In brief
The Commission’s Implementing Act on Joint Clinical Assessments for medicines for human use was finally adopted on 23 May 2024. This is the first out of six Implementing Acts for the EU HTA Regulation. With only seven months to go before the HTA Regulation becomes applicable, developers of medicines within the first wave of products subject to the HTA Regulation will need to consider their strategy for EU assessment and its interplay with national procedures. They need to gear up for the short dossier submission timelines and try to anticipate the scope for assessment and methodology despite remaining uncertainties.
As a point of concern, the Implementing Act does not foresee any meaningful involvement of health technology developers in the scoping and assessment process. This will not only have pharmaceutical companies worried about their procedural rights, the entire healthcare community has an interest to see more involvement of the developer of the product. It suffices in this respect to point at the EMA approval process, which has clearly demonstrated that active engagement between a regulator and company is critical to ensure smooth process, positive outcomes and timeliness.
As January 2025 looms, pragmatism and acknowledgement of each party’s role will be necessary if the JCA process is to deliver on its objectives of high quality HTA, elimination of duplication and providing patients with swift access to innovative products across the region.
More detail
Scope and Gradual Application EU HTA Regulation: The EU HTA Regulation[1] will soon become applicable for a first wave of products falling into its scope: As of 13 January 2025, medicines submitted to EMA with a new active substance claim and indicated for treatment of cancer and those regulated as advanced therapy medicinal products (ATMPs) will have to undergo joint clinical assessment at EU level. Throughout 2028 – 2030, the HTA Regulation will gradually become applicable to all centrally authorised medicinal products based on a full dossier.
Implementing Act – JCA Procedure for Medicines for Human Use: In preparation for the implementation of the HTA Regulation, the European Commission published on 23 May 2024 the First Implementing Act on Joint Clinical Assessments which sets forth the timelines and procedural steps for carrying out joint clinical assessment for medicinal products for human use (but does not cover medical devices).
The Implementing Act offers much needed clarification around the future outlook of the assessment process but also raises concern from a health technology developer’s perspective. Key concerns include:
Scope of Joint Clinical Assessment (JCA)
As per the EU HTA Regulation, the scope of a JCA must be inclusive of all Member States’ needs. A pragmatic approach to such requirement will, however, be needed if the Coordination Group is to meet the required standards of quality and timeliness under Article 4 of the EU HTA Regulation.
Moreover, under the Implementing Act involvement of the health technology developer in the scoping process is very limited. At the start of the procedure, the developer of a medicine must submit the SmPC and clinical overview of the marketing authorisation application. The developer is seemingly not expected to provide other information unless specifically requested by the Coordination Group and has no right to comment on the JCA scope unlike other stakeholders including clinical experts and patients.
An explanation meeting will be organised after the adoption of the scope if requested by the developer. This is a welcome softening of the wording in the initial draft which left the possibility of an explanation meeting at the discretion of the Coordination Group.
Timeline for dossier submission
The timeline for submitting the dossier under the Implementing Act is set at 100 days (up from 90 days in the initial draft). Given that the developer will only know the final scope of the assessment at the time the Commission requests the submission of the HTA dossier, the 100-days term may prove to be challenging; even more so for smaller companies which cannot rely on a network of local affiliates to help address the PICOs (Patient, Intervention, Comparator, Outcomes) that Member States may propose as part of the JCA scope.
Right to be heard
During the assessment phase, the involvement of the health technology developer is also kept at a minimum. In contrast with EMA’s approval process, there are no upfront opportunities for the developer to provide input during the JCA process, and additional information may only be provided at the request of the Coordination Group.
In accordance with Article 11 (5) of the HTA Regulation, developers are provided with the draft assessment report. The developer may signal purely technical or factual inaccuracies and any information they consider confidential. The developer cannot provide any other substantive feedback. The timeline for review is short: seven days, which in certain cases can be reduced to five days[2]. For flexibility reasons, the Implementing Act does not contain further guidance to what must be considered “technical or factual inaccuracies” and “confidential information”[3]. Experience with the first JCAs will be critical to understand what will be acceptable and what not.
Arguably, systematic non-involvement of health technology developers in the scoping and assessment phases of the JCA process may be considered a violation of the right to be heard, which is enshrined in Article 41(2)(a) of the Charter of Fundamental Rights. It may also be disproportionate for a developer having to address shortcomings of the EU assessment within 27 Member States national procedures with a risk of diverging outcomes.
What should companies do to prepare for EU HTA? Despite the current concerns and the many outstanding uncertainties, the EU HTA Regulation will be a reality in a few months’ time. Companies which do not have products falling within the first wave, will have the opportunity to monitor the first JCAs and take lessons from the experiences gained. However, companies faced with EU HTA next year, should be in active preparation mode not only trying to anticipate the scope for EU HTA (involving local expertise) but also considering any additional local HTA requirements.
Will EU HTA Regulation deliver on its promise? The objective of the EU HTA Regulation is to avoid duplication and divergency, supporting high quality HTA and ultimately to provide faster access to patients across the region. However, the way the process is being construed leaves the attainment of these objectives in doubt. It may even result in delays of pricing and reimbursement decisions as Member States delay procedures to take account of the EU output (as may be the case in Belgium). EMA process has learned that early and active engagement with the developer is critical to ensure smooth process, positive outcomes and timeliness. To make this procedure a success, pragmatism and acknowledgement of each party’s role will be necessary as well as Member States’ active buy-in and commitment not to delay national decisions on access. If not, the Regulation is at risk to only add administrative burden and delay patient access.
[1] 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/EU
[2] For instance, in case of an accelerated procedure
[3] Regulation (EC) No 1049/2001 will function as a basis as well as past practice of EMA
