Diego Sacristan
SVP, Head of International; CSL Behring
From your international position at CSL Behring, how do you perceive the evolution of market access models in recent years? What global trends would you highlight?
DS: With a rapidly expanding pipeline of advanced therapy medicinal products, so called ATMPs, across multiple therapeutic areas and a growing momentum behind personalised medicine, health systems are under mounting pressure to rethink traditional contracting approaches. This is particularly crucial in light of increasing price pressure due to the complex geopolitical climate.
In response, national health systems have been reimagining their health technology assessment (HTAs) processes to support timely access to innovation. This has included introducing novel payment models and embracing greater use of real-world evidence (RWE). In Europe, the introduction of the European Union (EU) HTA Regulation marks a significant step forward, aiming to harmonise clinical assessments across member states and streamline decision-making.
At CSL Behring, we’re actively supporting this transformation by work- ing closely with local authorities and key stakeholders to unlock alter- native, sustainable, outcome focused payment solutions. For example, HEMGENIX® has been leading the way through reimbursement agreements tailored to each country’s needs and financial capabilities, while still supporting innovation. These landmark agreements allow patients to benefit from this transformative treatment option as well as pave the way for other gene therapies to benefit from tailored outcome-based agreements.
An additional trend we have seen is digital health integration, with pay- ers beginning to use digital monitoring to collect real-world evidence. For example, in Denmark, the innovative outcome-based agreement for HEMGENIX® recognises the importance of monitoring treatment outcomes. To support this, Amgros established a new digital platform to enable clinicians to report the effectiveness of HEMGENIX®, which is essential for implementing the outcome-based agreement.
HEMGENIX® has been a pioneer as a gene therapy for hemophilia B. What lessons have been learned from its access process in the countries where it is already available?
DS: With pioneering treatments such as cell and gene therapies (CGTs), there are always going to be challenges and hurdles to overcome ahead of launch. Many national regulatory and Health Technology Assessment (HTA) agencies need to make adjustments to their models and methods to ensure they can fairly assess one-infusion treatments and their projected long-term durability. A lesson that we have learnt is that for this to happen, we need to engage in early and iterative dialogue with regulators, payers and govern- ments to pilot and advance novel HTA assessments, including those that recognise the use of real-world evidence to generate information on the overall value of the drug and to support outcome-based pricing and pay-for-performance arrangements.
Innovative access to gene therapies like HEMGENIX® requires early dialogue, real-world evidence, and outcome-based payment models
Additionally, we have found that innovative access pathways require efforts and flexibility from both sides and collaboration with all relevant stakeholders. For example, in Denmark we took a completely new approach to the reimbursement of gene therapies, making it the first Nordic and European country to adopt a performance-based model. The innovative outcome-based agreement, finalised with Amgros in October 2024, means that costs are incurred only as long as the gene therapy proves effective over the agreed long-term period.
Could you share concrete examples of how CSL Behring has innovated in access models in key markets such as Germany, the UK or Spain?
DS: We are proud to have reached milestone funding agreements with Germany, Denmark, Switzerland, Spain, the UK (including Scotland), Ireland and Austria. Thanks to these innovative access decisions,eligible people living with haemophilia B will be able to benefit from HEMGENIX®.
In the UK, HEMGENIX® is available through a first-of- its kind agreement. This was a landmark for the UK Government’s Life Sciences Vision and represents a step forward in evaluating CGTs in the UK. HEMGE- NIX® is the first gene therapy to receive a positive recommendation through the first ATMP pathway to use an innovative outcome-based payment model as described under the Voluntary Scheme for Branded Medicines Pricing, Access and Growth (VPAG).
HEMGENIX® is also available to patients in Ger- many through a novel national success-based reimbursement model. The agreement with the GKV-Spitzenverband addresses critical challenges such as the long-term efficacy of this one-time therapy and ensures that reimbursement is tied to the individual treatment success of each patient. This new offering had to be carefully discussed with a large number of decision-makers in politics, the healthcare system, healthcare professionals, and the National Association of Statutory Health Insurance Funds (GKV-Spitzenverband). Intensive dialogue was also required to ensure that long-term medical and economic aspects were adequately taken into account. The agreement reflects a solution that both enables access to therapy for patients and ensures economic viability for the healthcare system.
Here in Spain, the Interministerial Commission on the Pricing of Medicines published a positive recommendation for HEMGENIX® in September 2024, resulting in national reimbursement for eligible patients with haemophilia B. The performance-based model means that regions will only incur costs if the gene therapy proves effective in the long-term.
We are also very pleased to see that the first patients in Europe have been treated with HEMGENIX® in France, Denmark, Austria, the UK, Germany and Spain. At CSL Behring we are continuing to build positive momentum for HEMGENIX®, and are seeing increased interest and activity among healthcare professionals and patients. We have a number of ongoing discussions with stakeholders in European and international markets to expand access with tailored reimbursement solutions.
What specific challenges have you faced or are you facing with HEMGENIX®? And how is the company addressing them?
DS:
1. Current contract models
The nature of the single-dose therapy means that at the time of market launch, only clinical data with a limited study duration are available. This situation naturally raises the questions of how long the clinical effect will last beyond the study duration shown and how treatment failures should be dealt with. Current contract models provide single, upfront prices for the reimbursement of single therapies. However, these models face two key challenges:
- The financial viability of future single-dose gene therapies and the resulting financial burden for the healthcare system.
- The necessity of agreeing on a one-time / upfront price that is based on clinical trials of a limited study duration, since the question of long-term efficacy cannot yet be answered at the time of market launch.
We are proud of the flexible contracting solutions, such as outcome-based agreements supported by real-world evidence we have been able to implement so far. These agreements are tailored to each country’s needs and allow sustainable and affordable payment options for patients and healthcare systems. However, the implementation of these contracts and innovative agreements may take time, as healthcare systems can face challenges in finding practical solutions based on their local regulatory and access systems.
2. Infrastructure of specialised treatment centres:
Another challenge is balancing the value of these transformative therapies with the sustainability of the healthcare system. It is important to ensure that optimal infrastructure, resources, and expertise are in place to enable eligible patients to receive gene therapy and to continue the long-term care and follow up. This means we need to help educate physicians, patients, payers, and treatment centres about this one-time treatment. Additionally, governments need to invest in building up the expertise and infrastructure of specialised treatment centres.
What role does collaboration with health authorities, scientific societies, and patient associations play in the success of these new access models?
DS: Each country has its own unique healthcare system, requiring a tailored access pathway. How- ever, these countries share a common openness and agility to pilot pioneering funding solutions, paving the way for patients to access HEMGENIX®.
By working with health authorities, scientific societies, and patient associations we have been able to address three key shared factors:
1. Recognition of unmet need
People living with haemophilia B face more than just the physical symptoms of the condition, they also live under the persistent threat of spontaneous bleeds, even for things as simple as going up and down stairs. Its unpredictable nature, combined with the limitations it imposes on social activities due to the risk of pain, injury, and uncontrolled bleeding, can lead people with the condition to withdraw and feel isolated. Despite advancements in haemophilia B care, patients are still burdened by planning their life around infusions and injections. This means that people with haemophilia B are never free from thinking about their condition.
Working with patient associations and scientific societies has been key to helping health authorities understand that more needs to be done to improve the quality of life of people with haemophilia B. Securing access to HEMGENIX® provides patients with the potential to no longer need regular infusions and have fewer bleeding episodes. This means they may be able to experience fewer disruptions in their daily lives, providing the potential to move towards a haemophilia-free mind.
2. Innovative payment models
CSL Behring has supported stakeholders across the healthcare ecosystem to recognise the value of innovative payment models. By embracing these approaches, health authorities have positioned their healthcare systems at the forefront of innovation, while making informed and sustainable funding decisions. To ensure the longevity of these models, collaboration with scientific societies has been crucial. Their expertise has helped demonstrate how long-term follow-up and RWE can be effectively gathered to underpin and strengthen outcome-based payment and contracting frameworks.
3. Ensuring readiness and expertise
Healthcare professionals and treatment centres have been instrumental in preparing for the delivery of gene therapy to haemophilia B patients, ensuring the highest standards of administration and patient care. Patient associations have also played a vital role, working closely with clinicians and hub-and-spoke centres to support a smooth and informed pathway to gene therapy. By championing shared decision-making, they’ve also helped empower patients, making access to treatment a reality.
Looking ahead, how do you envision the evolution of access to innovative therapies? What role will CSL Behring play in that scenario?
DS: We understand that there is no one-size-fits-all solution, and we are fully prepared to tailor our innovative funding solutions to meet the unique needs and financial capabilities of each country, while still rewarding innovation. We are proud to be pioneering a way forward for ATMPs to achieve reimbursement and market access.
We’re just at the beginning of the innovation around healthcare access models. The technology is moving into this direction. So across multiple therapeutic areas, in our case, for Hemgenix, hemophilia B and across all the disease areas, the new technology brings a significant change in treatment paradigms. Our current focus is on bridging existing access models, which were developed for traditional therapies that measure value through volume – such as the number of pills or injections – with emerging models that emphasise outcomes. We’re shifting from volume-based reimbursement to outcome-based approaches that reward performance and evaluate the actual impact on individual patients. But look, in the future, there might be other models that we can explore. There are subscription models that have been explored. There are partnerships in early development of medicines. There is end-to-end healthcare value chain integration that can be considered in these access models.
Governments need to evolve their current contracting frameworks to implement outcome- based solutions that are feasible and flexible
At this point in time, we’re touching the surface through creating new models that can be layered on top of the traditional models to recognize these different therapies. But I see in the relatively near future a complete change in terms of how value is recognized and looking at deeper partnerships between authorities, doctors and healthcare systems and pharmaceutical companies that really recognize that shared value, that share risk-taking and that shared recognition of the unmet medical need for patients and the value of science. Only through these innovative and different approaches, can we ensure that there are the right incentives for science to continue evolving and universities and basic science finding pathways for the exciting technology that we have ahead of us to find a pathway into patients that really need it. And the number of unmet medical needs throughout all the therapeutic areas continues to be immense.
The success of HEMGENIX® regulatory approvals and reimbursement agreements may encourage further research and development in gene therapy, leading to more innovative treatments for other genetic conditions.
What message would you like to share with healthcare decision-makers regarding the advances and challenges health systems are currently facing in enabling access to innovative therapies internationally?
DS: It is important to recognise that national value assessment processes have not been designed to take into account the specific characteristics of one-off transformative therapies that replace existing lifelong chronic treatments. We therefore need appropriate models to assess and account for the projected long-term durability and potential cost savings of gene therapies.
Governments need to evolve their current contracting frameworks to ensure the implementation of alternative or outcome-based solutions is both feasible and flexible. This preparation is essential for the arrival of future gene therapies. By doing so, healthcare systems can maintain sustainability and reduce the time it takes for patients to access these innovative treatments, while also appropriately capturing the value of these medicines.
We are encouraged by governments sending a strong signal of how innovative and collaborative thinking can make gene therapy a reality for patients. However, we continue to see barriers to innovative contracting solutions in other countries, so it is important that healthcare decision-makers play a role to facilitate timely access to CGTs across other countries.
How is HEMGENIX® expected to change the current standard of care for haemophilia?
DS: By providing patients with a therapy with the potential for long-lasting protection that can reduce or eliminate the need for frequent care, lower the risk of comorbidities, limit hospitalisations and improve the overall quality of life, we are confident HEMGENIX® has the potential to provide significant long-term value to patients in a way that is financially sustainable for our healthcare system.
In Spain, HEMGENIX® is the first gene therapy for haemophilia B to be listed by the National Health System, marking a new treatment paradigm. The performance-based model means that regions will only incur costs if the gene therapy proves effective in the long-term.
Additionally, 4-year data from the Phase 3 HOPE-B trial, presented at the European Association for Haemophilia and Allied Disorders (EAHAD) Congress 2025, showed that a one-time infusion of HEMGENIX® continues to offer long-term durability, safety and greater bleed protection versus prophylactic treatment in adults with severe or moderately severe haemophilia B.
