Paying the bill of gene therapy

GENE THERAPY: Understanding the Science, Assessing the Evidence, and Paying for Value

Approximately 12-14 investigational gene therapies for additional ultra-rare conditions and some for more common conditions, such as haemophilia and sickle cell disease, are progressing through the developmental pathway and are expected to reach regulatory approval within the next 2-3 years

These therapies rely mostly on viral vector techniques, therefore they don't take into account the coming genome editing, the most disruptive one and the most recent as well. If this new technologies reach the market, how should be paid and applied?. This is what a recent report explains and gives details for decision makers. It is really welcome, the issue deserves a deeper understanding.

Situation in Europe
Glybera and Strimvelis, have been granted marketing authorization in the European Union by the European Medicines Agency (EMA):
- Glybera was approved by the EMA in 2012, but has since become the world’s most expensive short-term treatment (Adams, 2016), and as such has not been widely successful - it has only been used by one patient, with the prescribing clinician overcoming steep bureaucratic hurdles to obtain insurer funding (Abou-El-Enein et al., 2016a).
- Strimvelis received marketing authorization in 2016. Patients can currently only be treated in Milan, due to the treatment’s extremely short shelf life which dictates that cells must be infused back into the patient in less than six hours.

More efforts should be devoted to understand this emerging market and assess its value.

Caro Emerald

Rethinking income inequality and health (once again)

Income Inequality and Health: Strong Theories,Weaker Evidence

The inequality frame is usually flawed, and this is specially clear when the metholodogy and data to support the statements are biased. Let me suggest today this article that summarises perfectly common misunderstandings on this relationship. It would be a great input for a review and remake of recent papers.
The summary:

What is already known about this topic? A large body of research has examined the association between income inequality and average health. A separate body of research has explored income disparities in health. These two traditions should be seen as complementary, because high and rising income inequality is unlikely to affect the health of all socioeconomic groups equally. 

What is added by this report? Although plausible theories suggest that rising income inequality can affect both average health and health disparities, empirical tests provide only modest support for some of these theories. We argue that understanding the effects of income inequality on health requires attention to mechanisms that affect the health of different income groups, thus changing average health, disparities in health, or both. 

What are the implications for public health practice, policy, and research? Progress is likely to require disentangling direct effects of rising income inequality, which operate through changes in an individual’s own income, from indirect effects, which operate through changes in other people’s income. Indirect effects of rising income inequality may change a society’s political and economic institutions, social cohesion, culture, and norms of behavior, all of which can then affect individuals’ health even if their income remains unchanged.

PS. If someone needs an estimate of morbidity, please avoid inconsistent approaches. If someone needs policy guidance don't trust on cross-sectional data on such a difficult issue.

PS. My posts on health inequalities.

Anticipating public concern over genome editing

Genome editing: an ethical review

The Nuffield Council has released a key document on ethical implications of genome editing. You'll notice that it is an open document, a work in progress because technology is evolving. If you want an excerpt check this short guide.

It should be remembered that most prospective technologies fail, and that some lead to undesirable consequences, a fact often obscured by ‘whig’ histories that reconstruct the history of successful technologies and their beneficial social consequences. Scientific discovery and technological innovation is important but not inevitable. Most important among the factors shaping technological development is human agency. It is human agency, in terms of decisions that are made about directions of research, funding and investment, the setting of legal limits and regulatory principles, the design of institutions and programmes, and the desire for or acceptance of different possible states of affairs, that will determine whether, and which, prospective technologies emerge and, ultimately,
their historical significance.

Nuffield council work is of interest, meanwhile, China is already testing CRISPR technology in humans, no ethical concerns...

Josep Segú - Barcelona

The value of diagnostic information

The Value of Knowing and Knowing the Value: Improving the Health Technology Assessment of Complementary Diagnostics

Last summer the Office of Health Economics released an inspiring paper on the value of diagnostic information related to companion diagnostics and next generation sequencing genomic tests. I read it recently and its appproach sheds additional light on a difficult issue:

Traditional cost-effectiveness analysis conducted as part of HTA focuses on three key elements:
1. Life years gained
2. Improvements in patient quality of life
3. Cost-savings within the healthcare system (also called “cost-offsets”).
Elements 1 and 2 are often combined in the quality-adjusted life year (QALY) by HTA bodies.
Elements 1, 2, and 3, plus the cost of the technology, are then used to assess the  cost effectiveness of the technology.
The next element most often included is “productivity” or “time value”, reflecting gains and losses related to the value of the patient’s time either when receiving medical care or related to the impact of absenteeism or presenteeism due to illness. Another element - less commonly measured - is nonmedical cost-savings outside the healthcare sector, such as transport costs and family caregiving.
Based on our systematic literature review, we identified and defined five additional elements
related to the value of knowing and the value of information:
• Reduction in uncertainty - additional value from knowing a technology is more likely to work
• Value of hope - willingness to accept greater risk given a chance for a cure
• Real option value - the value of benefiting from future technologies due to life extension
• Insurance value - psychic value provided by invention of an innovative medical product and by the accompanying financial risk protection afforded by a new treatment
• Scientific spillovers - value due to other innovations that become possible once a new technology has been proven to work.

I have discussed many times such additional issues, specially the real option value. Unfortunately measurement of such items are not that easy. Anyway, it's good to take into account and let's hope new developments on this topic.

 

Photobook Phenomenon

Falsehood as ingredient of populist health policy

On Rumors: How Falsehoods Spread, Why We Believe Them, and What Can Be Done

Today I would like to suggest you to read Barack Obama in his farewell speech. It's an inspiring piece to understand US and democracies in general. Inequality, racism and polarization are the three key isues to tackle.
He said:

For too many of us, it’s become safer to retreat into our own bubbles, whether in our neighborhoods or college campuses or places of worship or our social media feeds, surrounded by people who look like us and share the same political outlook and never challenge our assumptions. The rise of naked partisanship, increasing economic and regional stratification, the splintering of our media into a channel for every taste – all this makes this great sorting seem natural, even inevitable. And increasingly, we become so secure in our bubbles that we accept only information, whether true or not, that fits our opinions, instead of basing our opinions on the evidence that’s out there.

This trend represents a third threat to our democracy. Politics is a battle of ideas; in the course of a healthy debate, we’ll prioritize different goals, and the different means of reaching them. But without some common baseline of facts; without a willingness to admit new information, and concede that your opponent is making a fair point, and that science and reason matter, we’ll keep talking past each other, making common ground and compromise impossible.

Without a common baseline of facts there is no possibility to talk to your political opponent. Nowadays, fake news like health privatization are continuously spreading without any foundation. Our health minister speaks incredibly about that in our Parliament. Such obsession has driven to submit a new unnecessary law on health contracting.We are now in an obsession focused policy era.
To understand this phenomena, Sunstein wrote a book in 2008, before thee term post-truth era, that explains the basics and what to do.
Cass Sunstein says in the book:

Rumors are nearly as old as human history, but with the rise of the Internet, they have become ubiquitous. In fact we are now awash in them. False rumors are especially troublesome; they impose real damage on individuals and institutions, and they often resist correction. They can threaten careers, relationships, policies, public officials, democracy, and sometimes even peace itself. Many of the most pervasive rumors involve governments—what officials are planning and why.

This small book has two goals. The first is to answer these questions: Why do ordinary human beings accept rumors, even false, destructive, and bizarre ones? Why do some groups, and even nations, accept rumors that other groups and nations deem preposterous? The second is to answer this question: What can we do to protect ourselves against the harmful effects of false rumors? As we shall see, part of the answer lies in recognizing that a “chilling effect” on those who would spread destructive falsehoods can be a truly excellent idea, especially if those falsehoods amount to libel.

Sensible people believe rumors, whether or not they are true. On the Internet, self-interested, malicious, and altruistic propagators find it increasingly easy to spread rumors about prominent people and institutions. Such rumors cast doubt on their target’s honesty, decency, fairness, patriotism, and sometimes even sanity; often they portray public figures as fundamentally confused or corrupt. Those who are not in the public sphere are similarly vulnerable.

Rumor transmission frequently occurs as a result of cascade effects and group polarization. Indeed, rumors spread as a textbook example of an informational cascade: imperfectly or entirely uninformed people accept a rumor that they hear from others, and as more and more people accept that rumor, the informational signal becomes very strong, and it is hard for the rest of us to resist it, even if it is false.

Group polarization also plays a large role, as people strengthen their commitment to a rumor simply because of discussions with like-minded others.

 If you want people to move away from their prior convictions, and to correct a false rumor, it is best to present them not with the opinions of their usual adversaries, whom they can dismiss, but instead with the views of people with whom they closely identify

The signal of the rumor may be so strong that though reality is absolutely different, and everybody is finally conceding it the value of truth. This is sadly what has happened to health privatization debate.

The value of health, and how to measure it

Valuing Health: WELL-BEING, FREEDOM, AND SUFFERING

Too often people talk about the value of health, and few are those that try to measure it. Now you have the opportunity to have a look at the book that summarises the state of the art on measuring health from different perspectives, clinical and research, epidemiology and economics (resource allocation). The implications of health in well being are explored, and the author says:

Valuing health states by their average consequences for well-being has the unfortunate implication that disabilities count as significant health problems only if the people who have them are significantly worse off than the people without them. With respect to disabilities, such as blindness, to which people adapt, this implication leaves the health analyst with a choice between asserting falsely that the blind necessarily have lower levels of well-being or asserting falsely that blindness is not a serious disability.
This unfortunate implication, coupled with the difficulties in measuring the value of health by eliciting preferences or by measuring subjective experience, raises doubts about the project of valuing health by its bearing on well-being, which chapter
10 explores. The value of health differs in important ways from well-being and indeed appears to be easier to measure than well-being.

And we all agree that health is a crucial factor for well-being, though its measurement is uncertain up to now.

PS. A wide review of the book.

On sugar as a toxic substance. How little is still too much?

THE CASE AGAINST SUGAR

Last book by Gary Taubes takes a difficult way, how to demonstrate sugar as a toxic substance for our health. Although he tries to show evidence for his words, he finally concedes the following conclusion:

Ultimately and obviously, the question of how much is too much becomes a personal decision, just as we all decide as adults what level of alcohol, caffeine, or cigarettes we’ll ingest. I’ve argued here that enough evidence exists for us to consider sugar very likely to be a toxic substance, and to make an informed decision about how best to balance the likely risks with the benefits. To know what those benefits are, though, it helps to see how life feels without sugar.

The "very likely" expression is crucial. Unfortunately we don't have a explicit causal explanation of the impact of sugar on metabolic syndrome, for example. I think that epigenetics will provide neew perspectives on the issue, however we will have to wait. Meanwhile reducing exposure is the best advice.

PS. On the sugar sweetened beverage taxes. And in this blog