LS&HC Horizons 2022 - Flipbook - Page 60
Hogan Lovells | 2022 Life Sciences and Health Care Horizons
60
Europe
Hospital exemption for academic gene and cell therapies in the EU
The hospital exemption rule (Sec. 3(7) Directive 2001/83/EC and Recital 6 Regulation
No 1394/2007) allows for the use of Advanced Therapy Medicinal Products (ATMPs) (the
so-called academic or non-industrial ATMPs) without a marketing authorization (MA).
At an EU level, academic ATMPs are defined as ATMPs prepared on a non-routine basis
according to specific quality standards and used within the same Member State in a hospital
under the exclusive professional responsibility of a medical practitioner, in order to comply
with an individual medical prescription for a custom-made product for an individual
patient. Although academic ATMPs are exempted from applying for and obtaining an
‘ordinary’ MA, their use/manufacturing still need to be approved by the competent
authorities of the Member States.
Hospital exemption in Spain shares with the Directive 2001/83/EC that leaves many
aspects open to interpretation (‘non-routine’, ‘custom-made’, etc.). The position in Spain
seems to be as follows: academic ATMPs serve to enable patients to receive therapies in
cases where no authorized medicinal products are available and should not become the
normal route to market ATMPs; rather, the goal should be to obtain a centralized MA.
These interpretations all suggest that co-existence between ‘ordinary’ ATMPs and academic
ATMPs sharing the same indication will likely not take place.
In the Netherlands, prior approval of academic ATMPs must be obtained from the
Healthcare Inspectorate (Inspectie Gezondheidszorg en Jeugd or IGJ). The request can be
made by completing a detailed application form as published by IGJ. The form requires
the applicant to provide information on, inter alia, the qualified person for batch release,
rationale for the need for the hospital exemption, clinical experience with the product and
pharmacovigilance.
Hein van den Bos
Mikael Salmela
Giuseppe Aminzade
Álvaro Abad
Partner, Amsterdam
Counsel, Milan
Partner, Paris
Senior Associate, Madrid
In Germany, the use of hospital exemption mainly requires that the procedure is done
in a ‘non-routine manufacturing’. However, the law defines this very broadly, mainly as
treatment procedures done in an amount of cases for which likely comprehensive data/
evidence cannot be expected. Thus, the demarcation to authorized ATMP is not really made
and co-existence of authorized ATMP and procedures in hospitals remains, to the detriment
of the authorized ATMP therapy.
France has implemented the exemption for academic ATMPs, and their manufacturing is
subject to detailed requirements and Agence Nationale de Sécurité du Médicament et des
Produits de Santé (ANSM) approval. Highlights of new rules being adopted in 2022 include:
(i) hospital preparations are no longer limited to specific cases covered by European
law or cases of rupture; (ii) automatic reimbursement by the health insurance when the
pharmaceutical product benefiting from a MA ‘is not available’; (iii) no clear restrictions on
manufacturing to limited purposes. The French government aimed to allow alternatives to
some of the gene therapies that recently received an unfavorable clinical assessment that
did not allow their access to the market, which is a direct competition to pharma companies
that have implemented a clinical development.
Moving to Italy, the main requirement for the exemption is the “preparation on a nonrepetitive basis”, which is defined as the non-routine preparation (even for a cycle of doses)
following specific quality requirements, to be used exclusively in a public hospital, under
the exclusive professional responsibility of a physician, in the execution of an individual
prescription for a specific product intended for a concrete patient. Similar to Germany,
the definition is very broad. Demarcation to authorized ATMP is not really made and coexistence between the two types of ATMPs remains.
Dr. Jörg Schickert
Partner, Munich
