The uncertainty over genomics sequencing value in clinical decision making

Assessing Genomic Sequencing Information for Health Care Decision Making: Workshop Summary

"The value of genetic sequence information will depend on how it is used in the clinic", key statement that needs some elaboration. This is precisely what the IOM report does, you'll find in their pages the current situation about how genomics may impact in decision making. In chapter 5 you'll understand how an insurer decides about coverage of such tests according to 5 criteria:

1. The test or treatment must have final approval from appropriate governmental regulatory bodies, where required;
2. scientific evidence must permit conclusions about its effect on medical outcomes;
3. technology must improve net health outcomes;
4. the technology must provide as much health benefit as established alternatives; and
5. the improvement in health must be attainable outside investigational settings.

Unfortunately, if you start from the first one, you'll find a complete lack of references by governmental bodies on the approval of such tests. Therefore, I can't understand from the chapter how successful they are on such process.
While reading the book you'll increase your uncertainty about outcomes and value of genomic tests instead of reducing it. This was my impression. Let's wait for future good news, again.

PS. Summary of the report:
"Clinical use of DNA sequencing relies on identifying linkages between diseases and genetic variants or groups of variants. More than 140,000 germline mutations have been submitted to the Human Gene Mutation Database and almost 12,000 single nucleotide polymorphisms have currently been associated with various diseases, including Alzheimer’s and type 2 diabetes, but the majority of associations have not been rigorously confirmed and may play only a minor role in disease. Because of the lack of evidence available for assessing variants, evaluation bodies have made few recommendations for the use of genetic tests in health care."

Consensus in health policy

Some months ago I posted on the same topic. Now you can read my short article in the annual report of the Col.legi de Metges.(text in catalan)

El consens en la política sanitària: el Pacte Nacional de Salut

La política sanitària i els valors socials. La immediatesa i dinamisme del món en que vivim ens porta massa sovint a considerar la política sanitària com el fruit de la decisió puntual, anecdòtica i controvertida del moment. Ens cal una mirada més panoràmica per comprendre que darrera tota política sanitària hi trobarem uns valors socials que li donen fonament. És a dir judicis de valor sobre allò que és bo per a la societat. En ocasions es referiran als objectius o resultats finals i en d’altres als mitjans per assolir-los. Si bé la seva rellevància és determinant per als objectius, en relació als mitjans cal tenir present a més a més la seva efectivitat, i per tant un judici expert sobre allò que de veritat funciona.

L’objectiu final de tota política sanitària descansa sobre la millora de la salut poblacional però va més enllà. L’any 2006 la Unió Europea va considerar que els sistemes de salut són un pilar de la protecció social, de la cohesió i de la justícia social, i assenyala que la universalitat, l’ accés a l’assistència de qualitat, l’ equitat i solidaritat són els valors europeus compartits[i]. En aquest marc i en el seu desenvolupament és on es fonamenta la política sanitària.

El consens en política sanitària. L’adopció de decisions públiques en relació a aquests valors essencials requereix d’un consens polític. Pertoca al parlament i al govern assenyalar què signifiquen exactament aquests valors compartits (objectiu) i quines decisions cal adoptar per assolir-los (mitjans). L’existència d’un consens social sobre aquestes qüestions esdevé una peça clau de l’engranatge. Més enllà de l’acord polític parlamentari, cal que tots els actors que participen en el sistema de salut remin en la mateixa direcció. Precisament un dels elements que es consideren factor d’èxit d’un sistema sanitari és el consens social[ii]. Ens cal doncs una política sanitària basada en el màxim consens possible per tal de tenir un sistema eficient i equitatiu.

La política sanitària basada en l’evidència. Distingir aquells mitjans que poden ser efectius per assolir objectius d’aquells que no ho són, esdevé una prioritat. Disposar d’evidència per tal de contrastar aquelles decisions que produiran el millor resultat és crucial, però alhora complex. La complexitat prové de les singularitats i del context on es desenvolupa cada política. Allò que ha funcionat en un lloc i moment determinat, pot ser difícil de ser reproduït en un altre. Malgrat aquest atenuant, saber allò que funciona amb un criteri expert i objectiu ha d’ajudar a millorar les decisions. Molts informes d’experts sobre reforma sanitària han tractat d’adoptar aquesta perspectiva, si bé amb impacte força limitat. La falta d’aplicació del consell expert té a veure almenys amb les dificultats d’establir consensos amplis i la comprensió del procés polític[iii].

El pacte nacional de salut i els àmbits de consens. La Comissió de Salut del Parlament de Catalunya va acordar el febrer de 2013 impulsar el treballs per assolir un acord per a la salut a Catalunya en el marc d’un model propi. En aquest acord es defineixen les bases del sistema sanitari català, estables i consensuades per tots els agents implicats. Es va precisar que els membres de la comissió serien els representants dels diversos grups parlamentaris i els agents que formen part del Consell Català de la Salut. Els treballs de la comissió es van desenvolupar en vuit grans àmbits temàtics. Es va prendre com a referent els 6 blocs inicials que segons l’Organització Mundial de la Salut han de constituir un sistema sanitari, i s’hi van afegir dos àmbits identificats com a claus pel sistema (la recerca i innovació, i el compromís ciutadà). Els àmbits han estat doncs: finançament i cobertura, professionals, prestacions i catàleg de serveis, model de serveis, avaluació i transparència, recerca i innovació, compromís ciutadà, i governança.

Entrar en el detall del contingut dels 83 acord va més enllà del que es pretén en aquest article, tant sols es farà una breu referència als 2 primers. En l’àmbit de finançament i cobertura s’assenyala amb claredat l’opció per un accés universal de la ciutadania al Sistema Nacional de Salut i alhora s’estableix un criteri de nivell de finançament públic en salut suficient i sostenible, que es relacioni amb el nivell de riquesa del país i que convergeixi amb la despesa de països amb producte interior brut per càpita equivalent i sistema sanitari similar.
En l’àmbit dels professionals s’ha considerat que la planificació de necessitats de professionals, les competències i capacitats acreditades han de ser objecte reconsideració atenent als canvis sociodemògràfics, econòmics i tecnològics. S’explicita l’èmfasi en el professionalisme com a criteri que guia la relació entre professionals amb la ciutadania, amb el sistema sanitari i els proveïdors, i la necessitat d’establir mecanismes per tal de fer efectiva la participació dels professionals en l’elaboració de polítiques i la gestió.

El nivell de consens i els propers passos. El procés per arribar a aquest conjunt d’acords ha estat fruit d’una elevada participació. Diversos motius van impedir que la totalitat dels representants confirmessin el seu acord al darrer moment. Cal assenyalar que en l’elaboració del document hi ha contribucions de tots, també d’entitats i grups polítics que no han pogut donar finalment el seu suport. Cal fer efectiu aquest consens que desitja la ciutadania, les bases perquè això sigui possible hi són, només cal teixir-les acuradament i amb generositat. El Parlament de Catalunya va donar l’opció de mostrar el sistema sanitari que desitgem a una àmplia representació social, ara ja hi ha les bases per a que això pugui transformar-se en realitat.

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[i] Council Conclusions on common values and principles in European Union  health systems, OJ 2006 No. C146/1.

[ii] Balabanova D, Mills A, Conteh L, et al. Good Health at Low Cost 25 years on: lessons for the future of health systems strengthening. Lancet 2013;381:2118-2133

[iii] Black, Nick. "Evidence based policy: proceed with care." BMJ: British Medical Journal 323.7307 (2001): 275.

Statistical vs. identifiable lives

Do We Really Value Identified Lives More Highly Than Statistical Lives?

The recent Ebola evacuated case exemplifies the concept created by Shelling a long time ago, the difference of how a society allocates resources according to 2 different rules:

In 1968, in a paper about valuing ways to reduce the risk of death, Thomas Schelling1 distinguished between “identified lives” and “statistical lives.” Identified lives are the miners trapped in a mine or the child with a terminal disease—specific people who need help now. Statistical lives are those people, unidentifiable before the fact and often after as well, who will be saved by a new safety regulation, public health program, or environmental standard. Schelling observed that people seem to be willing to pay more to save an identified life: “Let a six-year-old girl with brown hair need thousands of dollars for an operation that will prolong her life until Christmas, and the post office will be swamped with nickels and dimes to save her. But let it be reported that without a sales tax the hospital facilities of Massachusetts will deteriorate and cause a barely perceptible increase in preventable deaths—not many will drop a tear or reach for their checkbooks.

Really such a case goes beyond Shelling insight because of uncertainty and unavailability of effective treatment. Bioethics field has argued over what they called "rule of rescue", a different perspective of the same issue. In this respect, NICE statement helps to understand both views:

When there are limited resources for healthcare, applying the ‘rule of rescue’ may mean that other people will not be able to have the care or treatment they need. NICE recognises that when it is making its decisions it should consider the needs of present and future patients of the NHS who are anonymous and who do not necessarily have people to argue their case on their behalf. NICE considers that the principles provided in this document are appropriate to resolve the tension between the needs of an individual patient and the needs of present and future users of the NHS. The Institute has not therefore adopted an additional ‘rule of rescue.

The article by Louise B. Rusell reflects precisely the theoretical and practical controversy and ends with this paragraph:

Adjustments and controversies aside, the evidence provided by VSL estimates suggests that people’s willingness to pay for statistical lives may be consistent with their willingness to pay for identified lives. The apparent existence of 2 different decision rules may have been no more than an artifact of the economic method for valuing statistical lives in use at the time the distinction was proposed. Now that economists’ methods more fully reflect “the interests, preferences and attitudes to risk of those who are likely to be affected by the decisions,” their estimates of the value of a statistical life support the idea that there just may be a single rule: Identified and unidentified lives may be equally valuable. This is good news for decision makers who use cost-benefit and cost-effectiveness analysis to inform decisions.

The theoretical suggestion sounds good, nowadays the political decision making reality goes in the opposite way, at least close.

PS. A must read post on GCS blog about the same topic.

PS. Ebolanomics, the economics of ebola at the New Yorker. Nothing new, prizes instead of patents to promote R&D, a good idea with difficult implementation.

PS. How much would you pay for a quality adjusted life year?

Walt  Khun.  From Tita Cervera  St. Feliu exhibition

Inconvenient reasons

International statistics provide useful information on trends and allow to compare between countries. This is only true if all countries do their homework. In the last edition of  OECD health data, Australia, Japan, New Zealand, Spain and Turkey have "forgotten" to send 2012 expenditure  data. Maybe there are some inconvenient reasons behind that. Anyway, somebody should ask in Parliament about the delay and why they are not delivering the statistics..
Beyond that, I suggest a look at internal 2012 data, coming from Ministry. You'll find that there are some countries with 9.4% public health expenditure over GDP, while others like Catalonia spend 4.9%. Again, somebody should ask in Parliament if there is anybody in charge of the situation. I stop here to avoid any misinterpretation. Disconnecting is the right option, goodbye.

PS. By the way, today you can find in ARA a flawed op-ed. Check the current and right figures and the argument vanishes.

Good Music with Txarango, you may download it free at txarango.com

Health as a shared responsibility

During our lifetime we are exposed to risks, some of them are preventable while others not.
Prevention is a shared responsibility, individuals and society may affect the course of such potential health events. Today, while reading in the press about Ebola controls in the flight Conakry-Casablanca-Madrid I was really concerned about how governments are dealing with such an outbreak. If this is true and only three controls were really made, somebody should request explanations in Parliament. Individual opportunism may avoid to declare "yes" when they were asked if they felt bad.
Sounds naive. A responsible individual should say yes if he really feels bad, but he may also ask himself about cost and benefit of such answer...
On September 24th we are helding a roundtable on such issues: "Individual and collective responsibility on health". It may be of interest for the readers of this blog. If you finally come we may meet at the end of the session, Minorca deserves a yearly visit at least.

PS. Full programme, here

Enough is enough

If there is a grey area in medical devices and services regulation, this is the Laboratory Developed Tests one. Up to now, FDA has refused to define the rules of the game for 11,000 diagnostic tests performed at 2,000 labs in USA. This means that no official or external reviewer has analysed the clinical validity and clinical utility as it is done in any reagent and instrument. I can't understand why we have arrived at such a situation.
Fortunately NYT reports that on July 31st, FDA announced that this will change.

The agency said on Thursday that such discretion must end because circumstances had changed. Lab-developed tests once were fairly simple, often developed by a hospital for tests on its own patients. Now the tests can be complex and are being developed by  companies and marketed widely.

Some widely used commercial tests have never had to be reviewed by the agency. These include Myriad Genetics’ breast cancer risk test, the subject of a Supreme Court patent decision last year; the Oncotype DX test from Genomic Health, which is used to determine if women with early-stage breast cancer need chemotherapy; and noninvasive prenatal tests for Down syndrome that are rapidly catching on.

In this blog I have supported several times for a clear regulation of these tests . Just the other day when looking at the statements of FDA commissioner, I was astonished:

Just as drugs need to be safe and effective for treating diseases, medical devices used to help diagnose disease and direct therapy also need to be safe and effective, Faulty test results could lead patients to seek unnecessary treatment or to delay or to forgo treatment altogether.

These statement raise more concerns about what US regulator has done after all these years. And european regulation is still worse in this sense. I have explained such disaster previously and up to now there is no news. Some times I wonder why do we pay taxes, why do we have to be part of Europe. Enough is enough.

Failures and successes of the engines of democracy: Politics and Policymaking

Why Government Fails So Often: And How It Can Do Better

Our democracy finally has been created to deliver specific outcomes. However, citizens are increasingly unsatisfied with governments. Fortunately we can analyse this fact through a new book that attempts to disentangle the issue going deeper than usual in the roots of the problem:

Americans have a dismal opinion of the federal government’s performance, one that is only getting darker.4 Significantly, this growing antipathy is not antigovernment generally. Instead, it targets only the federal government; respect for state and local governments is both high and stable. Nor is this hostility toward the federal government in Washington a partisan matter. Instead, it is expressed by a majority of Democrats as well as Republicans. And perhaps most revealing, this disaffection long preceded the current political gridlock in Congress that many pundits see (wrongly, as I shall show) as the root of the problem.

You may change the word americans at the begining and place your nationality and this former statement could work. We should question what government failure or success means and the authors look at the literature on policy evaluation and find fewer references than expected.

Understanding government failure, then, presents complex challenges. Its funders, consumers, and ultimate appraisers—“We the People”—are more disgruntled than ever, and the social scientists who assess the evidence most rigorously find that these appraisers’ disapproval is amply warranted.

The author considers that government failures are rooted on recurrent weaknesses that  include unrealistic goals, perverse incentives, poor and distorted information, systemic irrationality, rigidity and lack of credibility, a mediocre bureaucracy, powerful and inescapable markets, and the inherent limits of law. This sounds familiar. Anyway, something should be done, and part 3 is entirely devoted to this issue. I would like to highlight the limits of the law as a constraint, others details are in the book that I strongly recommend. He says:

The very nature of public law places some severe limits—both constitutional and functional—on the effectiveness of the policies that it communicates and governs. Although most of these limits cannot be avoided, some of them might be eased.

I really think that we should explore new options for producing laws and assessing its performance, the current situation of the "democracy engine" is outdated.

PS .A review at WSJ.

 Many of our political debates are about what if anything government should do about the problems our society confronts. The combatants in these battles rarely stop to consider just what government actually can do.

 Peter H. Schuck has written an essential manual for 21st-century policy makers.

When bad science leads to bad policy

The Corruption of Peer Review Is Harming Scientific Credibility

Nothing new, scientific peer review is a process under suspicion. The Guardian illustrated this fact three years ago, and WSJ has repeated the same recently. The potential solution is to put into practice the falsiability process as Popper emphasized long time ago. The main difficulty is data access, however some journals have started to supply such data for researchers in order to confirm the results. My position about it is clear, as a referee I'll refuse to review more papers unless this option is possible for any submitted article. The potential harm is huge in certain fields and circumstances, as the Vioxx case illustrates.
The WSJ op-ed says:

Fixing peer review won't be easy, although exposing its weaknesses is a good place to start. Michael Eisen, a biologist at UC Berkeley, is a co-founder of the Public Library of Science, one of the world's largest nonprofit science publishers. He told me in an email that, "We need to get away from the notion, proven wrong on a daily basis, that peer review of any kind at any journal means that a work of science is correct. What it means is that a few (1-4) people read it over and didn't see any major problems. That's a very low bar in even the best of circumstances."
But even the most rigorous peer review can be effective only if authors provide the data they used to reach their results, something that many still won't do and that few journals require for publication. Some publishers have begun to mandate open data. In March the Public Library of Science began requiring that study data be publicly available. That means anyone with the ability to check should be able to reproduce, validate and understand the findings in a published paper.

Doctor crisis. What crisis?

The Doctor Crisis: How Physicians Can, and Must, Lead the Way to Better Health Care

Last May I saw this press release about a book by a physician from Kaiser Permanente. Initially I thought that it would be a book for those interested uniquely in US healthcare. I started reading "The Doctor Crisis" last week and still can't stop. It has captured my attention. His observations about the practice of medicine and the pressures that physicians are under, are similar in any developed country, maybe the intensity is not the same. Anyway, in the book there is a reference of a work by Sinsky et al.:  In Search of Joy in Practice: A Report of 23 High-Functioning Primary Care Practices, a must read:

The current practice model in primary care is unsustainable. We question why young people would devote 11 years preparing for a career during which they will spend a substantial portion of their work days, as well as much of their personal time at nights, on form-filling, box-ticking, and other clerical tasks that do not utilize their training. Likewise, we question whether patients benefit when their physicians spend most of their work effort on such tasks.³⁰ Primary care physician burnout threatens the quality of patient care, access, and cost-containment within the US health care system.

We set out in search of joy in practice. What we found were pockets of professional satisfaction.

I missed this article when it appeared last year.What they explain is in my opinion what exactly should be done. As Tom Bodenheimer says, “the Triple Aim should be a quadruple aim, with clinician and staff satisfaction a necessity to achieve the other three aims.”  Considering it as an input and not only as a goal itself is the right approach. More on Berwick's triple aim, at IHI.
I am only at the begining of the book, but I wanted today to reflect this critical issue of our health systems. Something should be done beyond the triple aim. Organizational innovation is required. Right now I am not able to perceive such effort around here.

PS. About the title, focusing only on physicians is a too narrow perspective for those who have to lead a better health care , why not "transdisciplinary professionalism"?

PS. A suggestion: their blog.

Morbidity adjusted life-expectancy

OBTENCIÓN DE LA ESPERANZA DE VIDA Y DESCOMPOSICIÓN EN ESTADOS DE SALUD A PARTIR DE INFORMACIÓN CLÍNICA

I have always considered that any estimate of healthy life expectancy that is build upon many assumptions and coefficients in the end it is difficult to understand. The global burden of disease and its use of DALYs is an extraordinary effort, though if you dig into the results you'll find methological difficulties.
An alternative to such estimates is just to show how the burden of mordibity is distributed across lifetimes. That is precisely what we have presented at the last Health Economics Conference. I believe that such estimation is a promising way to present population life expectancy and health. As far as this is the first attempt, there is still room for improvement.

Healthy and satisfied

Enquesta de salut de Catalunya

Latest data from the 2013 Health Survey shows that 81,1% of the population consider themselves as healthy, slightly better that in 2010 that was 79,3%. Regarding satisfaction, 86,9% of population is satisfied with public health services, again better than 2010 that was 84,7%. Though in 2012 results were a little bit better.
In the details of the results you'll find that obesity and overweight is the biggest issue to address in my opinion. There is still a lot to do on tobacco and alcohol, but data shows some improvement.
This health survey should be broadcast in the media and efforts to promote healthy behaviours should raise. Unfortunately nowadays media is focused on negative messages and this issue lies far from journalists' interests.

Positive and negative risk cultures

Risk Savvy

While reading The Guardian I find out that Nudge theories could fall from the mainstream.:

Though nudge-economics remains seductive, what once seemed like a panacea has come to look a bit more like a series of sticking plasters. Earlier this year the nudge unit was removed from direct government control, partly sold to the Nesta innovation charity run by New Labour guru Geoff Mulgan, a move which seemed to suggest the prime minister no longer viewed it as quite so central to his philosophy. That move has coincided with a backlash, or at least a critical analysis, of some of the tenets on which its brand of behavioural economics is based.

You already know from this blog I have devoted many posts to it. And I've said many times that its application is still in its beginings. However, if you look at the new book by Gerd Gigerenzer "Risk Savvy", maybe the perspective could be otherwise. He examines Kahneman works and gives a different view. The issue of two systems of the brain, A and B, when taking decisions is under criticism. He defends heuristics that in some sense use both when taking some difficult decisions.
His work goes beyond such criticism and it is an additional perspective on how we take decisions and the role of risk and uncertainty.
He considers that health sector is dominated by a negative risk culture, a way of doing that tries to hide errors and in such situations learning is much more difficult. On the other end of the spectrum are "positive error cultures that make error transparent, encorage good errors and learn from bad errors to create a safer environment". This is the case of commercial aviation. From his view, the use of check lists and safety measures should be boosted in many settings to improve efficiency.
Gigerenzer work is a good recommendation for summer reading. Wether he is able to convince you more than Kahneman, it's uncertain right now.

Big data, big opportunity

Learning from Big Health Care Data

Big Data is more than a buzzword, it raises high expectations about how the massive treatment of data may deliver new results. At NEJM you'll find an article that explains general implications for health care:

Two key “learning” applications of big health care data that hold the promise of improving patient care are the generation of new knowledge about the effectiveness of treatments and the prediction of outcomes. Both these functions exceed the bounds of most computer applications currently used in health care, which tend to offer physicians such tools as context-sensitive warning messages, reminders, suggestions for economical prescribing, and results of mandated quality-improvement activities

At JEP, you'll find an article by Hal Varian that shows the new challenges for econometrics:

Conventional statistical and econometric techniques such as regression often work well, but there are issues unique to big datasets that may require different tools. First, the sheer size of the data involved may require more powerful data manipulation tools. Second, we may have more potential predictors than appropriate for estimation, so we need to do some kind of variable selection. Third, large datasets may allow for more flexible relationships than simple linear models.

All in all, you'll be convinced that it is more than a buzzword.

PS. You may find an example of application of big data in our recent article in Gaceta Sanitaria.

Who sets the health policy agenda?

Making Health Policy

From this book:

In relation to policy making, the term agenda means: the list of subjects or problems to which government officials and people outside of government closely associated with those officials, are paying some serious attention at any given time . Out of the set of all conceivable subjects or problems to which officials could be paying attention, they do in fact seriously attend to some rather than others.

The crucial issue is who sets the policy agenda, how and why. Two main sources appear as agenda-setting: government and mass media-social networks. There are of course, additional groups and lobbyists that can influence such a process.
Nowadays we could consider that the recession and cutbacks has created a window of opportunity for some to discuss many foundations of our health system. In such a situation, the worst position is the delay on setting the list of topics to be addressed by the government, otherwise non-elected bodies try to mobilise efforts and decisions towards their interests that add to those of the opposition. Therefore, if you are interested on the basics of agenda-setting, have a look at chapter 4 and ask yourself who is in control of it. Are you comfortable with the answer?. If not, something should be done.