TRANSFORM Alliance

The #EuropeanCommission launched last week a public consultation on the EU Life Sciences Strategy, the first in a series of initiatives set to transform the European biotech ecosystem. TRANSFORM is pleased to see the Commission recognise the decline of EU competitiveness in key sectors such as orphan drugs and ATMPs, as well as the key factors negatively impacting the R&D landscape that supports these biotechnologies: ⚪ Insufficient translation of innovation into products with viable market prospects ⚪ Overlapping, time-consuming and complex regulatory requirements for the development, manufacturing and use of biotechnologies ⚪ Insufficient capital infusions into biotech start-ups Our multistakeholder alliance remains committed to supporting the European Commission and all other institutional partners on the journey to create better legislation for the sector. Stay tuned for more information on what TRANSFORM is doing to support the EU life sciences sector. Access the consultation webpage here: https://lnkd.in/esAwV9tx #ATMPs #biotech #biotechact #EuropeanLifeSciences #orphanmedicines #innovation

🇪🇺Today, the Commissioner for Health and Animal Welfare Olivér Várhelyi reiterated before the SANT Committee his commitment to fostering a competitive and resilient biotech ecosystem in the EU.   The structured dialogue touched upon the recently published #CriticalMedicinesAct, the implementation of the Beating Cancer Plan, and forthcoming legislative initiatives in the areas of #MedicalDevices, #HealthData, and #biotech.   🧬We were pleased to hear the Commissioner's continued commitment to making the upcoming #BiotechAct the centrepiece of his mandate: the Act is meant to boost the EU economy and enable effective translation of scientific discoveries into marketable therapies that can reach patients across the EU. The Act will also drive greater investment into the sector and advance treatment and screening solutions for rare disease and paediatric cancer patients.   TRANSFORM is closely following legislative developments in the medical biotechnology sector and the forthcoming consultation on the Biotech Act.   #MEPsforATMPs #cellandgenetherapy #rarediseases #orphanmedicines #competitiveness 

💡Share your insight! 🧬Join4ATMP launched earlier this week their expert survey on the clinical, regulatory and economic challenges of ATMP research and development. All stakeholders are invited to share their insight on: - pre-clinical development - GMP - clinical translation - economic hurdles - stakeholder awareness ➡️Access the survey here: https://lnkd.in/eDnkn6gs

Earlier this week, the #EuropeanCommission published its proposal for the #CriticalMedicinesAct, which it presented as an effective tool to facilitate patient access to critical medicinal products and other therapies when market deficiencies impact their availability.   The Act features provisions for ‘medicinal products of common interest’, including collective/joint procurement frameworks for medicinal products undergoing Joint Clinical Assessments under the HTA Regulation - covering ATMPs as of January 2025.   As a multi-stakeholder alliance committed to ensuring safe and timely patient access to critical, life-saving therapies, we appreciate the Commission’s ambition to strengthen patients’ rights to quality healthcare in Europe. We remain committed to supporting all institutional partners in developing novel and equitable access pathways across the EU, while ensuring that the provisions are fit-for-purpose, proportional, and supportive of the European innovation base. #ATMPs #orphanmedicines #rarediseases #innovation #cellandgenetherapy #criticalmedicines

This week, we are talking #UnmetMedicalNeeds (UMNs) with Matt Bolz-Johnson, ATMP & Mental Health Advisor at EURORDIS-Rare Diseases Europe. EURORDIS is a non-profit alliance representing over 1000 rare disease patient organisations from 74 countries. Together, the network advocates for policies and initiatives that can improve the lives of the 30 million+ European citizens living with a rare condition: EURORDIS engages at the national, EU and international levels, and develops evidence-based policy recommendations, such as their ‘Rare 2030’ foresight study, to support EU action in the areas of diagnosis, healthcare pathways, novel treatments, needs-led innovative research and UMNs in rare disease patients.        UMN is a crucial issue in rare disease policy, informing much of EURORDIS’s advocacy work and covering a wide spectrum of clinical and non-clinical factors impacting patients’ daily lives. What are some of its dimensions policymakers often overlook? Rare diseases are the definition of UMN due to the rarity of the conditions, their medical complexity, and their debilitating, life-changing or life-limiting nature. UMN is further magnified by the challenges and limited availability of diagnosis and treatments - 95% of rare conditions do not have an effective therapy. Key dimensions to be included in defining UMN should include population size, medical complexities, and the impact on patients' health outcomes and their quality of life, daily living, and ability to attend education or work - with the wider social and economic ramifications for society at large. The availability (or lack) of an alternative existing therapy and the ability to diagnose in a timely manner are also important factors. ‘High’ unmet medical need (HUMN) is terminology that is bound to create difficulties in assessment, but also can create confusion for our patient community who clearly see this bilateral division as “unfair”. Therefore, EURORDIS is supporting the removal of the HUMN terminology from policies.  TRANSFORM has been a strong advocate for the development of comprehensive yet flexible definitions of UMN. How could patients’ voices be captured in the development of such definitions? Any attempt to set out a singular definition of UMNs will fall short of adequately quantifying the real-life impact of rare conditions - it is a continuous process and not a standalone checklist. UMNs can only be quantified based on core principles where patient groups can provide the real world evidence on the impact of the condition. Early multistakeholder dialogue on specific diseases is needed, and patient groups can play a critical role in the co-development of guidelines for evaluating UMNs, by providing the evidence, public assurance and transparency of the decision-making process. EURORDIS has long experience in this area - we have been involved in patient engagement for many years. #ATMP #cellandgenetherapy #rarediseases

📣We are excited to share with you that The European Association of Hospital Pharmacists (EAHP) is now the newest member of the TRANSFORM Alliance! 🏥EAHP is a network of national organisations representing over 29,000 hospital pharmacists across 36 member countries. The association works towards the development of hospital pharmacy for the benefit of patients across Europe: EAHP serves as a platform for education, training and professional development, as well as a common voice for European hospital pharmacists and their advocacy efforts at both the national and EU levels. Multistakeholder representation is a core value for TRANSFORM, and we are thrilled to have hospital pharmacists' perspective featured in our advocacy efforts for safe and timely patient access to #ATMPs. Learn more about EAHP here: https://lnkd.in/eVVVpb6v #genetherapy #celltherapy #hospitalpharmacy #ATMP #rarediseases #orphanmedicines 

👋 Meet our new member! TRANSFORM is delighted to welcome GBS|CIDP Foundation International to the multi-stakeholder Alliance 🎉 Their knowledge and expertise will be invaluable for TRANSFORM’s advocacy to improve access to ATMPs for all European patients that need them. As an Alliance, we will keep ensuring the complexities and specificities of ATMPs are not forgotten in policy and legislative files 🧬 Welcome to TRANSFORM!

🎙️This week, ‘TRANSFORMTalks’ #Access for ATMPs with Joelle Khraiche, Director for International Advocacy at CSL Behring.   CSL is a global biotechnology company, operating as one integrated research and development organisation active across 10 countries with over 32,000 employees. CSL’s extensive portfolio – covering rare diseases, influenza vaccines and iron deficiency & nephrology – has been delivering life-saving treatments and vaccines to patients in more than 100 countries. CSL Behring, a CSL subsidiary named after Nobel Prize winner Emil von Behring, works to deliver advanced therapies' promise for a long-term cure in several disease areas, including rare genetic bleeding disorders and primary immunodeficiency. Cell and gene therapies are biological medicines for which comprehensive clinical data is harder to develop. How do hurdles in data generation impact market access further down the line? We see diverging approaches across countries when it comes to data requirements, making it challenging for companies to generate a suitable data package that can be adequately assessed - this can ultimately lead to patient access barriers. We need to progress on real-world evidence collection - integrated into a strong rare diseases data ecosystem at the European level - to provide robust data that can inform HTA and P&R decisions, support improved healthcare delivery, and help advance R&D for the benefit of patients. Solutions can be found if we establish early dialogue with all the involved stakeholders and create true willingness to partner and overcome hurdles. TRANSFORM has been a strong advocate for the implementation of novel payment models for ATMPs, such as outcomes-based models that integrate real-world evidence into national decision-making. Why are these novel payment models better suited to the ATMP market than conventional ones? National value assessment processes have been created without the specific features of one-off transformative therapies, which can replace existing lifelong chronic treatments, in mind. There is a need for new models to assess and account for the projected long-term durability and potential cost savings that ATMPs can bring. Flexible contracting solutions, such as outcome-based models supported by real-world evidence, can help governments manage budget impact. We are encouraged by Member States that send a strong signal on how innovative and collaborative thinking can make ATMPs a reality for patients. We call on the European Commission and the upcoming Danish Presidency to support the sharing of best practices in novel payment models. ➡️Learn more about CSL Behring here: https://lnkd.in/ewW-8PeE #ATMP #celltherapy #genetherapy #HTA #RWE #PatientAccess

🧠Rare neurological conditions cover a wide spectrum of #UnmetMedicalNeed in Europe and elsewhere: the treatment and management of these life-limiting or severely debilitating diseases pose several challenges other rare disease patient groups also struggle with: access to fast and accurate diagnosis, access to innovative therapies and deeply ingrained stigma.   🧬Ahead of #RareDisease Week, we were delighted to join the European Federation of Neurological Associations, our Alliance Member, for a timely discussion on policy responses to rare neurological challenges. Empowering patient voices is a core value for TRANSFORM, and we were touched by the many testimonials our colleagues from EFNA brought forward to the MEPs present in the room.   🇪🇺Our Co-Chair Billy Kelleher MEP, who hosted the event, emphasised the large scope for EU added value in the area of rare diseases: despite not having exclusive competence in health, the EU can support the harmonisation and development of benchmarks on several key items - such as #NewbornScreening - that can advance the state of care for the 30 million+ EU citizens living with a rare condition. Learn more about the event here: https://lnkd.in/eeXwYd5y   #MEPsforATMPs #orphanmedicines #rarediseases #neurology #ATMP #celltherapy #genetherapy

This week, the European Commission unveiled its 2025 Work Programme, announcing several initiatives at the intersection of public health and competitiveness that are set to influence ATMP research, development and access in the future. While it seems that the highly anticipated Biotech Act will be pushed back to 2026, the Critical Medicines Act, expected early this year, was prefigured by Commissioner Olivér Várhelyi to include provisions for equitable access to innovative therapies, potentially including ATMPs. The Strategy for European Life Sciences could have deep ramifications for the medical biotechnology sector, and the TRANSFORM Alliance hopes that the initiative will support investment into the sector, streamline R&D for ATMPs, and fully leverage available infrastructures for effective innovation uptake. The EU Start-up and Scale-up Strategy also holds greats potential for Europe’s small- and medium-sized ATMP developers, the backbone of the EU medical biotechnology ecosystem. Follow us on LinkedIn to learn more about the TRANSFORM positions on the forthcoming initiatives, and read the Commission 2025 Work Programme here: https://lnkd.in/disrWz3z #celltherapy #genetherapy #EuropeanCommission #ATMP #MEPsforATMPs #rarediseases