Cost-effectiveness of new (genomic) benefits, it's just the begining

HERC database of health economics and genomics studies 
Cost-effectiveness of cell-free DNA in maternal blood testing for prenatal detection of trisomy 21, 18 and 13: a systematic review

Just yesterday our government suddenly decided to introduce a new benefit in public insurance coverage: contingent DNA based non-invasive prenatal screening. And the question is: does someone know if this new benefit is cost-effective?
You can get the answer after reading this review article, and the summary is:

 In total, 12 studies were included, four of them performed in Europe. Three studies evaluated NIPT as a contingent test, three studies evaluated a universal NIPT, and six studies evaluated both. The results are heterogeneous, especially for the contingent NIPT where the results range from NIPT being dominant to a dominated strategy. Universal NIPT was found to be more effective but also costlier than the usual screening, with very high incremental cost-effectiveness ratios. One advantage of screening with NIPT is lower invasive procedure-related foetal losses than with usual screening. In conclusion, the cost-effectiveness of contingent NIPT is uncertain according to several studies, while the universal NIPT is not cost-effective currently.

If this is so, since uncertainty is the word that better reflects its current cost-effectiveness, why do the have introduced? Because they don't care about it. These are not the best days for a health economist (and for the society as a whole). Maybe it's just the begining of a new world without scarcity, and I can't figure out.

Manuel Anoro

Health care access in EU

BENCHMARKING ACCESS TO HEALTHCARE IN THE EU.Report of the Expert Panel on effective ways of investing in Health

If access is one of the main features of any health system, we do need better measures to understand the current situation. This is my conclusion after reading the report. Differences across EU countries (and within countries!) are huge in unmet needs , in copayments, in catastrophic expenditures... The surprising figure for me was that Spain is at the top of out of pocket expenditures as a share of household expenditures (3.5%) with Latvia. I have to check it. If so, bad news. Universal healthcare is being questioned. Does someone care about it?

Saul Leiter, Barcelona exhibition

Cost-effectiveness of genome sequencing (3)

Application of next-generation sequencing to improve cancer management: A review of the clinical effectiveness and cost-effectiveness

Once again, there is no need for cost-effectiveness if there is not a clear message on the analytical validity, clinical validity and utility of a diagnostic test.
A new article want to shed light on cancer and NGS, and says:

Our search for cost‐effectiveness studies on NGS in cancer care yielded 2037 articles. Only 6 articles included cost‐effectiveness studies of the application of NGS (targeted gene panel) in cancer

The 6 selected reports could be separated into 2 types. Three of the articles assessed the cost‐effectiveness of recommending patients receiving targeted therapy matching their genetic mutation identified via NGS; and the remaining 3 articles assessed the cost‐effectiveness of using NGS as part of the screening program to direct patients or high risk family members into prophylactic treatment

Two out of 3 articles in the “targeted therapy” group reported that NGS and targeted therapy was not cost-effective (Table 3A), using an ICER threshold of US$100 000 per Quality Adjusted Life
Year (QALY) gained. An ICER of less than US$100 000/QALYs gained is generally considered favourable for funding in the United States

Two out of the 3 articles in the “screening” group reported that the use of NGS was cost‐effective (Table 3B), that is, under US$100 000 per QALY gained.loser surveillance.

 In our evaluation of the effectiveness of NGS, we found that NGS is effective at identifying mutations in cancer patients, and we reported that 37% of the diagnosed patients proceeded to receive therapy matching their genetic profile. However, with only 6 articles available that assess the cost-effectiveness of NGS in various settings, it remains an area for future research to determine whether the technology is cost-effective in routine cancer management

Summary: the message is that there is no message with such a few observations!


Something is being missed...

Guillem Roma and Marta Roma

Value creators and extractors

The Value of Everything: Making and Taking in the Global Economy

The rethorics of value is usually plagued with deliberate misunderstandings. Specially, those that quote themselves as value creators may appear on a close look as a value extractors. This is precisely what the book of Marianna Mazucatto does. It identifies the patterns to assess value creation and extraction and the private of public and private roles.Chapter 7 on Extracting Value through the Innovation Economy is specially helpful. You'll find there the patents as a value extraction process or the pharmaceutical pricing discussed in detail. Therefore, a must read.

In modern capitalism, value-extraction is rewarded more highly than value-creation: the productive process that drives a healthy economy and society. From companies driven solely to maximize shareholder value to astronomically high prices of medicines justified through big pharma's 'value pricing', we misidentify taking with making, and have lost sight of what value really means. Once a central plank of economic thought, this concept of value - what it is, why it matters to us - is simply no longer discussed.

 The logical outcome of a combination of monopoly and rigid demand is sky-high prices, and this is precisely what is happening with specialty drugs. It explains why pharmaceutical companies enjoy absurdly high profit margins: in addition to the normal profit rate, they earn huge monopoly rents.59 A value-based assessment of the kind NICE carries out can be helpful because it reduces demand for the monopolists’ drugs and prevents them from charging whatever price they choose. The downside, however, is that increased elasticity of demand for drugs comes at the cost of leaving some patients without the medicines they need, because pharmaceutical companies may not cut their prices enough to treat everyone who needs the drug if doing so would reduce profit margins by more than the companies want.

The improvement of the healthcare access and quality

Measuring performance on the Healthcare Access and Quality Index for 195 countries and territories and selected subnational locations: a systematic analysis from the Global Burden of Disease Study 2016

While the global consensus on universal health care is a fact, healthcare access is really diverse. Among countries you can check the differences in the new article that has appeared in Lancet.

From 1990 to 2016, 186 of 195 countries and territories significantly increased their HAQ Index score, with several middle-SDI countries, including China, the Maldives, Equatorial Guinea, Peru, and Thailand achieving among the most pronounced gains. 

 In 2016, HAQ Index performance spanned from a high of 97·1 (95% UI 95·8–98·1) in Iceland, followed by 96·6 (94·9–97·9) in Norway and 96·1 (94·5–97·3) in the Netherlands, to values as low as 18·6 (13·1–24·4) in the Central African Republic, 19·0 (14·3–23·7) in Somalia, and 23·4 (20·2–26·8) in Guinea-Bissau. The pace of progress achieved between 1990 and 2016 varied, with markedly faster improvements occurring between 2000 and 2016 for many countries in sub-Saharan Africa and southeast Asia, whereas several countries in Latin America and elsewhere saw progress stagnate after experiencing considerable advances in the HAQ Index between 1990 and 2000. Striking subnational disparities emerged in personal health-care access and quality, with China and India having particularly large gaps between locations with the highest and lowest scores in 2016. In China, performance ranged from 91·5 (89·1–93·6) in Beijing to 48·0 (43·4–53·2) in Tibet (a 43·5-point difference), while India saw a 30·8-point disparity, from 64·8 (59·6–68·8) in Goa to 34·0 (30·3–38·1) in Assam.

 The measurement has been done with 32 causes from which death should not occur in the presence of effective care to approximate personal health-care access and quality by location and over time. This means that death registries should be available and reliable, and we know that there is no standard practice across countries. Anyway, the message is clear, most of the world is improving access to better healthcare and this is good news.

Aminata’ (2013) by Omar Victor Diop

Evorafrica exhibition

The p53 nightmare

p53 and Me

This week you'll find a short piece in NEJM, a story written by a physician on how detecting a genetic p53 mutation changed her views. Key message:

Genetic knowledge is power only if both clinician and patient are equipped to move beyond a result and toward action, even if that merely means living well with what we know. I believe we need an expanded definition of genetic counseling; we require more data, yes, but also more sophisticated and sensitive ways of assimilating such data. And not just into databases we can mine to see what happens to people like me, but into programs for learning to live with uncertainty.

A must read

The spanish flu, a century later

Pale Rider: The Spanish Flu of 1918 and How it Changed the World

Laura Spinney has made a great job with her latest book "The Pale Rider". For those that are interested on the largest recent epidemy and public health crisis -the spanish flu of 1918,- this is the book to read. I enjoyed specially the details of what should be avoided, and nobody cared about it. The conflict between religion and medicine. You'll not get the precise number of deaths, but it was an enormous tragedy in social terms.
The book also explains how physicians were exposed to the disease without any tools and how it was arriving to the remote and less inhabited places of the world.
It is specially helpful to recognise how vulnerable are all of us, still now. Highly recommended.

PS. You'll find it also in spanish, "El jinete pálido"

Theranos: a massive fraud and epic failure

The Theranos deception on CBS News 60 Minutes

The weirdest health financing system of the world (2)

Tracking Universal Health Coverage: 2017 Global Monitoring Report

If I had to summarise the best outcome of health policy in the last century in western countries, I would say mandatory health insurance. No doubt. And the joint report by WHO and WB reminds us that there is still a long way to achieve such goal for the whole population in the world.  Mandatory insurance is the most efficient way to solve the failures of the health insurance market. We al know the details and difficulties that arise as a result of information asymmetries and opportunistic behaviour.
Therefore the recommendation is clear, for those that already have a mandatory system, keep on it. This is precisely what hasn't happened here. In 2012 the system changed from universal towards a social security based membership funded by taxes. The weirdest health financing system of the world.

Maya Fadeeva with Club des Belugas

Measuring morbidity in large populations

MODELIZACIÓN ECONÓMICA DEL AJUSTE POR RIESGO DEL GASTO SANITARIO PER CÁPITA SEGÚN MORBILIDAD EN LA COMUNIDAD VALENCIANA

There is a unique study on measuring morbidity in a large population. In Valencia (4.7 million inhabitants) the Clinical Risk Group classification system has been applied. And you may find the results comparing the whole population, and one Department (Denia). The study shows details about the utilization and costs related with morbidity. Interesting application that replicates former ones. Epidemiologists, clinicians, policy makers and managers should be interested in using these approaches for taking decisions.

Pablo Picasso, by Arnold Newman

Changing the production function of diagnostic tests

Next-generation diagnostics with CRISPR

Last week while reading Science I noticed a short and crucial article. Up to now CRISPR technology was focused on gene editing, now we can say that its usefulness is widening into diagnostics. It may change completely molecular diagnostics of "infectious diseases through detection of Zika virus (ZIKV), Dengue virus (DENV), and human papillomavirus (HPV) in human  samples, and noninfectious diseases, such as detection of gene mutations in circulating cell-free DNA from lung cancer patients." The production founction of lab testing would change completely.
Several articles explain details about it. The fight for patents is going to start again on CRISPR diagnostics. And this are unfortunately bad news.
Anyway, Science article reminds us:

These emerging diagnostic tools will by necessity be compared to standard diagnostics to ensure sensitivity and specificity and will need to be field-tested to guarantee performance in patient care settings, as environmental conditions and end-user application might affect performance. Proven assays, if affordable, promise to improve care in resource-limited settings where undifferentiated febrile illness is the norm and where gaps or delays in diagnosis, targeted care, and infection control contribute to infectious disease mortality and spread.

More details in The Verge.

Cost-effectiveness of genome sequencing (2)

Application of next-generation sequencing to improve cancer management: A review of the clinical effectiveness and cost-effectiveness

If you want to go deeper on the issue, have a look at this article. It is focused on one disease, cancer and tries to combine clinical effectiveness and cost effectiveness. Sounds good. At the end you'll see that the number of available studies is limited (6), but that's the situation and these are the conclusions:

We report the rate of successfully detecting mutations from the clinical studies. The incremental cost-effectiveness ratio and sensitivity analysis outcomes are reported for the cost-effectiveness articles. Fifty-six articles reported that sequencing patient samples using targeted gene panels, and 83% of the successfully sequenced patients harboured at least 1 mutation.

 In our evaluation of the effectiveness of NGS, we found that NGS is effective at identifying mutations in cancer patients, and we reported that 37% of the diagnosed patients proceeded to receive therapy matching their genetic profile. However, with only 6 articles available that assess the cost-effectiveness of NGS in various settings, it remains an area for future research to determine whether the technology is cost-effective in routine cancer management.

PS. Today this blog has surpassed its 200.000 visits. That's great! Thank you for your loyalty.

Sally Mann, On the Maury, 1992, gelatin silver print, Private collection.

Washington National Gallery, current exhibition

Cost-effectiveness of genome sequencing

Are whole-exome and whole-genome sequencing approaches cost-effective? A systematic review of the literature

It is quite difficult to talk about value in genetic tests without any reference to analytical validity, clinical validity and clinical utility. Once these three issues are appropriately solved, then we need to assess costs. Cost effectivenes makes sense once this three steps are covered successfully.
An analysis of cost-effectiveness of whole genome/exome sequencing it sounds too generic if there is no reference to specific baseline that allows to estimate incremental cost-effectiveness ratios.
That's the reason why a recent article trying to review existing studies fails to achieve any conclusion.

The current health economic evidence base to support the more widespread use of WES and WGS in clinical practice is very limited. Studies that carefully evaluate the costs,
effectiveness, and cost-effectiveness of these tests are urgently needed to support their translation into clinical practice.

 Let's start focusing on the assessment of three key perspectives before entering into a black hole.

Diagnosing diseases and editing genes

CRISPR: The gene-editing tool revolutionizing biomedical research
The WIRED Guide to Crispr
A New Startup Wants to Use Crispr to Diagnose Disease

Take your time and read Wired and watch CBSNews. It will provide an exciting perspective of CRISPR. And just to end, imagine that diagnostic tests could be based on CRISPR while you read the last piece of WIRED.
We are closer than you may think.