From 12 January 2028, every EU orphan-designated therapy must navigate a 110-day Joint Clinical Assessment (JCA) timeline under Regulation (EU) 2021/2282:
- 20 days to finalise PICO (Art 8)
- 90 days to build and submit the dossier (Art 12)
- ≥ 45 days before CHMP opinion for assessor review
Teams often refer to Days 0–100 as the “100-day sprint” to focus on core execution, with a 10-day buffer on either side. Embedding structured patient engagement throughout this window transforms compliance into strategic advantage.
Why Patient Engagement Matters
- Regulators expect documented, structured patient input in PICO scoping
- Early insights help refine endpoints, comparators, and outcomes
- Authentic patient voices strengthen both the scientific case and the value narrative
Aligning Patient Activities with JCA Operating Models
Five Phases of a Patient-Centred 110-Day Sprint
Four Enablers for Success
- Governance & Traceability
- Operational Alignment
- Regulatory Precision
- Methodological Rigor
RARE-READY™ in a Nutshell
Developed by swii.ch health, RARE-READY™ is a four-module framework designed to embed audit-ready, patient-centred tools into every phase of your 110-day JCA sprint— Engage & Scope, Gather & Validate, Craft & Tell, and Deliver.
Embedding patient insight throughout the sprint transforms a regulatory deadline into a strategic asset.
Next steps: • Comment “Ready” for a one-page JCA Sprint Readiness Checklist • Comment “Preview” for a high-level snapshot of the RARE-READY™ framework
References:
- Arcà, M. et al. (2024). Patient Involvement in Joint Clinical Assessments: Emerging Expectations. European Journal of Health Technology Assessment.
- Regulation (EU) 2021/2282 on Health Technology Assessment.
- HTA Coordination Group (HTACG). (2025). Guidance on Patient Involvement in EU JCA.
- EURORDIS. (2023). Recommendations for Patient-Centred Evidence in Rare Disease HTA.
- Deloitte. (2023). Getting Ready for the New HTA Regulation in EU: What Pharmaceutical Companies Need to Know.