Gene Therapy & RWE Challenges

MarcusWelcome to Access Brief, the daily AI podcast on HEOR, HTA, and market access. I'm Marcus, with Sara. Today: gene therapy cost-effectiveness, real-world evidence in HTA, patient-reported outcomes, and digital health HTA methods. Let's get into it.

SaraStarting with gene therapy cost-effectiveness challenges. These therapies face fundamental economic hurdles. The one-time cure model conflicts with traditional cost-effectiveness frameworks. HTA bodies struggle to model lifelong value against upfront costs. We see evidence gaps in long-term durability data too.

MarcusCommercial framing is premature. Companies are pricing based on theoretical cures without real-world validation. The economics only hold if durability matches claims. HTA bodies are increasingly demanding longer-term RWE before accepting cost-effectiveness. The burden of proof should shift to manufacturers.

SaraThat's exactly the problem—shifting the burden ignores the reality of these technologies. HTA methodologies haven't caught up to the science. We need new value frameworks that account for curative potential beyond QALYs. Current approaches penalize innovation.

MarcusInnovation doesn't justify unsustainably high prices. The market can't bear these costs without system disruption. HTA bodies are right to demand evidence. The solution isn't new frameworks—it's better evidence generation.

SaraMoving to real-world evidence in HTA submissions. RWE is no longer supplementary—it's becoming primary evidence in dossiers. HTA bodies are accepting RWE for effectiveness when RCTs are impractical. This accelerates access for niche populations.

MarcusBut RWE quality remains inconsistent. Methodological gaps in data collection and analysis undermine credibility. HTA bodies are implementing stricter validation protocols. Submissions need more rigorous statistical justification to avoid rejection.

SaraThat's a commercial argument disguised as methodological rigor. RWE fills critical evidence gaps where RCTs fail. HTA bodies are increasingly recognizing its utility. The focus should be on standardizing RWE use, not restricting it.

MarcusStandardization requires robust governance. Without it, RWE becomes a black box. HTA bodies are right to proceed cautiously. Quality control must precede broader acceptance.

SaraPatient-reported outcomes in value assessments. PROs are gaining traction in HTA, especially for chronic conditions. They capture treatment impact on daily life that clinical endpoints miss. HTA bodies are incorporating PROs in decision-making frameworks.

MarcusPROs introduce significant measurement challenges. Variability in survey design and interpretation reduces comparability. HTA bodies are developing stricter validation criteria. Without standardization, PRO data risks being dismissed as subjective.

SaraSubjectivity is inherent in patient experience. The alternative is ignoring patient perspectives entirely. HTA bodies are evolving to accommodate this complexity. The value of PROs outweighs methodological hurdles.

MarcusEvolution requires evidence, not advocacy. HTA bodies need data showing PROs predict real-world outcomes. Without that, they remain supplementary at best.

SaraFinally, HTA methodologies for digital health. Digital therapeutics and apps are forcing HTA bodies to adapt. Traditional endpoints don't capture their impact. HTA bodies are exploring new value frameworks for these technologies.

MarcusAdaptation is slow and inconsistent. HTA bodies lack standardized approaches for digital evidence. Submissions face higher uncertainty. The field needs methodological clarity before digital therapies can demonstrate value reliably.

SaraClarity will emerge as these technologies mature. HTA bodies are actively developing guidelines. The process is iterative but necessary for innovation.

MarcusIteration shouldn't delay patient access. HTA bodies must balance rigor with responsiveness. The status quo disadvantages digital innovation.

SaraThe tension between evidence generation and access is inevitable. HTA bodies are navigating this carefully. We need patience and collaboration.

MarcusCollaboration requires shared goals, not competing priorities. HTA bodies and industry must align on evidence standards. Without that, progress stalls.

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