RNA Breakthroughs, Critical Medicines Act, and Evidence Judgment Era

MarcusWelcome to Access Brief, the daily AI podcast on HEOR, HTA, and market access. I'm Marcus, with Sara. Today: the Critical Medicines Act provisional agreement, EMA's first RNA veterinary vaccine approval, and ISPOR's evidence judgment shift. Let's get into it.

SaraThe Critical Medicines Act provisional agreement hit on 12 May. Parliament and Council have positioned EMA's Executive Steering Group on Shortages with an expanded mandate for supply chain vulnerability assessments across 200-plus active substances on the Union critical medicines list.

MarcusThe dependency numbers are stark. Eighty percent of imported pharmaceutical ingredients come from just five countries - China, US, UK, Indonesia and India - with China accounting for 45% alone. This formalises the MSSG role in conducting assessments to inform strategic projects and public procurement.

SaraHere's where I push back on the framing. We're treating this like a pure supply chain resilience play, but there's a market access angle everyone's missing. When you create a Union critical medicines list with vulnerability assessments, you're essentially creating a two-tier system. Products on that list will have different commercial dynamics.

MarcusHow so? The assessments are about supply security, not pricing or access pathways.

SaraSupply security becomes leverage. If your molecule is deemed critical and supply-vulnerable, that changes your negotiating position with payers. It's not just about diversifying manufacturing - it's about redefining what constitutes essential medicines and who controls that definition.

MarcusMoving to regulatory precedents. EMA's CVMP recommended authorisation for Nobivac NXT HCPChFeLV on 17 April - the first veterinary vaccine in the EU containing self-amplifying RNA as an active substance. Data from 15 studies and one field study with 142 cats showed adequate immune responses against five pathogens.

SaraThe veterinary precedent matters more than people realise. RNA technology validation in animal health creates regulatory pathway clarity that human medicine can leverage. We're seeing immunity starting around one week after vaccination with duration of immunity of 3 years for feline panleucopenia virus and 1 year for four other pathogens.

MarcusThe February CHMP highlights reinforce this RNA momentum. The committee recommended mCombriax - the first combined messenger RNA vaccine for people aged 50 years and older against COVID-19 and seasonal influenza. Plus conditional marketing authorisation for Ojemda for paediatric low-grade glioma and extended Dupixent use for chronic spontaneous urticaria in children.

SaraISPOR's 2026-2027 trends report captures the broader shift we're seeing. AI rises to number one from number three in the previous edition. Real-world evidence moves to number two, but the focus is shifting from access to data quality, transparency and methodological rigour.

MarcusValue-based healthcare appears for the first time at number three as it moves from concept to implementation. But here's the key insight from industry analyst David Miller: "We are no longer being evaluated on our ability to produce data; we are being judged on our ability to influence decisions under constraint."

SaraThat Miller quote is the entire game right there. The era of evidence judgment. But I think we're overestimating how ready the field is for this transition. Most HEOR teams are still optimised for data production, not decision influence.

MarcusDisagree. The constraint environment is forcing the evolution. Look at EU JCA capacity - the HTACG is planning 8 to 12 joint scientific consultations on medicinal products and 2 to 5 on medical devices. They estimate around 35 joint clinical assessments for new active substances in cancer treatment and around 15 for advanced therapy medicinal products. The constraint is built into the system.

SaraConstraint exists, but constraint doesn't automatically create judgment capability. You can limit slots and create bottlenecks, but that doesn't mean the evidence being produced is better tailored for decision-making. We're still seeing teams submit traditional dossiers to constrained systems rather than fundamentally rethinking evidence strategy.

MarcusThe medical device JCA expansion starting in June will test that thesis. Approximately 5 joint clinical assessments expected to start, and we'll see whether device manufacturers approach evidence differently than pharma.

SaraDevice manufacturers might actually be better positioned for this transition. They're used to working with limited clinical data and making compelling cases under uncertainty. Pharma's still addicted to the comprehensive dossier approach.

MarcusOne positive data point - NICE statins uptake shows evidence translation working. Atorvastatin at NICE-recommended doses increased from 4.2 million people in 2022/23 to 5 million in 2023/24. Among adults with CVD, 85% were on cholesterol lowering treatment in June 2024, up from 82% in June 2023.

SaraIf 95% coverage were achieved, around 17,300 cardiovascular events and 2,000 deaths could be avoided. That's evidence judgment working - moving from guideline to implementation with measurable outcomes.

MarcusThe question is whether we can replicate that systematic approach across the more complex interventions coming through RNA technology and advanced therapies.

SaraAnd whether HTA bodies can adapt their assessment frameworks fast enough to match the pace of innovation. The Critical Medicines Act and JCA expansion suggest they're trying, but the jury's still out on execution.

MarcusBack tomorrow on Access Brief. Show notes at outcomes-analytica.no.