LERU shares advice so that patients have a better access to advanced therapy

Unit of Cell Production and Tissue Engineering, at the Faculty of Medicine and Health Sciences. Located in CREATIO center, the only one to produce advanced genic therapy medical products. Photo: CREATIO/UB

The League of European Research Universities (LERU) has published a new report on how to improve patients’ access to Advanced Therapy Medicinal Products (ATMPs) in medical centers and university hospitals. In this report, LERU presents recommendations to improve ATMP within the academic context in order to ensure that EU institutions, national competent authorities and other relevant bodies work together on policies to help patients access these novel therapeutic products.

The report, named Advanced Therapy Medicinal Products, is signed by Josep M. Canals ( from the Stem cells and regenerative medicine group, Institute of Neurosciences of the University of Barcelona), Pauline Meij (Leiden University Medical Centre), Maeve Lowery (Trinity College Dublin) and Mike Scott (University of Cambridge).

According to Josep M. Canals, director of the Production and Validation Center of Advanced Therapies of the UB (CREATIO) and researcher at the Institute of Neurosciences of the UB (UBNeuro), the study goes over the current state of the production of advanced therapy products within academic environments, and takes “advice for universities, regulator entities and the pharmaceutical companies”. The report presents several actions to improve the best and fastest implementation of these innovative therapies in the health system”.

Many advanced therapy medicinal products (ATMP) in early stages are developed in the universities, and more than half of clinical studies at stages I/II are funded by university centers and/or university hospitals. This occurs because the professionals of these centers have a large experience on the study of diseases, do innovative research and have access to facilities which enable the transition of the medicine to clinical trials. However, only a part of the products continue the developing process and reach the patients. This happens largely due the lack of interaction between universities and pharmaceutical companies in Europe, plus the current European legislation, which does not favour it.

Leukaemia, Parkinson’s disease and Huntington’s disease, diabetes or multiple sclerosis are known and common diseases in our society. Advanced therapy medical products (ATMP) offer a pathway to treat these diseases and others, for which other products do not work, although the amount of patients treated with ATMP is still low. The long legislative course in the European Union makes it hard for the development of these products to carry on and end up with a science article or in the shelves of many researchers. Actually, there are only a few advanced therapy medical products that have been commercialized, and those are very expensive so its application and industrial exploitation is not an option. Therefore, many advanced therapy medical products that were registered have been removed from the market.

In the conclusions, the LERU paper calls for a better interaction among universities, regulators, industry and other relevant organizations to work on the development of the final product and ensure its support (funding too) during all stages of the process so as to implement programs of integrated clinical development to start soon in the therapy development of the product.

It also calls for a better dedication of the universities to work on innovative products aimed at medical needs that are not covered and products which are not attractive for the industry. One of the proposals is to create collaborations between universities, philanthropic donors and regulators.
 
EThe paper makes a special mention to hospital exemption, which is defined in the European regulation and nationally regulated, and which is applied to specific cases for customized products, such as ATMPs. In this sense, LERU calls for a development of common plans to standardize the production and clinical processes among different countries in the European Union in cases of hospital exemption. This procedure has received critiques for a potential competition to the industry but it is however, the only way patients can access these therapies.

In order to prevent this situation from happening, the report suggests specifying how to apply the exemption in universities and proposes two options: one focused on the production process, as a continuation of the treatment of patients between clinical development phases; and a second one for products which are not suitable for marketing authorization.

It also calls for transparency, regarding clinical trials with ATMP and the use of hospital exemption. This would enable active organizations of this field to gather expertize, success and failures, and increase the speed of innovation in this area.

Last, the report opts for including the ATMP development in postgraduate training programs and expert training in European universities, especially for LERU members.

Advanced therapy units


At the moment, Catalonia has different units of cell production and tissue engineering, one of them in the Faculty of Medicine and Health Sciences of the University of Barcelona (UB) and the only one able to produce genic therapy medical products. This unit, located in the Creatio center of the Faculty of Medicine and Health Sciences, participated in both academic and industrial translational projects in collaboration with pharmaceutical companies. Creatio worked on cell therapy products, cell vaccines, and genic therapy products, as well as the production of artificial tissues. At the moment, in collaboration with different hospitals and Spanish entities, it is producing lentiviral particles for the treatment of blood diseases such as leukaemia. These viruses are used in hospitals to work on a new medicine based on genetic cell modifications in the immune system, known as CAR T cells. The other two production units are located in Hospital Clínic de Barcelona and the Blood and Tissue Bank (BST).