Tag Archives: health policy

Being right when you’re wrong: on NICE and the Cancer Drugs Fund

Conceptual work by Yves Klein at Rue Gentil-Be...

When rules don’t work [Conceptual work by Yves Klein at Rue Gentil-Bernard, Fontenay-aux-Roses, October 1960, photo by Harry Shunk. Le Saut dans le Vide (Leap into the Void) (Photo credit: Wikipedia)]

 

 

 

 

 

 

Sir Andrew Dillon, the erstwhile leader of NICE as said that it is irrational for the Cancer Drug Fund to pay for drugs that NICE has turned down.

 

He’s right of course, it is irrational. But only if NICE’s logic is compelling.

 

The problem for Sir Andrew, and likemined people, is that there is another logic that trumps NICE’s rational world. Don’t get me wrong. NICE performs a useful, but technocratic, function with analytical assessments that any rational person would indeed want to know. Where we part company is believing that NICE’s logic is the final word on the matter. Which it isn’t.

 

Tasked, perhaps unenviably, with parsing the performance of medicines and clinical practice, cannot also mean that they are above challenge. Many of NICE’s rulings fly in the face, not of logic, but of our beliefs as humans. It is why we do things when the odds are against us, because not to do so would be wrong. If we think of the challenges NICE faces as wicked problems, that is complex problems with a multiplicity of solutions, it becomes self-evident that their logic is just one way of deciding and choosing. We could use other rules, other criteria. The Cancer Drug Fund is just such an approach. It is another matter whether we should have in place alternative funding approaches that individuals can avail themselves of (such as co-payments or co-insurance); for extraordinarily costly therapies, co-funding would not apply, so we’ll back to the problem anyway.

 

NICE has a troublesome relationship with the notion of ‘rule of rescue’ and so has decided to ignore it. There replacement, the “end-of-life premium” is really just a reweighting of the logic they use.

 

You see, the rule of rescue is what we might call a meta-rule — it is a rule that tells us if other rules are working properly, and importantly, as a moral imperative which tells us what to do. The rule is often invoked in a particular form: that people facing death should be treated regardless of cost. The rule as originally formulated is really about assisting identifiable individuals facing avoidable death (Jonson, 1986); the bioethicists and economists have shifted this to a cost-effectiveness approach, making it one about trade-offs instead.

 

The problem for healthcare systems is that all patients are becoming identifiable as medicines become personalised (medicines may become orphan drugs). The problem for the NHS is that it does not allow such people to rescue themselves because it prohibits any sort of co-funding or other arrangements. The only option is an opt-out (and private medical insurance has rules about pre-existing conditions). Given the funding priorities of the NHS, we should be reflecting not so much on how to make the pot bigger, but on using the money that is available better (there will never be enough money), and ways to introduce practical co-funding.

 

Since individuals have no other options in the NHS, the rule of rescue as a moral imperative will be violated and we will act, not out of analytical error (i.e. make a technical mistake), but unethically. You see, the NHS must be the healthcare system of last resort and therefore of rescue, otherwise, identified individuals are destined to a death sanctioned by public policy and is that a policy or healthcare system worth having?

 

We have seen a similar challenge to NHS/NICE logic recently with the King family and proton beam therapy, and the NHS will also use NICE logic to determine access. Whether beams or drugs, it is the same argument.

 

But why cancer? The main public policy question is why should cancer patients be given preferential treatment as against any other deserving group? This may in part be driven by the often astronomical costs of new cancer therapies themselves, which demarcate cancer patients decisively from equally deserving patients with less cost-contentious therapies. I have just finished some work on motor neuron disease, for which there is one specific medicine and life expectancy from diagnosis is 3 to 5 years, with median survival rates that are measured in months. NICE reportedly is developing guidelines for this disease. Costs are considerable, and at least in the UK, highlight the bureaucratic illogic of separate healthcare and social care, but that is another story.

 

The moral dilemma that the economists at NICE are trying to reduce to an equation is whether a new therapy is extending life, or delaying death. The Oregon approach collapsed when the hard choices emerged and people were unable to resolve this dilemma, which is not a quantitative issue, but one of how we value our humanity. Kierkegaard’s Concluding Unscientific Postscript speaks of the leap to faith as involving self-reflection and the emergence of scepticism. It is worrisome that NICE is so confident.

Further reading

 

Cookson R, McCabe C, Tsuchiya A. Public healthcare resource allocation and the Rule of Rescue. J Med Ethics. 2008 Jan 7 [cited 2014 Sep 4];34(7):540–4.
Jonsen, AR 1986, Bentham in a box: technology assessment and health care allocation, Law, Medicine and Health Care, Vol 14, pp172–4.
Richardson J, McKie J. The rule of rescue, working paper 112, Centre for Health Program Evaluation, Monash University

European Health Technology Assessment: time for Euro-NICE?

Sommelier F.I.S.A.R.

Quality control

NICE is the health technology assessment [HTA] agency in England, the one upon which others in many countries are modelled.  More generally, HTA is increasingly being used by governments for more than simple economic evaluation of medicines and devices, but is being used as a form of rationing of access to technologies (medicines or devices are considered technologies in this sense), and for budgetary control.  The more mundane scientific assessment of technologies alone, required for licensure, is gradually being replaced across Europe with a patchwork of regulatory HTA systems.  Their operation has a direct bearing on what technologies enter health system use in specific countries and therefore what benefits patients and clinicians derive from the use of those technologies.

So much is not new.

With release of the Commission’s findings in its Pharmaceutical Sector Inquiry Report, 9 July 2009, there is the opportunity to reflect further on HTA methods across Europe. The report notes that one reason given by industry why fewer novel medicines were reaching the market was uncertainty about financial rewards (a factor in whether technology assessment would lead to regulatory and reimbursement approval).  The report notes that with prescription medicines the patient is not the ultimate consumer, it being the doctor or pharmacist; to some extent this is true, but these clinicians are making decisions based on what products are on the market at any one time, and it is regulatory systems and the structure of formularies that determine this.  In more extreme cases, decisions are embedded in prescribing guidelines and substitution protocols, which a child could follow.

The report also misunderstands how HTA works, and this may reflect the fact that member states pursue HTA for various reasons.  Using the rather oblique term “added value” they see HTA as essentially linked to the “budgetary situation and health priorities of each member state”.  This is, of course, precisely the problem.  HTA IS an economic assessment, based on value add, but it is NOT defined purely in terms of affordability.  It provides information to decision-makers about what the economic and social costs are for different thresholds of health outcome — a polite way of pricing rationing.  In other words it informs decision-making, it does not determine it.  It is quite possible to arrive at a Europe-wide HTA finding on anything you like, and leave it up to member states to quantify in their own terms where this sits within their own national system of affordability and a desired health benefits.

I doubt the pharmaceutical industry would think of advocating for a European Health Technology Assessment Agency, using consistent scientific and economic standards applicable to all countries, thereby simplifying their own regulatory hurdles (HTA is referred to as a 4th hurdle), because they dislike the 4th hurdle in the first place.  But HTA is not going to go away, but misuse and abuse of HTA methods will likely continue.

My view is that the industry should advocate for such a system.

The argument is straight-forward.  A “Euro-NICE” would bring order to the HTA process, and establish EU-wide standards for the application of HTA methodologies and enhance regulatory certainty which industry should like.  It would have the additional benefit of creating a level-playing field amongst the different health systems, which would be a general benefit to patients.

It is important to understand that the pharmaceutical industry and individual member state health systems are locked in a very tight embrace, helped along by a country’s regulatory system and medicines pricing mechanisms.  Movement by either has an impact on the other — reimbursement policies signal industry behaviours, availability of medicines determines what clinicians get to use and the medicines budget, regulatory ‘style’ links to investment in research, research investment acts to attract the big brains.  It is a complex system, not a simple set of linear relationships (an impression one gets from the Commission Inquiry, unfortunately).  And complex systems can cause perverse behaviours, which are invariably embedded in the rules themselves, and particularly important rules are HTA practices, not codified with in the formal medicines regulatory system, but which condition different member state markets.

While there is action on European HTA, through EUnetHTA, it is necessary to distinguish between doing HTA itself, and how HTA is used in decision-making within national policy systems, and regulation.  A Euro-NICE would provide consistency in the former, to ensure transparency with the latter.

Swedish Presidency: e-health, flu and patient mobility

Recurrence plot of mathematicaly generated chaos

Map of a complex health problem

The Swedish presidency has identified a number of health priorities. I’d like to reflect on the following:

1. patient mobility directive,

2. e-health cooperation, and

3. flu.

Patient mobility adds cross-border health risk as people with diseases move around. Zoonoses, diseases caught from animals (and it is worth remembering that animals catch diseases from humans, for instance pigs have caught H1N1 from humans in Canada), are spread by mobility.  Perhaps the most dangerous animal other than humans for disease transmission are bats, flying in a 3d world, social animals with rather exotic immune systems. I’m all for patient mobility and see within this considerable opportunities to offer patients greater choice, drive quality improvements in national health systems, and so on.

E-health cooperation is important.  However, cooperation between health systems at some level is actually quite mundane:cross-border access to electronic health records, an EU-recognised prescription and cross-border dispensing, smart card for health and so forth. This has been the focus but real national progress is still weak, perhaps constrained by fear of unrestrained cross-border care itself.  E-health works when location doesn’t matters, that the patient matters more than where the hospital or doctor is located.  Underlying e-health are some really disruptive forces, such as unpicking the cartel-like behaviour and favoured position of particular health professions, and replacing professionally defined service structures with ones that reflect patient preferences, giving patients real power and control over health information, indeed, fully empowered patients and health consumers might actually drive the objectives of health policy, something that might upset the privileged role of the policy elites that gather for their usual policy pow-wows.

But, e-health for Europe means something a bit different, hence my inclusion of flu.  Cross-border vigilance of zoonoses becomes possible by bringing together satellite or similar sensor networks, real-time data transmission, and really useful mapping for visualisation.The current flu pandemic is really only one in name; there are far worse diseases currently ravaging human populations, killing more people in a day than the current flu has so far in total.  With climate change for instance, they are coming to a neighbourhood near you, and animals, insects, snails, and other hosts migrate north.

Of course, it goes without saying that such technical capabilities must be put into some context — why would we want to do this anyway?

Simply put, such a system enables free movement of people around Europe within a system of disease monitoring and decision-making that aligns the problems and necessary resources better.  Our public health models, we have learned with the current H1N1 pandemic shows that containment strategies don’t work.  Despite Treaty powers at national levels, health issues in a cross-border world transcend the ability of member states to act in a fully autonomous way, when their actions/inactions may affect the health of others (recall the UK and BSE crisis).

So, while each of these three threads is in and of itself worthy of significant hyperactive attention, the real opportunities lie when they are put together, seeing the linkages amongst the parts.

Perhaps one of the lessons that might come from the Swedish presidency is an understanding that in the modern world, the really important issues are complex, almost chaotic.

What is to fear from cross-border healthcare?

Simulated gravitational lensing (black hole go...

European policy: detected only in its absence?

The British have raised the issue that cross-border healthcare will only benefit the few:

All EU citizens, not just the wealthy or well informed, must be able to benefit

Baroness Howarth of Breckland, of the House of Lords’ EU Committee

Is this fear or concern justified?

The whole point of enabling European citizens to have access to healthcare in other EU member states, apart from it being the logical conclusion of substantial ECJ decision-making and rulings, is that it addresses directly improved access to healthcare.

Citizens will be able to seek healthcare anywhere and whenever their own healthcare system is manifestly unable to respond.  The UK’s National Health Service lacks a consumer-orientation, so behaves like a massive rigid system whenever it is challenged by patients and their own preferences.  This is evidenced most recently by the gyrations over NICE and access to life-saving medicines that are expensive, or the much praised ‘choice’ agenda, something those in other EU states largely take for granted.  This may also explain why the UK has had so much trouble getting used to the concept.

Their fears though do point to the importance of ensuring that all member states provide:

1. easily accessed information on EU health providers, presented in a way to facilitate patient decision-making;

2. unambiguous procedures to enable access to healthcare in other countries written in a way that explains how to access the entitlement;

3. defined administrative procedures that enable this entitlement and

4. clearly defined payment, reimbursement methods with transparent costs.

There is no point having a right that you can’t access.

It is also time to see Commission leadership in encouraging service providers to harmonise their information on EU healthcare providers, payment and reimbursement systems, and access procedures.   It is also time for commercial and other service entities to emerge to facilitate patient access to care, a sort of cross-border healthcare access broker.

And it is time to stop calling it ‘medical tourism’; this is serious business for the patient.

Cross-border healthcare: finally some progress

Japanese tourists at Riffelsee

Have we found the clinic yet?

Resistance is futile, say some, but the spread of Community protection for European health has taken an important step forward.

Some countries will be dismayed, others relieved.  Dismay in some because prideful to the end they cannot image the likely exodus of their patients to more congenial and responsive health systems and relief in others as the logic of a European health system, sharing scarce resources and providing a world-standard of healthcare is finally on the horizon.

Is it just medical tourism?  I think not.  Member states characterise it this way as though people shop around for a knee operation the way they might price a pair of shoes.  Healthcare matters to people and perhaps matters more than it does to governments who oversee health systems.  It is people who have the personal relationship with health professionals and have the needs for treatment.  For some governments, that people is what healthcare systems are about is a novel concept.

The willingness of people to seek healthcare in another country, unfamiliar, different language and culture, is stark testimony in many cases to the intransigence and bureaucratic bully-boy tactics of state-run health systems, more to ensure control than facilitate treatment.

Smaller member states perhaps understand this better as it is economic madness for them to try to run a comprehensive healthcare system for all possible demands, and instead work with other countries to pool resources.  Larger states, more congested with pride, will swallow the new cross-border paradigm with great difficulty and argue that economics (it is usually economics, too) will just make it not possible for them to afford this.

This is, of course, nonsense and posturing.  For a country not to reimburse a person for treatment in another country is just evidence that they would not pay for that person to be treated at home — but they would never say that.  Rather, cross-border patient movement is evidence of dissatisfaction with care, or inability to be treated because of administrative procedures and delay — and they would never admit that, either.

The proposed way forward is clear, the identified goals worthy and the challenges the right ones (data protection, quality standards, patient rights) to achieve these goals.

Health and Security: the civil liberties dimension

Barbed tape at a prison

Quarantine zone

While it is good to see progress at the European level to deal with threats to human health that do not recognise borders, the steps that may be taken to ensure security in the name of health many lead to greater threats to civil liberties.

Integral to coordinated responses to public health threats are efforts to quarantine populations, restrict movement of individuals (infected or not), and triage.  Where are the risks to civil liberties?

First, quarantining and free movement.  Establishing the boundaries of quarantine zones if notably difficult.  Toronto, with SARS, had great difficulty not just identifying where the boundaries should be, but in keeping people from wandering across them.  Officials lacked the legal authority to restrict freedom of movement as, of course, no crimes were being committed.  Indeed, in order to enforce quarantine boundaries, illiberal measures may be required.

Are quarantine zones to be identified by physical barriers with armed guards (to keep people in and others out)?  And what is to be done to infected people who try to ‘escape’?  What enforcement procedures are likely and when do they escalate to the use of lethal force?  We know from general behaviour of armed forces that they are not so easy to control when confronted with unruly citizens, and may actually be loathe to fire on their own citizens.  The challenge facing planners of quarantine zones is defining their scope: are they big enough to catch all likely infected people and then some who aren’t or are they small enough to ensure that no uninfected person is included, but risks missing some infected people?  The public health planners would prefer the former, despite creating the possibility that uninfected people would then be essentially trapped with infected people — a potential death sentence.  No surprise if some of these people might try to escape, to be confronted by what response from officials?

Second, is triage.  This is the decision-making about who lives and who dies.  Certainly in combat, soldiers understand the risks and accept that battlefield decisions are not easy to make.  In peacetime, such decisions take on a completely different complexion.  Public health threats such as pandemics are not combat, despite the rhetoric from governments that such threats much be met with a ‘war on disease’.  How, then, are the life and death decisions to be made?  Within a quarantine zone, would a large number of untreatable and infected people be left to die when numbers swamp the capacity of the health system to cope?  It must be remembered that a widespread pandemic would create chaos within health systems as health professionals fall ill or become trapped in quarantine zones.  Medical supplies will become scarce, and treatment facilities will be unable to cope with the demand at the rate at which it is appearing — people getting sick faster than people are being treated and getting better.

The risks are that we won’t consider these issues within the context of civil liberties and human rights, but use the excuse of a public emergency to enact essentially authoritarian measures, without reflection in the longer term of the consequences for our society.  The evidence, though, is before us: terrorists threats are met with authoritarian legislation which is often found by courts to violate human rights or constitutions.  Will health be the next victim?

Innovative medicines: it is about the patient, stupid!

The Clown

Clowning around with policy

The Innovative Medicines Initiative seeks to put right the chaotic treatment of pharmaceutical innovation by EU member states, and will likely fail.  The goose that lays golden eggs of medical innovation is cooked to death by member state medicines policies.

Increasing the speed of product development will come to a grinding halt as member states inconsistently apply economic evidence, and thereby stifle adoption of new medicines into clinical use to the benefit of patients.

Current regulatory thinking (i.e. EMEA) is dated as the priorities have moved beyond safety/efficacy issues to outcomes and cost-effectiveness.   NICE and its counterparts across Europe have yet to harmonise their methods, adding uncertainty as member states use economic evaluation as a 4th hurdle which varies in height depending on which country you’re in.  That economic evaluation is really just a code for controlling health expenditure and reimbursement, and is less about real value and outcomes in many member states only adds to chaos of the regulatory environment into which innovative products are thrown.

So, as we incentivise the early stages of medicines development and speed up the launch of drugs, we continue to tolerate a bottleneck on the utilisation and adoption of medicines themselves within member states.    The whole translational medicine process, from bench to bedside, is not just of faster benches, but of clear bedside clinical priorities.  We can incentivise the bench until the cows come home, but until we address the real disconnect between adoption and utilisation and link innovation priorities with real-world priorities, patients will still not have the medicines they need.  However, the statistics will undoubtedly show a rise in innovation!

Cross Border Healthcare: the good, the bad and the ugly

Lukschy as John Baxter in a A Fistful of Dolla...

Demanding better policy?

The good news appears to be some progress in raising the possibility of a European health system, the bad news is that apparently it is going to take a lot longer than patients in particular would like. Which brings us to the ugly truth that many member states are fiercely resisting progress on this front and the Commission is now delaying draft plans.

The EU is certainly big enough and mature enough to work toward EU standards of care, comparative performance information and consistent statistical reporting, all of which would do wonders to improve the overall quality of care European receive from their health systems.

But what do the member states have to fear? Some offer their citizens a patchwork of poorly integrated services. Others punish their citizens with waiting, waiting and more waiting. And others are blanketed in bureaucratic processes that serve only to increase costs, delay treatment, and keep people in positions of dubious authority. There is much we don’t know, such as whether there is in fact so much demand for cross-border healthcare that it will destabilise the ability of member states to control their budgets — this is highly unlikely in the near term anyway, and in the longer term there are far more important imponderables driving healthcare costs than a few patients having their operations done somewhere else.

There are good and compelling reasons why the EU needs to have a framework to enable cross-border healthcare.