TRANSFORM Alliance

Yesterday, the European Parliament’s new standing committee on public health (SANT) held its constitutive meeting and elected its Bureau. TRANSFORM welcomes the elevation of SANT to a fully-fledged parliamentary committee, seeing the EU deliver on its promise to make public health a European priority for the benefit of all Europeans. With forthcoming legislation - including the highly anticipated Biotech Act - set to completely transform the European ecosystem for medical biotechnologies, TRANSFORM looks forward to working with committee members and helping deliver on the promise this flagship legislative project brings for the future of EU competitiveness and European patients’ access to quality healthcare. We congratulate MEP Adam Jarubas for his election as Committee Chair, and TRANSFORM MEP Interest Group members Tilly Metz and Stine Bosse for their election as first and second Vice-Chairs, respectively. #MEPsforATMPs #cellandgenetherapy #biotechact #rarediseases #innovation #EuropeanParliament #SANT 

As a multistakeholder alliance, TRANSFORM closely follows the key issues at the top of our members' agendas: the TRANSFORM secretariat was delighted to attend the IPOPI - International Patient Organisation for Primary Immunodeficiencies PID Forum on the potential of #AI in #PID, hosted by MEP Andras Kulja. There's a lot of talk about the potential of AI in healthcare, but what are its concrete applications and challenges?   During yesterday's PID Forum, IPOPI showcased concrete examples of projects using AI to speed up and improve diagnosis in PID, which is still a critical issue for many people suffering from rare diseases.   Some challenges encountered included gaining hospital management and ethics committee approvals, accessing quality data to model algorithms, and transferring these models to different databases and datasets. Broader questions remain on the application of AI in healthcare, such as the required financial investments, insurance coverage, staff training, and willingness of healthcare professionals to integrate AI tools into their clinical practice. Amidst all this, there are the end-users, the patients, keen to reap the benefits of innovation but wishing to maintain a personalised and human approach to their health care while ensuring the protection of personal health data.   The AI Act will officially come into force in August 2026. Still, around 60 implementing acts will need to be developed to finetune and adapt this legislation to stakeholders' needs, including patients, clinicians, researchers, and industry. Events and conversations like those held yesterday in the European Parliament will help policymakers ensure upcoming legislative acts are fit for purpose and meet end-user needs. 

We continue our member spotlight series today, as ‘TRANSFORMTalks’ #regulatorysupport with Etienne Régulier, Vice-President of Market Access, Pricing and Public Affairs (EMEA) at Santen Pharmaceutical. Santen is a leading specialist ophthalmic pharmaceutical company, leveraging over a century of deep expertise to enhance the vision and quality of life of patients across more than 60 countries in the EMEA region. With the rapid evolution of technology driving innovative treatment and diagnostic methods, Santen remains at the forefront of addressing significant unmet needs in ophthalmology. Their diverse portfolio spans over-the-counter and prescription products, offering innovative solutions for a wide range of eye conditions. The ATMP space features highly innovative products. However, ATMP developers face many R&D challenges. What are some of these hurdles in the ophthalmic space?   The development and delivery of ATMPs in ophthalmology present several challenges: clinical trial design remains a key hurdle, with the rarity and variability of many ophthalmic diseases complicating patient recruitment and endpoint determination. Manufacturing is another challenge, requiring scalable processes that maintain product consistency and stability, while delivery methods such as intravitreal or subretinal injections must be carefully optimised for safety and efficacy in the delicate ocular environment. Additionally, navigating the regulatory landscape can be complex. Despite these obstacles, the promise of ATMPs in offering sight-saving solutions for patients with severe or previously untreatable conditions underscores the importance of addressing these challenges. ATMP developers face multiple hurdles in developing clinical data portfolios for their products. With the revision of the General Pharmaceutical Legislation, TRANSFORM has been advocating for the inclusion of ATMPs within the scope of EMA scientific and regulatory support. How significant can these tools be in helping developers overcome these hurdles?   Clearer guidance on clinical trial design, acceptable endpoints, and manufacturing standards can help developers address key uncertainties. Access to scientific advice and knowledge sharing with regulatory bodies like the EMA foster collaboration and bridge knowledge gaps, ultimately improving the robustness of clinical trials and accelerating timelines for ATMP patient delivery. Initiatives such as PRIME designation or tailored frameworks also encourage investment in high-potential but high-risk therapies. Furthermore, harmonising regulatory approaches across the EU simplifies compliance, particularly for smaller developers, enabling them to navigate the complex landscape more effectively. #ATMP #cellandgenetherapy #ophthalmology #innovation #EMA #scientificadvice

TRANSFORM prides itself on multistakeholder advocacy work for safe and timely access to advanced therapy medicinal products (ATMPs, or cell and gene therapies). As 2025 begins, we will introduce TRANSFORM members through a new series of interviews, TRANSFORMTalks, to showcase their work and perspective on how TRANSFORM supports ATMP research, development and patient access. Today, 'TRANSFORMtalks' with Giovanni Migliaccio from EPTRI about #collaborativeresearch. Giovanni is a member of the TRANSFORM Steering Group and represents academic researchers and developers active in the ATMP space. EPTRI is a research infrastructure designed to implement research strategies and access to key technologies, standardised models and analytical tools, and offer to public and private stakeholders qualified research services dedicated to the development of paediatric drugs, diagnostics, devices and clinical studies. EPTRI is pivotal in connecting academic research units with biotech and pharmaceutical companies, promoting tailored solutions for the paediatric population and implementing innovative collaborative models that lower the entry barrier for novel tools and practices. Paediatric research into innovative therapies faces several challenges. What is one of the primary hurdles, and how can TRANSFORM support the community in overcoming it? Most paediatric pathologies, apart from infectious diseases, are of genetic origin. These are the obvious targets for genetic diagnosis and therapies designed for the foetal and newborn populations, aiming to prevent the insurgence of degenerative traits and ensure a normal life for patients. In the rare disease field, targeted therapies are mainly driven by academic research institutions. Hence, there is an urgent need to secure support from public and private institutions to mitigate reduced interest and support from commercial partners. EPTRI has a pivotal role in connecting academic and biotech stakeholders to advance ATMPs, thus aligning with the TRANSFORM goal of facilitating discussions between stakeholders and policymakers to ensure timely access to cell and gene therapies. TRANSFORM has been a strong advocate for a collaborative R&D ecosystem in the EU. How important are the synergies between commercial and non-commercial ATMP researchers and developers? Many academic institutions do not have the capacity, expertise and funds to pursue existing regulatory pathways to drug development, and therefore the application for marketing authorisation remains largely outside their remit. EU-funded partnerships and many European Reference Networks supply some capacity and support. EPTRI's academic community further complements these efforts. However, as commercial sponsors have more significant experience with the intricacies of commercial development, bringing novel products to the market requires strong collaboration between industry, academia and public non-profit investors. #ATMP #cellandgenetherapy #orphandrugs

❄️With the holiday season in sight, we are taking this opportunity to wish all of you Happy Holidays! ⭐️2024 was a pivotal year for Europe: with a new European Parliament and College of Commissioners now in place, TRANSFORM will once again present a unified position on behalf of the patient, clinician, academic and industry representatives of our Alliance, on our key priorities - advancing patient care, boosting European competitiveness, and fostering a supportive environment for innovative therapies. 🥂See you next year!

🧬Why bring ATMP research, development and access issues to the EU agenda today? 🎙Hear what our Steering Group had to say on the need to tailor incoming EU legislation to the specificities of ATMPs. Martine Pergent from IPOPI - International Patient Organisation for Primary Immunodeficiencies and Giovanni Migliaccio from EPTRI joined us last month for the annual TRANSFORM MEP Interest Group Conference, alongside other patient, clinician, academic and industry representatives from the Alliance. 🇪🇺Multistakeholder dialogue is a core value for TRANSFORM and a key tool in the development of future-proof policy that can drive patient access to innovative therapies across the EU. ➡️Visit our website to read the full event report: https://lnkd.in/e5XfpZRH #MEPsforATMPs #EuropeanParliament #orphanmedicines #competitiveness #raredisease #cellandgene 

🎙“All this translates into an environment of business predictability. I think what would attract investment in this part of the world would be exactly this sense of business predictability.”   🧬Speaking during our second panel on the future development and delivery ecosystem for ATMPs, Francis Pang from Orchard Therapeutics laid out the perspective of small and medium-sized ATMP developers: in order to bring patient access earlier in the global launch sequence, more efforts are required in accelerating approval pathways, broadening reimbursement for ATMPs, and overcoming European fragmentation in the area of value assessment. However, another crucial step centres around cross-border healthcare: many ATMPs are transformative, but have limited therapeutic windows and we need to translate the right to cross-border healthcare into viable access pathways that are not too cumbersome and challenging for patients, clinicians and their resident healthcare systems.   Check out our website for a full report of the discussions our panelists held during the 2024 TRANSFORM MEP Interest Group: https://lnkd.in/e5XfpZRH #MEPsforATMPs #cellandgenetherapy #innovation #HTA #europeancompetitiveness

📑Missed the 2024 TRANSFORM MEP Interest Group Conference? Read our event report below to learn more about the key takeaways, messages and recommendations our panelists shared with the ATMP community at the start of the new European mandate. 🙌🏼Multistakeholder dialogue remains very much at the centre of our activity, driving engagement across the patient, academic, medical and developer communities.   🇪🇺Our event captured this ambition by bringing all these voices under one roof for a broad-scoped conversation focused on the current European ATMP landscape and regulatory environment, with an outlook on how present and future legislative initiatives could support a thriving research and innovation health ecosystem that supports timely access to advanced therapies. 🧬Visit our website to learn more about our MEP Interest Group: https://lnkd.in/eFWDMeuv #MEPsforATMPs #cellandgenetherapy #rarediseases #competitiveness #patientaccess EUCOPE - European Confederation of Pharmaceutical Entrepreneurs EURORDIS-Rare Diseases Europe European Federation of Neurological Associations IPOPI - International Patient Organisation for Primary Immunodeficiencies EPTRI Billy Kelleher MEP Stine Bosse

🎙“What we need to do first is to make sure that we have a good and clear definition of unmet medical need: we need to know the true impact of health conditions.” 🧬Speaking at the 2024 TRANSFORM MEP Interest Group Conference, Iva Galovic from the European Federation of Neurological Associations emphasised the importance of including the patient and caregiver perspective in the decision-making process. The Pharmaceutical Package offers a once-in-a-generation opportunity to deliver a regulatory and legislative framework for equitable access to advanced care. Patients’ experience in living with often severely debilitating chronic diseases can inform the provisions from the Package with a holistic picture on the invisible social, economic and emotional burdens these diseases bring to patients. Check out our website for more information on our MEP Interest Group: https://lnkd.in/eFWDMeuv #MEPsforATMPs #rarediseases #cellandgenetherapy #neurologicalconditions 

🎙“Challenges across Europe are affecting these three areas: we need to incentivise innovation, we need to balance it with sustainability and affordability, and we need to drive value and access in our healthcare systems.”   💊Adam Parnaby from Bristol Myers Squibb spoke during one of our panels on the need to future-proof EU healthcare by taking a holistic, drug lifecycle approach: while it is crucial that research and innovation are supported and incentivised, solutions must also be designed for the challenges awaiting developers further down the drug development process. New tools are needed to ensure that regulators and assessors are able to capture ATMP clinical evidence in a way that is then translatable to healthcare systems, allowing them to make intelligent, future-oriented reimbursement decisions.   🧬Make sure to follow our LinkedIn page for more forceful messages from the annual TRANSFORM MEP Interest Group Conference. Check out our website for more information on our MEP Interest Group: https://lnkd.in/eFWDMeuv #MEPsforATMPs #cellandgenetherapy #EuropeanParliament #innovation #competitiveness #HTA