Supportive Care RWE, Cell Therapy Budget Impact, Patient-Centric HTA

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 patient-centricity angle.

MarcusSame here. We're looking at the evolving role of real-world evidence in supportive care for rare diseases — where evidence gaps are creating access hurdles. Then the budget impact challenges for cell and gene therapies — a critical bottleneck for innovative therapies. And finally, the impact of patient-centricity on HTA methodologies in Europe — a shift reshaping value assessments.

SaraThat second one is particularly interesting to me — the budget impact question for cell and gene therapies is one where the field hasn't fully reconciled the one-time high cost with long-term value uncertainty.

MarcusExactly. Let's get into it.

MarcusWe're seeing a noticeable shift in how HTA bodies are approaching supportive care for rare diseases. Historically evaluated on clinical outcomes alone, there's now growing recognition of health economic value in improving quality of life and reducing hospitalizations. This responds to increasing supportive care options and system resource constraints.

SaraThat's one read — I'd frame it slightly differently. The part that gives me pause is that supportive care often doesn't fit traditional cost-effectiveness models. Outcomes are patient-reported and benefits are intangible. Payers struggle with valuation, seeing ad hoc approaches rather than unified methodology.

MarcusThat's fair, though I think payers would see it differently — they're increasingly using multi-criteria decision analysis to capture broader value. But you're right, the lack of standardization is a challenge. What's striking here is patient advocacy groups shaping evidence requirements, pushing for patient-relevant endpoints.

SaraAnd that connects to something I keep coming back to — opportunity cost. When allocating to supportive care, we're not allocating to curative therapies. The budget impact question remains unresolved, especially in fixed-budget systems.

MarcusExactly, and what's interesting is some HTA bodies now incorporate budget impact assessments for supportive care — a departure from past practice. This is one of those stories where healthcare delivery realities drive methodological innovation.

SaraI wonder if that's the full picture though — evidence bases for supportive care remain thin, with heavy extrapolation from other disease areas. That's a methodological concern needing addressing.

MarcusThat's valid. Evidence generation is often under-resourced, limiting data quality. But the trend is clear: health economics is central to evaluating these therapies, a positive development for patients. We're also seeing more real-world evidence to fill gaps.

SaraAnd that's key — real-world evidence often reflects patient experience better than trial data, but comes with confounding factors and standardization issues. The field evolves, but we're not there yet.

MarcusCell and gene therapies lead innovation but face HTA integration challenges. The primary issue is lack of long-term real-world data critical for assessing durability and cost-effectiveness. HTA bodies increasingly request this data, but generation takes years, creating access bottlenecks.

SaraThat's one read — I'd frame it slightly differently. What strikes me is the budget impact question. These are one-time, high-cost interventions with enormous opportunity costs. Payers struggle to justify expenditure without robust long-term value evidence unavailable at assessment.

MarcusThat's fair, though industry might argue initial clinical trial data suffices for value demonstration. But you're right, real-world data absence is a significant hurdle. What's striking here is some HTA bodies accepting interim data with reassessment commitments — a pragmatic approach.

SaraAnd that connects to something I keep coming back to — regulatory-HTA misalignment risks. A therapy might gain approval on short-term data, but HTA deferral until long-term data causes access delays. This story shows the system struggling with innovation pace.

MarcusExactly, and what's interesting is federated real-world evidence networks emerging to address this, though still in infancy. Challenges include not just data generation but quality and HTA relevance.

SaraI wonder if that's the full picture though — methodological challenges using real-world data for these therapies are substantial. Real-world comparators are often ill-defined, follow-up periods non-standard. That's a methodological concern needing resolution.

MarcusThat's valid. Real-world evidence base for cell/gene therapy is still developing, with much experimentation in usage. But the trend is clear: real-world evidence grows more important in HTA, benefiting patients. Industry-HTA collaboration to generate necessary data is increasing.

SaraEurope leads in incorporating patient-centricity into HTA methodologies. We're shifting from purely clinical outcomes to including patient-reported outcomes and quality of life measures. This is driven by patient advocacy and recognizing patients should define value, not just clinicians.

MarcusThat's one read — I'd frame it slightly differently. The part that gives me pause is operational challenges. Patient-reported outcomes are subjective and hard to standardize. Payers struggle with incorporating these into decisions, especially without clear meaningful improvement thresholds.

SaraThat's fair, though I think payers would see it differently — they're increasingly using qualitative evidence to supplement quantitative data. But you're right, lack of standardization is a challenge. What's striking here is some HTA bodies piloting new approaches, like involving patients in appraisal processes.

MarcusAnd that connects to something I keep coming back to — budget impact. Including patient-centric outcomes often increases therapy valuation, affecting system sustainability. The budget impact question remains unresolved.

SaraExactly, and what's interesting is the EU's Critical Medicines Act accelerating this shift by requiring patient involvement in HTA. This story shows policy driving methodological change.

MarcusI wonder if that's the full picture though — evidence for patient-centricity is still emerging. We see enthusiasm, but practical access impact isn't yet clear. That's a concern needing addressing.

SaraThat's valid. Evidence base for patient-centricity is developing with much experimentation. But the trend is clear: patient-centricity becomes more central to HTA, benefiting patients. Greater patient engagement in the entire HTA process is also emerging.

SaraA lot to think about today. I'll be watching how HTA bodies operationalize patient-centricity in coming months, especially budget impact integration.

MarcusSame — and for me the thread is tension between innovation evidence generation and system 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.