HTA Evolution & AI Governance

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. The Asia-Pacific HTA landscape is shifting faster than many realize.

MarcusSame here. We're looking at HTA methodological shifts in Asia-Pacific — how jurisdictions are redefining value assessment beyond traditional cost-effectiveness. Then RWE integration challenges in Latin America — why real-world data adoption is stalling despite regulatory pushes. And gene therapy HEOR strategies in Asia — how companies are navigating fragmented markets for ultra-orphan therapies. Finally, digital therapeutics HTA challenges — the evidence gaps that are delaying coverage decisions.

SaraThat second one is particularly interesting — the budget impact question there is one the field hasn't fully worked out yet. Latin America's RWE fragmentation creates massive opportunity costs for health systems.

MarcusExactly. Let's get into it.

MarcusStarting with HTA methodological shifts in Asia-Pacific. What's striking here is how rapidly jurisdictions are moving beyond cost-per-QALY thresholds. Japan's recent framework incorporating social value and South Korea's emphasis on patient-reported outcomes are creating a patchwork of value definitions that companies must navigate.

SaraThat's one read — I'd frame it slightly differently. What strikes me about that is how these shifts are fundamentally altering risk-sharing agreements. We're seeing more performance-based pricing models in Singapore and Malaysia, but the criteria vary so dramatically that HEOR teams are essentially developing country-specific value propositions.

MarcusThat connects to something I keep coming back to — the methodological flexibility in Asia-Pacific is creating both opportunities and headaches. The real question is whether this fragmentation will lead to more innovation-friendly environments or just increase development costs.

SaraI'd push back slightly on that. While the flexibility exists, the operational challenges are immense. HTA bodies in the region are struggling with inconsistent evidence standards, and that's creating real access delays for genuinely innovative therapies. The part that gives me pause is how this inconsistency affects patient access across borders.

MarcusFair point. And what's interesting is how some companies are responding by developing modular evidence packages that can be adapted across jurisdictions. But that raises its own questions about resource allocation — is this the most efficient use of HEOR budgets?

SaraThat's a practical industry perspective, though I think payers would see it differently. For systems already stretched thin, the burden of reconciling multiple value frameworks is unsustainable. The solution needs to come from greater harmonization, not just more flexible submissions.

MarcusExactly. And we're seeing some early signs of that through ASEAN HTA initiatives. The pace is slow, but the direction is clear. The patient perspective demands that we find a way to balance innovation with system sustainability.

SaraShifting to RWE integration challenges in Latin America. The gap between regulatory acceptance and actual HTA adoption is wider here than in any other region. Brazil's CONITEC has published guidance on RWE use, but implementation remains sporadic. Mexico's COFEPRIS accepts RWE for certain submissions, but health technology assessment bodies rarely incorporate it into final decisions.

MarcusThat's one read — I'd frame it slightly differently. What strikes me about that is how infrastructure limitations are the primary bottleneck. Many countries lack the data infrastructure to generate or validate RWE, and that's not just a technical issue but a capacity problem that requires significant investment.

SaraThat connects to something I keep coming back to — the opportunity cost argument. Health systems in Latin America are making coverage decisions based on limited evidence, not because they're opposed to RWE, but because they lack the resources to generate it properly. The part that gives me pause is how this perpetuates a cycle where only wealthy countries can access innovative therapies supported by robust RWE.

MarcusI wonder if that's the full picture though. There are examples like Colombia's use of RWE in oncology that show it's possible to overcome infrastructure challenges with targeted approaches. The key is developing context-specific RWE strategies rather than expecting one-size-fits-all solutions.

SaraThat's fair, though I think payers would see it differently. The issue isn't just generation but interpretation. Even when RWE is available, HTA bodies often lack the expertise to evaluate it appropriately, leading to inconsistent decisions. The solution requires both capacity building and methodological standardization.

MarcusExactly. And we're seeing some promising developments through PAHO's regional RWE initiatives. The challenge is scaling these efforts across the diverse health systems in the region. From an innovation perspective, this is a critical market where RWE could unlock access for many therapies.

SaraAbsolutely. The patient impact of these delays is significant, and the field needs to develop more efficient pathways for generating and validating RWE in resource-constrained settings. That's where the real opportunity lies.

MarcusNow to gene therapy HEOR strategies in Asia. The ultra-orphan therapies are creating unique value assessment challenges across the region. Japan's innovative pricing model for one-time treatments and China's pathway for ultra-rare diseases are creating distinct evidence requirements that companies must navigate carefully.

SaraThat's one read — I'd frame it slightly differently. What strikes me about that is how the long-term value arguments are being reshaped. In jurisdictions like South Korea and Taiwan, the focus is shifting from traditional cost-effectiveness to lifetime value and system-wide impact, which requires fundamentally different evidence generation approaches.

MarcusThat connects to something I keep coming back to — the market fragmentation is creating complex portfolio strategies. Companies are developing tiered pricing models that account for different health system capacities, but this raises questions about equity and sustainable reimbursement across the region.

SaraI'd push back slightly on that. While fragmentation exists, there are emerging patterns in how jurisdictions are approaching ultra-orphan therapies. The part that gives me pause is how some countries are implementing risk-sharing agreements that create access barriers for therapies that don't meet specific outcome thresholds, potentially limiting patient options.

MarcusThat's a valid concern. And what's interesting is how some companies are responding by developing real-world evidence programs specifically designed to address these outcome requirements. The challenge is balancing the need for robust evidence with the urgency of patient access.

SaraThat's a practical industry perspective, though I think payers would see it differently. The sustainability of these therapies depends on demonstrating clear value beyond just clinical outcomes. The solution requires more sophisticated value frameworks that can capture the full impact of these transformative treatments.

MarcusExactly. And we're seeing some innovative approaches through regional collaborations like the Asia-Pacific Rare Disease Network. The key is developing evidence strategies that are both rigorous and adaptable to different market contexts. The patient perspective demands that we find ways to make these therapies accessible while ensuring system sustainability.

SaraFinally, digital therapeutics HTA challenges. The evidence gaps are particularly pronounced here. While regulatory approvals are increasing, HTA bodies are struggling with how to value interventions that require ongoing patient engagement and generate data continuously rather than through discrete clinical trials.

MarcusThat's one read — I'd frame it slightly differently. What strikes me about that is how the reimbursement models haven't caught up with the technology. Traditional fee-for-service models don't work for digital therapeutics, and we're seeing inconsistent approaches to coverage across jurisdictions, with some countries creating specific pathways while others default to existing frameworks.

SaraThat connects to something I keep coming back to — the patient-reported outcomes are central to value assessment but difficult to measure consistently. The part that gives me pause is how the lack of standardized endpoints is creating coverage uncertainty that discourages investment in these potentially transformative therapies.

MarcusI wonder if that's the full picture though. There are examples like Germany's early coverage decisions for digital therapeutics in diabetes that show it's possible to develop appropriate value frameworks. The key is developing evidence packages that demonstrate both clinical impact and system-wide benefits.

SaraThat's fair, though I think payers would see it differently. The issue isn't just evidence generation but cost-effectiveness assessment. Traditional HTA models struggle with interventions that have upfront development costs but ongoing value generation. The solution requires new methodologies that can capture the dynamic nature of these interventions.

MarcusExactly. And we're seeing some promising developments through ISPOR's digital health special interest group. The challenge is developing consensus on appropriate endpoints and valuation approaches. From an innovation perspective, this is a critical area where the field needs to evolve quickly to realize the potential of digital therapeutics.

SaraAbsolutely. The patient benefits of these interventions could be transformative, but the field needs to develop more efficient pathways for demonstrating value. That's where the real opportunity for both innovation and access lies.

SaraA lot to think about today. I'll be watching how Asia-Pacific HTA frameworks evolve in response to gene therapies and digital interventions — the methodological innovations there could reshape global approaches.

MarcusSame — and for me the thread running through today is how regional fragmentation is driving both innovation and complexity in value assessment. The challenge will be finding balance between local adaptation and global harmonization.

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.