ATMP regulatory landscape survey

With the aim of identifying areas of ATMP development that require regulatory harmonisation and preparing a future workshop with stakeholders, we are reaching out to everyone for their opinions on the current ATMP regulatory landscape.

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Which of the following best describes your organization?

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Where is the main commercial activity of your company focused (pick more than one)?

What type/types of ATMPs do you develop (pick more than one)?

Have you or are you developing other types of medicinal products other than ATMPs (including, but not limited to, biologics (antibodies, vaccines etc.), small molecule etc.)?

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What is your most advanced level of experience developing cell and gene therapy products (pick one based on highest level)?

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What regulators have you previously had interactions with (pick as many as required)?

Which territories do you intend to develop and commercialise your ATMP (click as many as required)?

Which regulatory procedures have you performed in the development of your ATMP (pick as many as required)?

Have you ever conducted one of the following regulatory procedures with different regulatory authorities in parallel? (pick as many as required)

What is the overall EU regulatory experience of your company (scale of 1-5: 1 none, 2 limited experience, 3 average experience, 4 moderate experience, 5 very experienced)?

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What is the overall USA regulatory experience of your company (scale of 1-5: 1 none, 2 limited experience, 3 average experience, 4 moderate experience, 5 very experienced)?

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What is the overall Asia regulatory experience of your company (scale of 1-5: 1 none, 2 limited experience, 3 average experience, 4 moderate experience, 5 very experienced)?

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Which department or responsible party is most responsible for regulatory activities in your company?

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How important do you think the following areas are for regulatory harmonization to improve or accelerate development of ATMPs (scale of 1-5: 1 not important – 5 extremely important)?

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GMO requirements
Nonclinical: biodistribution requirements
Nonclinical/clinical: shedding requirements and immunosuppression
Nonclinical/clinical: immunogenicity requirements
Nonclinical/clinical: insertional mutagenesis
Long-term follow up (LTFU) of patients (efficacy and safety)
Genome editing requirements
ATMP classification
Combined ATMPs and incorporating medical devices
GMP issues for ATMP
Decentralised manufacturing
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How important do you think  the following general drug development areas (i.e. areas not only ATMP-specific) are for regulatory harmonization to improve or accelerate development of ATMPs  on a scale of 1-5 (1 not important- 5 extremely important)?
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Orphan Drug Designation (ODD) and exclusivity
Paediatric regulatory requirements
Clinical Trial / Investigational New Drug (IND) Applications
CMC and Manufacturing
Clinical data requirements for MAA/BLA
Biobanking
Use of databases / big data
GDPR Requirements
Clinical trials: Use of surrogate endpoints and biomarkers
Clinical trials: Development of companion diagnostics
Clinical trials: Use of Real World Evidence (RWE) and Registry studies
Use of Artificial Intelligence
Use of imaging / diagnostic techniques
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Please define how important you consider the following goals of the ARDAT project (scale of 1-5: 1 not important – 5 extremely important)

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Development of new models to better predict immunogenicity to ATMPs and the consequences for treatment safety and efficacy
Harmonisation / standardisation of methods used for assessment of pre-existing humoral immunity to ATMPs
Harmonisation / standardisation of methods used for assessment of pre-existing T cell immunity to ATMPs
Establishment of a central and sustainable biobanking infrastructure for patients receiving approved or investigational ATMPs to understand the clinical factors around pre-existing immunity and adaptive immune responses to ATMPs
Enhancement of our understanding of ATMP drug metabolism and long-term persistence
Establishment of a comprehensive database compiling published data on ATMP immunogenicity and immunosuppressive protocols
Establishment of a comprehensive database compiling published data on ATMP biodistribution and shedding
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