Commission Implementing Regulation (EU) 2024/1381 of 23 May 2024 laying down, pursuant to Regulation (EU) 2021/2282 on health technology assessment, procedural rules for the interaction during, exchange of information on, and participation in, the preparation and update of joint clinical assessments of medicinal products for human use at Union level, as well as templates for those joint clinical assessments

THE EUROPEAN COMMISSION,

Having regard to the Treaty on the Functioning of the European Union,

Having regard to 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⁽¹⁾, and in particular Article 15(1), points (a) and (c), Article 25(1), point (b), and Article 26(1) thereof,

Whereas:

1. Regulation (EU) 2021/2282 lays down a support framework and procedures for cooperation between Member States on health technologies at Union level and establishes the Member State Coordination Group on Health Technology Assessment (‘the Coordination Group’).

2. Under Article 15 of Regulation (EU) 2021/2282, the Commission is to lay down detailed procedural rules for the implementation of Articles 8 to 14 of that Regulation as regards conducting and updating joint clinical assessments. In particular, under Article 15(1), points (a) and (c), of Regulation (EU) 2021/2282, the Commission is to adopt detailed procedural rules for the cooperation, in particular by exchange of information, with the European Medicines Agency on the preparation and update of joint clinical assessments of medicinal products and for the interaction, including timing thereof, with and between the Coordination Group, its subgroups and the health technology developers, patients, clinical experts and other relevant experts during joint clinical assessments and updates.

3. In addition, under Article 3(7), points (d), (e) and (g), of that Regulation, the Coordination Group is to adopt further rules on conducting joint clinical assessments, namely methodological guidance on joint work, detailed procedural steps and the timeframe for the conduct of joint clinical assessments and their updates and guidance on the appointment of assessors and co-assessors for joint clinical assessments.

4. In order to ensure the highest scientific quality of joint clinical assessment reports, Articles 8(6) and 11(4) of Regulation (EU) 2021/2282 provide for the involvement of patients, clinical experts and other relevant experts in joint clinical assessments. Under Article 25(1), point (b), of Regulation (EU) 2021/2282, the Commission is to adopt, after consulting all relevant stakeholders, general procedural rules on the selection and consultation of stakeholder organisations and patients, clinical experts and other relevant experts in joint clinical assessments at Union level. Based on these rules, the Coordination Group, pursuant to Article 3(7), point (j), of Regulation (EU) 2021/2282, is to ensure appropriate involvement of stakeholder organisations and experts in its work.

5. Under Article 26(1) of Regulation (EU) 2021/2282, the Commission is to adopt the format and templates for the dossiers for information, data, analyses and other evidence to be provided by health technology developers for joint clinical assessments, as well as for joint clinical assessment reports and summary joint clinical assessment reports. This Regulation lays down those formats and templates, to ensure a uniform approach on the presentation of the evidence provided by the health technology developers to the Coordination Group and of the information contained in the joint clinical assessment reports.

6. In order to allow sufficient time for a high quality joint clinical assessment, that clinical assessment should start at the same time as the centralised procedure provided for under Regulation (EC) No 726/2004 of the European Parliament and of the Council⁽²⁾, that is to say upon the confirmation by the European Medicines Agency of a submission of a valid application for marketing authorisation in accordance with Regulation (EC) No 726/2004 or for a variation to the terms of an existing marketing authorisation in accordance with Commission Regulation (EC) No 1234/2008⁽³⁾. Therefore, it is appropriate to require the health technology developer to submit information necessary for the development of the assessment scope provided for in Article 8(6) of Regulation (EU) 2021/2282 (‘the assessment scope’) to the Commission acting as secretariat of the Coordination Group (‘the HTA secretariat’) at the same time as the health technology developer submits an application for marketing authorisation, or for a variation to the terms of an existing marketing authorisation, to the European Medicines Agency.

7. The joint clinical assessment of a medicinal product under Regulation (EU) 2021/2282 is carried out in parallel to the centralised procedure provided for in Regulation (EC) No 726/2004, and the Coordination Group is to endorse the joint clinical assessment report no later than 30 days following the adoption of a Commission decision granting a marketing authorisation for that medicinal product. The joint clinical assessment is to be discontinued, for example, where an application for a marketing authorisation or for a variation to the terms of an existing marketing authorisation is withdrawn, or where the outcome of the centralised procedure is negative for the application for a marketing authorisation or for a variation to the terms of an existing marketing authorisation. Therefore, the Coordination Group should be informed of submissions of valid applications for marketing authorisation and for a variation to the terms of existing marketing authorisations of medicinal products falling within the scope of Regulation (EU) 2021/2282 and of updates on steps in the centralised procedure, including changes in the envisaged timelines.

8. The assessment scope is based on the therapeutic indication(s) of the medicinal product. Therefore, with a view to enabling the subgroup on joint clinical assessments (‘the JCA Subgroup’) to update the assessment scope where deemed appropriate, while preserving the separation of the respective remits of the Coordination Group and the European Medicines Agency, the European Medicines Agency should inform the HTA secretariat on substantial questions or outstanding issues that might impact the therapeutic indication(s) proposed by the applicant for the medicinal product subject to a joint clinical assessment.

9. The assessor and co-assessor and the JCA Subgroup should get early access to the draft summary of product characteristics and to the assessment report referred to in Article 9(4), points (a) and (e) respectively, of Regulation (EC) No 726/2004.

10. Pursuant to Article 28, point (h), of Regulation (EU) 2021/2282, the HTA secretariat is to facilitate the cooperation, in particular through the exchange of information, with the European Medicines Agency, on the joint work referred to in Articles 7 to 22 of Regulation (EU) 2021/2282 related to medicinal products, including the sharing of confidential information. The exchange of information relevant to concrete joint clinical assessments and updates of joint clinical assessments should therefore take place through the HTA secretariat. The HTA secretariat should ensure that all the information it receives is communicated to the Coordination Group, its relevant subgroups and/or assessor and co-assessor, as appropriate, upon receipt thereof.

11. The health technology developer should be informed of the start of a joint clinical assessment, of the steps of that assessment, of its update, as well as of its re-initiation under Article 10(7) of Regulation (EU) 2021/2282. The health technology developer should also be informed of the decision of the Coordination Group to include the update of the joint clinical assessment in its annual work programme, pursuant to Article 14 of Regulation (EU) 2021/2282.

12. It is necessary to set out general procedural rules for the selection of patients, clinical experts and other relevant experts to be consulted during the joint clinical assessment. The selection should start as early as possible, whenever the Coordination Group, via the HTA secretariat, receives information about upcoming submissions of applications for marketing authorisations for the medicinal products referred to in Article 7(1) of Regulation (EU) 2021/2282.

13. The JCA Subgroup should aim to select patients, clinical experts and other relevant experts who have the required expertise in the therapeutic area of the joint clinical assessment, from a European or international perspective. These experts should be consulted during the joint clinical assessment.

14. In order to ensure that patients, clinical experts and other relevant experts take part in joint clinical assessments in an independent and transparent manner, free from conflict of interest, they should only be selected and involved in joint clinical assessments after the Commission has concluded on their conflicts of interest, in accordance with the rules set out in Article 5 of Regulation (EU) 2021/2282 and with the general procedural rules adopted under Article 25(1), point (a), of that Regulation. Only patients, clinical experts and other relevant experts who have signed a confidentiality agreement should be involved in joint clinical assessments.

15. The JCA Subgroup should give the opportunity to patient organisations, healthcare professional organisations and clinical and learned societies to provide their input on joint clinical assessments.

16. To achieve the objective that, during the development of the assessment scope, Member States’ needs are translated in the lowest possible number of sets of parameters for the joint clinical assessment in terms of patient population, intervention, comparators and health outcomes, the assessor, with the assistance of the co-assessor, should prepare an assessment scope proposal that will serve as a basis for Member States to express their needs.

17. In order to ensure that the assessment scope is inclusive and reflects Member States’ needs, the assessment scope proposal prepared by the assessor, with the assistance of the co-assessor, should be shared with the members of the JCA Subgroup. These members should consult national authorities and stakeholders in accordance with the procedural rules of the respective Member State.

18. The Commission should set out procedural rules in order to ensure that the assessment scope is developed respecting the right to good administration and taking into account information provided by the health technology developer and input received from patients, clinical experts and other relevant experts.

19. Article 10(1) and Article 11(1), point (a), of Regulation (EU) 2021/2282 set the timeframe for joint clinical assessments for medicinal products by reference to the timeframe applicable to the centralised procedure provided for in Regulation (EC) No 726/2004. The Commission should set the deadlines for the finalisation of the assessment scope and of the draft joint clinical assessment reports by the JCA Subgroup. These deadlines should refer to the main steps of the centralised procedure and should comply with the overall deadlines set out in Regulation (EU) 2021/2282. Where Article 11(1), point (a), of Regulation (EU) 2021/2282 does not apply, the Commission should set these deadlines with the aim of improving timely patient access to health technologies.

20. To contribute to the completeness and high quality of the dossier and the smooth conduct of the joint clinical assessment, the health technology developer should be given the opportunity to request an assessment scope explanation meeting with the JCA Subgroup.

21. The Commission should set deadlines that should give the health technology developer sufficient time to prepare and submit the dossier for joint clinical assessment of the medicinal product. The Commission should set the rules that provide a possibility to extend the deadline to submit the dossier in justified instances, without however going beyond the deadline specified in Article 10(1) of Regulation (EU) 2021/2282.

22. Similarly, the Commission should set deadlines that give sufficient time to the health technology developer to take the following actions: (a) provide missing information, data, analysis and other evidence as indicated in the Commission’s second request; (b) provide further specifications or additional information, data, analyses, or other evidence; (c) provide updates to previously provided information referred to in Articles 10(8) and 11(2) of Regulation (EU) 2021/2282; (d) signal technical or factual inaccuracies in the draft joint clinical assessment and summary reports and any information the health technology developer considers to be confidential.

23. The Commission should set deadlines for its assessment on whether the dossier submitted by the health technology developer for joint clinical assessment of the medicinal product meets the requirements laid down in Article 9(2), (3) and (4) of Regulation (EU) 2021/2282. The Commission should consult, as appropriate, the assessor and co-assessor when carrying out this assessment.

24. The Commission should set procedural rules to ensure that patients, clinical experts and other relevant experts are involved in the assessment process by being given the opportunity to provide input on the draft joint clinical assessment and summary reports.

25. According to Article 11(2) of Regulation (EU) 2021/2282, the health technology developer is to proactively inform the Coordination Group where new clinical data becomes available during the joint clinical assessment process. The Commission should set the deadline by which the health technology developer is to submit such new clinical data so that these data are considered in the draft joint clinical assessment and summary reports.

26. The Commission should set the deadline for finalising the revised draft joint clinical assessment and summary reports by the JCA Subgroup. This should ensure the delivery of joint clinical assessment reports in a timely manner and allow the Coordination Group to comply with the deadline for the conclusion of the joint clinical assessment set out in Article 11(1), point (a), of Regulation (EU) 2021/2282. The Commission should also set the deadline for the Coordination Group to endorse the revised draft joint clinical assessment and summary reports, with the aim of improving timely patient access to health technologies.

27. Where, during the centralised procedure, there is a change in the therapeutic indication(s) initially submitted in the application for marketing authorisation or for a variation to the terms of an existing marketing authorisation, the JCA Subgroup should decide whether the joint clinical assessment should continue or be restarted. The Commission should set the procedural rules that apply in case a new assessment scope is to be developed.

28. Certain specific procedural rules and deadlines should apply where the Coordination Group re-initiates a joint clinical assessment pursuant to Article 10(7) of Regulation (EU) 2021/2282 or where an update of a joint clinical assessment is carried out pursuant to Article 14 of Regulation (EU) 2021/2282.

29. In order to ensure transparency, traceability and professional secrecy, as well as to contribute to the procedural compliance of joint clinical assessment reports, any correspondence with and between the Coordination Group, the JCA Subgroup, the HTA secretariat, the health technology developer, patients, clinical experts and other relevant experts during joint clinical assessments should be sent in a digital format through the IT platform referred to in Article 30 of Regulation (EU) 2021/2282 (‘the HTA IT platform’).

30. This Regulation lays down, in accordance with Article 5(1), point (a), of Regulation (EU) 2018/1725 of the European Parliament and of the Council⁽⁴⁾, the rules for processing, through the HTA IT platform, personal data for the purposes of conducting joint clinical assessments and their updates. In particular, it specifies the personal data that may be processed through that platform, namely certain personal data relating to the patients, clinical experts and other relevant experts involved in joint clinical assessments and their updates and certain personal data relating to the representatives appointed to the Coordination Group and the JCA Subgroup, the representatives of health technology developers and the representatives of the members of the stakeholder network established pursuant to Article 29 of Regulation (EU) 2021/2282 (‘the HTA stakeholder network’). This Regulation also determines that the Commission is to be considered the controller of the processing of personal data through the HTA IT platform in the sense of Article 3, point 8, of Regulation (EU) 2018/1725. Any processing of personal data by the members of the Coordination Group and the JCA Subgroup and their representatives outside of the HTA IT platform is to take place in accordance with Regulation (EU) 2016/679 of the European Parliament and of the Council⁽⁵⁾.

31. The identity of the patient may reveal the patient’s health status in relation to the subject matter of the joint clinical assessment and therefore should be considered a special category of personal data under Article 10 of Regulation (EU) 2018/1725. Therefore, such data should only be processed where the criteria of Article 10(2), point (i), of that Regulation are met. This Regulation provides for suitable and specific measures to safeguard the rights and freedoms of the data subject. In particular, no personal data of patients shall be made publicly available. Moreover, under Article 5(6) of Regulation (EU) 2021/2282, the representatives appointed to the Coordination Group and the JCA Subgroup, as well as patients, clinical experts and other relevant experts involved in joint clinical assessments and their updates, are subject to a requirement of professional secrecy, even after their duties have ceased. Finally, this Regulation specifies that only patients, clinical experts and other relevant experts who have signed confidentiality agreements be involved in joint clinical assessments.

32. In order to ensure the possibility to verify whether the joint clinical assessments were conducted in the procedurally compliant manner, notably in the event of complaints or litigation, it is appropriate to provide for a retention period of personal data and for its review at regular intervals.

33. In order to ensure transparency, on the one hand, and the protection of confidential data for commercial reasons, on the other hand, the Commission should publish the joint clinical assessment and the summary reports, together with the documentation referred to in Article 30(3), points (d) and (i), of Regulation (EU) 2021/2282, after having considered the views of the JCA Subgroup as to the commercially sensitive nature of the information contained in this documentation, which the health technology developer has requested to be treated as confidential.

34. Joint clinical assessments of medicinal products are to be conducted from the date as of which Regulation (EU) 2021/2282 applies, that is, 12 January 2025. This Regulation should therefore apply from 12 January 2025.

35. The European Data Protection Supervisor was consulted in accordance with Article 42 of Regulation (EU) 2018/1725 and delivered an opinion on 4 April 2024.

36. The measures provided for in this Regulation are in accordance with the opinion of the Committee on Health Technology Assessment,

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