translating cell therapies: academic versus industry model
TRANSCRIPT
Translating cell therapies:Academic versus Industry model
Alexey Bersenev, MD, PhD
Advanced Cell Therapy Laboratory,Yale-New Haven Hospital, Yale University www.celltrials.info
The BioProcessing Summit, Boston, Aug. 4, 2015
• Academia vs. industry – clinical trials global trends
• Key differences between academic and industry models
• Gaps and solutions
Outline of the talk:
Global trends: sponsorship of clinical trials
http://dx.doi.org/10.6084/m9.figshare.1504124
Global trends: Total number of MSC trials
http://dx.doi.org/10.6084/m9.figshare.1504131
Global trends: Total number of trials, involved adipose-derived cells
http://dx.doi.org/10.6084/m9.figshare.1504586
Is industry in favor of allo- model?
NO!
Total number of registered clinical trials
http://dx.doi.org/10.6084/m9.figshare.1504311
Measuring output: published results of clinical studies in regenerative medicine –
2014 sample (n=116)
• focus on “first-in-human”, pilot studies
• early stages of development
• delivery: cell therapy as procedure
• favored model – auto?
• favored medical innovation regulatory path
• diseases with low prevalence
• low scale
• no attention to pharmacoeconomics, relies on “efficacy boost”
• focus on commercialization and technology transfer
• delivery: cell therapy as mass-produced drug
• favored model – allo?
• all phases of trials under FDA
• diseases with high prevalence
• large scale
• pharmacoeconomics will determine product development
Academic versus industry cell models – key differences:
academic industry
Medical Research
Personalized Medicine
First-In-Human
Serum
Cell Culture
Patient File/ Binder
Scale Out
Closed System
Cost of Goods
Automation
Large Scale
Bioprocess
Batch/ Lot
Unit Operations
Academia vs. Industry – cultural/ language difference:
Academia Industry
developer
FDA
local IRB
FACT
AABB
Traditional academic and industrial pathways
CAP
HCT/P
biologic/ drug
361
351
No GMP
cGMP
need for cell culture/ gene modifications
• Booming of stem cell and maturity of cell-based technologies on research side – ready for translation!
• Clinical resources in big academic hospitals – pioneering physician are eager to be the first
• Technological tools for clinical cell processing are available
• Pharma and Biotech are reluctant to enter the field on early stages
The need for academic GMP cell therapy facilities:
Academic GMP cell therapy facilities:
• Trend: increase number of academic facilities
• AABM – Association of Academic Biologics Manufacturers
http://www.aabmonline.org ~ 45 registered, mostly in US
• AGORA: ATMP GMP Open Access Research Alliance
http://agora-gmp.org/ - 8 centers listed
• EATRIS: European Infrastructure for Translational Medicine ATMP
Platform http://www.eatris.eu/ – 14 GMP facilities in EU
• Estimated total # of facilities in US 50-100 and EU – few
hundreds
• Usually not a subject of FDA inspection not always follow strict GMP standards
• Scale and capacity: much smaller than industry
• Acting as a hospital core facility must to accommodate all types cell/gene therapy trials
• Usually under-funded data not publishable, so no return
Academic GMP facilities – key differences:
Centralized vs. decentralized cell manufacturing facilities
cGMPcGMP
decentralized centralized
collection/ processing
OR/ APH
closed system/ automation
OR/ APH
releasedelivery
collection
delivery
processing
release
• Licensing —> Tech Transfer
• Contract manufacturing
• Validation studies
Academic GMP – Industry interactions:
Key to success in tech transfer:
1. Academics must understand needs of industry for
successful commercialization and regulatory
compliance
2. Industry to leverage the knowledge of academics
Academic GMP - more gaps:
• Lack of QC, QA and Regulatory
• Research-like mindset
• Training
• Funding: The cost of GMP compliance is underestimated in academia
Advice for academics:
• Invest time in developing cell therapy programs in academia – training, GMP facilities, QA/ regulatory affairs teams
• Start pre-clinical design and Phase 1 with pharmacoeconomics analysis think of cost-effectiviness, COGS, BLA, reimbursement
• Utilize (1) translational centers to help and (2) industry consultants
• Get involved in industry collaborations and tech transfers huge experience!