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Value for money

How is health technology assessment affecting market access decisions in Europe?

A small tree with dollar bills as leavesMuch has been written about the role of health technology assessment (HTA) in Europe, as it assumes an increasingly significant role in market access decisions: not only does it contribute to evidence-based decision making; it also assists in identifying those products that offer the best value for money.

HTA is a multidisciplinary process that summarises information on the clinical, social, economic and ethical consequences related to the use of a health technology in a systematic, transparent, unbiased, and robust manner. Its aim is to inform the formulation of health policies in order to provide patients with equitable access to safe, effective, and high-quality health technologies that achieve best value for money, based on the best available evidence.

The term 'health technology' covers a wide range of interventions used in healthcare and health promotion, including methods for prevention, diagnosis, treatment and rehabilitation, such as vaccines, pharmaceuticals, medical devices, medical and surgical procedures. HTA should be grounded in robust and transparent methods and processes, and be based on clear, standardised guidelines that outline evidence and methodological requirements.

HTA is used to provide policy makers and other key decision makers with evidence-based information on the relative costs and benefits of available treatments, based on a systematic assessment process. This not only provides patients and prescribers with the information they require to make the best treatment choices, but also enables payers to make decisions centred on value by, for example, maximising health for a given healthcare budget.

The importance of evidence
Any review of HTA needs to recognise the interrelationship between HTA, as a form of policy research that examines the short- and long-term consequences of the application of a healthcare technology, and evidence-based medicine (EBM), defined as clinical evidence analysis for individual practice guidelines and policy decision making.

HTA thus comprises an evaluation of the clinical evidence for the technology being assessed, an estimation of the magnitude of the clinical outcomes (outcomes analysis), an evaluation of costs (economic analysis), a comparison of health and economic outcomes (cost-effectiveness analysis), and an evaluation of ethical factors. EBM itself has two components: evidence-based individual decision-making, which focuses on the evidence pertaining to an individual patient and his or her management, which helps balance evidence with clinical judgment.

The second component is evidence-based guidelines or policies, which describe the importance of founding population-based policies such as clinical guidelines, reimbursement, formulary decisions, and performance measures, on robust evidence.

The history of HTA in Europe
HTA has been recognised as essential in improving the quality of healthcare across the different health systems in Europe. The first national HTA agency was established in Sweden in 1987 to provide independent and unbiased scientific technology assessments of healthcare interventions for both healthcare decision makers and patients.

Since then, in most European Union (EU) countries, programmes for HTA have been established either through the foundation of new agencies or institutes, or the establishment of HTA units in universities or other existing governmental and non-governmental bodies to inform a variety of decisions, from determining pricing and reimbursement, to setting health service standards.

In 2004, the European Commission requested the establishment of a sustainable network for HTA in Europe (EUnetHTA) in order to assist the member states to plan, deliver and monitor health services effectively, based on the best available scientific evidence on the medical, social and economic implications of health technology. A call for proposals from EU member states by the High Level Group on Health services and Medical Care led to the EUnetHTA Project, which was established in 2006 and co-funded by the European Commission.

Over three years, the project established an effective European network to connect public HTA agencies, research institutions and health ministries to enable effective exchange of information and support policy decisions on the use of health technologies in member states at national or regional levels. Sixty-three HTA institutions and organisations joined EUnetHTA: an open network with extensive communication facilities.

Following the success of the project, the EUnetHTA Collaboration was established in 2009 to "contribute to the generation of HTAs to inform policy and healthcare decision making in European countries so that new health technologies can be adopted and obsolete technologies abandoned in a well-informed and robust manner, hence bringing about high-quality, safe, accessible, sustainable, ethical and efficient healthcare for citizens across Europe."

Recent reviews comparing the different healthcare systems and the place of HTA in each European country have shown that there is widespread variation in the utilisation and diffusion of technology among (and within) countries. Some HTA agencies are more transparent than others about their deliberations, and there can be differences in how priorities are set; the degree to which stakeholders are permitted to provide input; communicate results; interact with national reimbursement authorities; and use decision analytic models and cost-effectiveness analyses. This can lead to suboptimal use of technology and potential inequities in patient access.

Informing the decision-making process
It has been acknowledged that international collaboration among HTA agencies can support the development of more efficient assessment processes, and facilitate knowledge transfer across different healthcare systems. However, there has recently been considerable discussion, particularly in the US as a result of the funding of comparative effectiveness research through the American Recovery and Reinvestment Act, as to who is best placed to conduct the assessments. In the ongoing debate, concern has been expressed over whether the US Department of Health, and specifically the Agency for Healthcare Policy and Research (AHRQ), has the resource to provide a centralised process that addresses the needs of the disparate stakeholders within the complicated healthcare system.

Clearly, the manufacturer has access to the most information on their products, yet HTA agencies such as the National Institute for Health and Clinical Excellence (NICE) in the UK and the Institute for Quality and Efficiency in Healthcare (IQWiG) in Germany prefer an independent review of data, which raises the ethical issue of whether agencies should conduct research that informs policy decisions made by that same agency (much in the same way that NICE makes recommendations to the UK Department of Health).

Regulatory agencies, meanwhile, require manufacturers to demonstrate only that new therapies are both safe and efficacious in order to receive market authorisation, and only require proof of superiority over existing products when it is unethical to withhold treatment from patients in clinical trials. This means that many new drugs are approved on the basis of demonstrated superiority over placebo. Healthcare providers are thus facing increased pressure to adopt new health technologies as soon as they become available, to ensure rapid access to innovative treatments.

However, even after regulatory approval, there is considerable uncertainty about the long-term benefits, risks and relative value of the technology in a real-life setting. While early decisions to adopt technologies into routine care may prove clinically or economically appropriate, delaying access to new treatments could withhold potential benefits to patients. To reduce the risk of inappropriate decisions, high-quality, timely assessments with monitoring procedures are essential for gathering additional evidence on the value of emerging technologies that are expected to have a major impact on healthcare.

Now that the era of the blockbuster drug has passed, and with fewer clinical unmet needs, many drug companies are seeking to target therapeutic niches or small subpopulations, such that the focus on HTA has increased. Many of the products that have recently been licensed are for orphan diseases, with price premiums that provide an acceptable return on investment for the developer, but which cannot hope to fall within any traditional cost-effectiveness threshold.

The question is whether these products offer good value for money to the healthcare provider, the patient and society as a whole. Decision makers must therefore find a balance between providing high-quality, innovative care on the one hand and managing healthcare budgets and safeguarding the basic principles of equity, access and choice on the other.

Improving patient access to new technologies
It is now the responsibility of the manufacturer to demonstrate the benefits of its new product in terms of improved clinical benefit, safety, quality of life, and value over and above that achieved with the current best standard of care. A formalised way of demonstrating the relative value of products is comparative effectiveness research, a form of HTA in which new products are compared with current standard care, in terms of their clinical outcomes, in a real-world setting. Comparative effectiveness research also incorporates an appraisal of a therapy's safety and effectiveness, measured in clinical, economic, and humanistic dimensions. However, this approach does not explicitly include an evaluation of relative cost-effectiveness, but an implicit analysis of relative value based on safety, effectiveness and quality of life benefits.

Since manufacturers may have an introspective view of the value of their own products, it may be beneficial to use an independent and impartial agency that can accumulate the relevant evidence to support an HTA and compile the dossier in a suitable and acceptable form for submission to the appropriate agencies. This approach can also provide a critical appraisal of the 'value story' to aid internal decision making before submitting the final dossier.

Part of the mandate of an HTA is the need to conduct a systematic review of the evidence available for the product and its comparators. It is in the interests of the developer to conduct this research as early in the development process as possible, since a meta-analysis of the data forms the most robust evidence source and is a key driver of policy decisions.

This is especially important in the absence of comparative head to head clinical trials, which are not a mandatory requirement of the regulatory process. Indeed, new techniques, such as indirect comparisons, are now being used to compare new products with existing standards of care, and are implicit in comparative effectiveness research. Where possible, a similar process should also be conducted using data from prospective observational or registry studies to demonstrate the effectiveness of a product in a real-life setting.

HTA is probably the key driver of market access in Europe
In summary, since HTA is pivotal in assessing the relative value of new products as they reach the market, the process can form an effective barrier to successful market access. The judicious use of appropriate methodology to compile the evidence to support the clinical and economic value of a new product and present this in a cogent, transparent and rigorous format to inform guidance from HTA agencies will optimise the market access of new products across EU member states.

The Author
Ian Johnson is technical director at Complete Market Access
He can be contacted at Ian.Johnson@complete-ma.com

To comment on this article, email editor@pmlive.com

22nd March 2010

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