On 31st October, Professor Aisling McMahon hosted a policy workshop in Maynooth University organised as part of the Irish Research Council funded "Advanced Cancer Care: Enhancing Systems and Structures to Deliver In-House Personalised Therapies for Patients via the Hospital Exemption" (ACCESS) project. This project is run in collaboration with Dr Frances Drummond and Breakthrough Cancer Research and funded by the Irish Research Council New Foundations scheme.
The ACCESS project focuses on the hospital exemption provision which is a pathway provided under Article 28(2) of Regulation 1394/2007/EC that enables medical practitioners, under certain conditions, to deliver ‘Advanced Therapy Medicinal Products’ (ATMPs), such as CAR-T therapies, prepared on a non-routine basis for individual patients in cases of 'unmet need'. The ACCESS project examines the scope of the hospital exemption provision under EU law and its operation in national States. The project is led by Professor Aisling McMahon, together with the project team which includes Sinéad Masterson, Alanna Kells and Lauren Kane. The research is considering the potential legal, regulatory and ethical issues that arise around the use of the hospital exemption pathway in specific contexts. At a policy level, this research aims to understand the extent to which this pathway could be used to deliver access to advanced ATMPs in the cancer context on a non-routine basis to patients with 'unmet need'.
This policy workshop was attended by over 25 stakeholders from a range of backgrounds including stakeholders with expertise drawn from clinical, regulatory, scientific, ethics and economics fields. The workshop commenced with an introduction to the ACCESS project and hospital exemption pathway delivered by Prof Aisling McMahon, and an overview of Breakthrough Cancer Research’s work and collaboration in this area delivered by Dr Frances Drummond. It then featured six presentations by leading experts on a range of topics related to the ATMP landscape and hospital exemption pathway, namely:
Session 1: Advanced Therapy Medicinal Products – Access for Patients and Emerging Research Landscape in Ireland
- “The emerging Irish research landscape for Immune Effector Cell Therapies as Living Drugs” – Prof. Nicki Panoskaltsis, Professor in Personalised Therapeutics, School of Pharmacy & Pharmaceutical Sciences, Trinity College Dublin; Consultant Haematologist, St. James’s Hospital.
- “CAR-T Therapies in Child, Adolescent & Young Adult Cancers: Current Objectives and Future Trend” – Professor Owen Patrick Smith, CBE, Hon FTCD, Professor of Child, Adolescent & Young Adult Oncology, Trinity College Dublin; Professor of Haematology, Trinity College Dublin; Visiting Full Professor, University College Dublin; NCCP National Clinical Lead for Children, Adolescent and Young Adult Cancers; Consultant Paediatric Haematologist, Children’s Health Ireland; Chair, European Society of Paediatric Oncology (SIOPE) - AYA Committee.
Session 2: Hospital Exemption in Ireland: Regulatory and Practice Perspectives
- “The Hospital Exemption Route: Regulatory Context” - Gráinne Power, Director of Compliance, Health Products Regulatory Authority, Ireland.
- “Hospital Exemption Route in Ireland: Insights from the Limbal Stem Cell Context”- Sandra Shaw, Chief Medical Scientist, Tissue Bank, Irish Blood Transfusion Service.
Session 3: Use of the Hospital Exemption Route in other European Countries
- “CAR-Ts in Italy: Public Ways and Trails” - Professor Luca Arnaudo, Syracuse University College of Law, New York, Distinguished Lecturer
- “Use of the Hospital Exemption Route: Insights from the Spanish Context” - Sara Varea, Advanced Therapies Medicinal Products Regulatory Unit, Clinical Pharmacology Department, Hospital Clínic Barcelona, Spain.
Each workshop session included roundtable discussions chaired by Prof McMahon on these issues which considered a range of clinical, regulatory, scientific, and ethical aspects related to the hospital exemption route for ATMPs for patients with ‘unmet need’. The workshop discussions will feed into a policy report which the project team are developing as part of the project. This report will focus on the hospital exemption route and challenges/opportunities around its use in the ATMP context for patients with unmet needs.
ACCESS Project
The ACCESS project aims to develop an in-depth understanding of, and policy report on the hospital exemption route, considering the potential opportunities and challenges around using this route for the provision of ATMPs in cases of ‘unmet need’, with a particular focus on cancer therapies. This project builds upon findings of an earlier IRC funded project led by Professor McMahon, run in collaboration with Breakthrough Cancer Research, which examined access and provision of CAR-T therapies in Ireland, and culminated in a report (2023). That report set out 10 recommendations around access and provision of CAR-T therapies, including the need for deeper examination of the hospital exemption pathway.
Funding Acknowledgment: The ACCESS project is funded by the Irish Research Council, New Foundations Scheme under grant number NF/2023/1763 (Project Title: Advanced Cancer Care: Enhancing Systems and Structures to Deliver In-House Personalised Therapies for Patients via the Hospital Exemption). This project is run in collaboration with Breakthrough Cancer Research (Civil Society Partner). Any omissions/errors remain the authors own.
