Universities play a major role in the development of ATMPs, largely because they have the necessary disease-specific expertise, capacity for innovative research and access to donor and patient material. However, universities still face challenges in bringing ATMPs to patients. At a launch event in Brussels today, LERU outlines how patient access to Advanced Therapy Medicinal Products (ATMPs) can be improved at university hospital medical centres.
Leukemia, Melanoma, Parkinson’s disease, Diabetes, Huntingdon’s disease, Multiple Sclerosis. These debilitating diseases are all well-known and are all too common within our society. Advanced Therapy Medicinal Products (ATMPs) offer a route to treating these diseases, and many other (including many rare diseases), where existing products are not effective, although the numbers of patients treated with ATMPs to date however is still very low.
In this Briefing Paper, The League of European Research Universities (LERU) outlines its recommendations to improve the development of ATMPs within the university medical centre context. We hope it will act as a framework for ensuring that the EU institutions, national competent authorities and other relevant bodies develop sound and workable policies which aid patient access to these novel and promising therapeutic products.
LERU calls for:
- Improved interactions between universities, regulators, industry and other relevant organisations. This should cover the whole product development pathway and ensure support (including financial) at all stages of development.
- A focus within universities on the development of new innovative products that address an unmet need and on products which are unlikely to be attractive to industry.
- Retention of the Hospital Exemption, but it should be refocused for use only in product development or for products not suitable for marketing authorisation.
- Increased transparency, both in respect to ATMP trials and the use of the hospital exemption. This will allow organisations active in this field to build upon others expertise, success and failures, thus increasing the speed of innovation in this area.
Dr. Pauline Meij, Head of Production ATMP/Qualified Person at Leiden University Medical Center and lead author of the paper said: ‘Collaboration, training, improving the use of the HE and increased transparency are all essential to create improved patient access to this complex group of medicinal products’.
Professor Kurt Deketelaere, Secretary-General of LERU said that “Universities are crucial players in this innovative ATMP area, but they could do more if certain hurdles were addressed. LERU therefore calls on the EU institutions and other relevant bodies to put in place measures to improve the role of universities and other stakeholders in this complex area, and to ensure patients get access to these much-needed therapeutic products.” Full article / Biomedical and Life Sciences Policy Group