Gene Therapy Access & RWE Guidance

SaraAnd I'm Sara. Always good to be here — and I'll say, I've had today's topics circled since this morning. The gene therapy coverage shifts alone are keeping me on my toes.

SaraThat second one is interesting to me — the FDA's RWE guidance for post-marketing studies could fundamentally change how we approach confirmatory evidence. But the operational burden on companies could be substantial.

SaraThat's a significant shift. Payers will need robust real-world data to justify coverage decisions, especially for therapies with durable effects but high upfront costs. The budget impact question here is one the field hasn't fully worked out yet.

SaraI'd push back slightly on that. This isn't HTA — it's coverage policy with financial risk-sharing. But the methodological rigor CMS is demanding is approaching HTA standards. The part that gives me pause is the operational feasibility for small manufacturers.

SaraThat's the core tension. We need better tools to model long-term value, especially for curative therapies. The budget impact isn't just about the first year — it's about decades of avoided costs.

SaraShifting to FDA's draft guidance on RWE for post-marketing studies. They're explicitly allowing RWE to support traditional confirmatory trials and safety assessments. This is a major validation of RWE's evidentiary value.

SaraThat's one read — I'd frame it slightly differently. This could accelerate approval timelines by reducing the need for additional randomized trials. But the methodological requirements for RWE in confirmatory settings remain stringent.

SaraThe historical precedent is mixed. Remember the challenges with RWE in accelerated approvals? The part that gives me pause is the potential for inconsistent application across review divisions.

SaraExactly. This could reshape how we approach post-marketing evidence generation. The budget impact for companies could be significant if they need to build new RWE capabilities.

SaraThis is long overdue. Digital therapeutics challenge traditional HTA models because their effects aren't always captured in clinical endpoints. The real question is how NICE will handle the dynamic nature of these products — updates and iterations that aren't typical for drugs.

SaraI'd push back slightly. The framework explicitly addresses budget impact through its system impact dimension. But the methodology for valuing continuous data streams versus discrete interventions remains underdeveloped.

SaraThe historical precedent is the digital technology appraisal pathway, which took three years to finalize. But the political pressure to address digital health is stronger now. We'll see if this moves faster.

SaraFinally, the EU Critical Medicines Act accelerating HTA timelines. The provisional agreement includes provisions to reduce assessment timelines for critical medicines, particularly in oncology and rare diseases.

SaraThat's a valid concern. The acceleration is tied to the Critical Medicines designation, which requires demonstrating unmet need. But the methodological rigor needed for robust HTA doesn't compress easily. The part that gives me pause is the potential for inconsistent application across member states.

SaraExactly. And from the payer side, we need to ensure that faster assessments don't lead to lower-quality evidence. The budget impact of suboptimal HTA could be substantial for health systems.

SaraThat's fair, though I think industry would argue that patient access justifies the timeline pressure. The key will be maintaining scientific rigor while addressing unmet need.

SaraA lot to think about today. I'll be watching how CMS operationalizes the Part D gene therapy criteria and whether it creates meaningful access barriers for innovative therapies.

SaraThanks so much for listening — really glad you're here with us.

SaraThanks for listening — see you tomorrow.