Health technology assessment (HTA) is the systematic analysis of the medical, social, economic, and ethical issues of a new health technology. Its aim is to provide evidence-based conclusions that support the decision-making process of health policymakers.
Over the past 15 years, HTA has developed on the European level. From EUR-ASSESS in 1994 through to the development of EUnetHTA and its accompanying network up to the recent elaboration of the EU HTA Regulation, this article shows milestones and developments.
Health technology assessment (HTA) is the systematic evaluation of properties, effects, and/or impacts of a health care technology. It covers medical, social, ethical, and economic dimensions and informs decision-making in health policy. HTA aims to identify the best medical practice and help decision makers invest in safe, effective technologies that are not harmful to patients or society at large.
Until recently, there were no structures in place that could provide a common framework for HTA across the EU. This resulted in a number of issues, including duplicative and repetitive work at the Member State level, delayed patient access to novel therapies, and increased costs for both companies and societies.
Therefore, the European Commission was determined to change this situation. It aimed to reinvent EU-wide HTA cooperation, with a particular focus on mandatory joint clinical assessments.
The aim was to streamline the process and share resources, while arriving at a single consensus view on assessments that would be binding across the entire EU. It hoped that this would help to avoid duplicative work by ensuring predictability in the processes.
In its earliest years, EUnetHTA gathered input from different stakeholder institutions, such as trade associations and research organisations. The results of this work were compiled in a set of reports, called the JA1 to JA3 series.
These reports, along with the corresponding methodology and web-based tools, formed the basis for EUnetHTA’s activities. These reports also contributed to the development of a legal and regulatory framework for HTA in the EU.
The resulting EU-HTA Regulation is now in force, and has the potential to secure timely patient access to new medicines by eliminating duplicative work at the national level. However, the regulation has been revised and rewritten several times over the past three years.
Health Technology Assessment (HTA) aims to inform policy-making in the health area by providing evidence on benefits and efficacy, clinical and technical safety, and cost effectiveness of new technologies. It is a multidisciplinary, comprehensive and systematic process that involves a wide range of stakeholders, including regulatory authorities, medical experts, patients and patient organisations, and other health care professionals.
In Europe, there are a number of government-appointed bodies responsible for HTA. They vary in their structure, function, remit, and approach. Some are dedicated to synthesising and critically reviewing evidence submissions while others are responsible for the appraisal function, which may include a more thorough review of the overall health impact of a new technology.
The European network for Health Technology Assessment was established in 2006 to link public national and regional HTA agencies, research institutions and ministries of health, enabling an effective exchange of information and support to policy decisions by the Member States. EUnetHTA consists of sixty-four partner organisations from 27 European Union and EEA countries plus Norway.
Since the launch of EUnetHTA JA1, which ran from October 2012 to September 2015, the network has developed a set of methods, tools, and proposals for integrating HTA into policy-making processes within the Union. In addition, the network has produced twenty-seven assessments which are being used in national processes across Europe.
In 2018 the Commission presented a proposal for a Regulation on Health Technology Assessment and amending Directive 2011/24/EU, which was quickly adopted by the European Parliament in 2018. It includes four key elements: 1) mandatory joint clinical assessments; 2) a new coordination group to coordinate these assessments; 3) continuing voluntary cooperation in areas not covered by the mandatory assessments in which individual EU countries will continue to be responsible (non-clinical aspects of health technologies, decisions on pricing and reimbursement); and 4) the establishment of a European Network of Health Technology Assessments (EUnetHTA) to ensure a constant exchange of information and knowledge between HTA institutions in Europe, to increase synergies between Member States, to streamline HTA methodologies, to increase transparency and evidence-based decision-making, and to ensure business predictability.
The European network for Health Technology Assessment (EUnetHTA) was established in 2006 and comprises over eighty organizations from thirty European countries. EUnetHTA’s main goals are to promote more effective use of resources, increase HTA input to decision making, strengthen the link between HTA and policy making, and support countries with limited experience in HTA.
The network has developed various tools for capacity building, such as a handbook on HTA capacity building and a toolkit for adapting existing HTA reports to other settings. Moreover, it has promoted a network-wide information service on new and emerging technologies.
In the fifth project phase, EUnetHTA aimed to improve the efficiency and standardization of joint work by setting up a quality management system (QMS). A dedicated work package was established to coordinate all QMS-related activities.
Several working groups defined processes and methods to support the assessment teams in producing high-quality assessment reports. The resulting concepts were integrated into a new web platform and made available to the members through a Companion Guide.
However, a number of needs expressed by the assessment teams could not be addressed and required further development and implementation in the future. Consequently, the project’s final evaluation report suggests that the development of a solid permanent QMS with structures and mechanisms for quality improvement and evaluation is essential to support future European HTA collaboration.
In addition to providing a framework for a sustainable and enduring EU network for HTA, the JA3 project also contributed to the capacity building of assessment teams in various European countries. In particular, a series of Standard Operating Procedures (SOPs) were developed in order to define the structure of assessment teams and their tasks. The SOPs include guidelines for the identification of assessors, internal and external review processes, and the collaboration with patients/patient representatives, healthcare professionals, and manufacturers/marketing authorization holders. The SOPs also provide a checklist for quality control, email templates, and links to relevant methodological guidelines and tools.
Health technology assessment (HTA) is a process that involves an analysis of the medical, social and economic issues related to a new health technology. It seeks to provide health policy makers with the best available information on a new drug or technology. It aims to avoid duplication of work between Member States and to ensure that new technologies are only added to the health package once they have been proven to be effective and cost-effective.
The HTA process begins with a submission by industry for a new drug or technology, and is followed by a clinical assessment of the evidence. This is carried out by a multi-disciplinary committee, which can include representatives from doctors and other healthcare professionals. It also takes into account wider input from stakeholders such as patients and payers in the context of the healthcare system.
Ultimately, this assessment results in a recommendation or conclusion on whether the new drug or technology should be included in the healthcare package for use in the country. This decision is then adapted to the national reimbursement system, and the technology may be used or removed depending on how it fits into the health package.
This process is often accompanied by a European Public Assessment Report (EPAR). It provides health technology assessment bodies (HTABs) with the best available scientific evidence on the medical, social and economic aspects of a new drug or technology.
The EPAR is designed to meet the requirements of both HTABs and regulators, and should therefore be optimised for both. This study aimed to identify opportunities for this by examining the requirements of both HTABs and EMA in the area of clinical evidence, through a literature review and expert interviews with regulators and HTABs.
High quality standards in HTA play an important role in ensuring that health technologies, such as medicines, are developed and used for their full potential benefit. This is essential for patients, the European healthcare system and the wider public. Poor evaluation standards, in contrast, can result in the uptake of technologies that offer little or no added value.
The European network for Health Technology Assessment (EUnetHTA) is a group of government-appointed organizations from European Union Member States, the European Economic Area and accession countries as well as a large number of relevant regional agencies and non-for-profit organizations that produce or contribute to HTA in Europe. It is designed to facilitate the exchange of information and knowledge between the different HTA bodies in the EU and to enhance the production of reliable, transferable and useful information on the short- and long-term effectiveness of health technologies.
Currently, there are three Joint Actions aiming at increasing synergies between HTA bodies in the EU and to develop common assessment methodologies and produce joint clinical assessments and full HTA reports. Several other projects are also underway to address specific issues such as the integration of real world data in HTA, joint scientific consultations and cost-effectiveness analysis.
However, the scope of HTA in Europe remains vast and it is not possible to solve all the underlying challenges by voluntary cooperation within a single project. Therefore, we conducted a semiquantitative survey with representatives of HTA bodies to identify the main issues and challenges that need to be addressed in order to successfully implement the European regulation on HTA.
We identified 19 domains related to four key areas processes, uncertainty, comparator choice and endpoint selection that urgently need to be addressed in order to ensure the success of the HTA regulation. These domains included: integrating evidence from clinical as well as real world data sources; generation of comparative evidence in small patient numbers; and including specificities of new health technologies for which some data may not be readily available.