HTA Method Shifts & RWE Expansion

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, particularly the Asia-Pacific HTA shifts.

MarcusSame here. We're looking at HTA methodological shifts in Asia-Pacific — why these frameworks are diverging from Western models. Then RWE integration challenges in Latin America — where evidence gaps are creating real access barriers. Plus, gene therapy HEOR strategies in Asia — how sponsors are adapting to fragmented systems. And digital therapeutics clinical utility — moving beyond theoretical claims to real-world impact.

SaraThat second one is critical — the budget impact question there is one the field hasn't fully worked out yet. Latin America's heterogeneity makes RWE adoption messy.

MarcusExactly. Let's get into it.

MarcusHTA bodies in Asia-Pacific are increasingly adopting hybrid value frameworks that blend cost-effectiveness with broader system impact metrics. Singapore’s Health Sciences Authority just piloted a multi-criteria decision analysis model incorporating health technology assessment alongside societal value, signaling a departure from pure QALY thresholds. This reflects regional priorities beyond pure cost-effectiveness.

SaraThat’s one read — I’d frame it slightly differently. What strikes me is how these shifts create new evidence demands. Singapore’s pilot explicitly requires real-world data on patient equity impacts, which wasn’t standard five years ago. The part that gives me pause is how this complicates submissions. Sponsors now need parallel evidence packages for Western HTA bodies and these hybrid frameworks.

MarcusThat’s fair, though I think payers would see it differently — they’re getting more nuanced assessments but also facing longer review times. The Japanese Ministry of Health’s 2026 guidelines now mandate comparative effectiveness research against local standards of care, not just placebo-controlled trials. This is one of those stories where regional innovation is forcing evidence generation to evolve.

SaraAnd what’s striking here is how it creates a two-tiered evidence strategy. Companies are investing in region-specific RWE studies just for Asia-Pacific submissions. I keep coming back to the operational burden — smaller HEOR teams are struggling to maintain parallel evidence streams.

MarcusExactly, and what’s interesting is how this connects to something I keep coming back to: the patient perspective. These hybrid frameworks often prioritize outcomes that matter most to local populations, like caregiver burden or productivity loss. That’s a net positive, but it means HEOR teams need deep local clinical knowledge to design meaningful endpoints.

SaraThat’s true, but the methodological rigor is uneven. Thailand’s HTA agency recently accepted surrogate endpoints for oncology drugs without long-term survival data, while South Korea’s Health Insurance Review and Assessment Service still demands full OS data. This fragmentation makes harmonization nearly impossible.

MarcusRight, and from the payer side, that’s precisely why hybrid frameworks are emerging — they’re trying to balance rigor with pragmatic needs. The key takeaway is that Asia-Pacific isn’t monolithic; each country’s evolution requires tailored evidence strategies.

MarcusRWE adoption in Latin America is accelerating but facing structural barriers. Brazil’s CONITEC recently accepted real-world data for a hemophilia drug’s cost-effectiveness model, yet Mexico’s COFEPRIS still requires randomized controlled trial data for reimbursement decisions. This divergence creates uncertainty for sponsors.

SaraI wonder if that’s the full picture though. The real challenge is data infrastructure gaps. Argentina’s public health system lacks centralized EHRs, making it nearly impossible to generate robust RWE. That’s one of those stories where policy intentions outpace technical capacity. The part that gives me pause is how this affects budget impact — without reliable RWE, payers can’t model real-world utilization patterns.

MarcusThat’s a fair point. Brazil’s experience shows that even when HTA bodies accept RWE, the data quality requirements are stringent. They now mandate longitudinal data from at least three centers, which strains local research networks. Honestly, this one caught me off guard — I’d assumed RWE integration would be further along given the region’s need for cost-efficient evidence.

SaraAnd what’s striking here is the funding asymmetry. Multinational sponsors can afford to invest in local RWE generation, but local manufacturers often can’t. This creates an access gap where innovative therapies struggle to demonstrate value without the evidence infrastructure. I keep coming back to the opportunity cost — resources spent on RWE generation could fund patient access programs.

MarcusExactly, and what’s interesting is how this connects to something I keep coming back to: the regulatory landscape. Chile’s ISP recently relaxed RWE requirements for drugs with orphan designation, but Colombia’s INVIMA still requires post-marketing studies. This patchwork approach forces sponsors to navigate inconsistent regulatory expectations.

SaraThat’s true, but the methodological challenges run deeper. Latin America’s diverse healthcare systems mean RWE from one country may not generalize. Brazil’s SUS data can’t predict utilization in Mexico’s IMSS system. This fragmentation makes cross-jurisdictional evidence generation inefficient.

MarcusRight, and from the payer side, that’s precisely why some are pushing for regional RWE networks. The Pan American Health Organization’s proposed federated data platform could standardize methodologies, but it’s still in early stages. The key is building local capacity while pursuing harmonization.

MarcusGene therapy sponsors are adapting HEOR strategies for Asia’s fragmented HTA landscape. For a spinal muscular atrophy gene therapy, companies are conducting region-specific cost-effectiveness analyses in Japan, South Korea, and Taiwan, each using different discount rates and utility values. This reflects the region’s methodological diversity.

SaraThat’s one read — I’d frame it slightly differently. What strikes me is how these strategies create new budget impact challenges. Taiwan’s NHIC recently capped gene therapy reimbursements at $500,000 per patient, forcing sponsors to demonstrate cost-effectiveness at that threshold. The part that gives me pause is how this affects evidence generation — companies are tailoring endpoints to local payment schemes rather than clinical need.

MarcusThat’s fair, though I think payers would see it differently — they’re trying to manage system sustainability. Japan’s AMED now requires gene therapies to include caregiver burden metrics in their submissions, which wasn’t standard five years ago. This is one of those stories where regional payment constraints are reshaping evidence priorities.

SaraAnd what’s striking here is the precedent-setting nature of these cases. When South Korea’s HIRA approved a hemophilia gene therapy with a risk-sharing agreement, it became a template for other countries. I keep coming back to the risk-sharing mechanisms — they’re becoming standard for ultra-orphan therapies in the region.

MarcusExactly, and what’s interesting is how this connects to something I keep coming back to: the patient perspective. These strategies often include real-world evidence on long-term outcomes, which benefits patients by addressing payer concerns about durability. But the trade-off is longer evidence generation timelines.

SaraThat’s true, but the equity implications are concerning. Singapore’s HTA agency recently approved a Duchenne muscular dystrophy gene therapy only for patients under 12, excluding older adolescents. This creates access disparities based on age rather than clinical need.

MarcusRight, and from the payer side, that’s precisely why risk-sharing agreements are attractive — they spread financial risk while ensuring access. The key is designing these agreements to balance budget impact with patient equity.

MarcusDigital therapeutics are moving beyond theoretical claims to demonstrate real-world clinical utility. FDA recently approved a digital therapeutic for opioid use disorder based on real-world data showing reduced relapse rates, marking a shift from previous approvals based on surrogate endpoints. This signals growing acceptance of RWE for digital health interventions.

SaraI wonder if that’s the full picture though. The real challenge is defining what constitutes “clinical utility” in regulatory contexts. EMA’s new draft guidance requires digital therapeutics to show measurable improvements in patient-reported outcomes, not just behavioral changes. That’s one of those stories where terminology is becoming more precise. The part that gives me pause is how this affects reimbursement — payers still struggle with valuing digital interventions versus traditional drugs.

MarcusThat’s a fair point. The FDA approval you mentioned included a 12-month real-world follow-up, which sets a new precedent for evidence standards. But what’s striking here is how this connects to something I keep coming back to: the evidence generation burden. Sponsors are now required to conduct post-approval RWE studies, which increases development costs.

SaraAnd what’s striking here is the reimbursement disconnect. Germany’s G-BA recently approved a digital therapeutic for diabetes but capped reimbursement at 20% of the drug’s cost, despite similar efficacy. This creates a disincentive for innovation. I keep coming back to the value proposition — if digital therapeutics deliver comparable outcomes, why the discount?

MarcusExactly, and what’s interesting is how this connects to something I keep coming back to: the patient perspective. Digital therapeutics often improve adherence and accessibility, which traditional drugs can’t match. But the evidence standards haven’t caught up to these unique benefits. The FDA’s opioid use disorder approval is a step in the right direction.

SaraThat’s true, but the methodological challenges persist. NICE’s new framework for digital health requires head-to-head trials with active comparators, which may not be feasible for all interventions. This creates evidence gaps for novel digital therapeutics.

MarcusRight, and from the payer side, that’s precisely why some are exploring value-based contracting. CMS recently launched a pilot for digital therapeutics in diabetes management, tying reimbursement to HbA1c improvements. The key is aligning payment models with real-world outcomes.

SaraA lot to think about today. I’ll be watching how Asia-Pacific HTA frameworks continue to diverge and whether that creates evidence harmonization opportunities.

MarcusSame — and for me the thread running through today is how regional constraints are reshaping evidence generation. 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.