Advanced Therapy Medicinal Products (ATMPs) are offering transformative, and often life-saving, treatments across a wide range of conditions. Yet while their value to patients is undeniable, their heavy price tags and clinical uncertainty present challenges for pricing and market access.
Because ATMPs typically target rare diseases, they are developed in small patient populations, rely on accelerated trials with limited follow-up, and can often deliver benefits through one-time interventions; all of which drive high upfront costs and uncertainty around durability. In addition, from 12 January 2025, the new EU HTA regulation (Regulation (EU) 2021/2282) introduced Joint Clinical Assessments (JCAs) for ATMPs, adding further complexity to the pricing and reimbursement pathway.
Payers fear an unprecedented budget impact, whilst market access teams must juggle disjointed national processes on top of JCAs, and executives must strike a balance between patient access and sustainable returns.
Gaining optimal market access in this new era requires more than just a breakthrough product; it requires careful strategic foresight, particularly when it comes to securing a price that is reflective of innovation. This article sets out a 5-step pricing playbook to maximise ATMP launch success.
The 5 steps
Step 1: Early integrated evidence & value story alignment
ATMPs face unique challenges: high upfront costs, small patient populations, and limited long-term data. These factors make payers cautious – they need confidence that they are funding treatments that truly deliver value. To achieve market access at a desirable price, a clear, evidence-backed value story is essential.
Defining this value story early, for both JCA and national assessments, is the first step to launch success. If the value of a product is not clearly and effectively communicated, payers will conduct their own assessment of its value, which will likely be less favourable.
A strong value story should be tailored to the product and its indication, reflecting the unmet need, treatment landscape, and the products unique attributes that translate into meaningful patient and system benefits. Taking into account the perspectives of other stakeholders (such as patients and clinicians, as well as payers) ensures the narrative is credible and will support pricing negotiations.
Once the narrative is clear, align evidence generation to support every message. Map trial design to JCA methodology to ensure relevant comparators and endpoints, and embed payer-relevant outcomes from the outset. Payers want to see validated, indication-appropriate measures that capture functional improvements. For any uncertainties or unsupported messages that remain, develop your real-world evidence strategy early to address and fill the evidence gaps. In summary, every value message must be anchored in evidence robust enough to withstand payer scrutiny!
Without this foundation, the value of an ATMP risks being underestimated, leading to payer uncertainty, tougher negotiations, and price concessions.
In short: Build and evidence your value story early to protect pricing power.
Step 2: Consider reference pricing and launch sequencing
Reference pricing systems across Europe create significant challenges for maintaining price integrity. Taking a strategic, dynamic approach to pricing and launch sequencing is critical for long-term success.
It is critical to model how reference pricing will unfold across priority markets. External reference pricing means lower prices in one country can quickly influence others, while internal reference pricing caps prices against comparators. Without anticipating these dynamics, even the strongest value story risks being undermined by an eroded price corridor.
Launch sequencing can mitigate these risks. Entering high-value markets first (such as Germany, France, or the UK) anchors a strong benchmark price. Lower-priced markets may need to be delayed to avoid premature undercutting. Stress-testing different sequencing scenarios helps balance early patient access, while protecting long-term price and return on investment.
In short: Anticipating reference pricing dynamics and sequencing launches strategically is essential to maintaining a desirable price across Europe.
Step 3: Innovative contracting and outcome-based agreements
High upfront costs and uncertain long-term outcomes make ATMPs a challenging proposition for payers. Even with a strong value story and carefully sequenced launch, affordability concerns can still hinder pricing ambitions. Innovative contracting offers a way to share risk with payers while safeguarding price.
These agreements align payment with performance, easing payer concerns. For example, annuity-style models spread costs over several years to smooth budget impact without reducing overall revenue. Milestone-based or pay-for-performance agreements link reimbursement to clinical or utilisation outcomes. The choice depends on product profile and market context: a one-time curative therapy may suit an annuity model, while a repeatedly administered therapy might align better with milestones tied to patient outcomes over time.
Success depends on early engagement with payers. Co-designing agreements and data collection frameworks builds trust and ensures contracts are workable in practice. At the same time, preparing adaptable templates helps maintain consistency across countries while allowing flexibility for local requirements.
In short: Innovative contracting is a powerful lever to secure patient access, mitigate payer concerns, and protect long-term pricing strength.
Step 4: Tailored stakeholder engagement
For ATMPs, national HTA appraisals build on the JCA, and how those interactions are managed can directly shape achievable price. The value story and evidence must be communicated convincingly, with emphasis on what matters most to each audience. Therefore, tailored stakeholder engagement is a critical element of pricing success.
The first priority is to translate the JCA report into national dossiers that reflect local priorities. A single evidence package will not be interpreted the same way by Germany’s AMNOG, France’s HAS, or NICE in the UK. Tailoring your communication is critical to sustaining pricing ambition across markets.
As well as HTA bodies, patients and clinicians are also critical stakeholders. Patient groups can validate unmet needs and reinforce societal value, while clinicians lend credibility to how a therapy will be used and the outcomes it can realistically deliver. When integrated into the value story, these perspectives add real-world context that supports value and justifies premium pricing. Early and meaningful engagement with these stakeholders can add credibility to the value story and strengthen pricing negotiations.
In short: Careful preparation and stakeholder alignment ensure your value story resonates across markets and translates into powerful pricing.
Step 5: Post-launch value optimisation and RWD strategy
Strategic pricing considerations should not end at launch – payers will continue to ask whether the therapy delivers on its promise. Therefore, post-launch value optimisation, anchored in a real-world data (RWD) strategy, is our final step.
This connects directly back to Step 1: while early planning must include RWE, it is only post-launch that these commitments are tested. Clinical trials for ATMPs are often short, small, and tightly controlled. RWE from registries, claims databases, and long-term follow-up is essential to demonstrate durability of effect, comparative performance, and system-level impact.
Validating trial outcomes in real-world populations reduces payer uncertainty and strengthens confidence that premium pricing is justified. Comparative effectiveness studies can demonstrate advantages over standard of care, while long-term follow-up confirms durability of effect – both of these can be invaluable when resisting downward pressure on price. In addition, evidence on healthcare resource use and system-level benefits can reinforce the economic case, demonstrating that investment in the product results in long-term savings.
Conclusion
A transformative ATMP alone is not enough to secure market access or a desired price; it requires clear demonstration and communication of meaningful value. From early evidence planning and value story development, through launch sequencing, innovative contracting, stakeholder engagement, and post-launch optimisation, each step plays a direct role in shaping the price that can be achieved and sustained.
Payers will continue to demand reassurance that high upfront costs are justified by long-term outcomes, and pricing success can’t be achieved without a robust strategy. By planning early, anticipating payer concerns, and adapting to national market requirements, companies are best placed to maintain both access and price.
If you are preparing an ATMP launch, now is the time to stress-test your pricing strategy. At Remap Consulting, we partner with companies to co-create tailored approaches that anticipate payer needs, withstand scrutiny, and deliver launch success.
Get in touch with us today to discuss how we can support your upcoming launch.