HTA research to support evidence-based healthcare

Proposals should develop new or improved methodological approaches and frameworks, and foster methodological consensus-building, to address all of the following areas:

1. Specific types or groups of health technologies: Help adapt existing HTA frameworks to reflect the specificities of particular types of health technologies⁹⁷ for which HTA is currently less established but gaining importance. Particular consideration should be given to the increasing role of combinations of technologies, co-dependent technologies (e.g. companion diagnostics) and personalised medicine[[Personalised medicine refers to a medical model using characterisation of individuals' phenotypes and genotypes (e.g. molecular profiling, medical imaging, lifestyle data) for tailoring the right therapeutic strategy for the right person at the right time, and/or to determine the predisposition to disease and/or to deliver timely and targeted prevention.]] in healthcare.
2. Selected therapeutic areas: The focus should be on therapeutic/disease areas where new products frequently face challenges in HTA, but a high unmet medical need persists. Methodological work and consensus-building should be aimed at key issues for relative effectiveness assessment, such as patient-relevant health outcomes, appropriate outcome measures, clinically relevant patient subgroups, and the current evidence-based standard of care. With regard to patient-relevant health outcomes, patient preferences and patient-reported outcome measures (PROMs) should be taken into account. Particular consideration should be given to strengthening synergies between HTA and clinical guideline development, with a view to more consistent reporting on the clinical/therapeutic value of health technologies.
3. Use of real-world data: Methodological work should address current concerns and uncertainties around the quality and suitability of real-world data (e.g. from diseases-specific registries and routine healthcare databases) for relative effectiveness assessment in HTA. It should also contribute to broader efforts for improving the collection, comparability and analysis of real-world data across Europe[[Synergies should be sought with related initiatives, e.g. EUnetHTA Joint Action 3 (Work Package 5), the EMA initiative for patient registries, and JRC activities on registries.]].
4. Implementation: In all of the above areas, part of the efforts should be directed at implementation of methodological work, using e.g. case studies or pilots. Involvement of HTA bodies in all of the above areas should ensure that the needs of HTA practitioners are addressed and uptake in HTA practice is facilitated.

The proposed consortium should bring together partners with relevant expertise from e.g. academia, HTA bodies, regulators, centres of expertise for clinical research and care[[For rare diseases, involvement of European Reference Networks (ERNs, see https://ec.europa.eu/health/ern/policy_en) should be considered.]], scientific and medical learned societies, and organisations involved in developing evidence-based clinical guidelines and systematic reviews in healthcare. The consortium should also seek input from relevant stakeholders such as patients, technology developers, healthcare providers and payers. The composition of the consortium should ensure a broad geographical representation of European countries. Gender equality aspects should be taken into account in carrying out the relevant research objectives and activities.

Proposals should complement or build on existing work, including results of EU-funded projects in the field of HTA⁷⁶. The consortium should closely liaise with EUnetHTA[[EUnetHTA Joint Action 3 is a European network of national/regional HTA bodies under the EU Third Health Programme.]] to avoid duplication, build on EUnetHTA existing work and create synergies with ongoing EUnetHTA activities and other relevant EU cooperation efforts.

The Commission considers that a proposal requesting an EU contribution between EUR 5 to 10 million would allow this specific challenge to be addressed appropriately. Nonetheless, this does not preclude submission and selection of proposals requesting other amounts. Should more than one proposal be selected, applicants are expected to collaborate and this should be indicated in the proposal.

Health technology assessment (HTA) is gaining increasing importance across Europe and the world as a tool to support evidence-based decision-making in healthcare. HTA aims to assess the added clinical/therapeutic value of a new health technology compared to the existing standard of care, under the usual circumstances of healthcare practice (relative effectiveness assessment). HTA can also assess additional aspects of added value (e.g. economic or organisational), depending on the specific context in which it is used.

European collaboration on HTA has increased in recent years, notably in the context of EU-funded projects[[EUnetHTA Joint Actions 1-3, AdHopHTA, ADVANCE-HTA, INTEGRATE-HTA, MedtecHTA, GetReal, ADAPT-SMART]], including work towards shared methodologies and the joint production of relative effective assessments. Despite recent progress, a number of methodological challenges remain in the field of HTA. This includes a need for methodologies that address the specificities of particular types of health technologies[[Health technologies are broadly defined to include e.g. pharmaceuticals, medical devices, in-vitro diagnostics, medical procedures, screening tests, vaccination programmes, eHealth, and other measures used for health promotion, disease prevention, diagnosis or treatment.]] and their increasingly combined use in healthcare. Better methodological agreement is also needed in particular therapeutic areas, including on important aspects of relative effectiveness assessment such as health outcome measures. Moreover, there is a need to resolve methodological issues related to the use of ""real-world"" data, to inform the assessment of effectiveness under the usual circumstances of healthcare practice.

Thus the challenge is to address these complex issues and needs, by bringing together methodological expertise from across the various relevant research communities. Such a collaborative effort should draw on the best available expertise and latest evidence, in order to develop methodological approaches that are scientifically sound, fit for purpose and fit for the future.

• New or improved methodological approaches, frameworks and consensus-building mechanisms to address the above-mentioned challenges for particular types and groups of technologies, therapeutic areas and real-world data use in HTA.
• Strengthened methodological quality of HTA by input of specialist expertise from the broader scientific, clinical research and evidence-based healthcare community.
• Improved methodological agreement between HTA researchers across Europe, increasing the impact of HTA on evidence generation, clinical guideline development and evidence-based healthcare.
• Contribute to strengthening EU cooperation on HTA, building on ongoing and planned efforts.