Is there any justification for interventions that aren't cost-effective?

Ethics, priorities and cancer

This is one of the most challenging questions nowadays. Anthony Culyer sheds light n this difficult issue in a recent article applied for cancer care. These are his nine  arguments:

Argument 1: the whole health maximisation assumption underlying the approach is misconceived. health care is about more than just promoting health. Other objectives commonly include financial protection (e.g. from the out-of-pocket expenseof costly interventions), innovation, and all those listed earlier
Argument 2: innovation is stifled by the strict application acost-effectiveness threshold that is too low
Argument 3: the use of standard outcome measures, like theEQ-5D QALY or averted DALYs, underestimates the health benefits of cancer treatments
Argument 4: the assessment of benefit excludes the beneficial effects that treatment and its consequences have on those who care for cancer patients
Argument 5: the opportunity cost argument is weak. There are always efficiency savings that can be found in any systemwhich mean that the alleged sacrifice of health represented by the threshold is spurious. the actual sacrifice is much smaller
Argument 6: cancer is a scary disease and people who suffer from it deserve to have access to treatments that would fail aconventional cost-effectiveness test
Argument 7: for some cancer patients a costly and not very effective treatment may offer a “last chance” to someone in despair. such a situation might exist if no intervention of any kind existed for these patients or if the patient suffered from a rare form of cancer
Argument 8: cancer is a “severe” disease and should accordingly be given a higher priority than less severe diseases
Argument 9: many cancer patients have a short life expectancy even with treatment. a quasi-utilitarian argument might cite the law of diminishing marginal value: even small gains for such people are to be valued more highly than the same gains of equivalent quality of life for people with an already long expectation of life. alternatively, there is the more direct emotional appeal “Our moral response to the imminence of death demands that we rescue the doomed proof"

These arguments fall into two broad groups. Some are questionsof social value: how should we value health gains of particular kindsand should we value them differently according as they accrueto different people? Others are questions of fact: would informa-tion about the quantitative size of the effects in question lead us to conclude that cancer is indeed a special case? The burden of proof in both cases lies with those making the assertion that cancer is, indeed, special. That burden of proof is not impossible to bear.

Is cancer a special case? The question may apply to many diseases and will provide more difficulties than answers. In the end any analysis relies on distributive justice principles and according to different views you'l apply different prioritisation criteria.

PS. The article was published in a cancer journal. I was surprised by the new perspective by Tony Culyer.

PS. What do you think about a new cancer inmunotherapy service that may cost $750.000???

Le Corbusier Guitariste (1960)

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The farce of confidential drug prices (2)

Payers’ experiences with confidential pharmaceutical price discounts: A survey of public and statutory health systems inNorth America, Europe, and Australasia

Some months  ago I posted on confidential drug pricing. I said that this was the end of cost-effectiveness as we have known. Now a new article reflects the evidence of my words:

Confidential price discounts are now common among the ten health systems that participated in our study, though some had only recently begun to use these pricing arrangements on a routine basis. Several health systems had used a wide variety of discounting schemes in the past two years. The most frequent discount received by participating health systems was between 20% and 29% of official list prices; however, six participants reported their health system received one or more discount over the past two years that was valued at 60% or more of the list prices. On average, participants reported that confidential discounts were more common, complex, and significant for specialty pharmaceuticals than for primary care pharmaceuticals.

If confidential discounts are huge (>60%), as they are, any cost-effectiveness analysis is adhoc and its obsolescence undermines any result. This fact is the recognition that the pricing system is not working and we are under a procurement system. As I said some months ago:The time to finish such farce has come.

Genome editing: understanding CRISPR-CAS9

Excellent speech by Salvador Macip at Grifols Foundation conference on CRISPR (in catalan):

Compensating behaviour after nudging

Nudges that fail

Cass Sunstein shows in his las published article that nudging may fail, and explains the reasons and what to do. Great, I was waiting for that, because we need to disentangle the current approaches to nudging. the article tries to shed light, but in the end, uncertainties remain.

The general point is that any form of choice architecture, including the use of default rules, may have little or no net effect if people are able to find otherdomains in which to counteract it. The idea of compensating behavior can be seen as a subset of the general category of strong antecedent references, but it points to a more specifi c case, in which the apparent success of the nudge is an illusion in terms of what choice architects actually care about (Hirschman,1991).
What matters is welfare, not effectiveness (Sunstein,2016). A largely ineffective nudge may have positive welfare effects; an effective nudge might turn out to reduce welfare. A strong reason for nudges, as distinguished from more aggressive tools, is that they preserve freedom of choice and thus allow people to go their own way. In many contexts, that is indeed a virtue, and the ineffectiveness of nudges, for some or many, is nothing to lament. But when choosers are making clear errors, and when third-party effects are involved, the ineffectiveness of nudges provides a good reason to consider stronger measures on welfare grounds.

 Therefore with this text Sunstein is landing to the practical difficulties on nudging. Highly recommended.

PS. Congratulations to Adam Oliver, C. Sunstein and G. Akerloff for the new journal.

Pissarro à Eragny - La nature retrouvée

Au Musée de Luxembourg maintenant

Controversies on QALYs

The Limitations of QALY: A Literature Review

After 50 years, valuing health using QALYs is still a daunting task. Basically the debate over ethical considerations, methodological issues and theoretical assumptions, and context or disease specific considerations is still alive. And I would add that it will remain as an open issue. Those that would like a simple metric for a complex issue will fail forever. And this pitfalls are translated to decision making when QALYs are the reference for resource allocation.
I'm unsure about what will be the next step. A recent article explains current limitations, but unfortunately I can't foresee alternative options for the future:

Debate continues to exist on whether QALYs should serve as the central means of health economics analysis. This review examines the potential shortfalls of QALYs, spanning current ethical, methodological, and contextual domains in addition to examining their suitability for regenerative medicine and future technologies. In the UK, NICE currently stipulates a threshold of £20 000 - £30 000 per QALY  when evaluating new therapeutics and/or technologies for NHS adoption, and has used this tool to apply a rational and transparent process to technological adoption for over ten years. Calculating QALY or cost effectiveness thresholds is particularly complex and debate has previously been publicized on whether the value of a QALY should be dictated by first proposing the worth of a QALY and setting the healthcare budget at or below that value, or alternatively, proposing a healthcare budget and then allowing the cost of a QALY to declare itself following purchasing decisions. With the advent of cellular based therapeutics and their comparably high upfront costs, the QALY calculation methodology may need refinement to realise the financial advantages and opportunity costs such interventions may convey – particularly considering the degree of uncertainty associated with them.

Meanwhile we should focus on improving comparative effectiveness of current and new technologies, specially those that are related to precision medicine.

 



 

Dr. Heisenberg's Magic Mirror of Uncertainty, 1998

Duan Michals exhibition in Barcelona

 

Are You What You Eat?

Are You What You Eat? Healthy Behaviour and Risk Preferences

I am not strictly a fan of economic experiments. They are useful, but usually researchers achieve conclusions from samples and settings that are far from what happens really in population and geographies. However, some days ago I was looking at an article that it seemed of interest. They try to:

estimate the degree of risk aversion for a sample of young healthy adults and we explore its links with a broad range of risky behaviours considered together. Second, as indicator of the overall quality of diet, we complement, for the first time, the BMI with the Healthy Eating Index (HEI), and we relate both to estimated risk preferences

Sounds good, because the use of Body Mass Index is absolutely outdated and its relationship with risk aversion is crucial. This is the summary:

Our results show that risk preferences significantly differ across young adults with different, not extreme, health conditions. In particular, they reinstate the importance of conducting analyses that look separately at the two sub-samples of female and male subjects . This allows disentangling the links and interactions between preferences and key health variables such as smoking, and also to fully account for the gender-specific effects of the BMI and of alternative indicators of healthy weight.

Second, in our sample young women do not show any significant robust associations between risk preferences and BMI. Third, for young men – but not women – the HEI index appears to be significantly and consistently associated with risk preferences: across all specifications, healthier nutritional habits, tend to be robustly associated with higher risk aversion. This, together with the lack of significance of BMI-based indexes, suggests that, for subjects with not extreme health conditions, there is a wide scope to use measures alternative (or complementary) to the BMI, as indicators of the overall quality of diet.

That's it. And his final recommendation:

 From a health policy perspective, our study suggests that in young adults who have not yet developed chronic or extreme health conditions, looking at a comprehensive nutritional indicator such as the HEI could provide more direct insights to the deeply rooted behavioural mechanisms that drive health behaviours than considering an indirect and increasingly questioned measure such as the BMI.

Since children's obesity is one of the main challenges for health improvement, someone should take into account this message.

PS. Eliciting risk and time preference, the 2008 key article.

 Nancy Vieira

Taxing unhealthy foods

The effect of prices on nutrition: Comparing the impact of product-and nutrient-specific taxes

Nowadays, many people is asking about evidence oon the impact of taxes for sugar sweetened beverages. The reason is that in Catalonia from May 1st. a new tax has been implemented.Two tax rates have been set in relation to sugar content: For drinks containing more than 8 grams of sugar per 100 ml: €0.12/litre. For drinks containing between 5 and 8 grams of sugar per 100 ml: €0.08/litre
A new article in the Journal of Health Economics sheds light on the issue:

Our main finding from the tax simulations is that nutrient-specific taxes have much larger effects on nutrition than do product-specific taxes, without causing a larger decline in consumer utility. The intuition for this result is that nutrient-based taxes have a much broader base, so it is more difficult to substitute away from any one good in response to such taxes. For example,a 20% tax on soda decreases total purchased calories by 4.84% and decreases sugar consumption by over 10%. However, a 20% sugar tax decreases total calories by over 18% and sugar by over 16%.The larger effect of a sugar tax on nutrition comes despite the fact that it has the same effect on indirect utility as a soda tax. Dueto their negative income elasticities and the patterns of own- and cross-price elasticities we find, taxes on snacks and packaged mealshave very small effects on nutrition. Fat and salt taxes, on the other hand, have much larger effects, decreasing calories by 19% and 11%, respectively. SSB taxes, which can be thought of as a hybrid price policy that targets a set of products based on their nutritional content, also are quite effective, reducing caloric intake by over 8%. However, these taxes are less-effective and only slightly less-distortive than a broad-based sugar tax.

If this is so, the next steps should be to review the initial impact and explore wether new approaches could be more succesful. Unfortunately the article doesn't explains the details of how to implement their result...

PS. On sugar

Ben l'oncle Soul

The challenges of medical practice variations

Medical Practice Variations

The title of this post is not original, it is really from a book published in 1990, 27 years ago! And Wennberg started such research on the 70's. What is new is the book "Medical Practice Variations" released last year. After all these years concerns have spread, methodological improvement is huge, and unfortunately evidence says that practice still shows wide range of variability. This is the main concern, what to do about it.
The description is excellent, 23 chapters and 527 pages reflect an effort of many years of several projects on the issue. A must read is the chapter 4, p. 53 by Enrique Bernal and his team: Medical Practice Variations in Elective Surgery. Variations may harm and produce waste, therefore understanding how to prevent low-value care is crucial. They say:

Two key steps in reducing low-value care, proposed by García-Armesto et al. (García-Armesto et al. 2013), are the following:
• Identifying those technologies ineffective in their usual indications or less effective than alternatives
– Dropping them from the benefits basket or making them subject to avoidable copayments
– Restricting indications to certain types of patients (choice guided by evidence of positive benefit/risk balance)
– Specifying and limiting the types of providers more suitable to offer each service (therefore substantiating indication becomes a requisite, discouraging irrelevant use)
– Capping the frequency or length of treatments
• Producing and making available guidance on a regular basis to reduce inappropriate use of procedures
– Highlighting and tackling unwarranted variations in elective surgery (naming and “shaming” to prompt query and change)
– Fostering best practices and improving coordination of care

As I said, a must read. Congratulations to the authors. Unfortunately the barrier is the price: $279. Notwithstanding that, health policy makers and managers should have it as a key reference for their decisions.

PS. If you want to know more about current projects, check the ECHO  website.

The tough figures of worldwide health spending

Evolution and patterns of global health financing 1995–2014: development assistance for health, and government, prepaid private, and out-of-pocket health spending in 184 countries

We live in a disparate world, and the range of health care expenditure per capita goes from $33 in Somalia to $9.267 in USA. These are tough figures, while in Somalia you'll understand that access is the problem, in USA disparity is inside, waste and access at the same time are the problems. The Lancet article shows the reality of world health expenditure. It worths reading it.

The availability of prepaid resources for health, such as government spending, is one of many determinants of access to health care, and can lead to population health gains. Economic development is associated with an increase in spending and specifically an increase in prepaid resources. This is at the core of the pursuit for universal health coverage. This research also points to countries that deviate from the trends, spending more or less than expected, based on their level of economic development. This information is valuable to planners assessing funding gaps and financing opportunities, and can be used to provide insight into what future health financing challenges are likely. Tracking changes in health financing patterns across time and benchmarking against global trends is vital to addressing missed opportunities, ensuring access to medicines and high quality services, and the pursuit of universal health coverage.

Gorgeous new album by Joan Miquel Oliver. Atlantis

ACOs state of the art

L’expérience américaine des Accountable Care Organizations:des enseignements pour la France ?

After all the efforts, ACOs coverage in US is right now only for 9% of population (28 million citizen). It seems a low figure. You can check the details of the current situation in an excellent report (en français) by IRDES. However, they consider that this approach could be useful for France, and I'm not so sure. The differences are huge to introduce something similar.

Parov Stelar - State of the Union

In Barcelona soon

Toolkit for comparative effectiveness

Methods in comparative effectiveness research

If comparative effectiveness is the new fram for valuing health technologies, then we need the appropriate toolkit. This is not new, I said the same in 2010 and afterwards in this blog. Right now there is a difference, you may read in this 600 pages book all the details about it (a chapter on machine learnisn is missing).
A clear understanding of comparative effectiveness is precisely what the authors of this report have neglected, unfortunately. It doesn't make any sense to start economic evaluation without an assessment of comparative effectiveness. It doesn't make any sense to back for QALYs as an accounting approach. Forget this guidelines, and suggest to read this book.

This volume covers the main areas of quantitative methodology for the design and analysis of CER studies. The volume has four major sections—causal inference; clinical trials; research synthesis; and specialized topics. The audience includes CER methodologists, quantitative-trained researchers interested in CER, and graduate students in statistics, epidemiology, and health services and outcomes research. The book assumes a masters-level course in regression analysis and familiarity with clinical research.

What is population health?

What does improving population health really mean?

Population health means the health outcomes of a defined group of people, as well as the distribution of health outcomes within the group.
Therefore, measuring and understanding the reasons for differences in health is a key factor for tackling them.
Kindig paper is the most relevant, and this one reflects the begining.
From the post by Kings Fund :

Approaching the golden age of epigenomics and epitranscriptomics

A new twist on epigenetics

If epigenomics is crucial to discard the genetic predestination paradigm, now we can add a new 'omics to the paradigm: epitranscriptomics. Last February, Nature published interesting news related to recent scientific developments:

The epigenome helps to explain how cells with identical DNA can develop into the multitude of specialized types that make up different tissues. The marks help cells in the heart, for example, maintain their identity and not turn into neurons or fat cells. Misplaced epigenetic marks are often found in cancerous cells.

 Chuan He and Tao Pan are two researchers that have been working on new ways of controlling gene expression

He and others have shown that a methyl group attached to adenine, one of the four bases in RNA, has crucial roles in cell differentiation, and may contribute to cancer, obesity and more. In 2015, He’s lab and two other teams uncovered the same chemical mark on adenine bases in DNA (methyl marks had previously been found only on cytosine), suggesting that the epigenome may be even richer than previously imagined.

The team had shown for the first time that RNA methylation was reversible, just like the marks found on DNA and histones.

Methylated adenine bases are the focus of research on gene expression.

Exercise as a socially contagious activity

Exercise contagion in a global social network

Disciplines as diverse as economics, sociology, medicine, computer science, political science and physics have recently become interested in the interdependence of behaviours across the human social network. In particular, scientists have begun to ask whether our health and other behaviours are contagious, in that our decisions and actions affect the decisions and actions of our peers. If behavioural contagions exist, understanding how, when and to what extent they manifest in different behaviours will enable us to transition from independent intervention strategies to more effective interdependent interventions that incorporate individuals’ social contexts into their treatments

A new  study offers some of the first hard evidence that health-related habits can spread — and so perhaps could be deliberately seeded and encouraged — by social influence and peer pressure. Previous research has sought such a contagious effect in factors such as obesity and smoking, but the results have been inconclusive.

Studies in social differences in health have a a new hurdle to tackle. How to boost social permeability? As Mackenback said in The Lancet on health inequalities: now it's personal.

PS. These are the results of the study in one figure: