Defending Price Under HTA Scrutiny: Why Evidence Must Be Designed to Match Payer Priorities 

Price ambition is only defensible when the evidence can carry it 

Manufacturers can no longer rely on innovation alone to justify premium pricing. HTA bodies and payers increasingly consider whether the innovation is relevant to the health system and how it fits among competing funding priorities, given the increasing budgetary constraints and growing pressure on finite healthcare resources.  

The achievable launch price is therefore not just a commercial position relative to competitors. It is an evidence-backed claim that must survive scrutiny across both clinical benefit and economic value within the context of healthcare efficiency gains.  

In this article, we explore recent case-studies and how evidence translated into willingness-to-pay, or how it failed to achieve that, and the underlying factors driving decision-making.  

Key learnings 1: Imperfect evidence can survive HTA scrutiny when economic value is compelling and defensible  

Mirvetuximab soravtansine for FRα-positive platinum-resistant high-grade serous ovarian, fallopian tube or primary peritoneal cancer after 1–3 prior systemic lines 

• Evidence: Direct evidence of superiority versus chemotherapy, showed improvements in both progression-free survival and overall survival 

• NICE criticism: Long-term survival extrapolation, utility assumptions and resource use were heavily scrutinised and rejected 

• How it survived scrutiny: Strong comparative evidence was supported by a 1.2 severity modifier rating, and survival and baseline age were anchored to a real-world NHS data set instead of the trial data, which improved generalisability 

• So what? All evidence uncertainties do not necessarily need to be resolved to secure willingness-to-pay. A strong case around economic value tends to be the pivotal factor for payer-decision making 

Seladelpar for previously treated primary biliary cholangitis, including associated pruritus 

• Evidence: Heavily dependent on ITCs and structural assumptions to demonstrate short-term biomarker and symptom gains, linking to long-term efficacy claims 

• NICE criticism: Lack of H2H data and discontinuation assumptions, along with pruritus utility values, were considered highly uncertain by NICE 

• How it survived scrutiny: Although only a more conservative version of the economic model was accepted, leading to not every patient-relevant benefit being captured, the economic value remained robust enough to demonstrate cost effectiveness under highly uncertain assumptions 

Key learnings 2: Traditional value arguments anchored around significant unmet need may still fail if the overall value proposition does not fit payer priorities  

Lecanemab for the early stages of Alzheimer’s disease who have one or no copies of ApoE4 

• Evidence: The product demonstrated a strong value story highlighting the significant unmet need and a lack of disease-modifying treatments, with evidence showing that lecanemab could slow disease progression in the early stages of Alzheimer’s disease  

• NICE criticism: NICE concluded that the long-term effectiveness was too uncertain, despite the short-term benefits demonstrated in trials. More importantly, the wider system resource burden for treatment delivery and monitoring was considered too significant to justify the incorporation of the treatment in the NHS 

• Why it failed scrutiny: The clinical benefit of lecanemab was recognised, but it did not translate into payer value and a compelling economic case once the full treatment pathway and health system impact were considered 

• So what? When the evidence base does not clearly link upfront cost to durable long-term outcomes and healthcare resource savings, high upfront prices are more difficult to justify 

Key take-away: A defensible pricing strategy starts with understanding payer priorities, since clinical benefit on its own may not be enough 

To build a price strategy that stands up to payer scrutiny, manufacturers should work backwards from the HTA decision problem. This means identifying the price-relevant value drivers, selecting the relevant comparators that payers will accept, defining the patient subgroups most likely to benefit and testing whether the asset evidence strategy supports the intended price in each market.  

Where uncertainty remains, manufacturers need a strong economic argument to counteract that, considering not only drug costs but also the wider impact on the healthcare system. Manufacturers that align clinical development, HEOR, market access and launch price strategy around payer evidence needs and funding priorities, are better positioned to reduce negotiation risk and secure sustainable access. 

What manufacturers should do now? 

• Treat early scientific advice as critical influence point, ensuring clinical, HEOR, regulatory, market access and affiliate teams align before opportunities to shape the assessment narrow 

• Stress-test evidence packages for assets with high uncertainty, particularly rare disease, paediatric, precision medicine, oncology and single-arm trial assets, where HTA conclusions may diverge across key countries 

• Refine your value proposition to contextualise uncertainty, cost effectiveness, equality considerations and managed access options to proactively mitigate access risks