@misc{9230679,
  abstract     = {{Advanced therapy medicinal products (ATMPs) are complex biological products that present specific scientific and regulatory challenges during clinical development. This thesis aimed to analyse consolidated Part I considerations raised during assessment of investigational ATMP clinical trial applications (CTAs) under the Clinical Trials Regulation (CTR) to identify recurring scientific and regulatory assessment patterns.
Consolidated Part I considerations were extracted from the Clinical Trials Information System (CTIS) for investigational ATMP CTAs submitted between 2022 and 2025, where Sweden or Denmark acted as Reporting Member State (RMS). A structured classification framework was developed to classify considerations into assessment categories, themes and specific issues. The considerations were subsequently analysed across assessment categories, request for further information (RFI) stages and exploratory subgroup characteristics. 
The analysis included 22 CTAs and 907 consolidated Part I considerations. Quality represented the largest assessment category, followed by clinical, regulatory, non-clinical and statistical considerations. Across categories, considerations focused mainly on product and process understanding, control of manufacturing and materials, patient safety, treatment justification, risk management, and documentation requirements. The findings also suggested that similar underlying scientific and regulatory challenges often appeared across multiple assessment categories from different regulatory perspectives. The exploratory analysis fur-ther suggested that assessment patterns may be influenced by overlapping factors such as sponsor type, trial phase, ATMP subtype and dossier maturity.
Overall, the findings showed that CTA assessment of investigational ATMPs mainly focused on how sponsors identified, justified and controlled uncertainties and potential risks before investigational ATMPs were authorised for administration to patients in clinical trials. The results support the view that investigational ATMP assessment under the CTR is product-specific, risk-based and case-by-case. By examining recurring assessment patterns at an earlier stage of ATMP development during clinical trial assessment, this study may contribute to a better understanding of the scientific and regulatory challenges while development strat-egies and supporting documentation are still evolving. Earlier identification of such recurring challenges may help sponsors address critical issues more proactively before late-stage marketing authorisation applications. The classification framework developed in this thesis may also support future analyses of larger CTIS datasets.}},
  author       = {{Stanoevska, Aleksandra}},
  language     = {{eng}},
  note         = {{Student Paper}},
  title        = {{Assessment Patterns in Clinical Trial Applications for Advanced Therapy Medicinal Products : Analysis of Consolidated Part I Considerations where Sweden or Denmark acted as Reporting Member State (2022-2025)}},
  year         = {{2026}},
}