Cellular and gene therapy (CGT) products offer promising potential to address unmet medical needs, and there is pressure to expedite the regulatory approval process for CGT therapies. However, there are many scientific and technical challenges within this area due to the variability and customizability of each product type. To overcome these unique hurdles, and to improve the odds of having a successful CGT clinical trial, consider the following^1-3:

• Product manufacturing is a crucial issue to be addressed
• Demonstrate the clinical benefit of CGT products through statistical analysis of primary efficacy end points
• Consider alternative trial designs, such as using a rarely occurring treatment outcome as an end point to maximize data from a small, possibly heterogeneous sample size
• Use stratification at the randomization stage or analysis of covariance at the analysis stage, or both approaches
• Use adaptive trial design to resolve the impact from high/unknown response rate variability
• Establish well-designed, high-performing virtual patient registries to combat small patient populations and allow for longitudinal and post-authorization safety and efficacy studies
• Establish risk management practices to identify key risks affecting patient safety and data integrity and to allow for the mitigation and control of key risks
• Consider expedited programs that provide special U.S. FDA assistance, such as Regenerative Medicine Advanced Therapy (RMAT) designation (for regenerative medicine therapies only)

Our Experience

• 20+ years of clinical product development
• 10+ years working on CGT products
• 35+ global CGT clinical projects
• Clinical trials: 12+ (Phase I-IV), 400+ sites, 3,100+ patients; proof of concept through FDA marketing approval
• Regulatory services: multiple approved IND and PMA applications
• Full-scope projects and targeted-scope projects

Therapeutic Indications

• Alzheimer’s disease
• Acute respiratory distress syndrome (ARDS)
• Burn wound healing
• Chronic obstructive pulmonary disease (COPD)
• Chronic wounds
• Chronic spinal cord injury (cSCI)
• Diabetic foot ulcer (DFU)
• Glioblastoma
• Hypertrophic scars
• Leukemia
• Metastatic gastrointestinal epithelial cancer
• Rheumatoid arthritis
• Pressure ulcers
• Regenerative medicine and accelerated recovery
• Stroke (ischemic, chronic)
• Venus leg ulcer (VLU)
• Wound healing from surgical procedures

References

1. FDA guidance for industry “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions” (Feb 2019)
2. FDA draft guidance for industry “Human Gene Therapy for Rare Diseases” (July 2018)
3. FDA guidance for industry “Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products” (June 2015)