Common Regulatory Issues for Stem Cell Products. Christopher A Bravery

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1 Common Regulatory Issues for Stem Cell Products 1 Christopher A Bravery cbravery@advbiols.com

2 Regulation of stem cell therapies Helicopter view: Need to show the product is of an acceptable and consistent quality Need to show the product is safe Need to show the product has efficacy There are no short-cuts, but decisions made early-on often have major ramifications later the rush to meet milestones for investment can lead to poor decisions. 2

3 PRODUCT/PROCESS QUALITY Product Development: Characterisation & Comparability Basic Research Drug Discovery Non-Clinical Development Stages of Product Development Phase I Clinical Development Phase II Phase III Market Approval Post Market Approval Disease Pathophysiology PoC Lead Candidate/s Research Process Pilot Process Investigational Product Development Process GMP Commercial Process Approved Product Scale Up/Out Product /Process Characterisation In-Process Controls/Process Parameters In vivo/ in vitro Models Biological characterisation (disease/product) Release Specifications/Stability Specifications Potency Assay (release) Biological characterisation assays 2010, Consulting on Advanced Biologicals Ltd 3

4 Audience Poll Q1 Who s somewhere between late pre-clinical to early first-in-man (for their product)? Q2 Where do you expect to be in 8 years time? 4

5 Biotech Historic Timelines 5 Overall years At least 8 years from first clinical use to submission of MAA. R PC I II III MAA L Basic Research Hard to calculate Pre-Clinical 1-3 Years Phase I (Safety) Years Phase II Years Phase III?3 5 years MAA Review (EMA/CAT) 1 2 years Launch (Not immediate)

6 Audience Poll Q3 How much money do you think you need to raise from PI to MAA? 6

7 Audience Poll Q3 What do you think your chances of success are? From Pre-clinical to Market 90% Fail! 7

8 Common Issues Facing SC Therapy Companies: A regulatory Perspective Unrealistic Timelines Everything takes much longer than you expect. There is a LOT of work to do outside of the clinical trials Manufacturing development/quality control Iterative process Need to demonstrate everything with own data Lack of Funding Inevitable corner cutting = increased risk of failure! 8

9 9 Common Quality (CMC) Issues

10 Common Quality (CMC) Issues 10 NATURE REVIEWS: Drug Discovery Vol. 7 Nov 2008

11 11 Common Deficiencies: Characterisation

12 12 Common Deficiencies: Characterisation

13 Further Reading 13 Note: Free Access

14 Further Reading Chapter 19: A CATalyst for Change: Regulating Regenerative Medicines in Europe. C. Bravery 14

15 Further Reading PAS-83 (BSi, 2006) Guidance on codes of practice, standardised methods and regulations for cell-based therapeutics. Currently being updated to include EU and US rules (2011) (free) PAS 84 (BSi, 2008) Regenerative medicine. Glossary Currently being updated (2011) (free) PAS 93 (BSi, 2011) Characterisation of cells and cell products (in preparation) Hopefully published later in 2011, should also be a free download. 15