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Journal round-up: PharmacoEconomics 39(5)

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The May 2021 (Volume 39, Issue 5) issue of Pharmacoeconomics features two economic evaluations, two economic burden/resource utilization studies, two country-specific valuation studies, and two systematic reviews. The reviews provide syntheses of ‘societal costs’ in women with breast cancer in LILAC (Latin American and Caribbean) countries and utility decrements in overweight Australian adults. There is also an article for a 20-person Delphi process to frame a decision problem focused on comparator identification and a lead Editorial about a proposal to create a Hispa-NICE (that is, Spanish NICE). I thought both of these last two articles were interesting for different reasons, which I will get into.

In his Editorial, Hispa-NICE: A Pipe Dream or a Realistic and Necessary Initiative?, Fernando Antoñanzas describes a December 2020 “Manifesto advocating for the creation of an independent authority for health practices and policies”, titled Towards a Hispa-NICE: Now or Never. First, let me say writing a ‘manifesto’ has more verve and drama than writing a ‘White Paper’, which is the usual, milquetoast alternative proffered in Canada. This manifesto [PDF] calls for a powerful tool, an independent body with good governance, to provide more coherent recommendations regarding what health technologies should be financed, based on the best available scientific evidence and considering social values. Essentially, a coordinating function that integrates some of the work of the Spanish Agency of Drugs and Medical Devices and regional HTA authorities. The problem it is attempting to deal with is the perceived arbitrariness in decision-making and priority setting of drugs, diagnostic tests, programs, or health interventions.

For me, this is interesting for two reasons. Firstly, it mimics many of the challenges involved with pluralistic health systems, of which Canada is one. When everyone is given a budget to do their own thing, inequity always looms its head. Coordination between healthcare systems requires courage and compromise. The original vision of the Canadian Agency for Drugs and Technologies in Health was actually to coordinate an HTA function across provinces (the Canadian Coordinating Office for Health Technology Assessment). To date, it has barely scratched the surface on this front, as the management of technology is too far removed from a coordinating authority. Even provincial Ministries have difficulty managing technology since private, not-for-profit hospitals and associated buying groups are the main deciders of what’s in and out. Similar challenges were revealed through the EUnetHTA project: governance has a bigger effect on how technology is adopted than science. I hope we start to see more focus on the role of governance in technology management.

The second interesting feature is that leaning toward vertical integration and centralized governance may be an inevitable consequence of two contemporary developments: the first is that electronic infrastructure has made it much easier to information share on a larger scale, and the value of big data cannot be ignored; the second is smaller, regional authorities are beginning to realize they are much less effective at extracting value from highly coordinated, global multinational life sciences companies than their larger, centralized counterparts. So, centralized healthcare systems have a stronger buying position and can measure things at scale with bigger data. The future is expensive, and good decisions require information. I wonder at what point countries begin to collectively bargain. It seems like a logical next step when monopolist and monopsonist titans try to sort out prices and fair game when buyers are up against more coordinated sellers.

The second article by Edna Keeney and Bristol-based colleagues that caught my interest was the use of the engagement of experts in prostate cancer early detection, treatment, modelling and the role of genetics in prostate cancer risk from the UK, Sweden, Finland, the Netherlands, and the US. They were asked to define a decision problem including key elements: who should be invited for screening, which diagnostic procedures, how frequently to screen? There were divergent views, and the modified Delphi procedure helped sort things out. I enjoyed this because the authors showed how a more disciplined approach to expert elicitation and stakeholder involvement is needed when conducting an economic evaluation. While I think it was appropriate to engage clinicians in this exercise, I certainly hope to see more descriptions of expert elicitation and stakeholder involvement supporting economic evaluation in the future. There is, I think, much more to be done in involving patients, the public, or community stakeholders, not only in defining decision problems but outcomes and others components of economic evaluation. See this article for more on these exciting developments in participatory research.

I have not too much to say about the other articles. You may be happy to hear stated preference surveys (time trade-off and discrete choice experiments) have been used to value the SF-6Dv2 in mainland China (TTO won!). Similarly, the EQ-5D-5L valuation study from Egypt has been retracted and updated (an error in the calculation was discovered).

The cost-effectiveness studies show us how vastly different the meaning of ‘cost-effectiveness study’ can be, depending on the approach. A more realistic approach would be to measure things in real-time with real data, as was done by a group of Ontario researchers. They followed 547 Ontarian patients prescribed afatinib, erlotinib, and gefitinib for non-small cell lung cancer and discovered afatinib produced 0.21 QALYS for an additional CAD$9745. This is interesting when you see that the original estimates based on RCTs were 0.21 QALYS and CAD$540. Good prognosticating, Boehringer Ingelheim!

The second cost-effectiveness analysis takes the more fantastical approach, extracting a 6-month post hoc analysis from a placebo-controlled trial and matching adjusted indirect treatment comparisons to show that using siponimod instead of interferon or best supportive care in secondary progressive multiple sclerosis leads to an addition CHF 84,901 while producing 1.591 more QALYs over a Swiss patient’s lifetime. What I don’t understand is why some comparators weren’t used in the analysis as it seems that some people with RRMS and taking a bunch of other therapies would develop SPMS. I’m sure I’m missing something. Perhaps they should have consulted the Bristolians.

In not-so-surprising news, late-stage Parkinson’s disease costs the German health system more than earlier stages, with age being a cost driver. Similarly, multiple myeloma costs the Portuguese authorities a lot of Euros, with drugs representing the lion’s share. While characterizing costs are important and certainly a necessary input into things like economic evaluations, I do hope these studies are not misinterpreted by some as useful for decision-making. Since decision-making is about choices, we probably want to understand what preferences people have for health or wealth rather than price tags.


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