June 27, 2017

Critical thinking in medicine

Testing Treatments
Els Tractaments, a prova

If you are interested in critical thinking about treatment claims, then now it is the time to read this book in english and catalan. You'll understand:
Why do we need fair tests of treatments?
What are fair tests of treatments?
What can be done to improve tests of treatments?
How can YOU help to improve tests of treatments?
And you'll find a review by Xavier Bonfill at Annals




June 25, 2017

Health as the capacity for action

Autonomy in Patient-Centered Care for Chronic Conditions

Currently the concept of health as the absence of disease is absolutely outdated. The goal of clinical care has moved towards health-related quality of life. I have to admit that this is more an aim than a reality, but many professionalos share this view. If this is so, then considering health as capacity for action becomes critical. Mark Sullivan explains this view in an outstanding book, specially on part 3 and 4.

The turn to HRQL arose from the recognition that observable characteristics of the body could not provide an adequate account of the overall burden of chronic disease on individual patients or on the population as a whole. But the HRQL concept merely tried to add the experience of the patient to a notion of health defined by observable damage to the body or objective disease. It is not adequate to talk about the subjective experience of objectively defined health states. Health is not an objective state because neither death nor disease are always bad for patients. Most patients now choose to limit the medical care they receive at the end of life because they do not see death as the worst possible outcome. Similarly, it is possible for older adults to live well despite multiple chronic diseases. HRQL has not provided a fundamentally new metric for health and has not succeeded in redirecting clinical trials or activity to a more patient centered set of goals.
Any adequate, comprehensive, and valid approach to health must focus on personal agency, or the capacity of the person to achieve his or her goals. This is the only way we will understand and achieve individual well- being. This personal agency is both a means to well- being and an intrinsically valuable component of it. We conceptualize health and well- being as capabilities rather than as objective or subjective states: “our capability to lead the kind of lives we have reason to value.” To explore and define a new sense of health as agency, we will need to understand the various ways in which health and action are related.
A deep view of bioethics and the concept of health, all together. Good read for this summer.




June 16, 2017

The value of lab testing in precision medicine


Before Jevons, economists were unable to think on marginal terms. If price should be related to marginal utility, then cost pricing nowadays is outdated. However, when someone suggests value pricing, you must ask immediately about what is value for him, and maybe it is not the same than for me. A paper on lab testing and its value suggests the following:
The value of a diagnostic arises not because of its direct effect on a patient’s health but because of the information it provides on a patient’s likely response to a particular therapy. Personalized diagnostic testing reduces – though does not eliminate – the trial-and-error associated with empirical medicine, where physicians and their patients try an initial set of therapies and decide to continue or discontinue them on the basis of realized efficacy and side effects. In this manner, personalized diagnostic tests transform medical care from what economists call “experience goods,” whose quality can only be determined through consumption, to “search goods,” whose quality can be learned before  consumption
Personalized diagnostic testing offers several advantages over an empirical, trial-and-error approach to medicine. These benefits include the avoidance of side effects, potentially reduced financial costs of therapy (e.g., if a patient is identified as a likely nonresponder to an expensive therapy and the alternative is cheaper), potentially reduced opportunity costs of time – not just in terms of physician visits but also time lost on an ineffective or even harmful treatment, and improved or earlier access to effective care. Not only do patients receive value from personalized testing and treatment, but providers and health care systems benefit by avoiding ineffective, or wasteful, health care that accompanies less targeted, traditional treatment approaches. Specifically, a diagnostic test will be most valuable when the therapy being evaluated is expensive relative to alternatives, when side effects are frequent and severe (thereby making the empirical approach relatively less safe), and when delay from an alternate therapy can severely harm an individual’s health (e.g., metastatic cancer)
The concept is clear, its measurement is still uncertain.

June 15, 2017

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)

June 9, 2017

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.


June 8, 2017

Genome editing: understanding CRISPR-CAS9

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



June 2, 2017

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

May 31, 2017

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
 

May 26, 2017

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.


May 23, 2017

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

May 18, 2017

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.

May 3, 2017

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

April 30, 2017

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

April 26, 2017

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.