Written by Pina Haberl, Senior Director & Chief People Officer
Disclaimer: (Last updated: May 22, 2025 | Subject to final G-BA plenary resolution in July 2025)
The EU’s Joint Clinical Assessment (JCA) and Germany’s AMNOG process are converging, but critical differences in evidence requirements remain. At today’s G-BA information session, key updates were shared – not only on Germany-specific aspects, but also on developments at the European level – including JCA integration, Joint Scientific Consultations (JSCs), and practical implications for rare diseases and ATMPs. Here’s a breakdown of what you need to know.
1. JSC Slots in 2025: High Demand, Limited Supply
The EU HTA Coordination Group (HTACG) has allocated 10 total JSC slots for 2025:
- 7 slots for medicinal products (drugs and ATMPs).
- 3 slots for medical devices/IVDs.
Current Status (May 2025):
- 3 slots already allocated (February–March 2025 submission period).
- Remaining slots:
- 4 for drugs (June 2025 submission period).
- 3 for devices/IVDs (June 2025 submission period).
Priority Criteria:
- Unmet medical need, first-in-class innovation, and EU-wide public health impact.
- For rare diseases, emphasize patient population size and cross-border relevance.
Parallel Advice: Allowed – But Coordinated
- Manufacturers may request both JSC and G-BA national advice, especially when EU-level consultation does not fully address German-specific needs (e.g., comparators, subgroups).
- However, the G-BA actively coordinates to avoid duplication. If a company applies for both JSC and national advice simultaneously, the G-BA will reach out to discuss how to focus the national advice on Germany-specific topics. For example, EU-level JSC might cover PICOs, while national advice focuses on German specific comparator needs in Germany or endpoint requirements.
Strategic Tips:
- Secure slots early for June 2025 – there is high demand
- National Advice Parallelism: You can apply for both JSC and G-BA national advice, but the G-BA may contact manufacturers to avoid duplication. Use national advice to refine comparator choices and trial designs.
2. Dossier Strategies:
- EU Data Referencing – A Gebot;
The G-BA considers referencing the EU JCA dossier in AMNOG submissions as an obligation (“Gebot”), meaning manufacturers are expected to refer to relevant sections of the EU dossier wherever possible. However, this does not mean that submitting a full, standalone AMNOG dossier is forbidden (“Verbot”). Manufacturers still have the flexibility to provide additional national evidence or submit a complete AMNOG dossier if needed. Aligning the national submission with the EU JCA is increasingly recognized as best practice, but German-specific requirements must always be addressed.
- AMNOG Dossier – National Add-Ons Still Apply:
Despite European-level assessments, national requirements remain. These include German-specific comparators, healthcare resource use and cost data, and population definitions based on local clinical guidelines. - German Comparators:
- The G-BA prioritizes therapies widely used in German clinical practice. For example, if the JCA includes a “basket of EU comparators,” the G-BA may focus on one German SOC.
- Cost and Resource Use:
- Submit detailed German-specific cost models, including GKV reimbursement rates, hospital resource utilization, and pharmacy pricing.
- Subgroup Analyses:
- Populations must align with German clinical guidelines (e.g., age, disease severity, genetic variants).
Strategic Tip:
-Use national consultations to clarify which comparators/subgroups the G-BA will prioritize
3. JCA Timeline Impact on AMNOG
The timing of JCA report availability significantly impacts the AMNOG process:
| Scenario | JCA Availability | AMNOG Implications |
| 1. JCA Available at Submission | Before AMNOG dossier submission | JCA report is integrated into the initial assessment. Reduces duplication but requires alignment of PICOs. |
| 2. JCA Delayed | Post-AMNOG dossier submission, but pre/during AMNOG hearing phase | JCA findings can be added as supplementary evidence, but G-BA may request additional analyses. |
| 3. JCA Unavailable | After AMNOG completion | AMNOG proceeds with national data, with no delays expected. JCA cannot influence the outcome, risking misalignment. |
Strategic Tip:
-Submit AMNOG dossiers on time, even if the JCA is delayed, and use the hearing phase to add late JCA inputs
4. PROs in Practice: SF-36 vs. EQ-5D
- SF-36 Reigns Supreme:
The G-BA has and still prioritizes SF-36’s Physical/Mental Component Scores (PCS/MCS) for HRQoL assessments.
- Threshold: A 15% improvement (e.g., 5-point change on a 0–100 scale) is considered clinically meaningful.
- EQ-5D’s Limited Role:
While EQ-5D is used in EU cost-effectiveness models, the G-BA rarely accepts its utility scores. The EQ-VAS is cited more often but lacks SF-36’s granularity.
Strategic Tip:
–Collect SF-36 for Germany and EQ-5D for EU cost-effectiveness. For rare diseases, validate disease-specific PROs early.
5. Surrogate Endpoints: EU Flexibility vs. German Rigor
- EU JCA: Accepts surrogates (e.g., PFS, biomarker levels) as the “highest available evidence.”
- AMNOG: Requires validation against patient-relevant outcomes (survival, morbidity, HRQoL).
Example – Nusinersen (Spinal Muscular Atrophy):
The JCA would accept motor function scores (CHOP-INTEND), but the G-BA would demand evidence of ventilator-free survival.
Strategic Tip:
– Plan post-hoc analyses or RWD studies to bridge surrogate-to-patient-relevant outcomes for Germany.
6. Orphan Drug Privileges: Stability Under Scrutiny
- €30M Threshold Intact (For Now):
Therapies under €30M annual revenue retain automatic added benefit recognition.
- But…
Critics like IQWiG argue this fosters “fictitious” benefits. Prof. Hecken noted:
“The €30M rule is pragmatic, but we must ensure it doesn’t hinder robust evidence generation.”
- Post-2028 Reforms:
Stricter evidence demands are likely, even for small-revenue orphan drugs.
Strategic Tip:
–For ATMPs and rare diseases, collect real-world survival/HRQoL data pre-launch to pre-empt reforms.
7. Redactions and Timelines: Practical Tips
- Redacted Data = Ignored:
Blacked-out information in JCA reports (e.g., proprietary costs) cannot be used in AMNOG. Submit unredacted versions separately.
- Literature Searches: Must be <3 months old at AMNOG submission. Schedule updates 2-4 weeks pre-submission to avoid delays.
8. Strategic Implications for Therapy Areas
Rare Diseases
- Use JSCs to align PICOs with German subgroups (e.g., genetic variants).
- Leverage RWD to compensate for small trial populations.
ATMPs
- Pre-validate surrogates (e.g., vector shedding) with long-term outcomes.
- Engage the G-BA early to clarify comparator therapies (often “best supportive care”).
Key Recommendations:
- Dual PRO Strategy:
- Collect SF-36 for Germany, EQ-5D for EU cost-effectiveness.
- For rare diseases, validate disease-specific PROs early.
- JSC Early Engagement:
- Target June 2025 slots – only 4 drug and 3 device slots remain.
- Consider G-BA advice as an additional option
- Emphasize unmet needs and German-specific evidence plans.
- AMNOG Dossier Planning:
- Identify national add-ons early (e.g., German comparators, subgroups).
- Use modular appendices to avoid duplication or submit a full AMNOG dossier if local teams prefer.
- Post-JCA Mitigation:
- Prepare supplementary analyses for AMNOG if JCA surrogates are rejected.
- Use the hearing phase to integrate late JCA findings.
Conclusion: Walking the Tightrope
The integration of JCA and AMNOG requires a careful balance between EU-level harmonization and German-specific requirements. For rare diseases and ATMPs, success will depend on early evidence planning, strategic use of patient-reported outcomes, and anticipation of evolving regulatory expectations.
As Dr. Petra Nies aptly put it: “𝗔 𝗽𝗮𝘁𝗵 𝗶𝘀 𝗺𝗮𝗱𝗲 𝗯𝘆 𝘄𝗮𝗹𝗸𝗶𝗻𝗴 𝗶𝘁.” The G-BA acknowledges that JCA implementation is a shared learning curve—dynamic and still unfolding. Agility, transparency, and early alignment will be critical for manufacturers navigating this shifting landscape.
Need Support?
At P4A, we support biopharma companies across the full market access journey – from early strategy and JCA readiness to national submissions and stakeholder engagement. Our tailored solutions, including the PICO+ service, help you align evidence generation with both EU and national requirements – from Joint Scientific Consultations to AMNOG dossier optimization. Contact us to future-proof your access strategy and bring innovation to patients faster.
