HTA Method Shifts & IRA Implications

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, I've had today's topics circled since this morning, especially the Asia-Pacific piece.

MarcusSame here. We're looking at HTA methodological shifts in Asia-Pacific — how they're diverging from Western models. Then the IRA's impact on HEOR evidence generation — forcing new data approaches. Plus, patient-reported outcomes in value assessments — where the gaps remain. And real-world evidence in HTA submissions — implementation hurdles.

SaraThat second one is particularly urgent — the budget impact question there is one the field hasn't fully worked out yet. Payers are demanding more longitudinal data, but timelines are collapsing.

MarcusExactly. Let's get into it.

MarcusStarting with Asia-Pacific HTA shifts. We're seeing countries like Thailand and Malaysia adopting hybrid value frameworks earlier than Europe. They're integrating budget impact more directly into early value assessments, not just cost-effectiveness. This creates a different evidence calculus for sponsors.

SaraThat's one read — I'd frame it slightly differently. What strikes me is how these frameworks are becoming more locally contextualized. Thailand's HTA body now requires country-specific utility weights, which forces sponsors to generate region-specific PRO data. That's a significant operational shift.

MarcusAbsolutely. And it connects to something I keep coming back to: the tension between global evidence harmonization and local adaptation. Sponsors are having to run parallel evidence generation tracks, which strains resources.

SaraThe part that gives me pause is the inconsistency across the region. Singapore adopts NICE-style thresholds, while Vietnam uses cost-minimization as a primary gatekeeper. This fragmentation makes regional launch strategies incredibly complex. How are companies navigating this patchwork?

MarcusThrough targeted HEOR investments. We're seeing more local RWE studies funded specifically for ASEAN submissions, often leveraging federated networks. But the timelines are brutal — sometimes 6-9 months for localized evidence packages.

SaraThat's fair, though I think payers would see this as necessary friction. The alternative is generic Western models that don't capture local disease burdens or healthcare systems. The question is whether the evidence burden is becoming disproportionate to the market size.

MarcusThat's the open question. Some argue it's creating two-tiered access, but others see it as value recognition for diverse populations. What's clear is that sponsors can't treat Asia-Pacific as a monolith anymore.

SaraShifting to IRA implications. The Inflation Reduction Act is fundamentally altering how sponsors generate evidence for Medicare negotiation. CMS is now requiring post-marketing real-world evidence for drugs entering Part D, particularly for oncology and neurology.

MarcusI wonder if that's the full picture though. The IRA's impact goes beyond just RWE generation. It's creating a bifurcated evidence landscape — drugs subject to negotiation need robust real-world data, while non-negotiable drugs focus on traditional endpoints. This forces companies to make strategic evidence allocation decisions.

SaraExactly. And what's striking here is the timing pressure. CMS expects 2-3 years of RWE within 18 months of launch. Sponsors are compressing evidence generation timelines, which raises methodological concerns. How are they ensuring data quality while meeting these deadlines?

MarcusThrough adaptive trial designs and leveraging existing RWE networks. But there's a risk of data overload — payers are struggling to synthesize multiple data streams. The real challenge is demonstrating clinical utility beyond just price negotiation.

SaraThat's where budget impact becomes critical. Payers need to see how these drugs fit into existing formularies and budgets. The IRA is accelerating the shift from pure cost-effectiveness to system-wide value assessments. Sponsors who ignore this are at a disadvantage.

MarcusRight. And from the payer side, it's about opportunity cost. Every dollar spent on a high-price drug is a dollar not spent on others. The IRA is making that trade-off more explicit. Sponsors need to build stronger value narratives around total system impact.

MarcusNow to patient-reported outcomes. HTA bodies are increasingly demanding PRO data as primary endpoints, especially in chronic conditions. But the methodology gaps are significant — particularly in defining minimally important differences and handling missing data.

SaraI'd push back slightly on that. The issue isn't just methodology; it's about interpretation. NICE's new PRO framework gives more weight to patient preference data, but EMA still treats PRO as supplementary. This creates divergent expectations for sponsors. How are companies reconciling these differences?

MarcusThrough targeted evidence generation strategies. For EU submissions, they're combining PRO data with clinician-reported outcomes. For US submissions, they're emphasizing PRO data in risk-benefit analyses. But the resource burden is substantial — sometimes requiring dedicated PRO studies.

SaraThat's one read — I'd frame it slightly differently. What's missing is the patient voice in defining what matters. HTA bodies are increasingly engaging patients in outcome selection, but sponsors often default to legacy PRO instruments. This disconnect can lead to evidence that doesn't reflect patient priorities.

MarcusThat's a fair point. The latest trend is co-designing PRO measures with patient advocacy groups. It's more resource-intensive but yields more relevant data. Payers are responding positively to these approaches in their value assessments.

SaraAnd the budget impact question here is significant. If PRO data show meaningful quality-of-life improvements, payers may be more willing to accept higher prices. But the evidence must be robust — no more cherry-picked PRO endpoints that overstate benefits.

SaraFinally, real-world evidence in HTA submissions. Adoption is accelerating, particularly for drugs with complex mechanisms of action. But the implementation barriers are substantial — data quality issues, lack of standardized endpoints, and payer skepticism about generalizability.

MarcusWhat strikes me is how RWE is moving from supplementary to primary evidence. We're seeing HTA bodies like NICE accepting RWE as the sole basis for reimbursement in certain oncology indications, especially when traditional trials aren't feasible. This is a paradigm shift.

SaraThat's true, but the methodological challenges remain. Payers are particularly concerned about confounding in RWE studies. The latest guidance from CADTH emphasizes the need for propensity score matching and sensitivity analyses, which increases the burden on sponsors.

MarcusExactly. And it connects to something I keep coming back to: the need for better data infrastructure. Federated networks are helping, but interoperability between healthcare systems remains a hurdle. Sponsors are having to invest heavily in data cleaning and validation.

SaraThe part that gives me pause is the timeline. RWE studies often take 12-18 months to generate and analyze, while HTA submissions have 6-month windows. This mismatch creates significant bottlenecks. How are companies managing this?

MarcusThrough proactive evidence generation. The most sophisticated sponsors are launching RWE studies alongside Phase III trials, creating a continuous evidence pipeline. They're also leveraging real-world data from multiple sources to strengthen the evidence base.

SaraThat's fair, though I think payers would see this as necessary. The alternative is making decisions with incomplete evidence. The key is ensuring RWE meets the same methodological rigor as clinical trial data. Otherwise, it risks undermining trust in the entire HTA process.

SaraA lot to think about today. I'll be watching how Asia-Pacific HTA frameworks evolve, particularly whether they start converging or diverging further. The fragmentation is creating significant access challenges.

MarcusSame — and for me the thread running through today is the growing evidence burden across all jurisdictions. Sponsors are being asked to do more with less time, which raises questions about sustainability. 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.