|WP7||POTENTIAL IMPLICATIONS: LESSONS LEARNT, POLICY RECOMMENDATIONS AND ADVANCES IN HTA|
Type of activity
Core research objectives
Summary of conclusions
In terms of the horizontal component of WP7, a detailed account of objectives, methods deployed, findings, policy implications, recommendations to stakeholders (regional, national, supra-national governments; international organisations; patients; health care professionals; procurers of medical technologies), contribution to the debate on efficiency in health care resource allocation, and application in the real world were produced.
While the horizontal aspect outlined the results of each of the previous work packages, the vertical component underscored a number of dimensions related to HTA, the resulting policy implications and the broader socio-economic context in which Advance-HTA operated, notably: (a) the theoretical and conceptual considerations addressed by Advance-HTA; (b) the importance of appropriate service user involvement in HTA evaluations; (c) the orphan medicine treatments and the value of HTA in industrial policy formation; and (d) the implications for global health and health care development.
1. Theoretical and conceptual considerations addressed by Advance-HTA
Some of Advance-HTA’s results in general show that differences in the evaluation criteria underlying how products such as innovative medicines are assessed can (over and above issues such as variations in national per capita GDPs) consistently and significantly influence whether or not a treatment is judged cost-effective in one country as opposed to another. This does not mean that the work undertaken by health economists and others involved in HTA leads to arbitrary decisions. But it does mean that if more or less value is ascribed to, say, the scientific originality of a therapeutic innovation (and so, implicitly, the unknowable but potentially important long term returns to communities from publicly or privately funded biomedical research investment) relative to the immediately demonstrable benefits generated for individual patients, then differing purchasing and treatment supply decisions will result. Similar points apply to the use or non-use of ‘adjusting’ factors such as disease severity and rarity or ‘end of life’ care applications.
From a pan-European policy perspective this raises social equity concerns and a number of wider economic and industrial policy questions, alongside more immediate health sector resource allocation issues. One important challenge for the future relates to ensuring a better balance between achieving short-term equity in health care areas where there is already evidence available relating to the promotion of allocative efficiency and the inevitably more uncertain task of defending public interests in long-term innovation, and the eventual achievement of fundamentally improved technologies for preventing and treating conditions such as cancers and neurodegenerative disorders. These are different goals, which in some circumstances conflict. Similar questions and possible consequences arise in fields ranging from how affordability criteria are set in relation to ‘cost per QALY’ and allied utility measures in different nations and/or contrasting spheres of social and economic activity in a single country, through to – not least in the rare disease and cancer care contexts – whether or not rationing choices should be made on the basis of Benthamite utilitarianism as opposed to Rawlsian concepts of social justice. The latter are more likely to ascribe premium values to treatments that benefit minority populations than the former.
Other important Advance-HTA findings range from new observations showing that patients tend to ascribe higher relative values to physical pain and mental distress reduction than observers who have not experienced relevant disease states directly, through to revealing the limited extent to which HTA techniques have to date been applied to the evaluation, pricing and purchasing of medical devices as compared to that of medicines and vaccines. Its existence stems from the relative lack of centrally accessible data on medical devices use and the outcomes attributable to them, and the fact that their employment is often intimately associated with other forms of hospital spending. The special focus of HTA on drug evaluations is also linked to the (in some ways questionable) Western pharmacological tradition of evaluating medicines as discrete molecular entities, as opposed to items that act in combination with not only additional drug treatments but many other medical, surgical, nursing, psychological and wider social inputs.
Advance-HTA’s findings indicate that further investment needs to be made in developing Health Technology Assessment tools for use in areas outside the narrowly defined pharmaceutical sector, in order to further increase the productivity of health care as a whole. This stretches beyond conventional technologies and includes diverse health technologies such as combination therapies and companion diagnostics, among others. They may also need to be adapted to accommodate the reality that drugs do not normally act alone to achieve the best possible outcomes, albeit the discovery of optimum combinations and administration strategies can – as experience in spheres such as the treatment of child leukaemias has shown – be a long drawn-out process.
2. The importance of appropriate service user involvement in HTA evaluations
From a public policy perspective some of the strongest findings of the Advance-HTA research discussed here stem from observations made in relation to preference elicitation, and in the rare disease treatment context. These include the discovery that people who have experienced conditions such as physical pain or depression (and perhaps other psychiatric states such as, for example, severe anxiety disorders) are likely to value their alleviation more highly than those without such direct experiences. At the same time reductions made in the degree of QoL lost recorded via instruments like the EQ-5D-5L in the context of problems that can more readily be accommodated, such as being unable to conduct self-care tasks autonomously, are likely to be less highly valued by patients with relevant experiences than they are by ‘naïve’ observers.
Although the likely impact of such variations on quality of life estimations should again not be over-stated, such variations are potentially significant with regard to the ways that HTA research might best be conducted. This is particularly so when they are combined with findings that demonstrate that in areas such as rare disease research it is often the case that patients and their family members have deeper and more accurate insight into their conditions than many clinicians, let alone academic or directly State employed economists.
Given this, the key policy related conclusions drawn here are not only that there are opportunities for technical improvements in the ways in which the quality of life related impacts of health care interventions are measured, but also that closer patient involvement in the governance and direction of HTA and other forms of health care research and evaluation remains an important priority. At worst, patient involvement can be little more than a form of ‘box ticking’. But done well it is likely to add significantly to the quality of HTA findings, and the ways in which they are understood and implemented.
3. Orphan medicinal treatments and the value of HTA in industrial policy formation
Advance-HTA’s findings with regard to rare disease treatment and the affordability and access to orphan medicines highlight the fact that if evaluations include criteria which relate to their low prevalence and variables such as the severity of distress and other exceptional burdens that inheritable monogenic disorders can impose on not only individuals but entire families and, occasionally, on specific ethnic/racial minorities, then they are more likely to be judged affordable than if more general assessment methodologies or processes are used.
In linked areas like some areas of cancer care (and probably in future some forms of dementia treatment) patient access is also likely to be affected by the existence or otherwise of additional HTA evaluation modifying factors. These could relate to not only the value of demonstrating social solidarity in situations where only relatively small numbers of people are involved, but also the social fact that at given points in history some disease may be popularly seen as deserving greater investment than others. Such prioritisation judgement may be driven by subjectively perceived fears or objectively based beliefs that some sorts of innovation are more likely to prove viable than others, despite having equal potential ‘worth’.
Two main sets of policy related ideas are worth discussing here. First, the European Union, like nations such as the US and Japan, has in recent decades recognised the important health equity and industrially funded research challenges associated with developing better treatments for rare indications. Special incentives have been put in place to promote relevant activities. The EU has also encouraged Member States to develop rare disease strategies, which they have done at varying speeds and with differing degrees of excellence. But there is in the short term at least little point in bringing new treatments for rare disorders to market if they are not subsequently made available to those who could benefit from them.
Some commentators may take this to imply that in the European context more effort should now be made to facilitate the timely production of orphan medicine assessments produced to an agreed Union-wide standard and implemented in a manner consistent with the spirit of the existing European legislation on rare diseases. Whether or not this will be achievable is uncertain. But Advance-HTA clearly identifies this as a question that will demand increasing attention during 2016 and beyond.
Second, and following on from the above, present-day orphan drug supply issues can also be seen as indicative of the limitations of approaches to HTA that fail to take into realistic account industrial policy issues. These range from, for example, current and future EU-wide balance of trade and employment concerns to the impacts that major variations in the levels of volume demand for innovative products such as new medicines have on the economics of their development and their sustainable supply at what are perceived to be affordable unit prices.
Some experts may argue that such factors cannot properly be taken into account when calculating ICERs and should not be considered when interpreting their product pricing and purchasing implications. But if this is the case it severely limits the practical utility of HTA findings. There is a strong policy case for arguing that even if ‘cost per marginal QALY’ figures should continue to be calculated in much the same way as they are at present, affordability thresholds could and should be systematically adjusted to permit better quality future decision making. If this is agreed, then more effort needs to be put into the ‘science and art’ of determining affordability thresholds across Europe, and more widely.
Concerns like these can be linked back to the ‘utilitarianism versus Rawlsian social justice’ debate noted earlier. Benthamite thinking may, rightly or wrongly, taken to imply that minority interests should be sacrificed if greater overall welfare gains can be made by pursuing alternative social goals. But John Rawls’ theory of justice indicates that in some circumstances the (apparent) sacrifice of majority welfare optimisation interests in favour of meeting the needs of the least advantaged in a community can offer a more desirable path towards achieving what is generally accepted as being the fairest possible society-wide distribution of goods and the welfare derived from them.
Whether or not there is a genuinely irreconcilable divide at the heart of this apparent dilemma is disputable. But for the purposes of this analysis it can be said that the Advance-HTA programme’s outcomes point to the dangers of failing to understand the significant of such philosophical issues in the context of the ‘cost per QALY’ calculations currently central to the cost-effectiveness assessments undertaken by European HTA agencies. At worst, ‘simplistic’ utilitarianism could lead to an undermining of social solidarity and the intent of European policies aimed at ends like encouraging rare disease research.
4. Implications for global health and health care development
Advance-HTA also revealed marked variations in the numbers of qualified health economists and other HTA practitioners in emergent as opposed to more mature economies, and in the ways in which HTA based findings are used to determine policies and decision making. The world-wide picture is to a degree complicated by the position of the United States. There both industrial and allied research investment arguments, together with policy questions relating to the acceptability of using quality of life related calculations to determine care access, have had a significant influence on the evolution of HTA and attendant disciplines like comparative efficacy studies. In broad policy terms the emergence of health economics and within it the field HTA can be taken to be related to the processes of global demographic and epidemiological transition. These seem in their later stages to be normally attended by increased public (including compulsory insurance supported) spending on UHC provision. As health outlays increase, so do concerns for equity, efficiency and service excellence. So too do investments in aspects of quality management and value-for-money improvement. However, it should not be uncritically assumed that these are always appropriate or in themselves cost-effective. It is debatable, for instance, as to whether or not individual emerging economies should currently be seeking to increase their indigenous HTA capacity, as opposed to drawing on evaluative work conducted elsewhere and concentrating attention on further building their clinical workforces and generic health sector management capacities. Nevertheless, they will either way benefit from becoming more able to use health care resources to optimal effect. Advance-HTA work undertaken in the Americas identified an increasing tendency for local courts to require public health care systems to supply medicines to individuals seeking better health care. In other parts of the world judicial actions appear to have been aimed more at improving treatment access by limiting intellectual property rights. It would not be appropriate here to make judgments on the desirability or otherwise of such interventions. However, their existence underlines the importance of being able to price and supply products such as innovative medical devices and medicinal products in ways that are consistent with individual human rights and collective interests in achieving better care standards and ongoing global efforts to improve the effectiveness of treatments.
The Advance-HTA project’s findings offer European decision makers a wide range of useful insights into how in future cost effectiveness evaluations of not only medicines but products such as medical devices (and perhaps in time interventions such as surgical operations) could be better conducted. They also raise important questions as to the extent to which in contexts like promoting enhanced outcomes in areas of cancer care and the treatment of rare monogenic diseases the criteria used for evaluating the ‘worth’ of therapeutic innovations might be standardised across the European Union, and how regionally or globally consistent approaches to adjusting for factors like GDP and disease prevalence variations could be instituted.
Technically, Advance-HTA’s results raise questions about how the affordability thresholds used in assessments can best be set, and the degree to which the extended use of MCDA techniques would materially enhance the validity and acceptability of currently established evaluation methods. They also highlight as yet unresolved uncertainties about the extent to which national policy decisions or individual treatment choices could ever be determined on the basis of HTA findings alone.
Principal Investigator: Dr Julio Lopez Bastida