LERU: improve patient access to Advanced Therapy Medicinal Products23 September 2019• NEWSITEM
How can patient access to Advanced Therapy Medicinal Products (ATMPs) be improved at university hospital medical centres? In a new Briefing Paper the League of European Research Universities (LERU), including Leiden University, makes a number of recommendations.
In the Briefing Paper, with LUMC researcher Pauline Meij as lead author, 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.
ATMPs and universities
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. Examples of ATMPs used in the LUMC are stem cell therapy for heart failure, gene therapy for the immune disorder SCID, virus-specific T cells after stem cell transplantation, mesenchymal stromal cells after kidney transplantation and T cell immune therapy for cancer.
Despite these developments and the unique position of universities, too few ATMPs reach the patient. Professor Kurt Deketelaere, Secretary-General of LERU: “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.”
More information and the Briefing Paper can be found at the LERU website.