With More Than 200 Cell and Gene INDs on File with FDA by 2020, Challenges Lie Ahead on Path to Approval

Tue, Jun 25, 2019 | By Sue Blecman, Senior Director, Business Development, Cryoport

 “By 2025, we predict that the FDA will be approving 10 to 20 cell and gene therapy products a year based on an assessment of the current pipeline and the clinical success rates of these products.” – Scott Gottlieb, Former FDA Commissioner 

With the cell and gene therapy boom, and five to 10 percent projected approval rates, what challenges lie ahead and what do you need to know now to forge a successful path to approval?  

One major challenge is the integrity of product from raw material to final product: Contamination, environmental changes, tracking discrepancies, lack of information, lack of accurate information and other inconsistencies during transport can compromise the integrity of patient, donor and final product.  The potential consequences are ineffective final therapies administered to patients – a result we deem unacceptable.  

 ISO/TC 276 U.S. will require enhanced traceability and regulatory requirements in support of logistics distribution due to the fragile aspect of drug product and disproportionate risk of improper cold chain management on the viability of therapies.   

With the many touchpoints from starter material to final therapies, the risks are great.  Cryoport created Chain of Compliance to implement traceability and risk mitigation throughout the entire transportation process.  

  • Engineering of equipment used 
  • 100% requalification and documentation of equipment and components before and after every shipment 
  • Near real-time monitoring and traceability throughout transit, not just pickup and delivery location and time 
  • Easily accessible reports to prove appropriate temperatures were maintained throughout transit 
  • Validated cleaning processes 
  • Data integration to follow the journey from start to finish 
  • Commercial scalability competencies to continue your success after approval 

We created Chain of Compliance to stay ahead of the changing landscape, address foreseen and unforeseen challenges to ultimately provide unmatched support for viable, safe, and effective cell and gene therapies that positively impact and save lives.

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