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Welcome to the CREATIC ATMP newsletter | |
| | | | Welcome to the Fifth edition of the CREATIC ATMP newsletter. This newsletter gives you a window to the rapidly evolving regulatory and ethical landscape for therapies utilizing genes-, cells and tissue. You will find a collection of news on regulatory developments and discussions spiced up with perspectives and analysis – all with a focus on social, legal and ethical issues.
The newsletter is published quarterly, with the next issue being scheduled for February. If you're interested in staying updated on the latest social, legal and ethical
news related to ATMPs, please sign up here. | | | | |
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| | | |  | | | | In this edition of the newsletter you can find:
- Monthly opinion
- The latest ATMP news
- Upcoming events
- Disparities in access to innovative medicines
- Research Papers
- Previous webinars available online
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Fit for patients, not templates | |
| | | | By PharmDr. Adéla Bártová, Health Economics Specialist at Masaryk University, Faculty of Medicine - CREATIC
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| | | | Honestly, European HTA methodologies are still built for large, randomised studies more than for the clinical reality of rare diseases and children. In our mapping of 29 HTA guidelines across the 27 EU Member States and the UK, 16 documents (in 13 countries) refer to rare diseases, while only 12 (in 10 countries) address paediatrics. The main difference is qualitative. Rare-disease texts more often explain how to handle single-arm studies and real-world evidence; several systems have even institutionalised flexibility through deliberative elements and adjusted, ICER-based cost-effectiveness thresholds. Paediatric guidance, by contrast, tends to stop at quality-of-life measurement—typically EQ-5D-Y and proxy reporting—without a full methodological pathway for comparative effectiveness and economic modelling. Paradoxically, that leaves evaluators without practice-aligned tools where evidence is scarcest, and heterogeneity is greatest.
This asymmetry arises not only from methodologies but also from incentives and market settings. Orphan-drug policies attracted investment and, in return, stimulated more responsive HTA approaches. By contrast, paediatric development often represents an extension of adult-focused products rather than a stand-alone commercial goal, making such studies less attractive for industry investment once the primary adult indication has been secured.Paediatric development, therefore,remains fragmented, with uneven age-specific utilities and weaker commercial pull. Yes, regulatory levers exist—such as formal frameworks for paediatric extrapolation and the EU Paediatric Investigation Plan—but compliance still hasn't produced reliable, age-specific evidence sets that HTA can assess without substantial reservations. In day-to-day decisions, evaluators are effectively asked to force small, heterogeneous patient realities into templates designed for other populations. That is the wrong direction of travel. Methods and assessments should adapt to clinical practice and individual cases—through explicit acceptance of non-randomised evidence where randomisation is not feasible, transparent external comparators, pre-specified adjustments and sensitivity analyses, and modelling that reflects how treatment is actually delivered.
Against this backdrop, the EU HTA Regulation (2021/2282)—in force since 2022 and applicable from January 2025—offers a pragmatic path forward. While complete harmonisation is neither realistic nor desirable, meaningful convergence on how we handle uncertainty is both achievable and necessary.
Read more about how Europe's HTA systems could evolve to better capture the value of ATMPs and paediatric innovations in this paper. | | | | |
| | | | Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or European Research Executive Agency. Neither the European Union nor the granting authority can be held responsible for them. | | | | | | | |
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|  | | | | This section delves into the latest news within the ATMP area, with a special focus on Social Science and Humanities (SSH) including legal and regulatory news. | | | | |
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| | | | .png) | European Union puts Advanced Therapies at the core of its new Life Science Strategy | | | | |
| The European Union's new Life Science Strategy highlights Advanced Therapy Medicinal Products (ATMPs) as a key driver for innovation, competitiveness, and patient access across Europe. The EU faces fierce global competition in this area, where the innovation gap, a fragmented ecosystem, and other structural barriers demand immediate action. The Council calls for a holistic approach to research and innovation, building on existing capacities across the entire value chain. In particular, the conclusions encourage the development of ATMPs and welcome the creation of a network of centres of excellence in this field.
Among the measures announced are:
- The creation of a network of ATMP
Centres of Excellence, supported through
Horizon Europe 2026–2027.
- Increased focus on multi-country clinical
trials to speed up patient access to
transformative therapies.
- Investments to strengthen
Europe's research, development,
and production capacity for ATMPs.
Read more here. | | | | |
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| | | | .png) | European Medicines Agency ATMP Approvals 2009-2025 | | | | |
| As of 2025, the European Medicines Agency (EMA) has authorised close to 20 ATMPs, most of them gene therapies for rare or serious conditions. In comparison, the US Food and Drug Administration (FDA) has cleared over 40 cell and gene therapies, reflecting differences in regulatory scope and pace of approvals between the two regions.
Click here to see the overview of authorised ATMP's
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| Regulatory T-cell scientist trio wins Nobel Prize | | | | |
| This year's Nobel Prize in Physiology or Medicine has been awarded to three scientists for their work on regulatory T-cells (Tregs), research that has fuelled a well-stocked pipeline of potential new therapies for cancer and autoimmune diseases.
Read more here.
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|  | Father created a drug to save his son from a rare disease, now other families are desperate to get it | | | | |
| | | | On April 2, 2019, Terry and Georgia Pirovolakis received devastating news: their youngest son, Michael, had been diagnosed with the ultra-rare hereditary disorder SPG50. Doctors told them there was nothing to be done - only to love him and make the most of the time ahead. This marked the beginning of the Pirovolakis family's quest to find a better future for Michael, a journey that led to the creation of Elpida Therapeutics, a non-profit dedicated to developing life-saving treatments for children with rare diseases.
Read more about Pirovolakis journey here.
The story also connects to Denmark, where two boys with the same condition were once meant to receive gene therapy in the US - a plan that fell through in 2016 when Trump came to power. Today, two doses of that therapy sit frozen in San Sebastián, Spain. A Danish hospital has now agreed to administer the treatment - if regulatory authorities approve it, and if the families can cover the costs themselves. Importantly, the hospital insists the treatment must be made available for all patients with the diagnosis, not just for a few. It is quite unconventional when a non-profit company knocks on the door of a hospital and offers a self-developed medicine that costs nothing for an extremely rare disease for which there is no treatment. Chief physician Alfred Peter Born from Rigshospitalet calls it "an exciting development," as it could pave the way for new models to bring treatments to patients with ultra-rare diseases.
Read more here (in Danish and behind paywall).
At the same time, crowdfunding and other patient-driven initiatives in the development of innovative therapies raise important ethical considerations—from safety and oversight to fairness and transparency. These dilemmas are highlighted in The Lancet Neurology editorial Crowdfunding for neuromuscular disease treatment: the ethical implications, and echoed by the European Medicines Agency, which recently warned against the uncritical use of unregulated advanced therapies. | | | | |
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| | | |  | Putting patients first: why transparency in industry partnerships matters (Guest blog) | |
| | | | When pharmaceutical companies and patient organizations join forces, transparency isn't optional — it's essential. In this guest blog from EFPIA, it is argued that openness in financial and non-financial ties fosters trust, accountability, and genuine collaboration. Discover why clear standards for disclosure are vital to keeping patient interests front and center.
Read more here.
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| | | | .png) | Innovation is not enough: ATMPs deserve innovative markets | |
| | | | Advanced therapy medicinal products (ATMPs) are redefining what's possible in modern medicine. Yet their path from lab to patient isn't straightforward. In this column, Wouter Boon reflects on the challenges these groundbreaking therapies face when entering systems designed for traditional medicines, and why true innovation requires not just new science, but new markets too.
Read more here.
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| | | |  | Better framework for international cooperation on ATMPs | |
| | | | Monitoring side effects and following up on the effect of Advanced Therapy Medicinal Products (ATMPs) over a long period of time creates issues in relation to patient self- determination and data pro-tection. Regulatory frameworks that support more seamless international collaborations from production to treatment and follow-up are the way forward, writes Jakob Wested.
Read more here (in Danish). | |
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| | | | .png) | An interview with Birgitte Klindt Poulsen – Chairman, Danish Medicines Council | | | | |
| Since stepping into the role of Chair of the Danish Medicines Council in early 2025, Birgitte Klindt Poulsen has been navigating the delicate balance between accelerating patient access to innovation and maintaining the rigour of independent, evidence-based evaluation. In this interview, she shares how the Council is adapting to a surge in applications, embracing real-world evidence, advancing European collaboration, and preparing for the growing complexity of personalised and advanced therapies.
Read more here. | | | | |
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| | | | .png) | A path to better include patients' perspectives in the regulation of medicines | | | | |
| EMA has published a draft reflection paper on patient experience data for public consultation, with the aim of opening a new chapter in how patient perspectives and needs are considered in the regulation of medicines across the EU. These are data directly reflecting patients' experience or preferences on treatments or outcomes, without any interpretation by a clinician or anyone else.
This draft reflection paper is open for public consultation until 31 January 2026.
Read more here.
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| | National developments | |
 | | | | | | | An overview of recent ATMP developments from across EU member states. This section highlights key national initiatives and regulatory developments shaping ATMP progress in individual EU countries.
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| | | |  | New possibilities for Advanced Therapy Medicinal Products in Estonia | | | | |
| On May 10, 2025, amendments to the national framework for Hospital Exemption came into force in Estonia. The key changes are: 1) no upper limit for patient number, provided that the treatment's benefit-risk balance is favorable and
2) authorization can now be extended for up to five years. These changes aim to make the Hospital Exemption more flexible and therefore expand the possibilities to prepare and use of advanced therapy medicinal products (ATMPs) in situations where no suitable authorized product is available or sufficiently available on the market.
Read more here. | | | | |
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| | | |  | Tracking a decade of phase I clinical trials in Italy: trends and insights from national and european registries amid regulatory change | | | | |
| The global clinical research landscape has expanded significantly over the past decade, yet Europe has witnessed a decline in its share of clinical trial activity, especially in early-phase trials. This study aims to characterize the evolution of Phase I clinical trials in Italy from January 2015 through the completion of the European regulatory transition under Regulation (EU) 536/2014 (30 January 2025), by analyzing trends in trial submissions, sponsor typologies, study designs, and participant populations.
Read more here.
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| | | |  | The first non-commercial CAR-T therapy performed in Poland | |
| | | | A major milestone in Poland's cancer-care landscape: the first non-commercial CAR‑T therapy has been administered to a patient with a relapsed aggressive lymphoma. This Car-T is the first academic CAR-T where cells were collected, produced, multiplied and administered in the country without the intermediation of a commercial pharmaceutical company.
Read more here (in polish and behind paywall) | | | | |
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| | | |  | Denmark gets personal with new medicines strategy | |
| | | | Denmark's new personalised medicines strategy could set an example for the rest of Europe, enabling a new generation of innovative treatments at scale. The Danish government and Danish Regions have launched a new strategy for personalised medicine, focusing on patient involvement and the ethical use of individuals' health data – it's designed to boost innovation and improve therapies.
Read more here.
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| | | | .png) | Belgium Approves New Early & Equitable Fast Access Procedure for Innovative Drugs | |
| | | | Starting from 1 January 2026, Belgium will allow the use of innovative therapies for severe or life-threatening conditions even before they receive official approval from the European Medicines Agency. The new procedure, named "Early and Fast Equitable Access," will take effect on 1st January 2026 and will provide temporary reimbursement of promising new treatments even before they receive marketing authorisation.
Read more here.
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| | Hospital Exemption | |
| | | | | | | This section covers recent developments and regulatory updates on the hospital exemption pathway for ATMPs in the EU. | | | | | | | |
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| | | |  | Joint Statement on the Hospital Exemption for ATMPs | | | | |
| Key stakeholders across the European healthcare ecosystem — including research centres, healthcare professionals' associations, and patient organisations at both EU and Member State levels — respond to the latest proposed changes to the Pharmaceutical regulation review currently in progress. In this they urge the co-legislators to integrate these five critical points:
1. Regulatory clarity and primacy of GMP
2. A dedicated pathway for manufacturing
under HE
3. Removal of the 'non-routine' term
4. Fit-for-purpose evidence
5. Cross-border exchange of HE-ATMPs
These five point are critical for a pragmatic, uniform, and ethical regulatory framework that truly allows fair access to quality and safe treatments at the lowest cost possible.
Read more here. | | | | |
| New EU study on the Hospital Exemption | | | | |
| Discover the latest insights from the EU's landmark study on the Hospital Exemption (HE). The general objective of this study is to give an overview regarding the national rules providing frameworks for HE and the functioning of HE in EU Member States. This HE study covers the implementation of HE in the EU from the entry into force of the ATMP Regulation (30 December 2007) until January 2025, with an additional in-depth focus on the 19 EU Member States. Moreover, it gathers information on other international regulatory frameworks for innovative products. Moreover, it gathers information on other international regulatory frameworks for innovative products. This study also aims to propose set of data to be considered for the national implementation report.
Read more here. | | | | |
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| | | | .png) | ATMPs prepared under hospital exemption: European Blood Alliance position paper | | | | |
| Although industry associations have generally supported tighter restrictions on the use of the Hospital Exemption, the European Blood Alliance (EBA), alongside multiple scientific societies and professional organizations, has expressed strong support for maintaining and strengthening this exemption.
Read more here.
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| Current State- Of- Play of the EU Advanced Therapy Medicinal Product (ATMP) Field | | | | |
| Fifteen years after the introduction of the EU Advanced Therapy Medicinal Products Regulation, this paper takes a critical look at its tangible impact on the ATMP field. It analyzes how the regulation has shaped product approvals, innovation, and market access, while identifying key gaps that continue to challenge developers and regulators alike.
Read more here.
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| The Hospital Exemption pathway for non-industrially manufactured advanced therapy medicinal products | | | | |
| The aim of this article is to discuss the regulatory challenge related to the fact that in the European Union, together with ATMPs that have received marketing authorisation, other ATMPs also exist. These are usually referred to as products prepared pursuant to the Hospital Exemption (HE) scheme.
Read more here.
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|  | | | Conference | 09-11-2025 to 12-11-2025 |
| | | Conference | 27-11-2025 to 28-11-2025 |
| | | Conference | 27-11-2025 to 28-11-2025 |
| | | Conference | 17-03-2026 to 18-03-2026 |
| | | Conference | 25-06-2026 to 26-06-2026 |
| | | Conference | 05-08-2026 to 7-09-2026 |
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|  | | | | This section provides a overview of key findings on why access to cutting-edge treatments still varies widely across the EU and the world — from regulatory bottlenecks and pricing pressures to national system constraints that leave some patients behind. | | | | |
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| | | | .png) | Disparities in access to gene therapy in the European Union: ethical and regulatory challenges | | | | |
| Gene therapies represent a significant advancement in modern medicine, offering potential cures for untreatable genetic disorders. However, equitable access to these innovative therapies remains a critical ethical challenge within the European Union (EU). This paper examines the adequacy of the EU's centralized market authorization framework, supplemented by alternative pathways such as the Hospital Exemption and Compassionate Use Program, in addressing access disparities.
Read more here.
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| Enabling manufacture and access to advanced therapy medicinal products in low- and middle-income countries | | | | |
| This paper shines a spotlights on one of the greatest ethical and practical hurdles we face: inequitable access. While ATMPs promise transformative outcomes, the authors highlight a stark divide between high-income countries (HICs) and low- and middle-income countries (LMICs), where
even ATMPs that apply to larger segments of the population and are currently being used in high-income countries (HICs), are not affordable for most of these countries' patients.
Read more here. | | | | |
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| | | | .png) | Early access disparities in innovative therapies across the US, EU, China, and Japan
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| Early access to innovative therapies offers a critical lifeline for patients with severe or life-threatening conditions, yet substantial disparities persist across the United States (US), the European Union (EU), China (CH), and Japan (JP). These disparities arise from diverging definitions of unmet medical need (UMN), heterogeneous timelines for regulatory review, and uneven implementation of Early and Expanded Access mechanisms.
This policy review provides a comparative analysis of regional frameworks, emphasizing how clinical trial geography, eligibility rules, and access mechanisms interact to shape pre-approval opportunities for patients.
While regulatory innovations such as the FDA's Breakthrough Therapy designation, the EMA's PRIME scheme, the PMDA's Sakigake program, and recent reforms in China have accelerated access in selected contexts, cross-border misalignments and fragmented health technology assessment (HTA) processes continue to generate inequities. The EU's new HTA Regulation (2021/2282) represents a step forward by embedding joint clinical assessment and real-world evidence, but persistent structural inconsistencies hinder timely and equitable uptake. By reframing uncertainty as a shared responsibility among regulators, developers, payers, clinicians, and patients, early access governance can be redesigned to promote equity, transparency, and responsiveness.
Read more here. | | | | |
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| | | | .png) | From laboratory to patient: how do we make ATMPs sustainable and accessible?
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| During a recent trade mission to Boston, I formulated a key challenge: if we really want to get advanced therapies (ATMPs) to patients – and to do so in a sustainable and affordable way – we need to think disruptively about development, production and valorisation.
In this column, Frank Luyten reflects on the future of Advanced Therapy Medicinal Products (ATMPs) and regenerative medicine, arguing that while these therapies hold immense promise for curing disease, delivering them sustainably and affordably will require truly disruptive thinking in how we develop, produce, and valorize them. Read more here.
Sustainability and affordability of medicines in more broad terms was also the issue in a recent Lancet editorial: Drug pricing and pharmaceutical innovation: a false promise. | | | | |
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| | | | .png) | New data shows no shift in access to medicines for millions of Europeans | | | | |
| New data from two reports, published by EFPIA, highlight the ongoing challenges of providing equal access to medicines for all Europeans. According to EFPIA's 2024 Patients W.A.I.T. Indicator Survey, the average time from European Medicines Agency (EMA) approval of a new treatment to patient access across Europe is 578 days – more than a month longer than 2023. The Patients W.A.I.T. Indicator shows persistent, significant variation in access to new medicines between countries across the EU e.g. In Germany, patients typically wait as little as 4 months, while in Portugal it takes almost 2.5 years. The CRA Report on the Root Causes of Unavailability and Delay to Innovative Medicines furthermore identifies the numerous interlinked reasons behind the delays and barriers that patients face.
Read more here. | | | | |
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| | | |  | | | | Appl Health Econ Health Policy, 16 October 2025 | | | | |
| Health Technology Assessment Guidelines and Recommen-dations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations
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| Due to limited clinical data, high uncertainty, and outcome variability, assessing therapies for paediatric and rare disease populations poses specific challenges, often requiring adjustments to standard health technology assessment (HTA) frameworks. This study examines how national HTA guidelines and recommendations across Europe reflect these demands, identifying methodological adaptations and country-specific disparities.
Read more here.
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| | | | .png) | | | | Front. Med., 05 August 2025, Regulatory Science | | | | |
| Implementation of a quality management system for decentralized manufacturing of cell and gene therapy products - technical and regulatory considerations
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| Decentralized manufacturing has emerged as a promising approach to improve the accessibility and scalability of cell and gene therapy products, particularly for autologous treatments. This paper proposes a comprehensive Quality Management System framework tailored to decentralized cell therapy manufacturing, integrating current Good Manufacturing Practice principles and regulatory oversight through a centralized Control Site model.
Read more here. | | | | |
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| | | | .png) | | | | Regenerative Therapy, Volume 30, December 202, Pages 358-370. | | | | |
| Current challenges and future directions of ATMPs in regenerative medicine | |
| | | | Although ATMPs have offered significant improvement for a variety of severe illnesses, their progressive development is faced with numerous challenges. Some of these challenges are current complexities in their manufacturing, such as, efficacy, scaling up, scaling out, product efficacy, packaging, storage, stability, and logistic concerns. This article reviews the current challenges in ATMP manufacturing and application, highlights the advancements in technology that are paving the way for improved therapeutic strategies, and offers future perspectives on overcoming these barriers.
Read more here.
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| | | | .png) | | | | Gene Therapy 36, nr. 17–18 (2025): 1154–58. | | | | |
| Regulatory Strategies for Accelerating the Translation of Gene Therapies to Clinical Practice: Focus on GMO Considerations | |
| | | | Despite significant advancements, the classification of gene therapy medicinal products (GTMPs) as genetically modified organisms (GMOs) under EU legislation imposes significant regulatory burdens, hindering early and timely patient access to such therapies. Current GMO regulations, originally designed for agricultural biotechnology, require environmental risk assessments (ERAs) and additional approvals, creating delays and increasing costs. This article examines the scientific and regulatory discrepancies in classifying GTMPs as GMOs.
Read more here. | | | | |
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| | | |  | | | | GRUR International, Volume 74, Issue 5, May 2025, Pages 399–415. | | | | |
| Addressing the Gene Therapy Bottleneck in the EU: Patent vs. Regulatory Incentives | |
| | | | Today, the US leads the field of personalised medicine gene therapies, while the EU lags behind. The implemen- tation phase of gene therapy technologies remains a signi-ficant bottleneck in Europe. The legal framework for gene therapies differs significantly between the US and the EU as methods for the treatment of the human body by therapy are excluded from patent protection in Europe. Taking the creation of R&D incentives and information disclosure as core objectives of modern patent systems, this article analyses the potential to repeal the existing patentability excep-tion to allow for patent protection for gene therapies as such.
Read more here. | | | | |
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| | This webinar brought together key voices from across the EU healthcare landscape to explore what it takes to successfully adopt decentralised trial elements. | | | | |
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| | On November 22, 2024, The CREATIC team held the first CREATIC LabtoP talk about bringing ATMPs from lab to patients. You can now listen to the podcast from the webinar and learn more about bringing ATMPs from lab to patients, with focus on the Hospital Exemption as a pathway.
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| | High-quality clinical evidence is the foundation of effective healthcare decisions. Watch this LinkedIn Live hosted by the European Medicines Agency where they discussed how Europe can enhance evidence generation in a rapidly evolving healthcare landscape. | | | | |
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| | | | | This newsletter is curated by the CREATIC project, views and opinions expressed in this newsletter are those of the author(s) only and do not necessarily reflect those of the European Union or European Research Executive Agency. Neither the European Union nor the granting authority can be held responsible for them. | | | | |
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