The application of Joint Clinical Assessments (JCA) under Regulation (EU) 2021/2282 (EU HTA) began in Jan 2025 for oncology medicines and ATMPs. All JCA procedures are initiated through a PICO framework, which defines the assessment scope - Population, Intervention, Comparator(s), and Outcomes - defined by national HTA bodies¹.

The resulting JCA assessment scope determines the data required from the Health Technology Developers (HTDs) and is critical for timely, high-quality JCA dossier preparation. To support this process, the Coordination Group on HTA (HTACG) published the “Guidance on the scoping process” in November 2024², followed by 6 “PICO simulation exercises” in February 2025^3-8, three of which focused on medicinal products. The published exercises simulated responses to PICO surveys to inform the development of the final guidance on the scoping process. We appreciate the opportunity to learn from these PICO exercises as the first wave of JCAs progresses.

While the goal of consolidating Member States needs into the lowest possible number of PICOs² is commendable, its execution lacks clarity and transparency as well as evidence-based principles in determining the final assessment scope. In particular, the interplay between population and comparator is an area of concern as these are the key drivers of the number of PICOs. The JCA may lose relevance at the Member State level if it leads to either an overwhelming volume of data or insufficient evidence where the consolidated PICO imposes an evidence standard that cannot be satisfied.     

Key Questions and Observations

1. Development of Initial PICO Proposals

The guidance outlines the overall scoping process but lacks transparency on how assessors and co-assessors formulate initial PICO proposals. These PICO proposals are critical as they guide Member State responses and shape the JCA scope from the outset.

While HTD input, clinical guidelines, and stakeholder contributions (e.g., patients and clinicians) are considered², the lack of transparency on how proposals are developed limits HTDs’ ability to anticipate the scope and prepare effectively.

We recommend formalising a consultation with HTDs during the drafting of proposed PICOs to ensure consistency and predictability.

2. PICO Consolidation Process

Although definitions and methodological elements (e.g. subpopulations, options to include multiple comparators) are introduced², the current consolidation process appears to allow significant flexibility, lacking transparency and standardised HTD engagement. The rationale behind consolidation decisions and Member State dialogue remains unclear in the published scoping guidance and PICO exercises.

This uncertainty undermines predictability for HTDs preparing JCA submissions.

3. Relevance of the JCA scope

Diverse standards of care across Europe may necessitate multiple PICOs. As JCA research questions grow increasingly complex, individual Member States may struggle to see their specific needs reflected. Excessively large JCA submission dossiers risk misinterpretation of results and may dilute the relevance of the JCA for national decision-making.

We believe that the objectives of the EU HTA Regulation can be achieved through a balanced and transparent approach that ensures the necessary relevant evidence without overburdening HTDs or HTA bodies.

Conclusion

EFPIA welcomes the scoping guidance and PICO exercises as important steps toward greater transparency. To fully realise the potential of the JCA process and ensure enhanced efficiency and relevance at the national level, further refinement is needed, particularly in how PICOs are proposed, consolidated, and communicated.

We recommend HTD input be routinely integrated into the draft PICO proposals, rather than only at the discretion of the assessors. Prior to the official revision of the Regulation, we would welcome continuous process improvements with timely communication via established channels such as FAQs.

We remain committed to working collaboratively with all stakeholders to advance and refine the EU HTA system—ensuring it delivers meaningful benefits for patients, supports sustainable healthcare systems, and fosters innovation across Europe.

References

1. Member State Coordination Group on Health Technology Assessment. Procedural Guidance for Joint Clinical Assessments of Medicinal Products. Version 1.0. Adopted November 28, 2024. Available from: https://health.ec.europa.eu/document/download/0929cd01-619d-4456-a1c4-d8e33f9e36bf_en?filename=hta_jca_mp_procedural-guidance_en.pdf. Accessed August 25, 2025.
2. Member State Coordination Group on Health Technology Assessment. Guidance on the Scoping Process. Version 1.0. Adopted November 28, 2024. Guidance on the scoping process. Accessed August 25, 2025
3. Member State Coordination Group on Health Technology Assessment. PICO Exercise MP 01: Durvalumab (Imfinzi®) for Adults With Advanced or Unresectable Hepatocellular Carcinoma (HCC) [published online Spring 2024]. Available from: https://health.ec.europa.eu/document/download/322ae969-f3ca-4f23-ab8e-1b4bdf22528f_en?filename=hta_pico_mp01_en_0.pdf. Accessed August 25, 2025.
4. Member State Coordination Group on Health Technology Assessment. PICO Exercise MD 02: EASEE SYSTEM for Adults With Focal Epilepsy Refractory to Two or More Antiepileptic Medications [published online Spring 2024]. Available from: https://health.ec.europa.eu/document/download/4aacd064-8276-4b12-8c33-7979b67d378d_en?filename=hta_pico_md02_en.pdf. Accessed August 25, 2025.
5. Member State Coordination Group on Health Technology Assessment. PICO Exercise MP 03: Adagrasib (Krazati®) for Adult Patients With Advanced Non-Small Cell Lung Cancer (NSCLC) With KRAS G12C Mutation After Disease Progression Following Prior Systemic Therapy [published online Spring 2024]. Available from: https://health.ec.europa.eu/document/download/63868893-b079-4ea6-bf94-af2cd9f19c65_en?filename=hta_pico_mp03_en_0.pdf. Accessed August 25, 2025.
6. Member State Coordination Group on Health Technology Assessment. PICO Exercise MD 04: Edwards EVOQUE Tricuspid Valve Replacement System for Patients with Severe Tricuspid Regurgitation [published online Spring 2024]. Available from: https://health.ec.europa.eu/document/download/288f7898-fbcd-4e64-b7be-70b6b38006bc_en?filename=hta_pico_md04_en.pdf. Accessed August 25, 2025.
7. Member State Coordination Group on Health Technology Assessment. PICO Exercise MP 05: Etranacogene dezaparvovec (Hemgenix®) for Adult Patients with Severe and Moderately Severe Haemophilia B [published online Spring 2024]. Available from: https://health.ec.europa.eu/document/download/dcaede60-e13c-45d2-9e1d-f2257ba81444_en?filename=hta_pico_mp05_en_0.pdf. Accessed August 25, 2025.
8. Member State Coordination Group on Health Technology Assessment. PICO Exercise MD 06: PDA Stenting with a Bioresorbable Stent in Neonates and Infants with Cyanotic Congenital Heart Defects [published online Spring 2024]. Available from: https://health.ec.europa.eu/document/download/c62fd888-631f-4a0b-8764-cc5f5706ddc5_en?filename=hta_pico_md_6_en.pdf. Accessed August 25, 2025.