HTA bodies: what are they and why do they matter?
In this summary article, we explore health technology assessment (HTA) bodies – taking a look at what they are, how they consider real-world evidence, which ones you need to know about and more.
What are HTA bodies?
As the name suggests, HTA bodies are organizations that perform health technology assessments, meaning systematic assessment of the structure and effects of a health technology, which include medicines, medical devices or any technology designed to tackle health issues. This assessment often includes both direct and indirect consequences of the technology and is an integral part of the decision-making process regarding what technologies should be made publicly available.
It must be noted, however, that the scope of assessment of HTA bodies varies across both organization and country, from little additional evaluation post regulatory approval to full assessment of the clinical, economic, contextual and budget impact of the new technology. HTA procedures can also vary in their attitudes to transparency and stakeholder engagement.
Despite this, one important area of HTA assessment is cost-effectiveness analysis, where the HTA organization considers the economic value of the prospective health gains to the population brought about by the new health technology. This ties in closely with issues of reimbursement, whereby HTA bodies provide recommendations regarding which health technologies should be financed or reimbursed by the relevant healthcare system in the area of interest.
As such, the HTA provides a framework to establish whether or not the prices of health technologies are reflective of the advantages they offer to patients. This is important, as setting health technology prices that are set too low may lower the motivation of manufacturers to invest in research and development, but pricing that is set too high leads to inefficient levels of innovation and limited patient access to drugs. Indeed, payers will utilize value assessments provided for by HTA bodies to determine whether the price of novel healthcare interventions are justified or rather just overpriced.
Collaboration between HTA bodies and other healthcare organizations
Collaboration between HTA bodies and regulators is essential for allowing patients to access new health technologies and consequently serve public health. This cooperation is important in reducing developmental resources by increasing efficiency of medical development programs and also facilitates decision-making processes by widely sharing assessment information [1].
Across many European countries, Canada, Australia and the US, formal ties of cooperation have been founded between HTA and regulatory bodies [1]. In such countries, methods including adaptive pathways to licensing, parallel submission and post-authorization data review have been trialed as ways of enhancing effective collaboration [1].
However, achieving cooperation can be difficult. Indeed, a recent study has indicated that several barriers can affect effective cooperation between HTA bodies and regulators, including legal, organizational and resource-related factors. The authors suggested that these limitations must be addressed, in addition to investigating the long-term benefits of current collaborative methods, to enhance effective cooperation between HTA and regulatory bodies.
Why do we need HTA bodies and where does real-world evidence come into play?
HTA bodies generate information about the qualities of healthcare interventions and their impact on patients. This information can be utilized to improve a variety of standards and guidelines concerning the quality of healthcare services provided to patients, including clinical practice guidelines, manufacturing standards, clinical laboratory standards, adverse event reporting, facility design standards and other factors impacting healthcare standards.
One distinctive characteristic of HTAs is their frequent inclusion of comparative effectiveness research, which is the generation and compilation of evidence that compares the benefits and harms of differing technologies to prevent, diagnose, treat and monitor diseases in real-world settings in order to advance the delivery of healthcare [2]. This is intended to solidify the evidence base that is utilized for assisting patients, clinicians, healthcare managers, policymakers and others to make better healthcare decisions for the public.
Examples of comparative effectiveness research include directly assessing alternative interventions, as opposed to one intervention against a placebo effect, measuring the effectiveness of an intervention in practice within a real-world setting, as opposed to solely within trials, and analyzing certain subgroups of the population to understand more about different responses across patient types.
Another cornerstone of many HTAs is a focus on patient-centered outcomes research. This research focuses on a patient’s experience of their disease or illness, with an emphasis on the biopsychosocial perspective, considering the patient as a whole person. Additionally, thought is given to the sharing of power and responsibility within the patient’s healthcare experience, in addition to the patient–doctor relationship during the treatment process.
As such, patient-centered outcomes research may examine patient outcomes including: survival, functional status, quality of life, quality of death, symptoms, pain, nausea, psychosocial well-being, health utility and patient satisfaction [2]. Patient-reported outcomes, which are self-reported by patients themselves, are one way of obtaining data pertaining to patient-centered outcomes. Overall, patient-centered medical approaches can have a favorable effect on the safety, effectiveness and other related healthcare outcomes.
Want to know more about the latest HTA updates? Why not read these articles:
- RWE and HTA – a snapshot of recent developments with Sreeram Ramagopalan: April–May 2022
- Considerations when utilizing quantitative bias analysis for unmeasured confounding in HTA: an interview with Seamus Kent
- RWE and HTA – a snapshot of recent developments with Sreeram Ramagopalan: February–March 2022
What HTA bodies should I know about?
The UK
In the UK, the Health Technology Assessment Programme, which is part of the National Institute for Health Research, provides an output of sophisticated research relating to the effectiveness, costs and impacts of health technologies for those involved in the national health service (NHS) [3].
Such research often directly affects decision-making bodies including National Institute for Health and Clinical Excellence and the National Screening Committee. The Health Technology Assessment Programme also constitutes a core component of the ‘National Knowledge Service’, which aims to increase the quality of clinical practice in the NHS [3].
Europe
Since 2008, the European Medicines Agency (EMA) has been working closely with HTA organizations. The collaboration has involved regional and national HTA bodies providing recommendations on which health technologies should be financed or reimbursed by the healthcare system within a particular European Union country or region [4].
For the period 2021–2023, a joint work plan has been put together to allow EMA and European Network for Health Technology Assessment 21 to focus on several priority areas of healthcare practice, including joint scientific consultation for robust evidence generation, exchange of information on assessments of medicines and generation of patient-relevant data and information to support decision-making, amongst others [4].
This is in preparation for January 2025, when it has been decided that European cooperation between medicine regulators and HTA bodies will be governed by the Regulation on Health Technology Assessment (EU) 2021/228.
The US
The US is interesting in that, in contrast to many developed countries, it does not have a nationally implemented HTA body to assess novel health technologies and their pricing. Such lack of a centralized process is within the context of a general preference for market-oriented solutions and a decentralized insurance system within the US [5]. Additionally, given disparities in covered populations across payers, moving towards a national HTA body approach could be difficult in the US [5].
However, several key agencies in the US pursue health technology evaluation, including the Agency for Healthcare Research and Quality, which conducts HTAs through various processes, including the Evidence-based Practice Center program. Further, Medicaid agencies started the Drug Effectiveness Review Project in 2003, which had the aim of creating comparative effectiveness reviews, which are utilized in several states as the key evidence base when determining the coverage of new health technologies.
In more recent times, the Patient-Centered Outcomes Research Institute (PCORI) was established in 2010, which funds comparative effectiveness research with the goal of considering patient perspectives throughout the research process. This has the aim of bringing patient outcomes research to the organizations who directly conduct health technology assessments. In 2019, the US Congress renewed funding for PCORI for 10 further years.
Lastly, the Institute for Clinical and Economic Review, founded in 2006, has more recently emerged as a key organization evaluating the clinical and financial value of prescription drugs, medical tests and other health technologies. This non-profit, independent organization’s profile was raised in 2015, following their launch of the Emerging Therapy Assessment and Pricing program, and now consists of three appraisal committees that each conduct two to five healthcare evaluations annually.
