Why teams need upstream readiness
Europe now assesses evidence together. When Member States contribute, differences in evidence, outcomes and insight become visible in ways organisations rarely see internally.
This is especially important in rare diseases, where data is limited, expectations vary and interpretation differs across countries.
Recent HTA Coordination Group updates in late 2025 have brought clearer expectations on subgroup analyses, evidence completeness and PICO justification. These changes make early readiness even more important for rare-disease programmes.
Upstream clarity is now essential – long before timelines tighten.
What RAREready provides
1. We review your existing evidence, outcomes and insight through a JCA lens
This shows how your work may be interpreted once many countries contribute to the Joint Assessment.
We help teams understand what “complete and up-to-date evidence” means under the latest rules, so there are no surprises when the first dossier version is submitted.
2. We show where interpretation may differ or raise questions
This includes outcomes, patient insight, clinical expectations and internal assumptions.
3. We create a shared understanding across your functions
Medical, Access, Evidence, Clinical and Patient Engagement work from the same upstream view, which reduces confusion and strengthens early decisions.
4. We help affiliates understand their role and how to contribute early
We give affiliates clear and realistic guidance on what to watch for and how to share insight without drifting into HTA work.
5. We define an upstream readiness path that avoids HTA territory
You gain a realistic view of what your current work already supports and where expectations may not align once the JCA process begins.
Our work includes optional AI-enhanced tools that help map PICO expectations, identify potential evidence gaps and bring clarity to early team discussions. These tools run securely within standard office environments.
Who RAREready™ supports
- Rare disease programme leads
- Medical Affairs
- Patient Engagement & Advocacy
- Evidence and Clinical teams
- Market Access and Policy
- Affiliates
- US biotechs preparing for Europe
If you need early clarity and alignment before JCA timelines begin, RAREready is built for you.
What we do not do
- We do not design PICOs.
- We do not provide HTA strategy.
- We do not shape evidence or submissions.
- We do not replace HEOR or market access consulting.
We do not interpret HTA guidance for decision-making; we help teams understand how their existing work may be viewed in a joint-assessment context and where clarification or alignment may be needed.
Our focus is upstream clarity and organisational readiness.
From Our Expert Blog at swii.ch
Explore recent insights from the team behind RAREready
EU JCA is underway
A shared EU evaluation of evidence has begun. See what it means for rare-disease sponsors and why patient evidence will define future success. → Read more on Swii.ch Health
5 Patient Evidence Myths That Undermine Your EU JCA Strategy
Common pitfalls that sponsors should avoid. → Read more on Swii.ch Health
EU JCA 2025 Explained: What Rare Disease Sponsors Need to Know Now
Oncology in 2025. Orphan drugs in 2028.
For smaller sponsors, late patient evidence planning won’t work.
We’ve set out the timelines, expectations and what to do now. → Read more on Swii.ch Health
Europe’s Shift to Patient Evidence
What the first JCAs tell us about how patient evidence will be viewed in rare-disease assessments. → Read more on Swii.ch Health
Next Steps
A 20-minute call is often enough to understand your needs and identify the right starting point.
Book Your EU JCA Blueprint Call.
Contact us
We’re here to help rare-disease innovators prepare effectively for EU JCA and patient-centred evidence needs.
EU JCA & HTA Regulation (HTAR)
Frequently Asked Questions
The JCA is an EU-level comparative clinical effectiveness assessment under Regulation (EU) 2021/2282. It runs in parallel to EMA review and provides a single evidence base for national HTA and reimbursement decisions. Learn more in our About RAREready overview.
As of October 2025, JCA applies to oncology and ATMP products. It will expand to orphan medicines in 2028 and all centrally authorised medicines in 2030. Read more on EU HTA Regulation.
JCA covers clinical effectiveness and safety. Cost-effectiveness, budget impact and reimbursement remain national. JCA creates a shared evidence base but does not replace national HTA. See how we support sponsors with JCA strategy planning.
PICO stands for Population, Intervention, Comparator and Outcomes. It defines the JCA scope. If pivotal trials don’t align with likely PICOs, sponsors may face evidence gaps and extra national work. Explore our PICO alignment approach.
Patients and clinical experts contribute during scoping and assessment, bringing disease insight and lived experience. The HTA Secretariat manages selection, conflict-of-interest checks and confidentiality. We help sponsors build structured patient engagement frameworks.
A JSC gives early feedback from HTA bodies on comparators, outcomes, evidence plans and patient engagement strategies. Early JSC engagement helps anticipate PICO diversity and strengthen JCA readiness.
Discover how we support early JSC preparation.
Orphan products enter scope in 2028, but many sponsors are preparing now. Rare diseases often involve small populations, single-arm trials and diverse comparators. Early planning reduces risk and delays. Learn more about our RAREready 4D framework.
Yes. PED and validated PROs help show why outcomes matter to patients. Regulators and HTA bodies expect early and systematic patient input to support clinical benefit claims. Explore our patient evidence approach.
Experts are sourced through ERNs, EMA databases and patient networks. They sign COI and confidentiality agreements. Their input informs PICO scoping, evidence interpretation and assessment discussions. Learn more from EU HTA guidance.
Engage early with JSC, anticipate multi-country comparators, integrate patient input, and align trial design with JCA guidance. Build flexible national HTA packages from one strong JCA evidence base. Start with a Blueprint Call.
Start in early Phase II. Once pivotal designs are locked, adaptation options shrink fast. JCA timelines are tight, so early alignment avoids rushed and reactive evidence generation. Our 100-Day Sprint is designed for compressed timelines.
JCA readiness is cross-functional. Market Access alone can’t deliver it. Teams that align Medical, HEOR, Regulatory and Patient Engagement early are best positioned for success. See how our framework supports team alignment.
No. Late amendments and evidence gaps cost more. Early patient strategy strengthens submissions, reduces rework, and accelerates timelines—turning compliance into a competitive advantage. Learn more about RAREready’s patient engagement modules.
Yes. EU guidance supports structured, transparent patient engagement. Using COI declarations, engagement frameworks and clear facilitation keeps collaborations compliant and credible. Learn more from EMA guidance on patient involvement.