HTA Thresholds & Geopolitical HTA

MarcusWelcome to Access Brief, the daily AI podcast on HEOR, HTA, and market access. I'm Marcus, with Sara. Today: emerging market HTA thresholds, orphan drug RWE requirements, digital therapeutics clinical utility, geopolitical HTA collaboration, and precision medicine HEOR. Let's get into it.

MarcusEmerging markets are lowering cost-effectiveness thresholds to address budget pressures. Countries like Thailand and Brazil now accept thresholds below $5,000 per QALY for high-burden diseases. This creates tension with manufacturers who argue it undervalues innovation. The risk is that price suppression stifles future R&D investment. SARAH: But Marcus, these markets have finite resources. Lower thresholds reflect local economic realities and prioritize affordability over Western standards. Ignoring this risks excluding therapies that could address unmet needs. The question is whether global equity should override commercial viability.

MarcusOrphan drugs face rising RWE requirements from HTA bodies. The EU and US now demand post-marketing real-world data to confirm initial trial results. This adds years to evidence generation and delays patient access. SARAH: The delay is justified. Orphan drugs often have small trial populations, so RWE fills evidence gaps. But Marcus, manufacturers complain it creates uncertainty. The real issue is whether RWE should be mandatory or case-specific. Some therapies have robust phase III data—why force redundant RWE?

MarcusDigital therapeutics struggle to demonstrate clinical utility in HTA. Most apps rely on surrogate endpoints like symptom reduction, not hard outcomes. HTA bodies reject these as insufficient for reimbursement. SARAH: That’s too rigid. Digital tools offer scalable interventions, but traditional HTA frameworks don’t accommodate them. Marcus, we need adaptive endpoints that reflect real-world impact, not just mortality. The evidence bar shouldn’t be higher for innovations.

MarcusGeopolitical tensions are fragmenting HTA collaboration. The EU’s HTA Regulation faces pushback from non-EU countries over data sharing. Meanwhile, US trade policies influence HTA decisions in Asia. SARAH: Collaboration is eroding. Marcus, data sovereignty concerns are legitimate. HTA bodies shouldn’t be pressured by political agendas. But the consequence is inconsistent assessments. We need neutral frameworks, not geopolitical bargaining chips.

MarcusPrecision medicine is reshaping HEOR evidence generation. Biomarker-driven trials require smaller cohorts but raise heterogeneity challenges. HTA bodies struggle with generalizability. SARAH: Precision medicine demands new HEOR methods. Marcus, subgroup analyses aren’t enough. We need dynamic evidence models that evolve with real-world data. Otherwise, we’ll misvalue therapies that benefit niche populations. The system must adapt to innovation, not suppress it.

MarcusThat’s all for today. Back tomorrow on Access Brief. Show notes at outcomes-analytica.no.