A few days spent at the Health Technology Assessment International (HTAi) 2019 conference in Cologne this week gave a sense of how the practice of health technology assessment (HTA) needs to develop beyond 2020.
The issues and debates at HTAi this year showed that the simple HTA processes some of us grew up with earlier in our careers are now in need of a refresh.
Changes in society and health care systems around the world that mean that HTA needs to evolve if it is to be fit for purpose for the next phase of the 21st century.
One theme coming out of the conference was that the days of just working out whether a medicine was 'cost-effective', perhaps through the magic of a cost/QALY equation, and leaving it at that are well and truly gone.
The range of additional factors already being incorporated into the decision of whether to fund a medicine, device or diagnostic in a health system has led to the development of new methods such as multi-criteria decision analysis (MCDA).
(HTA experts, perhaps not always noted for their simplicity in communication, of course now had to graduate to a four letter acronym to explain a new process).
When I look at it, at one level MCDA simply operationalises previously informal factors that were often part of reimbursement and purchasing decisions such as budget impact, ethical factors, supply chain integrity and quality issues.
They just weren't given the same formal recognition even though they could sometimes ultimately determine whether or not patients received treatment in the end.
The difference now is that these MCDA processes are being developed to formally incorporate these wider issues into payer decision making.
Ringing in the changes
Putting greater emphasis on the budget impact on payers of a treatment has come to the fore as the widespread adoption of universal health coverage, together with a range of new treatments and diagnostics coming through the pipeline, has forced evaluators to consider the financial impact on payers' budgets in a much more overt way than in the past.
Perhaps this has been adopted in part because the HTA evaluators themselves were stung by payers rejecting or complaining about funding new therapies even when they were clearly cost-effective.
The range of new technologies and therapies now being developed and available to patients – such as new CAR-T medicines, cell therapies, new antibiotics and treatments for rare diseases – are all testing existing HTA methods and systems developed at a time when simpler chemical, small molecule medicines were the norm.
Can an HTA model that was developed at a time when blockbuster cholesterol reducing medicines or blood pressure pills were all the rage cope with personalised cell therapies to cure rare diseases based on genetic testing and just-in-time production individually tailored for each patient?
Many of these new therapies work very well, are often cures and the benefit provided to the patient and society is delivered up front after a short course of treatment.
The wave of lifetime cures and the resulting disruptive economics coming from these, first forewarned by the arrival of cures for hepatitis C a few years ago, is now crashing into staid HTA-based evaluation systems used to dealing with incremental improvements.
It is a challenge for evaluators and decision makers determining on behalf of society the value of curing someone's life-threatening disease up front.
Digital technologies are providing challenges and opportunities that we are still only just starting to understand and leverage, but they challenge the traditional methods and frameworks of HTA. The explosion in digital health apps in the last few years is a case in point.
And even social media, often looked at with derision by hard-nosed HTA experts used to dealing with clinical trials, got some positive feedback at HTAi.
There were stories told of trends in medicine adverse events being picked up by Twitter trends weeks before official adverse event data and social media helping to understand that multiple sclerosis patients were switching medicines because they changed doctors.
Whereas once real world data was seen as a poor cousin to clinical trial data, increasingly now with electronic health records, devices and apps it is becoming a real force in HTA.
This is particularly so when clinical trials are increasingly becoming more expensive and difficult to run in the context of targeted individualised therapies in rare diseases where patient populations are quite small.
The blurring of the lines between medicines, devices and diagnostics is presenting all sorts of challenges for HTA systems, particularly those that continue to assess these components separately in separate systems.
With so many co-dependent technologies appearing in healthcare now, it begs the question how such separated processes can continue to operate with a straight face.
Patient and industry engagement is another key issue. How to engage patients and the broader community in HTA decisions is becoming increasingly important when funding decisions and ethical choices need to be made about which therapies to use over others.
While not a new issue, the increasing difficulty of some of the choices together with greater empowerment means that patients and the community should be engaged in the process.
And working out the best way to get industry constructively and appropriately engaged in HTA processes is an important ongoing issue.
As more developing and emerging countries look to introduce universal health coverage, HTA is being looked at by many as a tool to help ensure the most efficient spend of healthcare expenditure.
Many sessions at HTAi focussed on the needs of emerging markets and how HTA might be relevant to them.
Where once upon a time it was thought that HTA was beyond these countries, for many this is increasingly now not the case.
And, finally, the internationalisation of HTA is continuing in tandem with the globalisation of health policy.
There is more discussion today of international cooperation between countries on HTA methods, tools and even joint assessments, perhaps led by experiments in Europe.
The debate about whether HTA needs to be context-specific at the national level is now moving to a discussion of which ‘bits’ of HTA can be done collaboratively at the international level and which bits are still dependent on local country-level factors.
Whether this is a good thing or not remains to be seen and doesn't come without some issues.
As the Polish vice minister for health explained, EU efforts to trial joint HTA evaluations across European countries have raised concerns in Eastern Europe because those countries have different levels of income and health spending compared to their counterparts in Western Europe.
What’s old is new again
At one level, many of these issues are not new to HTA.
Things like budget constraints, increasing patient engagement, electronic technologies, the development of new therapies, real world data, emerging markets and globalisation have been debated in sessions at various HTA conferences for a long time.
Some of us have been in and around these conversations for years.
The difference today is that there is a growing realisation and political momentum now to ensure that these issues are appropriately dealt with going forward.
Are we ready for it?
In short, no.
But don’t take my word for it.
That’s what the head of the OECD’s health division more or less said in her keynote address to the HTAi conference, arguing that health systems are slow to adapt to change.
Perhaps the final word should go to the senior official from the European Medicines Agency – pointedly not from an HTA agency – who challenged a roomful of HTA wonks with this:
“If HTA wants to remain relevant it needs to change radically. The scope will have to change. The idea that a national HTA agency wants to stay as a stand-alone agency is ludicrous.”
The feeling and theme at many sessions at the conference was that HTA and health systems need to get better at adapting to change.
Time for a reboot, perhaps.