FDA RWE Guidance & CMS Star Ratings

MarcusWelcome to the Access Brief — your daily briefing on what's moving in HEOR, HTA, and market access. I'm Marcus, and it's great to have you with us today.

SaraAnd I'm Sara. Always good to be here — and I'll say, today's topics are hitting at a critical moment where evidence generation is colliding with real-world policy pressures.

MarcusExactly. We're looking at FDA's draft guidance on using real-world evidence for post-marketing studies — which could fundamentally reshape how we confirm drug value post-approval. Then CMS's proposed changes to Medicare Advantage Star Ratings methodology — that's a direct pivot toward value-based metrics. And ICER's new framework for ultra-orphan gene therapies — addressing a notorious evidence gap. Finally, NICE's updated guidance on real-world evidence for technology appraisals — signaling deeper RWE integration.

SaraThat CMS Star Ratings shift is particularly interesting — the budget impact implications of tying quality metrics to reimbursement could create new access barriers if not calibrated carefully.

MarcusAbsolutely. Let's get into it.

MarcusStarting with the FDA draft guidance on RWE for post-marketing studies. This is significant because it formalizes pathways for using real-world data to confirm clinical benefits after approval, especially for accelerated approvals. The key question is how this will interact with the IRA's price negotiation triggers.

SaraRight, and what strikes me is the tension here. Payers will demand RWE to confirm value, but manufacturers face RWE collection challenges in rare diseases. The guidance mentions 'fit-for-purpose' data standards, but doesn't specify how that translates to reimbursement decisions.

MarcusThat's precisely the gap. The FDA emphasizes data quality and methodologies, but HTA bodies like NICE have their own RWE acceptance criteria. I wonder if this will lead to divergent evidence requirements across jurisdictions.

SaraThe part that gives me pause is the timeline. The guidance suggests RWE studies can be conducted within 2-3 years post-approval, but for complex conditions like Alzheimer's, that might be too short to capture meaningful outcomes. Payers will question the robustness.

MarcusAgreed. And historically, RWE acceptance has been inconsistent. What's new here is the explicit FDA endorsement for post-marketing confirmatory studies, which could shift payer expectations. Though I'd push back slightly — the guidance still limits RWE to supportive evidence rather than primary endpoints.

SaraThat's a crucial distinction. From the payer side, we need RWE to stand alone for reimbursement decisions, not just supplement clinical trial data. This feels like a half-step toward that goal.

MarcusFair point. But the industry will see this as progress toward faster evidence generation. The real test will be how CMS incorporates this guidance into coverage decisions. Moving on to CMS...

SaraSpeaking of CMS, their proposed changes to Medicare Advantage Star Ratings methodology are a seismic shift. They're adding new quality measures for chronic disease management and social determinants of health, with direct implications for reimbursement adjustments.

MarcusAbsolutely. What's fascinating is how this ties into value-based contracts. If plans can achieve higher Star Ratings through better outcomes, they might be more willing to cover innovative therapies. But the budget impact could be substantial if every plan starts demanding outcomes-based payments.

SaraExactly. And what I'm watching closely is how this interacts with the Part D Redesign. Higher Star Ratings could trigger bonus payments, but if plans prioritize these new metrics over drug access, we might see formulary restrictions for high-cost drugs.

MarcusThat connects to something I keep coming back to — the operational burden. Collecting real-world data for these new metrics will require new infrastructure. HEOR teams will need to design studies that align with both FDA guidance and CMS requirements, which is no small feat.

SaraThe methodology shift also raises questions about equity. Social determinants measures could disadvantage plans in underserved areas, potentially creating perverse incentives. Payers will need robust adjustment methodologies to avoid that.

MarcusThat's a valid concern. Though I'd frame it slightly differently — this could actually drive better patient outcomes if implemented well. The challenge is ensuring the metrics don't become another barrier to access. Now, turning to ICER's gene therapy framework...

MarcusICER's new framework for ultra-orphan gene therapies addresses a critical evidence gap. They're proposing a separate assessment pathway with modified cost-effectiveness thresholds and emphasis on long-term outcomes, recognizing these therapies' unique value profiles.

SaraThis is long overdue. The current one-size-fits-all approach has failed for ultra-orphan drugs. What ICER is signaling is that budget impact isn't the only metric — we need to consider broader system value and innovation incentives.

MarcusAbsolutely. But the practical implications are complex. How do you quantify 'broader system value' in a way that's consistent across HTA bodies? The framework mentions multi-criteria decision analysis, but that's notoriously subjective.

SaraThat's where RWE becomes critical. These therapies require decades of follow-up data, which we don't have. ICER is suggesting modeling approaches, but payers will demand real-world confirmation. It creates a chicken-and-egg problem for evidence generation.

MarcusExactly. And from an industry perspective, this framework could encourage more investment in ultra-orphan therapies, but only if payers actually adopt the higher thresholds. The proof will be in how NICE and others respond. I wonder if this signals a transatlantic convergence on rare disease value assessment.

SaraThat's one read — I'd frame it slightly differently. This feels more like an acknowledgment that traditional HTA methodologies break down for these therapies. The question is whether this becomes a precedent for other high-cost innovations.

MarcusFair point. The real test will be implementation. If ICER's framework leads to more consistent coverage decisions, it could be transformative. But if it just creates another layer of complexity, we're back to square one. Finally, NICE's RWE guidance...

SaraNICE's updated guidance on real-world evidence for technology appraisals is a significant evolution. They're explicitly allowing RWE to address evidence gaps in clinical trials, particularly for real-world effectiveness and long-term outcomes.

MarcusThis aligns with what we're seeing globally — HTA bodies moving beyond RCT limitations. What's notable is NICE's emphasis on 'fit-for-purpose' RWE studies, which could accelerate evidence generation for drugs with incomplete trial data.

SaraBut what strikes me is the methodological rigor they're demanding. The guidance requires predefined statistical analysis plans and sensitivity analyses, which adds development time. For companies with patent cliffs, that's a real commercial concern.

MarcusThat's a fair point. Though I'd push back slightly — the clarity could actually help manufacturers plan better evidence strategies. The bigger question is how this interacts with the new £50k threshold consultation. Will RWE studies become mandatory for drugs above that threshold?

SaraThat's precisely the tension. Payers want RWE to confirm value, but manufacturers need predictable pathways. The guidance suggests RWE can supplement clinical data, but doesn't specify when it becomes mandatory. That ambiguity creates risk.

MarcusAnd it connects back to the FDA guidance we discussed earlier. If NICE and FDA have different RWE acceptance criteria, we'll see divergent evidence requirements. The industry will need to navigate that carefully. What's your forward-looking take on this convergence?

SaraI'll be watching how NICE operationalizes this in their first few appraisals under the new guidance. If they accept RWE for decision-making, it could shift the entire value narrative. If they treat it as supplementary, we'll continue seeing access challenges for innovative therapies.

MarcusSame — and for me the thread running through today is how evidence generation is becoming more decentralized but also more complex. The opportunities are real, but the operational burdens are growing. Worth sitting with.

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

MarcusWe'll be back tomorrow. Show notes and transcripts at outcomes-analytica.no. See you then.

SaraThanks for listening — see you tomorrow.

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