Health technology assessment (HTA) is pivotal in the intricate landscape of today’s contemporary healthcare system. By meticulously and transparently consolidating data regarding the application of innovative technologies, HTA contributes to safeguarding patient safety, enhancing effectiveness, and maximizing overall value of healthcare services. This evidence-driven methodology supports informed decision-making and the formulation of health policies, ultimately resulting in the best possible outcomes. The Health Technology Assessment Regulation (HTAR) established by the European Union (EU) represents a significant advancement by creating a cohesive HTA framework across the EU.[1]
Enacted by the European Commission, this pioneer legislation will provide a single unified framework for the evaluation of medical novelties across the member states, which ensures their safety and accessibility in order to bring about breakthroughs in patient care. The EU HTAR became effective in December 2021 and shall fully come into function from January 2025. It is not just a mere update in the procedures of HTA, but a strategic shift towards an effective healthcare system in the EU. Conceived to unify fragmented HTAs, it streamlines processes to eliminate redundancies and expedite the delivery of cutting-edge health technologies to patients. By fostering innovation and ensuring faster access to treatments, the EU HTAR establishes a common framework for collaboration among national HTA bodies in the EU, enhancing market access for healthcare technologies across Europe.[2-4] The journey of the EU HTAR began in 2018, culminating in its adoption in December 2021. Among the key features of the EU HTAR are the Joint Clinical Assessments (JCA), Joint Scientific Consultations (JSC), horizon scanning activities, and voluntary cooperation in areas outside the scope of mandatory cooperation.[5]
The JCA is a joint initiative of the European Medicines Agency (EMA) with national HTA agencies, aiming at assessing clinical benefit and evaluating the cost-effectiveness of drugs with a view to their pricing and reimbursement at the national level. The JSC, on the other hand, streamlines logistics to maximize gains by optimizing opportunities for mutual understanding between regulators and HTAs. This promotes optimal and robust evidence generation fit-for-purpose for both regulators and HTA bodies, ultimately resulting in benefits for public health by enabling access to medicines that are effective for European patients.[6-7]
The long-awaited draft implementing act on procedural rules for JCA of medicinal products was published in March 2024 confirming the timelines and procedures previously laid out by the European Commission. The implementing regulation specifies that to allow sufficient time for a high-quality JCA, the clinical assessment should be initiated at the same time as the marketing authorization procedure, specifically upon the EMA’s confirmation of a valid submission of an application for marketing authorization. Additionally, the final JCA report shall also be endorsed by the HTA Coordination Group (HTACG) no later than 30 days after the date of adoption of the European Commission decision granting marketing authorization for the medicinal product under evaluation. If changes occur during the regulatory process, such as changes in the therapeutic indication compared to the initially submitted one or a variation to the terms of existing marketing authorization, timelines for the JCA may be significantly delayed, depending on the extent of the impact of such changes.[8]
Implementing the EU HTAR presents challenges, such as harmonizing methodologies and ensuring the active participation of all stakeholders in the JCA and JSC processes. For pharmaceutical and medical device makers, the regulation implies a pivot towards a more centralized European procedure for clinical evidence evaluation, impacting how they approach data generation and submission for the HTA process. There could also be some important challenges brought to the pharmaceutical manufacturers by the virtue of the new EU legislation. One major concern is the potentially onerous data requirements, which include multiple PICOs criteria (Population, Intervention, Comparator, and Outcome) for off-label treatments. One of the most important challenges that should be expected is the management of timelines because the final PICOs will only be known about 90 days prior to the JCA submission deadline, which typically leaves very little time to complete the evidence-generation process through meticulous and resource-intensive procedures such as systematic literature reviews, indirect treatment comparisons, and cost-effectiveness analysis using complex pharmacoeconomic models. This means that early engagement with local affiliates and key external stakeholders (payers, healthcare providers, patient organizations) will be crucial to understanding differences in clinical practice between Member States that will impact the scope of work.[9]
Concerns have been raised about the impact on smaller companies and the overall attractiveness of Europe for investment in healthcare innovation. The workload and limited engagement with industry during the development of the JCA process present a paradox: while the original goal was to reduce the overall workload, it might have the opposite effect, particularly as the process will be needed for all new oncology and Advanced therapy medicinal products (ATMPs).[8]
In conclusion, the new EU HTAR signals a transformative era in EU regulatory oversight, driving towards a more integrated and efficient approach to the HTA process. Manufacturers must proactively address the procedural and evidence-generation challenges ahead. While the new framework presents initial hurdles, it promises streamlined evaluations, reduced redundancy, and accelerated access to innovative treatments across Europe. Successful navigation of EU HTAR implementation will require active engagement from all stakeholders, including regulatory bodies, healthcare providers, manufacturers, and patients.
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References:
- Wild C, Stricka M, Patera N. Guidance for the development of a National HTA-strategy. Health Policy and Technology. 2017 Sep 1;6(3):339-47.
- European Commission. Regulation on Health Technology Assessment. Retrieved from Regulation on Health Technology Assessment – European Commission (europa.eu).
- European Commission. Questions and Answers: Adoption of Regulation on Health Technology Assessment. Retrieved from QANDA_21_6773_EN.pdf (europa.eu).
- Desmet T, Brijs M, Vanderdonck F, Tops S, Simoens S, Huys I. Implementing the EU HTA regulation: Insights from semi-structured interviews on patient expectations, Belgian and European institutional perspectives, and industry outlooks. Frontiers in Pharmacology. 2024 Apr 10;15:1369508.
- The European Parliament and the Council of the European Union. Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on Health Technology Assessment and amending Directive 2011/24/EU2021 22.12.2021. Available online: https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32021R2282&from=EN.
- EU Joint Clinical Assessments: Theory vs Practice. Retrieved from PharmaBoardroom – EU Joint Clinical Assessments: Theory vs Practice. Accessed on 4 Jun, 2024.
- Klaar S. Joint Scientific Consultations – Advice for EU Health Technology Assessment. NDA Whitepaper. 2024
- European Commission. The EU regulation on health technology assessment: what’s in it and why it matters? https://health.ec.europa.eu/document/download/855247c3-cda9-4645-99f7-7098836f314c_en?filename=ev_20240409_co01_en.pdf.
- Open Health. European Health Technology Assessment Regulation: Implications for the industry and the 5 key areas for successful implementation. EU HTAR – What are its implications for the industry + infographic (hubspotusercontent-na1.net).