HCA Warning & Leqembi Approval

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, the stories we're covering today really cut to the core of how system pressures and innovation are colliding in real-time.

MarcusSame here. We're looking at HCA Healthcare's profit warning and what it signals for broader US access challenges. Then the subcutaneous Leqembi approval and its HEOR implications. Also, the FTC settlement with CVS Caremark over insulin pricing. And finally, ICER's expansion of scientific advice.

SaraThat second one on Leqembi is particularly compelling — the Alzheimer's space has been a minefield for real-world evidence, and this approval forces us to confront that head-on.

MarcusExactly. Let's get into it.

MarcusHCA Healthcare's profit warning is a stark reminder of the financial pressures in the US healthcare system. They cited rising bad debt and an increase in uninsured patients, which points to underlying access issues that aren't going away.

SaraAbsolutely, and what's striking is that this isn't just HCA — it's a sector-wide signal. When a major for-profit system like HCA struggles, it reflects broader challenges in affordability and coverage gaps, especially for high-cost therapies.

MarcusThat's true. The question for us is how this impacts evidence generation. If systems are under financial stress, will they be more or less willing to participate in real-world studies for expensive new treatments?

SaraI wonder if that's the full picture though. While financial stress is real, many systems are also investing in RWE partnerships to demonstrate value. It's not just about cost but about proving that expensive treatments reduce downstream costs.

MarcusThat's a fair point. The part that gives me pause is the sustainability of those partnerships. If systems are cutting costs, will they prioritize RWE for expensive therapies? It's a delicate balance.

SaraAnd that connects to something I keep coming back to: the need for more flexible evidence models that can adapt to system constraints. We might see more focus on pragmatic trials that generate data without overburdening providers.

MarcusExactly. The takeaway is that access challenges are now directly influencing evidence strategy. We'll be watching how other systems respond and whether this leads to new models for evidence generation.

MarcusThe FDA's approval of subcutaneous Leqembi is a step forward for Alzheimer's patients, offering a more convenient administration option. But from an HEOR standpoint, this raises important questions about long-term real-world effectiveness and cost-effectiveness.

SaraAbsolutely, and the key here is that while the clinical trial data showed a modest slowing of cognitive decline, the real-world evidence is still nascent. How do we demonstrate value when the outcomes are so nuanced and the population is complex?

MarcusThat's where the evidence strategy needs to evolve. We'll need robust RWE to show not just efficacy but also the impact on caregiver burden and healthcare utilization — the indirect costs that payers care about.

SaraI'd push back slightly on that. Payers are increasingly skeptical of RWE for neurodegenerative diseases because of the heterogeneity and lack of standardized endpoints. The challenge is building consensus on what constitutes meaningful evidence.

MarcusThat's fair, though I think the subcutaneous formulation could be a game-changer for adherence, which in turn affects outcomes. If we can demonstrate better adherence in real-world settings, that could strengthen the value proposition.

SaraAnd that connects to something I keep coming back to: the importance of patient-reported outcomes. If patients and caregivers experience a better quality of life with easier administration, that's a value driver we can't ignore.

MarcusExactly. The HEOR community must be proactive in designing studies that capture these nuances. The approval is a milestone, but the real work on evidence generation is just beginning.

MarcusThe FTC's $125 million settlement with CVS Caremark over insulin pricing practices is a significant development. It addresses alleged anticompetitive behavior in the PBM space, which has been a barrier to insulin access for patients.

SaraAbsolutely, and what's notable is the size of the settlement and the focus on restitution. This could lead to more scrutiny of PBM pricing models, which have long been criticized for lack of transparency. The budget impact for health plans is substantial.

MarcusThat's true. For HEOR teams, this means we might need to re-evaluate how we model the cost-effectiveness of insulin products, especially as biosimilars and new formulations enter the market. The dynamics are shifting.

SaraAnd from a payer perspective, the settlement signals that regulators are taking a harder look at the entire supply chain. We could see more pressure for value-based contracting that ties pricing to real-world outcomes and savings.

MarcusThat's a forward-looking thought. If PBMs are forced to change their practices, it might open doors for more innovative pricing models that align with value. But the question is whether this will actually improve access or just shift the cost burden.

SaraThat's the tension. The settlement is a step in the right direction, but we need systemic change. I'll be watching how other PBMs respond and whether this leads to broader reforms in insulin pricing.

MarcusICER's expansion of its scientific advice program is a response to the growing complexity of novel therapies, especially in gene and cell therapy. They're offering earlier engagement to help sponsors align evidence generation with HTA needs.

SaraThat's a smart move. The challenge with these high-cost, one-time treatments is the uncertainty around long-term outcomes. Early scientific advice can prevent missteps in trial design and ensure the evidence meets payer expectations.

MarcusExactly, and it's not just about the evidence itself but how it's framed. ICER is helping sponsors anticipate the value assessment process, which could lead to more efficient submissions and faster access.

SaraI'd frame it slightly differently though. While this is positive, the real test is whether payers actually use the advice to inform their coverage decisions. There's a history of disconnect between scientific advice and final HTA recommendations.

MarcusThat's fair, though I think ICER's growing influence in the US could change that. If they can demonstrate that early engagement leads to better outcomes for both patients and systems, it might become the standard.

SaraAnd that connects to something I keep coming back to: the need for global harmonization in evidence standards. If ICER's model works, could it influence other HTA bodies? That would be a significant shift.

MarcusExactly. The expansion is a signal that the HEOR community is adapting to the complexity of innovation. We'll be watching how sponsors leverage this and whether it translates into real-world access.

SaraA lot to think about today. I'll be watching how HCA's profit warning influences payer attitudes towards high-cost therapies and whether it leads to new evidence requirements.

MarcusSame — and for me the thread running through today is the growing recognition that access challenges are now shaping evidence strategy. We need to be more proactive in generating data that addresses system pressures.

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.