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New actions underway seek to find a consensus on the role of HTAs in the decision-making chain, including with clinical trials.
Everyone agrees that there should be more health technology assessment (HTA). That’s the easy part. The real challenge is defining what sort of HTA, and how it should overlap with other elements of the decision-making chain -including with clinical trials. And that is where Europe is at present trying to look ahead and seek some sort of consensus. But it’s a tough-ask anywhere in the world-and in Europe it is, as usual, even tougher, because it means getting agreement among dozens of countries that each has its own views. In the EU there are more than 50 national and regional HTA bodies, all embedded in different institutional settings.
That is why the European Commission has just published what it calls an inception impact assessment on strengthening EU cooperation on HTA. Cooperation is perceived as an effective way of maximizing the use of resources-and making efficiency gains that can palliate the remorseless rise in spending occasioned by Europe’s ageing population, the increased incidence of chronic disease and the cost of complex new technologies.
The scope of the reflection is broad: drugs, devices and medical, surgical and radiation procedures, and also measures for disease prevention, diagnosis or treatment. And it takes account of both approaches to HTA-the rapid relative effectiveness assessments (REA) that cover clinical domains and measure the medical/therapeutic added value of a technology, and the full assessments that also include cost-effectiveness, budget impact, ethical, legal considerations, and impact on patients or the organization of healthcare systems.
In exploring possibilities for closer cooperation, the EU is between the rock of respecting diverse national approaches and the hard place of tackling budgetary constraints on healthcare. There is no doubt about the budgetary constraints. But there are plenty of questions over the different approaches. In the last 20 years, most member states have introduced their own HTA systems at national or regional level, and they differ widely in procedures and methodologies-in everything from data requirements for the submission dossier to the choice of comparator, and the way in which the added therapeutic value is expressed. While clinical trial data is a core requirement for all agencies, industry indicates that differences exist in the type of trials requested. Safety data, quality of life data and economic analyses are commonly requested, but not by all HTA bodies. Real-world evidence and additional studies may also be required.
Diverging methodologies lead to diverging outcomes, so informal attempts to get them to work more closely together have been underway for a decade. And since 2013, health ministries or national HTA authorities have met regularly-although on a voluntary and non-binding basis-in the framework of an HTA network. The network has been supported by a series of short-term programs to boost scientific and technical cooperation. These so-called joint actions have focused on developing common methodologies, on piloting joint REA and full HTA reports and on developing and maintaining common IT tools.
Outputs of the joint work so far include some standardized framework for HTA assessments, related methodologies and tools covering clinical and non-clinical elements, literature reviews and early dialogues and scientific advice on development planning and study design. There has also been some move toward supporting member states in providing objective, reliable, timely, transparent, comparable and transferable information, and easing information exchange. The cooperation led to the production of about 20 joint reports, including REA and full HTA, and some 20 early dialogues between technology developers and HTA bodies, which help industry to design the studies in terms of regulatory and HTA requirements.
The latest of these joint actions has just started, and will run until 2020. It involves 75 partners from 29 countries, and it plans to generate 80 joint reports and 35 early dialogues, as well as increased uptake of the joint work at national level. It will also review current guidelines, models, methodologies and tools, and develop new ones, to promote continued HTA collaboration at the EU level beyond the end of the project in 2020.
However, despite the developments in joint work over recent years, national authorities still carry out their own national assessments, because the scheme is entirely voluntary, and national uptake remains at the discretion of each member state. So they can-and do-choose to ignore all the joint work, and the intended cooperation is further impeded by legal, organizational and even linguistic barriers. As a result, uptake has been the exception rather than the rule.
When the costs of HTA are taken into account, the consequent duplication doesn’t come cheap: each national HTA assessment is estimated to cost around $30,000 to national bodies and $100,000 to the industry. And these figures are only for REAs (they need to be more than doubled when full HTAs are undertaken), and they do not include the expenditures of collecting additional data to meet the differing requirements of national HTA bodies.
Over the last two years, EU ministers have repeatedly acknowledged the importance of HTA and called for continued support for cooperation, with reinforced attention to its use in the context of personalized medicine. The European Parliament has echoed the calls, and the HTA network itself has urged support for joint work over the long-term.
The Commission, too, has pointed to the “very high” fragmentation of HTA systems in the EU, and identified the lack of “binding mechanisms for mutual recognition of joint assessments” as one of the major shortcomings of the current HTA system. Patients, health professionals and public health organizations have added their voices to the calls for strengthened HTA coordination at the EU level, to avoid unnecessary duplications of efforts and promote evidence based health policies.
So, too, has the pharmaceutical industry, which seeks consistency of the data requirements and clinical assessments, although with some reservations over full HTA at the EU level. Health insurers and other payers also want to be involved in HTA cooperation, particularly so that requirements on clinical evidence and cost-effectiveness of technologies can be aligned. And at the international level, the World Health Organization has urged its members to develop and apply HTA and to strengthen inter-country collaboration to obtain efficiencies.
The Commission is now seeking views on whether it should continue to fund the efforts to promote cooperation and convergence after the current program runs out in 2020. Without EU funding beyond 2020, the current cooperation will not continue or will be very limited, it believes, and the achievements of the cooperation to date-on common tools, methodologies and joint assessments-are at risk. But the Commission suggests that longer-term funding rather than a further series of five-year programs might be more effective in ensuring voluntary cooperation.
Another option under consideration is moving beyond purely voluntary cooperation, and introducing a legal framework for HTA cooperation. This could provide a mechanism to ensure that the efforts of national bodies in collecting data were compatible, shared and used, and allow for the production of joint REA reports. In a still greater shift toward legally binding cooperation, member states might not only jointly produce REAs, but could be obliged to use them. And an even greater level of mandatory engagement could see joint production of full HTA reports-covering economic, ethical, legal and organizational issues, too.
Don’t hold your breath in the hope that this will provide all the answers either. The document frankly admits that “no comprehensive analysis of the impact of HTA on resources has been concluded thus far.” To add to the confusion, the Commission states boldly: “This inception impact assessment is provided for information purposes only and can be subject to change. It does not prejudge the final decision of the Commission on whether this initiative will be pursued or on its final content and structure.” The only thing that can clearly be seen in the HTA mist at present is the mist.
Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium