Mandatory EU HTAR Implementation: Part 1

Are You Ready for Mandatory EU Health Technology Assessment Regulation (HTAR) Implementation Beginning January 2025?

Pharmaceutical companies need to be prepared for the new European Health Technology Assessment Regulation (HTAR), which will take effect in January 2025. The aim of the HTAR is to accelerate access to new drugs and reduce unnecessary duplication of work. To support this goal, the HTAR will support European Union (EU) member states (MSs) to jointly determine clinical effectiveness at a pan-EU level. It is crucial for companies to establish new internal timelines and processes to bring drugs, and eventually also medical devices, to the European market. Importantly, companies’ internal resources, skills, and key roles need to be well defined and aligned with this new way of working.

Health Technology Assessment

Health Technology Assessment (HTA) is a multidisciplinary field that determines the health impact of new or repurposed drugs or new technology based on information concerning medical, economic, social, and ethical issues related to the use of the technology and its alternatives. This evidence-based information supports policymakers in formulating health policies that are safe, effective, patient-focused, and cost-effective. It is also used by national health authorities to help make decisions on which technology should be reimbursed at the national level.

Prior to the HTAR, each MS has its own HTA procedures, criteria, and timelines. This fragmentation led to inefficiencies, divergent decisions, and inequitable access to health technologies.

Aims of the New HTA Regulation

The new HTAR aims to foster innovation and ensure faster patient access to new treatments by enabling a common framework, and to encourage collaboration among national HTA bodies to support a joint perspective on the clinical aspects of the development of medical products, in vitro diagnostics (IVDs), and high-risk medical devices. As such, a streamlined HTA process is expected to enhance market access for healthcare technologies throughout Europe.

The new HTAR enables cooperation between the European Commission (EC) and the EU Member States by:

• Accelerating the process for patient access
• Ensuring European consistency in assessment of new technologies
• Avoiding duplication

Key Elements of the New HTAR Process

The HTAR covers Joint Clinical Assessments (JCA), Joint Scientific Consultations (JSC), horizon scanning activities, and voluntary cooperation in areas outside the scope of mandatory cooperation. Table 1, below, summarizes the key elements of the HTAR process.

Joint clinical assessments will cover safety and clinical effectiveness and outline diverging scientific opinions but will not give recommendations on added value or reimbursement, which remains the scope of the MS. By having the safety and clinical effectiveness already covered on a European level, this will help national HTA decision-making.

A regular HTA evaluates the evidence on new health technologies including medicines, procedures, or medical devices, usually on a national level. It is a multidisciplinary field that addresses the health impacts of technology, information about medical, economic, social, and ethical issues related to the use of health technology, and alternative technologies.

The new HTAR will facilitate the assessment of the epidemiological, safety, and clinical impact of a new technology relative to its alternatives, based on the elements of the population, intervention, comparator(s), clinical outcomes (PICO) framework at an EU level. The economic impact related to the use of health technology, as well as alternative technologies, will remain the responsibility of the Member States.

The PICO components will be determined by the EU Member States before being consolidated by the EU Coordination Group (CG). After the PICO components have been determined by the EU MSs, pharmaceutical companies will then prepare and submit a JCA taking the PICO components into consideration. The first part of the JCA dossier will be prepared by the Sponsor and other parts will be compiled by the CG designated by the MSs.

Timelines for the JCA Preparation

A JCA dossier will need to be submitted to the European Commission (EC) at least 45 days prior to the date of the expected opinion of the Committee for Medicinal Products for Human Use (CHMP) and will be published 30 days following EU marketing authorization (Figure 1). The report will be made publicly available, including an outline of diverging scientific opinions. However, it will not include recommendations on added value or reimbursement, which remains the responsibility of the MS.

HTAR Implementation Timeline

The implementation of the HTAR will be a staggered process. As of 2025, pharmaceutical companies with health technologies in the EU within the setting of cancer and/or as an ATMP will be required to prepare and submit a JCA dossier. Next in line will be orphan drugs, planned for 2028, and as of 2030, all drugs, in vitro diagnostics (IVDs), and high-risk medical devices will be required to prepare and submit a JCA dossier. Vaccines, biosimilars, and generics are not in scope of the HTAR.

The Importance of Joint Scientific Consultations (JSCs)

Pharmaceutical companies will also have an opportunity to engage in JSCs to obtain guidance on the clinical evidence requirements of a JCA. A JSC also takes effect in January 2025; however, unlike the JCA, it is not required. However, although it is not mandated, a JSC is strongly recommended to make sure the work and preparation of the JCA is accurate and in line with the view of the HTAb representatives. The JSC can either be consultations jointly with EMA representatives or only with HTAb representatives. The JSC will include a meeting with the regulators, after which Sponsors will be provided with a document containing recommendations. Several criteria (e.g., high volume, high unmet need, high impact on the health care system, etc.) need to be met in order to qualify for such consultations.

Before the JSC is implemented in January 2025, a JSC can be requested in parallel with EMA Scientific Advice via the Parallel EMA/HTAb Scientific Advice procedure.

Such procedures aim to foster early dialogue between pharmaceutical companies, EMA representatives, and HTAbs to facilitate the exchange of relevant information and create a better understanding of the new technology being assessed.

Current Challenges of the HTA Regulation

With the upcoming mandatory requirement of a JCA dossier submission to the European Commission beginning in January 2025 and Parallel EMA/HTAb Scientific Advice consultations already taking place, pharmaceutical companies developing oncology drugs and ATMPs need to start thinking and planning now for their EU product launch.

With the deadline of JCA dossier submission 45 days prior to the CHMP opinion, the JCA dossier preparation will also need to be considered and developed well ahead in time. The JCA dossier needs to account for all PICOs identified from the MS(s), and a systematic literature review will need to include EU epidemiology data for the past five years. Additional indirect treatment comparisons will make the dossier extensive and are likely to have an impact on the evidence generation strategy.

ProPharma Can Assist with All of Your European Regulatory Affairs Consulting Needs

ProPharma is uniquely positioned to provide comprehensive support for the upcoming HTAR initiatives in January 2025. Our team combines in-house specialists in both regulatory affairs and HTA, enabling us to offer clients unrivalled joint support throughout the entire process.

Furthermore, our consultants remain at the forefront of the field by closely monitoring the latest EU guidance and actively engaging with national stakeholders, regulatory bodies, and HTA organizations.

In relation to the new HTAR and the mandatory requirements of a JCA, ProPharma’s expert team of local regulatory affairs consultants can support you with:

• Parallel EMA/HTA body Scientific Advice meeting preparation and execution
• Meeting preparation and participation for a JSC (following its January 2025 implementation)
• Preparation and submission of the JCA dossier
• National pricing and reimbursement activities that will remain a requirement for reimbursement in each EU member state
  
  

Next, read Part 2 of our Mandatory HTAR Implementation in the EU series.