CHMP Momentum, Critical Medicines Act, HTA Capacity Scaling

MarcusCHMP delivered eight approvals at their May meeting, including nerandomilast for idiopathic pulmonary fibrosis and progressive pulmonary fibrosis. That brings them to 36 positive opinions year-to-date. The pulmonary fibrosis space is finally seeing movement after years of limited options.

SaraConditional approval for alpelisib in PIK3CA-related overgrowth spectrum also caught my attention. These rare disease approvals are critical for establishing EU HTA precedents before the regulation expands. Each one tests how joint clinical assessments handle complex benefit-risk profiles in small populations.

MarcusThe regulatory momentum is undeniable, but I'm watching how these approvals translate into HTA preparedness. Thirty-six positive opinions means thirty-six potential JCA candidates once the scope expands. Are companies building dossiers that can handle both EMA's scientific standards and HTACG's clinical assessment framework?

SaraThat's the fundamental tension. EMA approval gets you market authorization, but it's no longer sufficient for access. The evidence packages that satisfy CHMP won't automatically work for joint clinical assessments. Companies need parallel evidence strategies from Phase III planning forward.

MarcusThe Critical Medicines Act reached provisional political agreement on May 12th. EMA welcomed it as building on their extended mandate to reinforce manufacturing capacity and supply resilience. This isn't just about availability – it's about creating a framework where critical medicines get prioritized treatment.

SaraBut here's where it gets interesting for market access. If medicines are designated as critical, do they get expedited HTA pathways? The Act focuses on supply chain resilience, but the commercial implications could be significant. Priority designation could mean faster joint clinical assessments or different benefit-risk thresholds.

MarcusI'm not seeing evidence of expedited HTA treatment yet. The Act complements pharmaceutical legislation but doesn't explicitly create HTA fast tracks. We're still dealing with the same 17 scientific advice slots and capacity constraints.

SaraTrue, but political agreements create momentum. If Parliament and Council prioritize supply resilience, that pressure flows to HTA bodies. The designation process could become a de facto access accelerator even without explicit pathways.

MarcusThe HTAR Capacity Building Programme is expanding with five eLearning modules available in English plus six other EU languages starting end of May. This represents serious investment in sustainable HTA capacity ahead of the five-year expansion.

SaraMultilingual capacity building is essential, but I question whether eLearning modules address the fundamental bottleneck. We need more assessors, more coordinators, more infrastructure. Training existing people better doesn't solve the throughput problem.

MarcusFair point, but you have to build competency before you can scale personnel. These modules standardize assessment approaches across member states. Without common methodological foundations, adding more assessors just creates inconsistent outputs.

SaraI'll grant you that standardization is necessary. But while we're building perfect training programmes, companies are struggling with 17 scientific advice slots for all of Europe. The capacity constraints are immediate and growing worse as more sponsors recognize they need early HTA engagement.

MarcusThe HTA Stakeholder Network membership got extended for all current members by another three years starting May 4th. No resignations from members or observers – that's remarkable stakeholder engagement in a system still finding its footing.

SaraIt shows confidence in the long-term framework, but also reveals how much work remains. Stakeholders are staying engaged because they recognize the system will eventually matter enormously for European access. But we're still in the early implementation phase with significant operational challenges.

MarcusHTACG estimates 35 joint clinical assessments for new oncology active substances and 15 ATMPs in 2026, plus the first 5 high-risk medical device assessments starting June. That's ambitious scaling from the current limited scope.

SaraThose numbers highlight the disconnect between ambition and capacity. Thirty-five oncology JCAs with existing infrastructure constraints? We're setting up for significant delays unless capacity building accelerates dramatically.

MarcusThe system is maturing, but operational realities are limiting actual impact. Companies are building parallel evidence strategies because they can't rely on EU HTA timelines aligning with commercial needs.

SaraExactly. Until capacity matches scope ambitions, EU HTA remains a valuable but unpredictable component of access strategies rather than the central coordinating mechanism it's designed to become.

MarcusThe framework is solidifying, but implementation bottlenecks are defining real-world outcomes more than policy intentions.

SaraEvidence strategies need to account for both the system's eventual impact and its current limitations. Back tomorrow. Show notes at outcomes-analytica.no.