miércoles, febrero 11, 2026
More
    Inicio Blog Página 4

    CARLOS MARTÍN SABORIDO – BEYOND CLINICAL BENEFIT: ADVANCING VALUE-BASED FINANCING FOR RARE DISEASES

    Por
    newsRARE
    -

    Carlos Martín Saborido

    Director of the Health Technology Assessment Agency at the Instituto de Salud Carlos III, Spain

    From your experience in institutions such as NICE in the United Kingdom and the European Commission, how do you assess the current state of outcome-based payment strategies applied to orphan drugs in Europe?

    CMS: In the UE, some countries are using the same tools for orphan drugs as for common drugs, to address both clinical and financial uncertainty. However, some Member States are focusing only on financial uncertainty. So, I think there is no unified or standardized approach to assessing orphan drugs across Europe.

    So far, I think that the situation remains very variable. You mentioned NICE, which indeed has a very clear position on how to address orphan drugs. They clearly distinguish between clinical and financial uncertainties, for example, using the Innovative Medicines Fund.

    Still, I’m very optimistic. In the next three to four years, when the Joint Clinical Assessments (JCAs) are more widely integrated into national systems, we may start to see some common trends in the way orphan drugs are assessed across Europe.

    Which European countries would you highlight for successfully implementing outcome-based financing models for medicines targeting rare diseases? What best practices could be transferred to other healthcare systems?

    CMS: It’s somehow complicated to say what constitutes a successful implementation of these outcome-based financing models, mainly because some Member States do not share enough information to properly evaluate how the agreements have actually worked. As a result, it’s difficult to assess the success of these models in practice.

    However, we can comment on their implementation, how these models are being put into place, even if we don’t yet know how effective they are. In this regard, Italy is worth highlighting, due to the regional registries they have established, and Finland as well, thanks to the real-world evidence systems integrated into their hospitals. Both countries are very focused on gathering insights and generating evidence from real-world data to assess how these outcome-based financing models are functioning.

    We are moving in the right direction, but we still need much more trust and cooperation between Member States

    It’s also important to clarify that your question refers specifically to rare diseases, not necessarily to orphan drugs. Outcome-based financing models applied to medicines used in rare diseases are not always applied to orphan drugs. This distinction matters, as certain orphan drugs lose their orphan designation over time when they start targeting larger patient populations. So, in some cases, these outcome-based financing models are not directly addressing rare diseases per se, but rather orphan drugs which may be used for rare diseases or for low-prevalence conditions.

    Digitalization and interoperability of health data are key to measuring real-world health outcomes. Is Europe technologically and regulatorily prepared to widely implement outcome-based payment models?

    CMS: I would say this is not happening widely across the European Union as a whole, although some Member States are indeed very advanced in this regard. The main limitation probably comes from the lack of data sharing. To successfully implement outcome-based payment models, we need not only strong data digitalization but also widespread access to that data.

    I’d also like to mention the European Health Data Space. With its vision and the potential for secondary use of health data to support outcome-based agreements, I think we are moving in the right direction. That said, and to be fair, we still need much more trust and cooperation between Member States.

    Within the framework of the new European regulation on joint health technology assessment (HTA), how do you think the incorporation of these innovative payment models could be harmonized among European countries?

    CMS: It’s a complex question, especially since you’re referring to joint health technology assessment, so you may be alluding specifically to the JCAs under the new regulation. The main expectation from this regulation is to establish a common synthesis of clinical evidence. What we aim to achieve with the new HTA regulation is the definition of shared outcomes that can be measured and included into innovative payment models.

    I think this is something that needs to be emphasized in the coming months: we need to acknowledge that the Joint Clinical Assessment (JCA) will define a set of outcomes that are relevant at the European level. These are the outcomes that should serve as the basis for innovative payment models. This is the only way to ensure that the data generated is comparable and usable across Member States, and this is essential for harmonizing outcome-based models across European countries.

    We need to recognize that both the number and type of outcomes required should be driven by the Joint Clinical Assessment. Otherwise, each Member State may select different outcomes for their innovative payment models, which would hinder alignment and comparability across the EU.

    From an international perspective, what structural or methodological barriers still persist for the adoption of value-based models in the field of orphan and advanced therapies?

    CMS: This question overlaps with the topic of advanced therapies, so I’ll try to address it more broadly, although I believe we’re actually discussing distinct but related issues.

    The first challenge is the definition of value, what exactly do we mean by it? Value is not limited to clinical benefit; it’s not just about efficacy or safety outcomes. Many other factors come into play when assessing value. So, I would say the first methodological barrier is precisely the lack of a common, agreed-upon definition of value. The second barrier is the ability to collect data that reflects that value. We need a clear and consistent methodology for capturing such data.

    One critical aspect of defining value is understanding the real burden of rare diseases and the impact that new technologies have on that burden. This is extremely challenging. In Spain, for instance, we’re working on a project called Argos, which aims to collect data on the resources patients use to live with a rare disease. I believe this kind of information is essential for accurately defining value.

    If we are able to gather the right data, define value properly, and share information efficiently, we can move forward

    In addition, there’s also a structural barrier related to data collection: the lack of a shared data collection system and coordinated infrastructure. For some orphan drugs and rare conditions, the European Reference Networks are already collecting and sharing common datasets. This is a valuable model that could be replicated in the context of pricing and reimbursement. Moreover, we should aim to collect relevant data directly from patients, not only from clinicians, as this remains a limitation in many cases.

    If we are able to gather the right data, define value properly, and share information efficiently, we can move toward robust value-based and outcome-based agreements.

    How can it be ensured that these financing models always prioritize clinical benefit and equity in access over other interests, especially in rare diseases where evidence is more limited?

    CMS: This is a serious misconception, the idea that we should prioritize clinical benefits above all else. Of course, we need to set priorities, but they should not be based solely on clinical outcomes. In fact, in the previous two questions we’ve already discussed the broader concept of value. I believe that focusing exclusively on clinical benefit is a trap. If we prioritize only clinical outcomes, we risk overlooking treatments that truly deliver meaningful value.

    Take, for example, a new drug or technology that may offer only modest clinical benefits, but significantly improves the lives of caregivers by simplifying care or easing daily routines. That treatment should be reimbursed, not just because of its clinical efficacy, but because of the overall value it brings to patients and their support networks.

    When we talk about rare diseases, it’s rarely just about clinical benefit. It’s also about how a technology improves the quality of life for informal caregivers, parents, and others who support the person living with the condition. So I apologize if my position seems disruptive, but I firmly believe that clinical benefit alone should not be our sole priority. We must prioritize value, and, equally important, equity.

    We must adopt a holistic view, considering not just clinical benefits, but the broader value a new therapy or technology brings

    Looking ahead, what opportunities do you identify to strengthen European cooperation around assessment, reimbursement, and outcome-based financing of orphan drugs, with the goal of achieving more equitable and efficient access to innovation?

    CMS: You mention three main areas here, assessment, reimbursement, and outcome-based financing. I believe it’s essential to approach them step by step.

    The first priority must be collaboration to establish a shared value framework for assessment. Only once that foundation is in place can we meaningfully move forward with discussions on reimbursement and financial models. Without a common framework for value assessment, we risk creating inconsistencies across Member States, with some approving drugs based solely on clinical benefit, while others reject them due to differing interpretations of that benefit. That’s why defining a shared value framework is not only the first step, but the most critical one.

    Regarding outcome-based financing in the context of orphan drugs, it’s important to recognize that it may not always be the right solution. What we truly need is innovation in financing, and that doesn’t necessarily mean outcome-based models.

    Returning to the earlier point on clinical outcomes: if we rely exclusively on outcome-based agreements and focus only on measurable clinical results, we may end up tracking outcomes that provide little or no real clinical benefit. In many cases, what we need is a value-based financing model, not merely an outcome-based one.

    Sometimes, value-based systems will include outcomes, particularly when the value lies in clinical efficacy or effectiveness. But other times, the value may lie elsewhere. Therefore, we must adopt a holistic view of patients with rare diseases, considering not just clinical benefits, but the broader value a new therapy or technology brings. This is why collaboration is key: only by working together can we build a robust and consistent value framework for assessment. Only then can we move forward with reimbursement models and value-based agreements.

    So, when it comes to rare diseases, we likely need to shift toward financing systems that are value-based, not purely clinical or outcome-driven. Otherwise, we risk missing a substantial part of the value these new technologies offer.

    MANEL FONTANET SACRISTÁN – PAYMENT BY RESULTS AND REAL-LIFE DATA: TOOLS FOR A SUSTAINABLE SYSTEM

    Por
    newsRARE
    -

    Manel Fontanet Sacristán

    Medicine Access Coordinator. Rational Use of Medicines Division. Medicines Department. Catalan Health Service. Government of Catalonia

    From your experience at CatSalut, how would you describe the evolution of payment by results models applied to orphan drugs in recent years? What differences do you observe with respect to other European countries?

    MF: At CatSalut, the application of managed access models has always been a clear line of work, not only in the field of orphan drugs. In fact, we have published a guide with recommendations and areas of application. Therefore, in general terms, it is already a priority area for development within the framework of incorporating innovation in the Catalan health system.

    In the particular case of orphan drugs, two relevant circumstances converge. On the one hand, due to the idiosyncrasy of rare diseases and their low prevalence, the evidence generated often does not reach the ideal level that we would like to have. This, of course, can be improved, but it can hardly match the evidence we obtain under more frequent conditions.

    On the other hand, the small size of the market means that the cost of these drugs is, in general, very high. These two circumstances, therapeutic uncertainty and financial uncertainty, make orphan drugs particularly suitable for the application of management measures in their incorporation into health systems, such as payment-by-results models.

    From our perspective, we are pleased to note that in recent years there has been a progressive increase in the number of medicines financed through these access models, which makes it possible to better align the price of drugs with their therapeutic value and, at the same time, to better manage the economic resources allocated to them.

    It is important to bear in mind that, since the adoption of the European regulation in 2000, the number of authorised orphan drugs has exceeded 250. We are pleased to see how these access tools have been progressively implemented and have facilitated the incorporation of innovation in health systems.

    With regard to comparison with other environments, especially in Europe, there are differences. There is no single reference model in Europe. This is partly due to the confidentiality of funding processes in each Member State, which limits access to comparative information. But it is also true that there are divergent positions: while some countries, such as Italy, have historically favored payment-by-results models, others have adopted strategies that place less emphasis on this approach.

    These methodologies favour the rapid, effective and sustainable incorporation of medicines into health systems

    In our view, the key issue is to identify the uncertainties (both therapeutic and financial) and to try to address them through models that allow them to be managed. We must also take
    into account a key aspect that explains many of the differences in the application of these models in Europe: transaction costs. These models require a significant implementation effort, with a consider- able workload, and this partly explains the differences in their deployment and diffusion in different countries.

    Outcome-based agreements seek to link the price of therapies to their actual clinical effectiveness. From your perspective, what are the main opportunities and challenges posed by their application in the field of rare diseases?

    MF: The main opportunity, in my view, is that these models enable the cost of a therapy or intervention (such as a drug) to be directly linked to the price paid by the health system. This represents a direct tool for managing uncertainty. The more certainty a system has about the decision it needs to make, the more quickly and comfortably it can make that decision.

    These are therefore methodologies that favour the rapid, effective and sustainable incorporation of medicines into health systems. In that sense, I think they is a great opportunity.

    Moreover, these models generate some positive externalities that, although they are not the main reason for their implementation, they do contribute to building a health system oriented towards the continuous collection of health outcomes through- out the life cycle of the medicine. In other words, beyond the direct benefits of the model itself, there are additional effects that help to consolidate a system focused on measuring outcomes, whether drug treatments or other interventions.

    However, the challenges involved are also significant. We are talking about an environment of uncer- tainty, with limited knowledge compared to what happens in more prevalent diseases. Therefore, defining a payment-by-results scheme is not always straight- forward. It is necessary to establish variables that clearly delimit what is considered a «response» to treatment, to do so in a clinically meaningful way, and also to define reasonable time horizons for both parties: both for the funder and for the pharmaceutical company. And this, I insist, is not easy.

    On the other hand, information systems, while functional in many cases, often exhibit limitations or areas for improvement in the collection of real-world data. These challenges stem less from technical deficiencies and more from the substantial effort demanded of the professionals tasked with managing and populating these systems

    These systems must collect the health outcomes that then allow the assessment of compliance with payment-by-results agreements. And all this work entails significant transaction costs, both for healthcare professionals and for the administration and the pharmaceutical companies themselves.

    In our environment, we view these models favourably, but we also recognise their challenges: from agreeing on a clinically valid and measurable response criterion in the appropriate time frame, to having sufficiently sensitive information systems to capture the results. All this, without forgetting the operational burden for all actors involved in the implementation of these schemes.

    Internationally, there is growing interest in integrating real-world data into these funding models. What role do you think real- world evidence should play in making these arrangements more effective and reliable?

    MF: Real-world data is undoubtedly one of the priority areas to develop, especially in those contexts where, due to the rarity of diseases, it is not always possible to generate evidence under the ideal standard of knowledge, such as clinical trials.

    Real-world data is undoubtedly one of the priority areas to develop, especially in those contexts where data are scarce

    I would distinguish two levels of application. Firstly, at the ex ante level, i.e. at the moment when the funding of a medicine is being assessed. At that point, I think it is essential to take into account all available evidence, including real world evidence. However, it is also important to weigh the weight and strength of that evidence against other sources which, from a methodological point of view, may be more robust, such as clinical trials themselves.

    Therefore, I agree that real-life evidence should be considered during the initial decision-making process, especially in settings where data are scarce and any additional information can be useful to make more informed decisions. But I insist: they must be properly assessed, giving them a weight proportional to their quality and methodological strength, which is probably lower than that of the available clinical trials.

    Secondly, at the ex post level, real-life data are also very relevant. One of the key orientations of the system should be the continuous review of previously made decisions. Therefore, having data generated after the drug has entered the system is essential for learning, generating new knowledge and adjusting the decisions taken, also with regard to funding conditions, not only to the clinical use of the drug.

    This requires, again, that we are able to assess the quality of the evidence we are generating, or are able to build, with the current information systems. And this is where another fundamental challenge comes in: to have systems that are sufficiently prepared to produce real world evidence of quality.

    We need that evidence to be robust enough to be of real use for decision-making. Ex post evaluation of the use of medicines should allow us to review, adapt and improve our funding decisions based on knowledge generated in real practice.

    What international experiences or best practices would you highlight as reference models in the implementation of payment by results strategies for orphan drugs?

    In terms of international strategies, although they do not strictly refer to the payment by results model, I do believe that there are prior stages that are necessary before applying any access measure. The first is to assess the therapeutic value of a medicine, i.e. to understand what its real clinical contribution is. From there, we can start to build the most appropriate financing system.

    In this respect, I believe that a very useful step for- ward will be the new European Regulation on Health Technology Assessment (HTA), which proposes a single joint clinical assessment at European level. This assessment will be more agile, agreed between the different countries and, therefore, can become a first key element in the construction of access and funding, even in cross-border contexts.

    As for other tools or international experiences that we value positively from the Catalan health system, perhaps there is no single reference model, but there are several interesting ideas. One of them is undoubtedly the link between the price of the medicine and its therapeutic value. The approach systematically applied by NICE (National Institute for Health and Care Excellence) in the United Kingdom, which introduces economic evaluation as a methodological basis for decision-making, seems to us to be very relevant. Through this analysis, a clear relationship is established between health outcomes and the costs associated with the intervention.

    There are also other interesting European experiences reported in the literature. For example, Italy has widely applied payment by results schemes.

    Another valuable experience, mentioned above, is that of the Netherlands. There, they work with the idea of adapting access conditions as new evidence is generated in real clinical practice.

    This approach makes it possible to adjust decisions on the funding of a medicine according to the data that are obtained in its daily use. This willingness to continuously adapt based on real world evidence seems to us to be particularly useful and perfectly applicable in our context as well.

    Digital technologies and integrated data systems are key tools for implementing and monitoring these models. Do you think that European healthcare systems are technologically prepared to support them? What barriers still exist?

    MF: I think the area of information systems is one of the biggest challenges facing health systems today. Are they ready to implement managed access models, such as payment by results schemes? I guess the answer is yes, because they are being applied, they are being implemented and, in many cases, successfully. Therefore, the formal conclusion should be that they are indeed ready.

    Including patients not just as observers, but as active participants, is a prerequisite for a fairer, more effective and responsive system

    However, it is also true that these information systems need improvements: advances in automation, in automatic data capture, in interoperability… All of this to reduce the workload that currently falls on health system professionals when these schemes are implemented.

    Payment by results models are already being used in our environment and in different European countries, but they entail a considerable operational effort. Therefore, transaction costs are relevant, and this poses a major barrier to scaling up these measures. We cannot apply these models to all medicines entering the system: there would simply not be the capacity, in terms of workload, to manage them all. So there is also a clear need for improvement of information systems, to reduce the effort required and facilitate the implementation of these models within the health system.

    The challenges are basically the same as those faced by all health systems: the need to capture adequate data, to ensure interoperability between different environments… And this is precisely what most systems are working on today.

    In addition, European regulations will also mark an important step forward. Initiatives such as the European Health Data Space and the general adaptation of information systems to these new requirements will be a key stimulus. All this will contribute to making these tools, which we are already applying, easier to implement, more scalable and extendable to a larger number of medicines.

    And this secondary use of data will undoubtedly also help us to generate much more knowledge, thanks to the pooling and integration of information from different environments.

    Collaboration between funders, industry, regulatory authorities and patient organisations is essential for the success of these innovative formulas. How could this collaboration be strengthened at the international level to ensure equitable access to orphan drugs?

    MF: I believe there are a number of areas where international collaboration could be strengthened, many of which are already under development and are likely to be enhanced in the coming years.

    One of them is the early dialogue between all the actors involved in the healthcare system. I think it is very interesting to generate early dialogues between regulators, funders, developers, patients and healthcare professionals. Spaces in which the expectations and needs of certain stakeholders can be anticipated, alongside the possibilities and constraints of others.

    This type of dialogue has already begun to be facilitated at the European level, for example, by the EMA, through initiatives such as the EUnetHTA network, among others. There is a certain degree of development of these spaces for early conversation,and they will surely be reinforced with the entry into force of the new European Joint Clinical Assessment regulation, which will contribute to the regulatory harmonisation that we so desperately need.

    Having a more harmonised, agile and predictable regulatory framework will undoubtedly facilitate the introduction of innovation in the system. This will allow us to better align the expectations of all takeholders and reduce uncertainties in key clinical and economic decisions.

    Another element that could also be very valuable is the development of more open models of innovation and discussion, with effective participation of all stakeholders and with spaces that allow ideas and approaches to be gathered from different perspectives.

    In fact, the European regulation itself envisages the possibility of establishing regulatory sandboxes in the field of orphan drugs. These regulatory tests can help us to be more agile and to find innovative responses to facilitate the incorporation of new treatments, especially in a field as complex and in need of solutions as rare diseases.

    Looking ahead, what do you think should be the key priorities at European and global level to ensure that payment by results models really contribute to a more sustainable and patient-centred ecosystem in the field of rare diseases?

    MF: I believe that there is no single measure, but rather a set of actions that should be leveraged in a complementary way, and that all of them together would contribute to improving access and strengthening the performance-based funding tools that medicines have within the system.

    One of the building blocks is the evaluation of medicines. This should be the first step before any funding decision is taken. In this sense, it is key to deepen the current evaluation models, both in the framework of the European regulation and, in the case of Spain, in the context of the future Royal Decree on Health Technology Assessment (HTA). Ensuring continuity and robustness in this assessment model is essential, particularly in the case of orphan drugs

    Another key aspect is to strengthen the current infra- structures of our information systems. This is essential to reduce the workload of professionals, facilitate data collection and better exploit the secondary use of the information generated. These data not only allow us to generate clinical or epidemiological knowledge; they are also essential to implement managed access models, such as payment by results schemes.

    In addition, it is necessary to establish mechanisms for periodic review of the decisions taken. Knowledge is not static: it evolves as medicines are used and rela-world data are generated. Therefore, being able to observe results and adjust decisions on an ongoing basis is an essential part of the process.

    And finally, regarding how to incorporate patient-centred innovation: the key is precisely to ask patients. Listen to their experience and integrate them into decision-making processes at all possible levels. Including patients not just as observers, but as active participants, is a prerequisite for moving towards a fairer, more effective and responsive system.

    DIEGO SACRISTAN – NEW MARKET ACCESS MODELS FROM AN INTERNATIONAL PERSPECTIVE

    Por
    newsRARE
    -

    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.

    MARC CZARKA-TOWARDS IMPLEMENTING NEW PAYMENT MODELS FOR THE REIMBURSEMENT OF HIGH-COST, CURATIVE THERAPIES IN EUROPE: INSIGHTS FROM SEMI-STRUCTURED INTERVIEWS

    Por
    newsRARE
    -
    TOWARDS IMPLEMENTING NEW PAYMENT MODELS FOR THE REIMBURSEMENT OF HIGH-COST, CURATIVE THERAPIES IN EUROPE: INSIGHTS FROM SEMI- STRUCTURED INTERVIEWS

    Desmet T, Michelsen S, Van den Brande E, Van Dyck W, Simoens S, Huys I. Front Pharmacol. 2025 Jan 20;15:1397531. Doi: 10.3389/ fphar.2024.1397531.

    MARC CZARKA

    CEO. EuroPharMedTech (EPMT)

    SUMMARY

    The article explores how Europe- an healthcare systems, specifically Belgium, could adopt new ways to pay for very expensive, one- time treatments like gene and cell therapies. These advanced therapies can offer long-term or even curative benefits, but their high upfront costs create major challenges for public health budgets. Traditional payment systems aren’t built to handle this kind of financial burden, which is why alternative models, like out- come-based spread payments (OBSP), are being discussed. In these models, payment for the treatment is spread out over time and tied to how well the treatment actually works for patients.

    The goal of the study is to understand what’s needed to make these kinds of payment models a reality. More concretely, it aimed to elicit opinions on and insights into the governance aspect of implementing OBSP in Belgium for the reimbursement of innovative therapies. To get a clear picture, the authors conducted 33 in-depth interviews with a wide range of stakeholders, including doctors, hospital pharmacists, health system managers, policymakers, legal experts, patient representatives, pharmaceutical company staff, and people from Belgium’s public health insurance system. These conversations took place between July and October 2020 and were analyzed using a structured approach that allowed the team to identify key themes and challenges. Statements were allocated into six main topics: payment structure, spread payments, outcome-based agreements, governance, transparency, and regulation.

    Seven key conditions must be met for OBSP models to succeed, from robust data to transparency and external governance

    Interviews revealed the necessary conditions that, fulfilled together, are seen to be sufficient for the successful implementation of OBSP, including consensus on pricing, payment logistics, robust data infrastructure and financing, clear agreement terms (duration, outcome parameters, payment triggers), long-term patient fol- low-up solutions, an external multi-stakeholder governance body, and transparency regarding agreement types. From the interviews, the authors found that seven conditions need to be in place for OBSP models to work properly.

    1. Everyone involved needs to agree on the price of the therapy and how its value is assessed.
    1. The way payments are broken down over time has to be carefully planned.
    1. Strong systems need to be in place to collect and analyze data about patient outcomes, since payments depend on whether the treatment is effective.
    1. The contracts that govern these agreements have to be clear, especially about how long payments last, what counts as a successful outcome, and when payments should stop or be adjusted.
    1. There must be systems to follow up with patients over the longterm, which is often difficult in real-world settings.
    1. An independent organization should be responsible for overseeing the process, to make sure it runs smoothly and fairly.
    1. Transparency is essential, stakeholders emphasized that the terms of these agreements, and how decisions are made, should be open and clearly communicated.

    Even though there is strong interest in implementing OBSP models, the study shows there are still a lot of barriers. For example, there’s no agreement yet on who should take responsibility for different parts of the process, like managing the data or covering financial risks if the therapy doesn’t work. There are also technical and legal challenges when it comes to tracking patient outcomes over time, especially if patients change healthcare providers or move between regions. In addition, there is no central authority currently in place to coordinate these efforts, and many existing agreements are kept confidential, which makes it harder for others to learn from past experiences or build better systems.

    To help move things forward, the authors propose a roadmap or checklist based on the seven key conditions mentioned above. This framework can help policymakers and other actors understand what pieces need to be in place before an OBSP model can be successfully launched.

    COMMENT

    Thomas Desmet’s 2025 article explores the barriers to implementing outcome-based spread payments for high-cost therapies in Europe. While the topic is timely and relevant in light of rising pharmaceutical expenditures, the article ultimately offers limited novel insight and leaves several critical issues unaddressed.

    Strengths

    • The authors rightly recognize OBSP as a potential tool to increase access to innovative therapies while possibly mitigating financial risk for payers.
    • The use of semi-structured inter- views allows for a diversity of stakeholder perspectives, touching on pricing, governance, and infrastructure challenges.

    Critical Shortcomings

    • Delayed Publication Timeline. The interviews were conducted in 2020, yet the article appeared only in 2025, a five-year gap that raises legitimate concerns about the relevance and currency of the In a field characterized by rapid policy developments, particularly around gene therapies and payment innovation, such a delay undermines the practical utility of the study’s conclusions. No explanation is given for this timeline.
    • Response Rate and Representativeness. Although 90 stakeholders were contacted, only 33 agreed to be interviewed (a ~37% response rate). The article does not specify how respondents are distributed across stakeholder categories — a major omission given the potential for imbalanced representation. Without such information, the findings risk reflecting a non representative or skewed sample, especially in a setting where perspectives can vary dramatically between industry, regulators, and healthcare providers.
    • Linguistic and Cultural Bias. All interviews were conducted in Dutch or This choice systematically excludes participants with limited proficiency in these languages, most notably native French speakers in Belgium. Given that language proficiency in professional contexts varies across regions and sectors, this methodological decision introduces a significant linguistic bias. The absence of French-language participation is neither acknowledged nor critically examined, undermining the inclusivity and national representative ness of the analysis.
    • Lack of Theoretical or Policy Innovation. While the article reiterates well-known challenges, such as the need for data infrastructure, transparency, and governance, these insights are not new to the literature. Nor does the study offer concrete policy solutions, implementation strategies, or comparative insights from jurisdictions where OBSP has been piloted. As such, the article contributes more as a consolidation of stakeholder sentiment than as a springboard for action or reform.
    • Identified weaknesses. The author’s level of introspection is limited but not non-existent. He acknowledges certain weaknesses himself including variability in responses and lack of “generalizability” to other countries.

    Desmet’s study engages with a real and pressing policy issue, but its limited originality, methodological blind spots, and much delayed publication dilute its impact. One is left with the impression of a paper that fulfills a publication requirement more than it drives the discourse forward. While it gathers useful quotes and clusters familiar themes, it does not significantly advance the conversation around sustainable reimbursement models.

     

    PATRICIA CUBI-MOLLA-ALTERNATIVE PAYMENT MODELS FOR INNOVATIVE MEDICINES: A FRAMEWORK FOR EFFECTIVE IMPLEMENTATION

    Por
    newsRARE
    -
    ALTERNATIVE PAYMENT MODELS FOR INNOVATIVE MEDICINES: A FRAMEWORK FOR EFFECTIVE IMPLEMENTATION

    McElwee F, Cole A, Kaliappan G, Masters A, Steuten L. Appl Health Econ Health Policy. 2025 Jul;23(4):535-549. doi:10.1007/s40258-025-00960-1

     PATRICIA CUBI-MOLLA

    Senior Principal Economist, OHE Honorary Senior Research Fellow in the Department of Economics at City St George’s, University of London

    SUMMARY

    The article presents a necessary reflection in today’s context: how can healthcare systems ensure access to innovative medicines without compromising their finan- cial sustainability? This question is especially relevant when con- sidering highly personalized therapies with high costs and limited evidence, such as gene therapies (noting that approximately 80% of rare diseases have a genetic origin), CAR-T cell treatments, and targeted oncology therapies.

    The authors propose the implementation of Alternative Payment Models (APMs) as a response to this challenge. Instead of reimbursing a drug in a standard way regardless of the patient, timing, or outcome, APMs allow for linking payment to clinical outcomes, spreading costs over time, or adapting the price to each therapeutic indication. Based on a literature review, the authors present a structured framework for implementing APMs, which includes four main steps:

    • Step 1: Identifying the main problem to be solved
    • Step 2: Assigning the appropriate payment model to each type of problema
    • Step 3: Assessing implementa tion feasibility, considering legal, administrative, or technological barriers
    • Step 4: Collaboration between payers and manufacturers, which is key to finding mutually accept- able solutions

    In Step 1, the authors highlight the main challenges to facilitating access to innovative medicines, including: budgetary impact, uncertainty about effectiveness, misalignment between the actual clinical value of the drug and the evaluation criteria (e.g., having to assess the effect of nearly curative therapies only in the short term), and decision-making constraints (e.g., not allowing differentiated pricing across patient subgroups or indications).

    Identifying the main problem is crucial to determine the most appropriate APM (Step 2). For example: outcome-based payment for drugs with clinical uncertainty; instalment-based payment for therapies with high upfront costs; or subgroup-based pricing when there are differences in effective ness.

    One of the key messages of the article is that many technical obstacles—such as the lack of data infrastructure or the complexity of contracts (Step 3)—can be over come if stakeholders share a clear understanding of the problem and align on objectives (Step 4).

    The article also illustrates the proposal with real-world examples: outcome-based agreements for oncology treatments in the U.S. and Spain; combined schemes with instalment payments in Italy for gene therapies; and subscription models for antivirals in Australia.

    COMMENT

    In the field of rare diseases, the implementation of more flexible and tailored payment models is not a new topic. If we revisit the first issue of newsRARE (from 2016), we already find clear references to APMs: «Mechanisms are needed that allow payment based on outcomes, in order to finance these treatments and thus guaran- tee patient access,» as well as the importance of including financial sustainability in decision-making: «The high amortization cost is compounded by the chronic nature that characterizes many rare diseases.» Nine years later, we can affirm that the adoption of alternative payment schemes tailored to the context of RDs is not just desirable—it is essential.

    Although the article focuses on innovative medicines in general, its framework is particularly relevant for orphan drugs (and it is worth noting that the authors do not include any specific term related to ‘Innovation’ in their literature review that would prevent applying their findings to other contexts). In fact, orphan drugs present specific characteristics that exacerbate the challenges described in the article. Perhaps the most significant is the low prevalence of RDs, which makes it difficult to obtain robust evidence from controlled trials or clinical registries, generating con- siderable uncertainty regarding effectiveness. In contrast, although the total number of patients is small, orphan drugs represent a high budget impact per patient for payers, especially due to the chronic nature of many RDs. Furthermore, many advances in rare diseases arise from drug repurposing or indication extensions, making heterogeneity among patients another relevant consideration.

    In this context, APMs can become an essential mechanism to ensure access to therapies for RDs without jeopardizing the sustainability of the healthcare system. For example, when there are doubts about clinical effectiveness, an out- come-based payment agreement would allow reimbursement only if the expected benefit is achieved in practice (point 5F in Figure 1 of the article). If the treatment cost is very high upfront (as with gene therapies), installment-based payment allows the expense to be spread over time (point 1A in Figure 1). The article cites Luxturna and Zolgensma—gene therapies used for the treatment of RDs—as examples, contrasting the actual payment mechanisms established in some countries with those recommended by the authors’ proposed model.

    These models can also address some of the challenges identified in Reference Centers, Services, and Units (CSURs). As recent studies in Spain have pointed out, CSURs face chronic shortages in funding and specialized personnel, which limits their ability to care for patients referred from other regions. Moreover, the complexity and heterogeneity of many RDs demand a more efficient and coordinated use of resources. APMs can support this by linking expenditure to outcomes achieved and optimizing investment.

    Finally, while the article identifies obstacles such as the need for real- time data and legal complexity, it also emphasizes that these can be overcome. What truly makes the difference is the willingness to collaborate, mutual trust, and clarity of objectives. For APMs to succeed, it is not enough to design sound models: conditions must be created for them to be credible, acceptable, and applicable.

    PILAR PINILLA-DOMINGUEZ-NICE’S ROLE IN BRINGING THE BEST CARE TO PATIENTS FAST WHILE ENSURING VALUE FOR THE TAX PAYER

    Por
    newsRARE
    -

    Pilar Pinilla-Dominguez

    Associate Director of the National Institute for Health and Care Excellence (NICE) International and Education Services

    NICE have been developing evidence-based recommendations on best practice and rigorously assessing new medicines and technologies for use in the NHS in England for over 25 years. Under the NHS constitution, patients have the right to medicines and treatments that NICE recommends. And the NHS is legally obliged to fund treatments NICE recommends in its health technology assessment (HTA) programme, technology appraisals and highly specialised technologies. So, it’s vital that NICE only recommends treatments that are both clinically and cost effective. This helps make sure the NHS uses its resources fairly and effectively[1].

    Over time NICE has found itself navigating increasingly complex terrain, particularly in the evaluation and adoption of treatments that offer transformative potential for patients but whose high (and sometimes also upfront) costs and long-term uncertainties pose significant challenges for HTA, reimbursement and patients. Examples of these include certain treatments for ultra-rare conditions or advanced therapeutic medicinal products.

    NICE’s approach to evaluating rare and ultra-rare diseases

    NICE’s standard HTA methods and processes are designed to be flexible, and adaptable for all technologies and conditions. They are therefore suitable for most technologies that treat rare diseases and small populations. NICE’s HTA structured decision-making framework considers the clinical and cost effectiveness of new therapies. It considers a therapy to be ‘a good use of NHS resources’ if it’s associated with an incremental cost-effectiveness ratio below £20,000 to £30,000 per quality-adjusted life year gained. However, it also accounts for other factors beyond clinical and cost effectiveness, including health (in)equalities, severity (where health gains in more severe conditions are valued more than in those for less severe conditions), uncaptured benefits, non-health factors (where applicable), or the level of uncertainty associated with the evidence available for the technology. In general NICE will normally be more cautious about recommending a technology if the evidence presented is less certain. However, NICE also acknowledges that there are certain technologies or population for which evidence generation is particularly difficult. This includes rare diseases, paediatric population or innovative and complex technologies. In these specific circumstances, NICE may be able to make recommendations accepting a higher degree of uncertainty while considering the nature, scale and consequences of the decision uncertainty and the risks to patients and the NHS[1, 2].

    .

    Despite this flexibility, NICE recognises that some therapies for ultra-rare conditions may require a deviation from the standard HTA approach as there is a risk of delivering results that are not equitable for these populations. This is done through the highly specialised technologies programme, which is designed to be used in exceptional circumstances, is flexible and considers a much higher incremental cost-effectiveness threshold for guiding decisions. Through this programme, NICE aims to strike a balance between the desirability of supporting access to treatments for ultra-rare diseases and the resulting inevitable reduction in overall health gain across the NHS[3].

    A Framework for Innovation and Access

    NICE’s HTA approach has evolved and adapted throughout the years. It has done so alongside broader policy frameworks, such as pricing agreements like the 2024 Voluntary Scheme for Branded Medicines Pricing, Access and Growth (VPAG, an agreement between the Department of Health and Social Care, NHS England, and the Association of the British Pharmaceutical Industry (ABPI) to guide pricing for branded medicines). VPAG explicitly supports the use of commercial flexibilities and managed access agreements to enable earlier access to promising therapies while further evidence is gathered through managed access agreements[4].

    In parallel, the NHS Commercial Framework for New Medicines, outlines how NHS England collaborates with industry to negotiate enhanced commercial arrangements. NICE supports these negotiations. These include confidential discounts, and other more complex types of arrangements. The framework encourages early engagement and flexible pricing strategies, particularly for high-cost, high-impact therapies. The preferred option are always simple commercial arrangements such as simple discounts on list prices[5].

    NICE’s Commercial and Managed Access Programme

    NICE’s Commercial and Managed Access Programme plays a pivotal role in operationalising these policies. It facilitates structured engagement between companies, NHS England, and NICE at multiple stages of the appraisal process. Managed access agreements are particularly relevant for some promising therapies that have a plausible potential to be cost effective but due to uncertainty on their clinical evidence at the time of evaluation, cannot be recommended for routine use in the NHS. These agreements allow conditional NHS funding while additional data is collected to address uncertainties in clinical or cost-effectiveness. These uncertainties must be mitigated during a pre-specified period of time through further data collection. NICE then re-evaluates the therapy after the period for data collection and will then recommend or not recommend the therapy for routine use in the NHS. All managed access agreements must have a data collection agreement and a commercial agreement and they are designed to be used in exceptional circumstances only because of the costs and risks of all parties involved[6].

    Case Study: Etranacogene Dezaparvovec for Haemophilia B

    A recent example that illustrates the strengths and challenges of this approach is etranacogene dezaparvovec (Hemgenix), a gene therapy for adults with moderately severe or severe haemophilia B. Conditionally recommended in NICE Technology Appraisal 989, the therapy is available through a managed access agreement that includes a commercial component[7].

    The therapy offers a one-time infusion that delivers sustained expression of Factor IX, potentially eliminating the need for lifelong prophylactic treatment. However, uncertainties remain regarding the durability of effect and long-term safety. The managed access agreement allows eligible patients to benefit from the therapy while these questions are addressed through ongoing data collection, which brings important difficulties. This case exemplifies how NICE, in collaboration with NHS England and industry, is using flexible mechanisms to enable access to high-cost therapies while mitigating the risk for patients and the NHS.

    Looking Ahead

    As more disruptive therapies enter the pipeline, NICE’s experience can be helpful for others. The success of the approaches taken depend on transparent governance, robust data infrastructure, and sustained collaboration across stakeholders. Having a strong policy foundation is also critical. Ultimately, HTA should be understood as a dynamic enabler of access and innovation. By allowing for flexibilities and arrangements, where relevant and justified, HTA can help to ensure that the promise of innovative technologies translates into real-world benefits for patients, while safeguarding the sustainability of the public health care systems.

    REFERENCES

    1. National Institute for Health and Care Excellence (NICE): How NICE makes its decisions, https://indepth.nice.org.uk/how-nice-makes-its-decisions/index.html

    2. National Institute for Health and Care Excellence: NICE health technology evaluations: the manual, https://www.nice.org.uk/process/pmg36/chapter/introduction-to-health-technology-evaluation

    3. National Institute for Health and Care Excellence (NICE): Highly specialised technologies: NICE prioritisation board routing criteria, https://www.nice.org.uk/process/pmg46/resources/highly-specialised-technologies-nice-prioritisation-board-routing-criteria-15301445581/chapter/the-vision

    4. Department for Health and Social Care, Association of British Pharmaceutical Industry: 2024 voluntary scheme for branded medicines pricing, access and growth, https://www.gov.uk/government/publications/2024-voluntary-scheme-for-branded-medicines-pricing-access-and-growth

    5. NHS England: NHS commercial framework for new medicines, https://www.england.nhs.uk/publication/nhs-commercial-framework-for-new-medicines/

    6. National Institute for Health and Care Excellence: NICE Managed Access, https://www.nice.org.uk/about/what-we-do/our-programmes/managed-access

    7. National Institute for Health and Care Excellence: NICE Technology Appraisal 989: Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B, https://www.nice.org.uk/guidance/ta989

    VI EDICIÓN DEL CURSO DE VERANO newsRARE 2025 Transformando el abordaje de las enfermedades raras: DEL DATO AL IMPACTO SOCIAL

    Por
    newsRARE
    -

    El próximo 17 de septiembre de 2025, en la Universidad de Castilla-La Mancha (Facultad de Ciencias Jurídicas y Sociales de Toledo) se celebrará el VI Curso de Verano newsRARE 2025: “Transformando el abordaje de las enfermedades raras: del dato al impacto social”.

    A lo largo de la jornada se desarrollarán tres mesas de debate  y se entregará el  V Premio Protagonista del Año 2025.

    Fecha: 17 de septiembre de 2025

    Lugar: UCLM, Facultad de Ciencias Jurídicas y Sociales de Toledo

    Horario: 10:15 – 17:30 horas

    Inscripciones gratuitas: VI EDICIÓN DEL CURSO DE VERANO newsRARE

    Agenda: Agenda

    VOL 10 – NÚM 1 – ABRIL 2025

    Por
    newsRARE
    -

    Descarga el número en PDF

    DESCARGAR

     

    MEDICAMENTOS HUÉRFANOS E INDICACIONES APROBADAS POR LA EMA DESDE 1 DE ENERO 2025 HASTA 31 DE MARZO DE 2025

    Por
    newsRARE
    -

     

    EL CNIO TRATA CON ÉXITO EN EL HOSPITAL LA PAZ UNA ENFERMEDAD RARA AUTOINMUNE CON UNA TERAPIA CAR-T

    Por
    newsRARE
    -

    Una niña con una enfermedad autoinmune rara, dermatomiositis anti-MDA5, ha sido tratada con éxito en el Hospital La Paz con una terapia CAR-T, marcando un hito en la medicina pediátrica.

    Con una mortalidad cercana al 60 % y sin tratamientos específicos, la enfermedad afecta gravemente al sistema inmunitario y los pulmones. Ante la falta de opciones y el ingreso de la paciente en UCI, el equipo médico decidió administrarle la CAR-T ARI-0001, desarrollada por el Hospital Clínic de Barcelona, mediante uso compasivo. La terapia consiguió reiniciar de forma segura el sistema inmunológico, permitiendo la retirada de tratamientos convencionales. Un año después, la niña sigue en remisión, respira por sí misma y mejora progresivamente su capacidad motora.

    El caso, publicado en Med, es el primero en aplicar esta terapia en una enfermedad autoinmune pediátrica no oncológica. La investigación, liderada por la Unidad Mixta de Oncohematología Pediátrica IdiPAZ-CNIO, ha permitido estudiar los cambios inmunológicos tras el tratamiento, sentando bases para su uso en futuros casos. Este avance ha sido posible gracias a un equipo multidisciplinar del Hospital La Paz, el CNIO, CRIS contra el Cáncer, el Hospital Niño Jesús y el Clínic de Barcelona.

    Artículo de referencia: París-Muñoz A, Alcobendas-Rueda RM, Verdú-Sánchez C, Udaondo C, Galán-Gómez V, González-Martínez B, et al. CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease. Med. 2025 Apr 25:100676. doi: 10.1016/j.medj.2025.100676.

    1...345...55Página 4 de 55

    NO TE PIERDAS

    Un proyecto promovido por la Fundación Weber

    Conoce la actividad de la Fundación https://weber.org.es/

    Conoce a nuestros patrocinadores:

    Contacto: fundacion@weber.org.es

    © Copyright - newsRARE

    Política de privacidad | Aviso legal