At Pharma Design Limited, we empower developers of therapies for rare diseases and cancer to navigate the evolving European regulatory landscape. Recent EU and UK reforms offer significant advantages through joint advice mechanisms, enabling sponsors to align regulatory and market access strategies early. This article explores the process and why it’s a game-changer for achieving faster approvals and reimbursement.
Differently from other Regions, European Regulatory Agencies have historically never been involved with pricing & reimbursement decisions and they still aren’t. Other governmental Agencies take a leading role in assessing a fair market price for new pharmaceutical products. This dichotomy mandates the need for two separate processes in Europe, one for a Regulatory approval (MAA) and one for a Market Access assessment (HTA). These two processes have been managed separately leading to painful delays in price negotiation and reimbursement.
The Joint Advice Process: EMA and MHRA HTA Collaboration
Joint scientific consultations provide integrated feedback from regulatory (EMA/MHRA) and HTA bodies (e.g., HTACG for EU, NICE for UK), reducing silos and ensuring evidence meets both approval and reimbursement needs.
- EMA/HTACG Joint Scientific Consultation (JSC): For EU, request during open periods (e.g., January-February 2026) via the HTA IT Platform. Submit the JSC Request Template justifying unmet need and innovation. Timeline: 6-9 months to final report, involving scoping, assessment, and a discussion meeting. Documents needed: Briefing package with questions on evidence generation, endpoints, and economic models.
- MHRA/NICE Joint Advice: In the UK, request rolling basis via MHRA form (no ILAP required for basics). Process: 2-4 months, with separate but coordinated advice letters. Ideal for aligning MAA with HST/STA evaluations.
These mechanisms are particularly relevant for rare diseases (e.g., orphan designations) and cancer treatments, where data immaturity or small populations challenge traditional assessments. Sponsors gain clarity on endpoints like QALYs or progression-free survival, de-risking future submissions.
The Case for Concomitant MAA and HTA Preparation
Preparing MAA and HTA plans together saves time by integrating clinical data into economic models upfront. Under EU HTA Regulation (2021/2282), JCAs run parallel to MAA for ATMPs/oncology from 2025, mandating simultaneous dossiers. In the UK, the Aligned Pathway (2025 launch) synchronizes MHRA licensing with NICE appraisals.
Staggered vs. Joint Assessment Comparison
- Staggered Approach: MAA first (210 days EMA standard), then HTA (9-18 months post-approval). Total to reimbursement: 15-30 months. Delays arise from evidence gaps identified late, leading to resubmissions or appeals.
- Joint/Concomitant Approach: Parallel reviews cut overlap; e.g., JSC feedback refines MAA data for HTA. Potential savings: 3-12 months to reimbursement (e.g., UK Aligned Pathway targets 6 months faster NICE guidance). For rare/cancer therapies, this means quicker patient access and revenue.
By leveraging joint advice, sponsors like those in oncology or rare diseases achieve streamlined market entry. Pharma Design Limited has helped clients save 20-30% on timelines.
Concurrent MAA and HTA Strategies: Accelerating Reimbursement for Rare Diseases and Cancer Therapies
In today’s regulatory environment, concurrent preparation of Marketing Authorisation Applications (MAAs) and Health Technology Assessments (HTAs) is essential for therapies targeting rare diseases and cancer. At Pharma Design Limited, we’ve seen this approach transform timelines, driven by EU reforms and UK collaborations. This article highlights the processes and time-saving benefits.
EU and UK Joint HTA/Scientific Advice: A Unified Path
- EU JSC (EMA/HTACG): Mandatory JCAs for oncology/ATMPs from 2025 run alongside MAA, with dossiers submitted simultaneously. Request JSC early via HTA IT Platform (open periods like January 2026); documents: JSC Template, briefing on evidence/endpoints. Timeline: 6-9 months, yielding aligned regulatory/HTA insights.
- UK MHRA/NICE Joint Advice: Rolling requests via MHRA; 2-4 months process. The Aligned Pathway (full 2026) synchronizes MAA with HST/STA, incorporating patient/commercial input.
These enable sponsors to address unmet needs in rare diseases (e.g., orphans) or cancer (e.g., targeted therapies) holistically, ensuring data supports both approval and value assessment.
Time Savings: Staggered vs. Concomitant
- Staggered: Sequential MAA (210 days) then HTA (9-18 months) totals 15-30 months to reimbursement, with risks of evidence mismatches delaying access.
- Concomitant: Parallel processes cut 3-12 months; e.g., EU JCA/MAA overlap and UK Aligned Pathway enable reimbursement within 6-9 months post-approval. Recent funding trends (e.g., €100M+ for rare disease biotechs) underscore the need for efficiency.
Concurrent planning, informed by joint advice, accelerates market access while optimizing resources.