LSHC Horizons Brochure 2024 - Flipbook - Page 21
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | Cell, Tissue, and Gene Therapies
21
Collaborative strategies for ATMP development through
Investigator-Initiated Trials
Advanced Therapy Medicinal Products
(ATMPs) are revolutionizing the
pharmaceutical industry, challenging the
traditional regulatory landscape. Unlike
conventional medicines, ATMPs demand a
re-evaluation of clinical research practices,
particularly within the EU where they adhere to
specific regulatory frameworks.
For industrial sponsors, conducting ATMP
research is complex. However, for individual
investigators, initiating such research appears
daunting. Investigator-Initiated Trials (IITs)
offer a promising avenue for exploring
new therapeutic areas. These trials, led by
investigators, can address unmet medical needs
and provide patients access to treatments
outside of industry-sponsored trials. For
pharmaceutical companies, IITs generate
additional data, speeding up the discovery
process and aiding in market access. IITs are
also a way for smaller pharma companies who
lack resources for conducting wide clinical trials
to support the development of their research.
Typically, IITs allow investigators freedom in
study design and methodology. Pharmaceutical
companies’ roles are generally confined
to supplying the product and sometimes
financial support. In return, they gain
access to data and intellectual property rights.
However, this dynamic shifts with ATMPs.
Their complexity demands more involvement
from pharmaceutical companies than mere
product supply and financial backing.
Mikael Salmela
Partner
Paris
ATMP development requires specialized skills
and training, from samples collection to patient
treatment. This includes:
• detailed protocols for cell or gene collection,
often necessitating specific training from
pharmaceutical companies;
• sophisticated manufacturing and logistics
involving third-party providers adept in
handling sensitive materials under stringent
regulations (e.g., human cells and genes,
hazardous materials, genetically modified
organisms, products that must be carried
out at specific temperatures and storage
conditions, products with very short lifetime);
• centralized manufacturing, often in the U.S.,
involving complex international transport
arrangements;
• high manufacturing costs; and
• complex liability questions in case of
patients’ safety issue during the IIT.
These factors present practical challenges
for IITs involving ATMPs. The classic
model of limited pharmaceutical company
involvement may be impractical. Instead,
a more collaborative approach is needed
where investigators retain research initiative
and flexibility, but with substantial practical
support from pharmaceutical companies and
innovation in funding given the potential
costs involved in setting up such trials. This
collaboration is essential for the successful
Joséphine Pour
Senior Associate
Paris
development and delivery of ATMPs, ensuring
these innovative treatments reach patients
efficiently and safely. The EU clinical trial
regulation allows for such collaboration
without the pharmaceutical company
necessarily being classified as a sponsor
or co-sponsor, and the new draft Soho
Regulation which should be endorsed
in 2024 should not impact this materially.
