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).
