Thanks a lot for your support in advance!
The CARAT and RESTORE teams

more information on the EU initiatives is provided at:
CARAT homepage; RESTORE homepage

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* 1. Background

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* 2. What is your professional background? Please select most relevant answer.

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* 3. Describe your previous experience with ATMP/CAR T trial applications: please select the most relevant answer.

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* 4. Describe your research interests: several answers are possible.

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* 5. Do you regard the following technological challenges as obstacles for translation of ATMP/CAR T cells into the clinics?

  1 (low obstacle) 2 3 (medium) 4 5 (high obstacle) N/A
Cell engineering (e.g. vector design, gene transfer technologies, cell expansion, cell activation etc.)
Manufacturing process
Availability of vector material
Safety /quality of raw materials
Quality control (and assays)
Relevant in vitro preclinical models
Relevant in vivo preclinical models

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* 6. Do you regard the following regulatory aspects as obstacles for translation of ATMP/CAR T cells into the clinics?

  1 (low obstacle) 2 3 (medium) 4 5 (high obstacle) N/A
Manufacturing license application
GMP requirements in general
Documentation for GMP processes
Clinical trial application approval
Ethical vote application
Investigational medicinal product dossier (IMPD)
Duration of approval process
General insecurity about regulatory requirements

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* 7. Do you regard the following clinical trial related aspects as obstacles for translation of ATMP/CAR T cells into the clinics?

  1 (low obstacle) 2 3 (medium) 4 5 (high obstacle) N/A
Patient acquisition
Inclusion criteria, definition of target population
ATMP manufacturing
Dosing of IMP
Dealing with side-effects
Patient monitoring
High costs for ATMPs and patient treatment
Reimbursement e.g. by payers
Administrative burden

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* 8. Do you regard the following ethical aspects as obstacles for translation of ATMP/CAR T cells into the clinics?

  1 (low obstacle) 2 3 (medium) 4 5 (high obstacle) N/A
Transfer genetically modified cells into humans
Life-long persistence of genetically modified cells in recipient
Side-effects e.g. cytokine release syndrome, neurotoxicity, Graft versus Host Disease (GvHD)
Socio-economic burden for health care systems through high costs of ATMP/CAR T cell products
Lack of appropriate reimbursement models
Limited access of patients to novel Advanced Therapies
Dependency on pharma/biotech industry
Unrealistic public expectations on therapeutic potential of Advanced Therapies

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* 9. Do you regard the following general deficits as obstacles for translation of ATMP/CAR T cells into the clinics?

  1 (low obstacle) 2 3 (medium) 4 5 (high obstacle) N/A
Lack of templates/blueprints
Lack of guidance through the application processes e.g. for manufacturing license or clinical trial application
Lack of funding respectively financing
Lack of efficacy of cell products
Lack of safety of cell products
Lack of research data e.g. on mode of action of CAR T cells or ATMPs
Lack of transition of ATMPs toward front-line care

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* 10. Are there other specific painpoints (personal experiences) for translation of ATMPs/CAR T that you would like to mention?

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* 11. Would you like suggest possible solutions that should be implemented for translation of ATMPs/CAR T cells?

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* 12. Do you have any further comments, questions, or concerns?

0 of 12 answered
 

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