Click / tap a segment to view Policy Asks.
Ensure that the patient and caregiver perspective is included throughout the development pathway of ATMPs – i.e. conduct of clinical trials, evidence generation and value assessment – with transparent feedback on how input is factored into decision making. Provide funding for cross-disciplinary education to equip patients, caregivers and healthcare professionals with the knowledge to contribute to technical discussions around transformative therapies.
Stimulate a collaborative R&D ecosystem for ATMPs, with patients, healthcare providers, universities, research centres and industry involved in identifying research priorities and effective incentives to promote innovation in areas of patient medical need.
Ensure that the EU remains attractive for the development of ATMPs, so that patients benefit from early access to innovation. Promote a broad understanding of medical needs that captures the evolving nature of technological advances, and ensure that the Clinical Trial Regulation is fully implemented and adhered to in all Member States, avoiding the introduction of additional national requirements for clinical trials outside of the Regulation.
Implement, via the proposed Regulation on Substances of Human Origin (SoHO), consistent and harmonised definitions for classification criteria of blood, tissues and cells (BTC) (Also referred to as Substances of Human Origin (SoHO)) and ATMPs, to avoid divergent interpretations and decisions across Member States and ensure a level playing field for all stakeholders.
Uphold high safety and efficacy standards for ATMPs, with equivalent regulatory oversight for ATMPs authorised under the centralised procedure and ATMPs manufactured under the Hospital Exemption in a clinical setting. Introduce EU regulatory guidelines on quality with equivalent standards for use of the Hospital Exemption, accompanied by European registries to record its use, giving greater transparency for patients, regulators and healthcare professionals.
Ensure the European Health Data Space enables the secondary use of data and sharing of real-world evidence in the full lifecycle of ATMPs. It should develop interoperability standards for health data sets, thereby facilitating pooling of data to increase reliability. TRANSFORM welcomes DARWIN EU, which will strengthen EU-wide real-world evidence to support regulatory, HTA and healthcare professional decisions. It should be inclusive of all stakeholders. See Data Analysis and Real World Interrogation Network (DARWIN EU) | European Medicines Agency (europa.eu)
Facilitate better and earlier healthcare professional and patient involvement in the collection of real-world data to address uncertainty on the long term effectiveness and safety and thus improve patient and health system outcomes. Invest in the infrastructure needed to create and harmonise data collected in patient registries to enhance monitoring of outcomes over the longer-term for patients treated with ATMPs.
Establish greater alignment between regulators, HTA bodies and payers on evidence generation requirements and use of real-world evidence throughout the development pathway.
Early dialogues such as those between the European Medicines Agency (EMA) and ATMP developers should be extended to be more iterative and inclusive of the full range of stakeholders, i.e. HTA bodies, payers, academia, researchers and patients/caregivers.
Consider a dedicated “EU Fund for real-world evidence collection” for the establishment, operation and maintenance of real-world evidence registries, to support the generation and use of data and RWE for ATMPs from the point of (conditional) marketing authorisation.
Ensure effective implementation of the EU HTA Regulation, to ensure timely and efficient evaluation of, and patient access to, innovative therapies when first assessments become mandatory in 2025 for ATMPs. Joint Scientific Consultations should be conducted early, in multi-stakeholder (e.g. patients, industry, regulators, academia) dialogue, so that advice can influence evidence generation plans, and the opportunity for advice should be offered to all developers that will undergo a Joint Clinical Assessment at EU level. Given the highly specialised nature of ATMPs, assessments should be conducted as efficiently as possible using appropriate methodologies which recognise the specificities of ATMPs. Duplication between EU and national assessments and/or requirements should be avoided.
Develop EU guidelines on newborn screening, genetic testing and diagnostics, to be included in the proposed European action plan on rare diseases. The ERN Expert Platform for Newborn Screening would be well placed to support this, and to position the EU as the central point for information sharing on good practices from existing national programmes. See further Screen4Rare’s Call to Action June 2022: https://screen4rare.org/calltoaction/ https://screen4rare.org/ern-expert-platform/
Optimize and develop new access pathways, ensure effective cross-border cooperation to enable timely and effective patient access to ATMPs by patients in multiple centres of excellence. Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare
Establish a pathway for ATMPs that can be launched and delivered through a number of designated centres in the EU, alongside with adequate support for patients and their families.
Leverage learnings, both the opportunities and challenges, from regional collaborations (e.g. Benelux-AI, FINOSE) to promote access to ATMPs for patients across countries. Benelux-AI has successfully led coordination efforts on Horizon Scanning and HTA, but discussions were less successful on the pricing & reimbursement of therapies. Other voluntary collaborations are increasingly inactive, like Valletta and the Visegrad Group.
Support national governments to share learnings on novel payment models and funding approaches for ATMPs, including pay-for-performance and annuity-based payment models, outcomes-based agreements, and risk sharing agreements. Consideration should be given to differentiated pricing to better align medicines’ prices with countries’ ability to pay.
Create a multi-stakeholder platform, involving EU Member States, the European Commission, European Parliament, industry, patient representatives, physicians and healthcare insurers and payers, to develop concrete, operable, transparent mechanisms to facilitate access to cross-border healthcare, based on current EU cross-border healthcare related legislation.