Who Knows What HTA Really Is?

Given the crucial role that health technology assessment (HTA) has acquired in the increasingly tense debate on drug prices and the related issues of the value of medicines and access to care, it comes as something of a surprise to learn that there is still no agreement on just what HTA is. But that is what was starkly revealed by an announcement in mid-August: a collaboration among the most respected organizations in the field has just kicked off to reflect on a definition.

The European Network for Health Technology Assessment  (EUnetHTA) will be working alongside the World Health Organization and ISPOR, the Professional Society for Health Economics and Outcomes Research. They will be sharing the work with regional HTA networks in Asia and America, and two global HTA organizations, the International Network of Agencies for Health Technology Assessment (INAHTA), and Health Technology Assessment International (HTAi).

As the announcement admits: "There are many different variations of the HTA definition used today." With masterly understatement, it goes on to make what would seem to be a rather unnecessary point that "it is in the interest of the international HTA community to create a consolidated definition that is clear and concise and reflects the current and future role of HTA to support health system sustainability." 

Of course. And not just in the interest of the HTA community, either, it might have added. Because agreeing on a definition is clearly in the interest of everyone and every organization whose options are influenced by HTA opinions, decisions and developments – and that includes drug developers, policymakers, healthcare authorities, payers, physicians, and ultimately, patients.

"Work is now underway to review existing definitions and develop the updated version", says the announcement, which encourages members of the participating organizations to make contact if they have "ideas or suggestions to offer".

This artless invitation makes a sharp contrast with the animated discussions now reaching a crescendo on just how to bring the many different HTA agencies in Europe to work more closely together. Over recent months, the European Union's lawmakers in the European Parliament and the Council of Ministers have been energetically debating draft legislation that was proposed by the EU's officials in the European Commission.

And it has to be said that the debate is not going well. The proposal, published in early 2019, ran rapidly into trouble with national politicians and MEPs who saw it as a power-grab by the European Commission, and an intrusion into the well-established sovereign right of each EU country to make its own decisions about what healthcare it chooses to deliver. Patient and civil society groups and most political groups in the European Parliament gave it qualified support, conditional on making the system immune to regulatory capture or undue influence by pharmaceutical companies. But the most virulent opposition came from heavyweight HTA agencies themselves, who feared that their own high standards might be compromised by the mandatory collaboration the Commission envisages. "A centralized HTA assessment would not do justice to the very heterogeneous national health systems," the boss of Germany's G-BA agency said as soon as the proposal was released.

After that, the controversy escalated, with the German parliament's health committee leading a broad front of national opponents concerned that the proposal intrudes into national prerogatives. At a public session of the EU Council of Health Ministers in June, clear opposition was voiced by many countries. Germany flatly rejected the mandatory elements as an interference with national sovereignty over health matters and on pricing and reimbursement. France said it could not accept mandatory use of the envisaged joint assessments or any prohibition on national clinical assessments. The Netherlands said "the far-reaching Commission proposal raises concerns", particularly with its mandatory elements. And Denmark, Finland, Italy, Portugal, Spain, Sweden and the UK followed a similar line. 

Other concerns raised included distaste for industry involvement, questions over which products should be jointly assessed and how the assessments would be kept up to date, and diverse national patterns in the use of comparators and treatments. France and Germany followed up their criticisms with a position paper demanding that "Substantial amendments must be made". A five-page document rejects many of the mandatory aspects of the proposal, and aims to limit the European Commission's role in the new system.  In the European Parliament too, criticisms have also rained down on the proposals, often on similar issues of clashes between national sovereignty and joint assessments, but also on questions of feasibility, methodology and procedures. By October, the Parliament should reach its verdict – but the Council is likely to take much longer.

But the essential difficulty of different systems remains, right at the heart of the European debate. Oncologists in Germany want to know how subgroups for HTA are defined. The Spanish health minister says it is "time for a new solution" to the challenge of coping with HTA across 17 autonomous regions in Spain, which means "some treatments are denied to patients on the basis of their cost on the basis of a decision made sometimes by individual hospitals". The European drug industry remains firm in its view that without some mandatory elements, there is no prospect of making HTA more effective – and easier for drug firms to manage. At present, harmonization of national approaches to HTA is still patchy in respect of new technology and new trial designs, according to Adam Parnaby, Director of Market Access Policy at Celgene. "Industry supports any measure that will speed access to new medicines, and has consistently pointed to diverse HTA decisions across Europe as a challenge. Improving uptake of joint HTA reports could remove another layer of delay", said an official at Europe's main drug industry association, EFPIA

Meanwhile, the vigor of what has become an HTA industry is evident from the proliferation of its activities. Just a cursory glance at the current panorama reveals that HTAi is currently on a recruitment drive "in accordance with its strategic plan". At Health Technology Wales, in the UK, "an exciting opportunity has arisen for an experienced and enthusiastic senior health economist ." Switzerland's Federal Office of Public Health is now boosting the health technology assessment department it set up only last year, "to increase the quality of care and reduce costs".

And Europe remains central to many of the international activities too. The HTAi 2019 meeting will be held in Cologne, Germany next June 15 - with the support of two of Germany's key HTA institutions, and with an agenda fixed on future development of HTA worldwide in 2020 and beyond in the face of "digitalization, disruptive innovations, and the questioning of established evidence requirements." HTAi's global policy forum took place in Barcelona in January this year, and its 2017 annual meeting in Rome. Since last year the European Medicines Agency is pioneering a new system of consultations in parallel with EUnetHTA, to allow medicine developers to obtain feedback from regulators and HTA bodies on their evidence-generation plans to support decision-making on marketing authorization and reimbursement of new medicines at the same time. And that is a further step forward from its earlier pilot on regulatory-HTA parallel advice and ten years of cooperation with individual HTA bodies.

EUnetHTA itself, now in the final phase of its existence as a focus for voluntary cooperation among HTA agencies in Europe – in expectation of agreement on a new EU system – is continuing its low-level technical work: over recent weeks it has produced a joint assessment of glucose monitoring systems in patients with diabetes mellitus and a project plan for assessing stool DNA testing for early detection of colorectal cancer.

The evident question that underlies all these efforts, these controversies, these discussions, however, is "who really knows what HTA is supposed to be doing?". If there is no international consensus on a definition, then how is an essentially international industry to have any confidence that a dependable and aligned system will emerge, in Europe or elsewhere. How can a sensible debate take place without agreement on what is under discussion? And if that is not a realistic prospect, what is the point of all this expense of time and energy in discussing an undefined activity?