Adaptive HTA: striking the right balance between rapid assessments and local health impact
What is an HTA and how is it used?
Health technology assessment (HTA) is a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to inform decision-making in order to promote an equitable, efficient, and high-quality health system.
An HTA agency is often an independent institution and may be under the umbrella of the Ministry of Health, the National Insurance Agency or other public institutions and can be conducted by those entities, academic centers or other expert organizations.
The outcome of such assessments are used in different ways by countries to inform whether a new health technology should be made available and funded by a particular country’s public health system.
While HTAs are primarily used to evaluate medicines today, leveraging mainly Randomized Controlled Trials (RCTs) and Real-World Evidence (RWE) where appropriate, they can conceptually assess all types of technologies and care approaches in health care, including prevention programs, advanced medical technologies, medicines, vaccines and other forms of care.
Countries use HTAs to inform the allocation of healthcare resources to address the needs of their patient populations. But HTAs can have serious drawbacks when they aren’t tailored to the local context or/and healthcare system design. If they are not implemented or managed effectively in the context of a society’s goals and preferences, HTA can risk exacerbating healthcare inequity, misguided cost-containment and reduced patient access to medicines.
Conducting HTAs to any reasonably high standard requires significant amounts of time, data, expertise, resources, and institutional support, and these assessments have to date primarily been employed in high- and upper-middle-income countries. However, in recent years, a number of lower-middle-income countries (LMICs) with emerging healthcare infrastructures have looked to institute their own more expedited, tailored to their needs and resources available, form of HTA, an adaptive health technology assessment or aHTA. aHTAs attempt to provide the same sort of guidance as traditional HTAs, but in far less time and by drawing on considerably less data and limited resources.
The promise and challenges of aHTAs
Many LMICs face the dual challenge of managing healthcare resources while meeting a growing demand for care, often with limited local data sources. In that context, a structured approach to conducting aHTA analysis in a more streamlined fashion than traditional HTAs may be appealing. Proponents of aHTA suggest that they “may leverage or adapt available international data, economic evaluations, models and/or decisions from the published literature or established HTA agencies to expedite policy decisions while adequately accounting for concerns of transferability and uncertainty.”
However, these approaches are still in their infancy, and there is only very limited peer-reviewed literature evaluating their accuracy and effectiveness. What little research does exist is far from conclusive. A recent study in the BMJ Global Health journal, for instance, through the application of selected aHTA approaches, adapted findings of globally published economic evaluation to Indian context and concluded that it is critical that decision-making reflects local circumstances, cost structures and clinical practice, among other factors. Direct transposition of HTAs across different health care ecosystems is of limited value for local reimbursement decision-making.
For example, if a public-sector health system declines to cover a new cancer therapy, a heart medication, or another health technology based on information outside the specific country context, patients could be denied access to their best chance at survival because the decision was based on the data and values of another country. A well-developed aHTA framework – grounded in local epidemiological, cost, and ideally, clinical data – is needed to support decision-making in allocating resources and making priority treatments available to local patients.
Furthermore, in the absence of a well-established local decision-making framework, the shorter timeframe and less robust engagement systems typical of aHTAs would also provide fewer opportunities for stakeholders to be consulted during the appraisal process and provide input to the recommendation of an assessment before it results in a substantive policy decision.
In order to increase aHTA relevance, access to relevant datasets (clinical and potentially also economic) and the availability of local experts in related scientific fields (such as statisticians, health economists) are of the utmost importance. An aHTA process should consider local clinical and epidemiological data as well as country specific treatment pathways in its evaluations. It is also critical to ensure that patients are engaged in the process of conducting aHTAs, to enable those to reflect on their experiences and local health needs and preferences.
For countries choosing to adopt aHTA, three principles for aHTA design and implementation can support more appropriate, local decision-making.
Principles guiding the use of aHTA
Principle 1: Keep patient outcomes front-and-center
The primary aim of any HTA, including aHTA, should always be the improvement of patient- relevant outcomes. This patient-centered mindset should inform the concept of “value” at the heart of any HTA. For instance, it’s often tempting to analyze the value of a specific intervention by weighing its short-term financial cost against its immediate effects on patient health. Yet, such a narrow measure of value ignores the wider implications of a given intervention over the long term.
An approach that captures the full value of medical technologies and is patient-focused demands aHTAs that are sensitive to considerations such as health-related quality of life, the wider and long-term benefits of medicines and vaccines, the broader societal and caregiver benefit, as well as the long-term benefits of investing in innovative medicines on evolving standard of care, among other clinical, economic and humanistic value.
Principle 2: Transparency and stakeholder input
One of the best ways to improve the reliability and soundness of an aHTA is to build transparency into the process. Many components of the assessment – including the methods, assumptions, and design choices underlying an aHTA – should be made available for input from stakeholders such as clinicians, and patient representatives. Sensitive data and information, of course, would remain confidential.
It is crucial for countries to establish clear, consistently enforced procedures that allow experts and other stakeholders to raise legitimate concerns and to appeal conclusions derived from an aHTA. For example, health authorities can hold public deliberation sessions where participants can provide insight into the experiences of people living with a certain health condition. Additionally, stakeholders can provide input into the selection/prioritization of medicines to undergo aHTA, which can be important for countries with constrained resources or HTA capabilities. This can help improve the odds that any shortcomings in an aHTA are identified and corrected.
Principle 3: aHTA should be conducted in the local context
While there is an appetite for aHTA to help with decision-making in resource-limited settings, it is important to recognize that if all relevant health system resources are not in place, an aHTA is considerably more prone to extrapolations and assumptions than a traditional HTA.
For example, a scoping review of aHTA methods published last year in the journal Value in Health, highlights just one of the many ways in which aHTAs can lack accuracy if they solely refer to other systems without considering local health care structures. As the authors note, “common reference countries for aHTA practitioners include the United Kingdom, Australia, and Canada, but these are not representative health systems.”
It is crucial, then, that aHTAs consider closely both the quality and quantity of available local data before undertaking the assessment.
Looking ahead
LMICs are seeking effective evidence-based strategies when allocating and prioritizing health resources across therapies. An adaptive Health Technology Assessment (aHTA) approach may be attractive due to shorter time frames, fewer needed resources and data compared to traditional HTA. However, to ensure better informed local decision-making, aHTA should appropriately leverage local data, account for local health priorities and needs, focus on what’s meaningful to patients and their outcomes and implement cross country best practices, including a transparent process and early stakeholder engagement.
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