Edward Mc Gettrick and Sharon Gorman of Pfizer, writing in the January 2023 edition of DIA’s Global Forum magazine, examine the scope and impact of the European Union’s new Regulation on Health Technology Assessment.
In December 2021, the European Commission (EC) adopted the Regulation on Health Technology Assessment (EU HTA Regulation; No. 2021/2282). Its main objectives were to strengthen the quality of HTAs across the EU, ensure efficient use of resources, avoid duplication of industry and national HTA body efforts, and ensure long-term sustainability of EU HTA cooperation. The EU HTA Regulation formally entered into force on 11 January 2022 and will apply to Oncologic & Advanced Therapy Medicinal Products (ATMP) products after a three-year implementation period (i.e., 12 January 2025) in the first instance, followed by Orphan Medicinal Products (OMPs) in 2028, and all other Centrally Approved Medicines from 2030 onward.
In practice, sponsors developing a new molecular entity (NME) in either oncology or an ATMP who expect to submit a marketing authorization application to EMA after 12 January 2025 will need to prepare their NME and subsequent line extensions under an EU-Joint Clinical Assessment (JCA). JCAs are relative clinical and safety effectiveness assessments that will be conducted by designated national HTA bodies at the EU level. Sponsors will be required to submit their dossier for JCA, which will then serve as the basis for national value assessments and price negotiations.
In the intervening period (2022-2024), implementation of the EU HTA Regulation will be mainly driven by EUnetHTA21, a consortium of 13 HTA bodies contracted by the EC to support the development of assessment methodologies and procedural guidelines for use by industry, agency assessors, patient representatives, healthcare professionals, and other experts. The EC, which maintains oversight and ownership, will then put forward the EUnetHTA21 deliverables (methods, procedures, templates, etc.), for further refinement/adoption by the Coordination Group or by implementing and delegated acts, before they constitute the basis for the more granular rules, procedures, and methodologies of any future system.
It is imperative that implementation of the EU HTA Regulation does not impede patient access to innovative medicines across all EU Member States. This paper reflects on elements of the HTA Regulation critical to successful implementation and highlights some potential pitfalls.
Key Elements of the EU HTA Regulation
The key elements of the EU HTA Regulation are:
- Phased introduction of JCAs.
- Single “core” submission of clinical data for JCA, including mandatory reuse at national level.
- Single JCA report as input into national HTA processes.
- Introduction of Joint Scientific Advice opportunities (i.e., Joint Scientific Consultations [JSC] with numerous national HTA bodies with/without EMA).
Impact of Joint Clinical Assessments
One of the most important elements of the EU HTA Regulation is the introduction of EU-level JCA. These joint assessments by experts from national HTA bodies at the EU level are the scientific compilation and description of a comparative analysis of the available clinical evidence of a health technology (e.g., a medicine) in comparison with one or more health technologies or procedures. In effect, they are relative clinical and safety effectiveness assessments. The parameters under JCA scope will include the patient population, the intervention, relevant comparators, and health outcomes (PICO).
JCA will impact all Centrally Authorised Products and certain high-risk medical devices. The assessment will then serve as the basis for national value assessments and price negotiations. Despite starting after the EMA regulatory assessment, JCA will run in parallel to, and continue after, the regulatory assessment has concluded. The JCA dossier will be submitted to HTA bodies approximately 45 days prior to CHMP opinion, and the JCA report will be published 30 days after the EC decision (i.e., day 307 of the regulatory procedure). Based on these timelines and the current strain upon the EU system, forward planning to ensure that the required national HTA body resources are available to undertake each JCA will be critical to ensuring timely patient access.
JCA are envisaged to generate high-quality scientific reports that will support Member States in making more evidence-based and timely decisions on patient access to new medicines. Industry hopes that JCA will bring greater clarity and predictability concerning HTA clinical evidence requirements such as further alignment and harmonization of European-focused PICO, particularly regarding populations and comparators.