Perspective in Health Technology Assessment: reflections from The Royal Mile
HTA Policy Futures
I recently spent two days in Edinburgh, attending Health Technology Futures. A gathering of international thought leaders convened by Eric Low, Sean Tunis, Jean Mossman and Carole Longson. The aim was to consider the complexity of the UK and global HTA policy landscapes with a view to identifying opportunities to improve what HTA does and how it does it, primarily in the United Kingdom.
Amongst many proposed improvements for the future of HTA, was a suggestion that the perspectives of patients, carers and health care professionals should be given greater prominence in the evidence and greater weight in decision making. This is an important issue as the perspective adopted for an evaluation determines which outcomes and costs are included in the analyses to arrive at an estimate of whether the additional costs of a technology are justified by the additional outcomes. As I sat listening to the discussions, I realised three things: (1) there are many legitimate perspectives that HTA could consider than these three, (2) that these perspectives are in tension with one another in ways that are frequently not acknowledged, and (3) that there was some confusion regarding stakeholders and perspectives in the HTA process.
My aims in this post are to; (a) attempt a more or less comprehensive set of perspectives that could be adopted for evaluations that inform HTA, (b) provide a differentiation between Stakeholders in an HTA process and Perspectives for evaluations in HTA, and ( c) highlight how these perspectives can be in tension with each other, the issues the tensions create, and how that relates to the use of reference case measures of value in evaluations that adopt an population perspective..
It is worth noting at the outset that these observations are all made from the perspective that health technology assessment informs resource allocation decisions in solidarity-based health care systems. In these health systems opportunity cost is captured in valued outcomes foregone by other members of the covered population. In health systems that are financed by individual private insurance systems, opportunity cost is captured in alternative consumption and investments foregone by the individual/household. The shorthand way of thinking about the difference is that solidarity-based health care systems consider the supply side cost effectiveness threshold to value health care interventions, whilst individual private insurance funded systems consider the demand side willingness to pay.
Demand and supply side: perspectives or stakeholders
Health technology assessment can be thought of as a mechanism by which a health system operates in the market for health technologies. Manufacturers offer their technologies in the market and HTA is one of the mechanisms through which health systems say how much they are willing to buy at the price offered, or make counter offers about how much they would buy at a different (usually lower) price. This is HTA as Economics 101.
To understand how the health care system decides how much it will pay for a given technology we ned to think about the objectives of the health care system. There are many ways of thinking about the objectives of health care systems, but a model that has a degree of traction in policy discussions is the Quadruple Aim, developed by the Institute for Health Improvement. Health care systems are concerned with individual health, population health, efficient use of available health care resources and the ‘joy in work’ of the people who work in them. Perspectives for evaluation flow directly from the aims of the system.
The patient is clearly a relevant perspective given the first aim. Aiming to improve population health provides justification for considering family and/or carer health spillovers - which can be conveniently termed the household perspective. Aiming to improve the ‘joy in work’ of those providing care justifies the care providers perspective; covering individual clinicians, care teams and health care institutions. Promoting the efficient use of health care resources provides justification for adopting health care institution and health care system. It also provides an additional justification for the population health perspective, as greater efficiency implies more health being provided to the covered population from the available resources. In some systems the payer is distinct from the health care system, and to the extent that the payer’s aims are not coterminous with the health care system’s, analyses could adopt the payer perspective.
At this point, we have identified seven possible perspectives for evaluation. In solidarity-based health care systems, where the government will often determine the resources that are available to the system, and sometimes specify non-health objectives for the system, a government or public purse perspective can be appropriate.
Perspectives or Stakeholders
Careful readers will note that the manufacturers, who are obviously stakeholders in the HTA process, are not included in the list of perspectives for the HTA evaluation. Perspectives are driven by the objectives of the health care system and manufacturers have different objectives to the health care system. They are suppliers to the health care system, with the objective of maximising the return on their investment in developing their products, which is dependent upon the degree to which their product helps the health care system achieve their aims. As stakeholders, they have a legitimate interest in ensuring the methods and process of HTA enable procedurally just assessments of how well their products do this, but they do not set the aims of the health care system and therefore do not provide a perspective for the analyses. Other stakeholders that would not be considered as perspectives include patient advocacy groups, government agencies such as regulators and indeed the HTA agency.
Perspectives in tension
It may be tempting to think of different perspectives like Matryoshka (Russian) Dolls. The most granular perspective is encompassed by the next most granular; so, the patient’s perspective is a component of the household perspective, and the household perspective, and ultimately the household perspective is a component of the population perspective but sadly this is not true.
The volume of condition specific outcome measures demonstrates that health is conceived of differently by different groups, and the value attached to improvements in the same domain of health can differ between patient groups. By extension both the concept of health and the value attached to specific domains of health can be expected to be different from the household and the patient perspective. Ethics, specifically autonomy and non-malfeasance, requires that decisions should not attach a positive value to domains of health that the patient attaches negative value to, but these considerations do not require that the household attaches the same values to specific domains of health as the patient, nor do these principles stop the household from attaching positive or negative values to domains of health to which the patient attaches zero value. For example, a patient may not value the impact of a technology on the duration of carer responsibilities, while the household may. Without any consideration of resource utilisation, a health change may have a positive value at the patient level but a negative value at the household level.
The logic underlying this patient-household tension applies equally to the individual clinical professional, care team and health care institution. In addition, the differences in the scope and value of health effects, these three provider perspectives are likely to attach value to any changes in the process of care that are new technology will generate. Disruptions to established workflows can impact on the therapeutic relationship and other determinants of the joy of work, even if they do not impact on the cost or efficiency of the process of care. The value attached to these changes may be positive or negative, but the pertinent fact is that their consideration can lead to the value of technologies diverging significantly from the value for both patients and households.
Adopting a health system perspective, which is generally considered the default perspective for HTA processes, brings a specific version of the value of health tension to the fore. The health system is there for every member of the covered population. It knows the domains health and the value of those domains differ across that population. At the same time, the principles of procedural justice - especially transparency, consistency and rationality - require that it applies a more-or-less standard approach to evaluating technologies. The principle is that whatever is valued for the patient who benefits should also be valued in considering the impact on population health. Again, ethics requires that it does not attach a positive value to effects that the patient would attach a negative value to, but it is not required to consider every effect that the patient attaches a value to, and it can consider effects that the patient does not value. This is the foundational justification for the use of a reference case measure of the value of health effect of a technology, such as the EQ-5D-5L with the UK general population value set.
Highly granular perspectives are not costless
For many innovative health technologies, the most efficient pathway to market access passes through system level health technology assessment processes. To understand what the alternative looks like, consider market access in Canada. With a national regulator (Health Canada), and a national HTA agency (Canada Drug Agency). However, health system drug funding decisions are made by the ten provinces. The average time from Canadian regulatory approval to public reimbursement was reported as 678 days by Innovative Medicines Canada (IMC). The equivalent statistic for the UK, according to IMC was 154 days. Whilst these data are from 2018, the structural difference persists.
An advantage of health care system level HTA process is the difference it can make in time to market access. This is valuable to both patients and manufacturers. This type of tension is not confined to the patient versus health system perspectives (most versus least granular). Processes that reflect health care providers aims, whether individual, team or institutions, we would expect to have a greater influence on clinical behaviour, which is what is required for patient access and company revenue. There are tensions, arguably irresolvable tensions between having a transparent, consistent and appropriate characterisation of value and the desire for more granular perspectives. This challenge is further complicated by the under-explored but likely complex relationships between perspectives and the speed and scope of patient access.