EU HTA Regulation: Mastering Joint Scientific Consultations for Market Access Success

Joint Scientific Consultations (JSCs) under the EU HTA Regulation are critical for aligning EMA and HTA expectations early in development. Sponsors should aim for late Phase II/early Phase III, prepare a robust briefing dossier, and anticipate differences between regulatory and HTA perspectives. A well-executed JSC can de-risk reimbursement hurdles and accelerate patient access.

Key Steps in the JSC Process – With Practical Examples

1. Request Submission

Sponsors apply to the HTA Coordination Group during defined request windows. Acceptance is confirmed within ~15 working days. Example: A cell therapy developer submitted its JSC request in Q2 2025 to align with EMA’s SAWP calendar. Early engagement secured a slot before Phase III protocol finalization.

2. Briefing Package Preparation (D –30)

Submit a comprehensive dossier including trial design, endpoints, and specific questions. Tip: Use the HTA common template and PICO framing for clarity.

Example: An oncology sponsor included a proposed comparator and asked: “Would HTA bodies accept PFS as a primary endpoint if OS is collected in an extension study?” This proactive question avoided later disputes.

3. Clarifications & Validation (D –15 to D 0)

HTA and EMA review the dossier and request clarifications. Sponsors submit an amended package by Day 0.

Example: A gene therapy company was asked to add health-related quality-of-life measures (EQ-5D) to its pivotal trial plan—an HTA priority often overlooked by sponsors.

4. Expert Input & Issue Lists (D +30 to D +40)

External experts (clinicians, patient reps) provide feedback. EMA and HTA draft Lists of Issues highlighting critical concerns.

Example: For a rare disease ATMP, HTA bodies questioned the feasibility of a single-arm design and suggested an external control cohort using registry data.

5. Joint Discussion Meeting (D +60)

A 2–3 hour virtual meeting co-chaired by EMA and HTA representatives. Sponsors present responses and discuss contentious points.

Example: In one JSC, EMA accepted a surrogate endpoint for accelerated approval, while HTA insisted on OS data for cost-effectiveness modeling. The sponsor agreed to collect OS in a post-launch registry—an outcome that satisfied both parties.

6. Outcome Documents (D +70)

Sponsors receive EMA’s official Scientific Advice letter and HTA’s consolidated outcome document. These guide Phase III design and evidence generation.

Example: A biologics developer revised its trial to include an active comparator after HTA feedback, avoiding a potential negative reimbursement decision later.

Strategic Best Practices – With Real-World Insights

1. Plan Early & Align Teams

Start JSC planning as soon as Phase II data show a path to registration. Involve regulatory, clinical, and HEOR teams.

Example: A mid-sized biotech formed a cross-functional JSC task force six months before request submission, ensuring alignment on trial design and value proposition.

2. Ask Targeted Questions

Focus on comparators, endpoints, and evidence gaps—not pricing.

Example Questions: “Is Comparator X acceptable for EU HTA bodies, or should we consider Y?” “Would HTA accept PFS as primary if OS and QoL are secondary?”

3. Anticipate EMA vs. HTA Differences

EMA may accept placebo-controlled trials; HTA demands active comparators and QoL data.

Example: A sponsor planning a placebo-controlled oncology trial used the JSC to agree on an indirect comparison strategy for HTA purposes.

4. Prepare for Divergence & Document Decisions

Create a matrix of recommendations and mitigation plans.

Example: When HTA requested subgroup analyses by biomarker status, the sponsor added stratification to its Phase III design—avoiding later criticism.

5. Leverage Post-Launch Evidence

HTA bodies often expect registries or long-term follow-up.

Example: A CAR-T therapy developer agreed to a European registry during JSC discussions, enabling conditional reimbursement in Germany and France.

6. Logistics & Compliance

Meet all deadlines, ensure dossier quality, and manage EMA fees.

Example: A sponsor missed the HTA IT platform setup deadline, delaying validation by two weeks—highlighting the importance of early technical preparation.

Conclusion

The EU HTA Regulation makes early integration of regulatory and HTA requirements essential. JSCs are your opportunity to align expectations, optimize trial design, and avoid reimbursement roadblocks. For sponsors of biologics and ATMPs, mastering JSC strategy is not optional—it’s the key to accelerating patient access across Europe.

Ready to navigate the new EU HTA landscape? MF BIOTECH offers hands-on expertise to help you prepare for JSCs and design evidence strategies that satisfy both EMA and HTA requirements.

References

European Commission. Regulation (EU) 2021/2282.

HTA Coordination Group. Procedural Guidance for JSC (Nov 2024).

EMA Scientific Advice Working Party materials.