LSHC Horizons Brochure 2024 - Flipbook - Page 29
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | Cell, Tissue, and Gene Therapies
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The hospital exemption scheme for advanced therapy medicinal
products in reform of the EU pharmaceutical legislation
Advanced therapy medicinal products
(ATMPs) are medicines for human use that
are based on genes, tissues or cells. They offer
ground-breaking new opportunities for the
treatment of diseases. Like any other medicinal
product (with a few exceptions), ATMPs must
obtain the relevant marketing authorization
before they can be placed on the market in
European Union (EU) Member States (MSs).
As an exception to the general rule, MSs
may authorize the provision of an ATMP
without marketing authorization under the
so-called hospital exemption scheme. Hospital
exemption (HE) products must be “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 custommade product for an individual patient”.
Within the context of the reform of the
EU pharmaceutical legislation, and having
expressly admitted that there are great
differences in the application of HE among
MSs, Article 2 of the European Commission’s
(Commission) Proposal for the new Directive
on medicinal products for human use
(Proposed Directive) introduces measures to
improve the application of ATMPs prepared
under hospital exemption (ATMPs under HE):
Álvaro Abad
Senior Associate
Madrid
• By way of derogation from Article 1(1)
(rules for the placing on the market,
manufacturing, import, export, supply,
distribution, pharmacovigilance, control and
use of medicinal products), only Article 2
shall apply to ATMPs under HE.
• The manufacturing of ATMPs under HE
shall require approval by the MS and MSs
shall (1) notify any such approval, as well
as subsequent changes, to the European
Medicines Agency (EMA) and (2) inform the
EMA and other MSs if an approval is revoked
due to safety or efficacy concerns.
• ATMPs under HE shall comply with the
requirements equivalent to the good
manufacturing practices (GMP) and
traceability for ATMPs, as well as with
pharmacovigilance requirements equivalent
to those provided at EU level.
• Approval holder shall collect and report data
on the use, safety, and efficacy of ATMPs
under HE at least annually and the MS shall
review such data, verify compliance with
GMP, traceability, and pharmacovigilance
requirements and transmit it to the EMA,
who shall set up and maintain a repository
of that data.
• The Commission shall adopt implementing
acts to specify (1) the details of the
application for the approval; (2) the format
for collection and reporting of data; (3) the
modalities for the exchange of knowledge;
and (iv) the modalities for preparation
and use of ATMPs under HE on a nonroutine basis.
These measures have aroused the interest
of many stakeholders, who have already
expressed their position on the matter,
including a joint position paper from the
Alliance for Regenerative Medicine (ARM),
European Federation of Pharmaceutical
Industries and Associations (EFPIA),
EuropaBio, European Confederation of
Pharmaceutical Entrepreneurs (EUCOPE)
and International Society for Cell & Gene
Therapy (ISCT).
