US Pricing Dynamics & Biomarker Therapies

MarcusAccess Brief. I'm Marcus, with Sara. Today we're looking at the FTC's settlement with CVS Caremark over insulin pricing manipulation — a clear signal that PBMs are now in the crosshairs of US regulators. The US drug discount program surging to $100 billion — creating massive pricing complexity for HEOR teams. And Celcuity's FDA approval for a biomarker-driven breast cancer therapy — accelerating the need for real-world evidence in subpopulations. Sara, where do you want to start?

SaraThe PBM settlement is just the beginning of a much larger reckoning in the US pricing ecosystem.

MarcusThe FTC's settlement with CVS Caremark over insulin pricing manipulation is a landmark moment. They're alleging CVS inflated insulin costs by steering patients to more expensive pharmacy partners and restricting access to cheaper alternatives. This follows a broader US crackdown on PBMs' role in drug pricing. For HEOR teams, this reinforces the need to model PBMs' impact on net prices and patient access, especially for high-cost chronic therapies.

SaraBut Marcus, this settlement doesn't address the root cause of PBM power — the opaque rebate structures that allow them to capture value at every step of the supply chain. The behavioral changes mandated are just surface-level fixes.

MarcusI don't think that's right. The behavioral changes are precisely what matter here — forcing transparency in pharmacy networks and removing barriers to cheaper alternatives. That's the fundamental shift, not just financial penalties.

SaraYou're missing the point here. The financial penalties are trivial compared to the billions PBMs extract through spread pricing. Without addressing the rebate structures, we're just rearranging deck chairs on the Titanic.

MarcusThat contradicts what we saw with previous PBM settlements — the evidence there showed that behavioral changes eventually lead to more significant market shifts over time. The transparency requirements in this settlement are more substantive than past agreements.

SaraTransparency without structural change is meaningless. PBMs will find new ways to game the system. The real HEOR challenge is quantifying their influence on cost-effectiveness analyses when they control so much of the pricing landscape.

MarcusRegardless, this is a clear signal for HEOR teams to incorporate PBM dynamics into their models. We need to develop new methodologies that capture the true cost of PBM interference with patient access.

SaraAgreed, but we also need to be realistic about the timeline. These changes won't happen overnight, and in the meantime, our HEOR models need to account for the current PBM reality.

MarcusThe US drug discount program sales surging to $100 billion in 2025 presents significant challenges for market access strategists. This expansion of eligibility and complex pricing structures complicates traditional pricing strategies, as discounts may not translate to net prices paid by payers. HEOR must now model program-specific price erosion and quantify program outcomes to justify value.

SaraBut Marcus, this $100 billion figure is misleading because it doesn't account for the actual savings passed to patients. The program may be growing, but its effectiveness in reducing costs is questionable.

MarcusThat's the obvious read but misses the strategic implications. Regardless of patient savings, the sheer scale of this program forces us to rethink how we approach value demonstration. We can't ignore $100 billion in sales when developing HEOR frameworks.

SaraI don't think that's right. The real issue is that these programs create artificial price points that distort traditional cost-effectiveness analyses. Our HEOR teams need to develop methodologies that isolate the true value proposition separate from these discount structures.

MarcusThat contradicts what we've seen with other discount programs — the evidence there showed that when properly modeled, these programs can reveal underlying value that traditional pricing models miss. The key is developing appropriate adjustment factors.

SaraThe evidence also shows that these programs often lead to higher overall healthcare costs, not savings. Our HEOR models need to capture that broader system impact, not just narrow drug-specific benefits.

MarcusRegardless, this surge in discount program sales demands a strategic response. We need to develop HEOR frameworks that can properly account for these program dynamics in our value propositions.

SaraAgreed, but we also need to be more transparent about the limitations of these programs in our submissions. HTA bodies are increasingly skeptical of discount program claims without robust real-world evidence.

MarcusCelcuity's FDA approval for its HER2-low breast cancer drug represents a significant shift toward biomarker-driven therapies. Their proprietary diagnostic-guided approach has triggered acquisition speculation, validating the growing importance of companion diagnostics in HEOR strategies. This acceleration requires RWE to confirm real-world outcomes in biomarker-defined subpopulations, critical for HTA submissions.

SaraMarcus, this approval highlights a fundamental tension in HEOR: how do we demonstrate cost-effectiveness in increasingly fragmented patient populations defined by biomarkers?

MarcusThat's precisely the challenge. Traditional cost-effectiveness models struggle with small, biomarker-defined subpopulations. We need new methodologies that can properly value these targeted therapies.

SaraI don't think that's right. The evidence shows that biomarker-defined populations often have better outcomes, which can offset the higher per-patient costs. Our challenge isn't the methodology, but generating sufficient real-world evidence to demonstrate those outcomes.

MarcusThat framing is too generous to the current state of evidence generation. We're still years away from having robust RWE for most biomarker-defined subpopulations, especially for novel diagnostics like Celcuity's.

SaraYou're missing the point here. The strategic advantage is that companies like Celcuity are generating this evidence earlier in the development process. That's what makes them acquisition targets.

MarcusRegardless, this approval signals a broader trend toward personalized medicine that HEOR teams must adapt to. We need to develop new frameworks that can properly value these targeted approaches.

SaraAgreed, but we also need to be realistic about the evidence requirements. HTA bodies are increasingly demanding real-world confirmation of biomarker-driven benefits, not just surrogate endpoints.

MarcusAkero's post-merger development of a long-acting IL-23 inhibitor for psoriasis demonstrates how M&A can accelerate differentiated therapies. The drug's reduced dosing frequency creates a clear value proposition that HEOR must quantify through adherence benefits and quality-of-life improvements. This requires comparative effectiveness against existing biologics using RWE and head-to-head trials.

SaraBut Marcus, reduced dosing frequency is becoming table stakes in the psoriasis market. The real HEOR challenge is demonstrating meaningful differences beyond just convenience.

MarcusThat's the obvious read but underestimates the commercial implications. In crowded markets like psoriasis, even small advantages in adherence can translate to significant market share gains.

SaraI don't think that's right. The evidence shows that patients value reduced dosing, but payers are increasingly skeptical of premium pricing based solely on convenience. Our HEOR models need to capture more than just adherence metrics.

MarcusThat contradicts what we've seen with other long-acting therapies in autoimmune diseases — the evidence there showed that reduced dosing correlates with better long-term outcomes and lower overall healthcare costs.

SaraThe evidence also shows that these benefits are often marginal compared to the price premiums being requested. Our challenge is to demonstrate that the quality-of-life improvements justify the cost.

MarcusRegardless, this post-merger development strategy highlights how M&A can create opportunities for differentiated value propositions that HEOR teams can leverage.

SaraAgreed, but we also need to be more sophisticated in how we model adherence benefits. Traditional approaches often overestimate the impact of reduced dosing frequency on real-world outcomes.

MarcusThe US pricing landscape is becoming increasingly complex, demanding more sophisticated HEOR approaches to navigate PBM influence and discount program dynamics.

SaraBiomarker-driven therapies and post-merger development strategies are creating new opportunities for value demonstration, but require evidence generation that keeps pace with innovation.