Towards HTA Harmonization; the European Commission Launches a Public Consultation on Draft Legislation Concerning the Conduct of Joint Clinical Assessment

With less than a year before Regulation (EU) 2021/2282 on health technology assessments (the “HTA Regulation”) enters into application, the European Commission (“EC”) has launched a public consultation on a draft Implementing Regulation concerning the conduct of joint clinical assessments (“JCA”). Members of the public and other interested parties have until 2 April to provide feedback on the EC’s draft.

What are JCAs?

The HTA Regulation, which entered into application on 12 January 2024, seeks to streamline health technology assessment (“HTA”) procedures for certain medicinal products and medical devices throughout the EU.

By way of reminder, HTAs assess the added therapeutic value and relative effectiveness of health technologies compared to existing treatments, and they are used by national authorities to inform pricing and reimbursement decisions. During HTAs, technologies are assessed for various qualities including clinical effectiveness, cost-effectiveness, and associated ethical and societal considerations. Whilst certain assessments must be conducted at the Member State level (taking into account, for example, budgetary constraints and public perception), the clinical effectiveness of a technology is not subject to the same constraints.

With a view to promoting cooperation and efficient use of resources, and amongst other aims, the HTA Regulation seeks to facilitate JCAs which national HTA bodies can rely on when making national pricing and reimbursement decisions.

The proposed JCA procedure

According to the draft Implementing Regulation, the day-to-day management of the JCA procedure will be handled by the JCA subgroup within the Member State Coordination Group (the “Coordination Group”). Individual JCA procedures will be led by an assessor and co-assessor appointed by the JCA subgroup.

As currently drafted, the Implementing Regulation sets out the following process for the conduct of JCAs:

• Development of an assessment scope. When submitting a centralised marketing authorisation (“MA”) application, health technology developers must provide the HTA Secretariat with their draft Summary of Product Characteristics (“SmPC”) and the clinical overview of their MA application. Using this information, the assessors will prepare a draft assessment scope covering four key parameters: patient population, intervention, comparators and health outcomes. The wider JCA subgroup and Member States will then be asked to comment on the draft before it is finalised no later than 20 days after the European Medicines Agency’s (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) adopts its list of questions.
• Conduct of the JCA. The health technology developer will then submit an electronic dossier of information responding to the scope. Following their review, the assessors will draft an assessment report and a summary report, which will then be shared with selected patients, clinical experts and other experts who have (i) the required expertise in that therapeutic area, (ii) no conflicts of interest, and (iii) have signed a confidentiality agreement, for them to provide comments. The health technology developer will have the opportunity to correct any factual or technical inaccuracies and to request (with sufficient justification) the protection of confidential information. The JCA subgroup must then finalise and send the revised reports to the Coordination Group no later than the date of adoption of the EC’s decision on the MA. In turn, the Coordination Group then has no longer than 30 days to endorse the reports or re-initiate the JCA process.
• The JCA decision. JCAs will be published by the EC, subject to the views of the JCA subgroup and the developer’s requests for certain information to be treated as confidential. The national HTA bodies in the EU will be required to “give due consideration” to the reports. In practice, this means that the reports should “be part of the documentation of authorities or bodies involved in HTA activities”. The finalised JCA reports will not be legally binding; national HTA bodies will be free to perform supplemental clinical analyses, for example, by using comparators which are more commonly used in their healthcare systems.
• Stakeholder cooperation. Throughout the JCA process, the EMA and Coordination Group are required to closely cooperate. For example, when the EMA receives an MA application, it must notify the HTA Secretariat when the MA application is validated, and also where there are any substantial questions or outstanding issues that might impact the therapeutic indication of the health technology. Importantly, the EMA is required to send the HTA secretariat a copy of the draft SmPC and the CHMP’s assessment report within seven days of adoption by the CHMP of its final opinion.
• HTA IT platform. All correspondence between the Coordination Group, HTA Secretariat, JCA subgroup, the health technology developer, patients, clinical experts, and other relevant experts must be sent via the HTA IT platform. This platform will also facilitate public access to the finalised JCA reports. The latest version of the EC’s Implementation Rolling Plan (dated February 2024) indicates that the first release of the IT platform went live in October 2023, and that two further releases are currently “Planned”, however, no firm dates are provided.

As with all public consultations, the text of the Implementing Regulation may be subject to further amendment.

What happens next?

Prior to the HTA Regulation entering into application in January 2025, the EC must pass five other implementing acts. These cover the management of conflicts of interest; cooperation by exchange of information with the EMA; joint scientific consultations for medicinal products; joint scientific consultations for medical devices; and joint clinical assessments for medical devices. The latest of these is not due for publication before Q4 2024. With this in mind, uncertainty around how the HTA Regulation will actually function is likely to persist until the end of this year. Thankfully, when the HTA Regulation does enter into application, it will be subject to a progressive phase-in approach which should help to ease industry’s transition to the new procedures.