpose, Methods, Good Practices and Acceptance in HTA, by Uwe Siebert, Felicitas Kühne and Nick Latimer. Felicitas Kühne explained that causal inference methods aim to estimate the effect of a treatment, risk factor etc. on our outcome of interest, controlling for other exposures that may affect it and hence bias our estimate. Uwe Siebert and Nick Latimer provided a really useful overview of the methods to overcome this challenge in observational studies and RCTs with treatment switching. This was an absolutely brilliant course. Highly recommended to any health economist!

ISPOR conferences usually start early and finish late with loads of exceptional sessions. On Monday, I started the conference proper with the plenary Joint Assessment of Relative Effectiveness: “Trick or Treat” for Decision Makers in EU Member States, moderated by Finn Børlum Kristensen. There were presentations from representatives of payers, HTA agencies, EUnetHTA, pharmaceutical industry and patients. The prevailing mood seemed to be of cautious anticipation. Avoiding duplication of efforts in the clinical assessment was greatly welcomed, but there were some concerns voiced about the practicalities of implementation. The proposal was due to be discussed soon by the European Commission, so undoubtedly we can look forward to knowing more in the near future.


My next session was the fascinating panel on the perils and opportunities of advanced computing techniques with the tongue-in-cheek title Will machines soon make health economists obsolete?, by David Thompson, Bill Marder, Gerry Oster and Mike Drummond. Don’t panic yet as, despite the promises of artificial intelligence, I’d wager that our jobs are quite safe. For example, Gerry Oster predicted that demand for health economic models is actually likely to increase, as computers make our models quicker and cheaper to build. Mike Drummond finished with the sensible suggestion to simply keep calm and carry on modelling, as computing advances will liberate our time to explore other areas, such as the interface with decision-makers. This session left us all in a very positive mood as we headed for a well-earned lunch!

There were many interesting sessions in the afternoon. I chose to pop over to the ISPOR Medical Device and Diagnostic Special Interest Group Open Meeting, the ISPOR Portugal chapter meeting, along with taking in the podium presentations on conceptual papers. Many of the presentations will be made available in the ISPOR database, which I recommend exploring. I had a wonderful experience moderating the engaging podium session on cancer models, with outstanding presentations delivered by Hedwig Blommestein, Ash Bullement, and Isle van Oostrum.

The workshop Adjusting for post-randomisation confounding and switching in phase 3 and pragmatic trials to get the estimands right: needs, methods, sub-optimal use, and acceptance in HTA by Uwe Siebert, Felicitas Kühne, Nick Latimer and Amanda Adler is one worth highlighting. The panellists showed that some HTAs do not include any adjustments for treatment switching, whilst adjustments can sometimes be incorrectly applied. It reinforced the idea that we need to learn more about these methods, to be able to apply them in practice and critically appraise them.

The afternoon finished with the second session of the day on posters. Alessandro Grosso, Laura Bojke and I had a poster on the impact of structural uncertainty in the expected value of perfect information. Alessandro did an amazing job encapsulating the poster and presenting it live to camera, which you can watch here.


In tomorrow’s blog post, I’ll tell you about day 2 of ISPOR Europe 2018 in Barcelona. Tuesday was another big day, with loads of outstanding sessions on the key topics in HTA. It featured my very own workshop, with Rob Hettle, Gabriel Rogers and Mike Drummond on communicating cost-effectiveness analysis. I hope you will stay tuned for the ISPOR meeting round-up part 2!

Rita Faria’s journal round-up for 22nd October 2018

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

Economically efficient hepatitis C virus treatment prioritization improves health outcomes. Medical Decision Making [PubMed] Published 22th August 2018

Hepatitis C treatment was in the news a couple of years ago when the new direct-acting antivirals first appeared on the scene. These drugs are very effective but also incredibly expensive. This prompted a flurry of cost-effectiveness analyses and discussions of the role of affordability in cost-effectiveness (my views here).

This compelling study by Lauren Cipriano and colleagues joins the debate by comparing various strategies to prioritise patients for treatment when the budget is not enough to meet patient demand. This is a clear example of the health losses due to the opportunity cost.

The authors compare the costs and health outcomes of various prioritisation schedules in terms of the number of patients treated, the distribution by severity and age, time to treatment, impact on end-stage liver disease, QALYs, costs and net benefit.

The differences between prioritisation schedules in terms of these various outcomes were remarkable. Reassuringly, the optimal prioritisation schedule on the basis of net benefit (the “optimisation” schedule) was the one that achieved the most QALYs and the greatest net benefit. This was even though the cost-effectiveness threshold did not reflect the opportunity cost, as it was set at $100,000 per QALY gained.

This study is fascinating. It shows how the optimal policy depends on what we are trying to maximise. The “first come first serve” schedule treats the most patients, but it is the “optimisation” schedule that achieves the most health benefits net of the opportunity cost.

Since their purpose was not to compare treatments, the authors used a representative price depending on whether patients had progressed to cirrhosis. A future study could include a comparison between drugs, as our previous work found that there are clear differences in cost-effectiveness between treatment strategies. The more cost-effective the treatment strategies, the more patients can be treated with a given budget.

The authors made the Excel model available as supporting material, together with documentation. This is excellent practice! It disseminates the work and shows openness to independent validation. Well done!

Long-term survival and value of chimeric antigen receptor T-cell therapy for pediatric patients with relapsed or refractory leukemia. JAMA Pediatrics [PubMed] Published 8th October 2018

This fascinating study looks at the cost-effectiveness of tisagenlecleucel in the treatment of children with relapsed or refractory leukaemia compared to chemotherapy.

Tisagenlecleucel is the first chimeric antigen receptor T-cell (CAR-T) therapy. CAR-T therapy is the new kid on the block in cancer treatment. It involves modifying the patient’s own immune system cells to recognise and kill the patient’s cancer (see here for details). Such high-tech treatment comes with a hefty price tag. Tisagenlecleucel is listed at $475,000 for a one-off administration.

The key challenge was to obtain the effectiveness inputs under the chemotherapy option. This was because tisagenlecleucel has only been studied in single-arm trials and individual level data was not available to the research team. The research team selected a single-arm study on the outcomes with clofarabine monotherapy, since its patients at baseline were most similar in terms of demographics and number of prior therapies to the tisagenlecleucel study.

This study is brilliant in approaching a difficult decision problem and conducting extensive sensitivity analysis. In particular, it tests the impact of common drivers of the cost-effectiveness of potentially curative therapies in children, such as the discount rate, duration of benefit, treatment initiation, and the inclusion of future health care costs. Ideally, the sensitivity analysis should also have tested the assumption that the studies informing the effectiveness inputs for tisagenlecleucel and clofarabine monotherapy were comparable or if clofarabine monotherapy does not represent the current standard of care, although it would be difficult to parameterise.

This outstanding study highlights the challenges posed by the approval of treatments based on single-arm studies. Had individual-level data been available, an adjusted comparison may have been possible, which would improve the degree of confidence in the cost-effectiveness of tisagenlecleucel. Regulators and trial sponsors should work together to make anonymised individual level data available to bonafide researchers.

Researcher requests for inappropriate analysis and reporting: a U.S. survey of consulting biostatisticians. Annals of Internal Medicine [PubMed] Published 10th October 2018

This study reports a survey of biostatisticians on the frequency and severity of requests for inappropriate analysis and reporting. The results are stunning!

The top 3 requests in terms of severity were to falsify statistical significance to support a desired result, change data to achieve the desired outcome and remove/alter data records to better support the research hypothesis. Fortunately, this sort of requests appears to be rare.

The top 3 requests in terms of frequency seem to be not showing a plot because it does not show an effect as strong as it had been hoped; to stress only the significant findings but under-reporting non-significant ones, and report results before data have been cleaned and validated.

Given the frequency and severity of the requests, the authors recommend that researchers should be better educated in good statistical practice and research ethics. I couldn’t agree more and would suggest that cost-effectiveness analysis is included, given that it informs policy decisions and it is generally conducted by multidisciplinary teams.

I’m now wondering what the responses would be if we did a similar survey to health economists, particularly those working in health technology assessment! Something for HESG, iHEA or ISPOR to look at for the future?