Developing a new drug is a costly undertaking, which can cost several hundreds of millions. Except for the largest Pharma companies, any other company will need external funding at some stage in the development of a drug. A solid regulatory strategy in early phase clinical research is essential to convince your investors to further fund the clinical development of your compound. Depending on your compound (chemical, biotech product, advanced therapy) and the type of investors (private investors, government funding bodies or a larger pharmaceutical company), different aspects of the regulatory strategy need to be more developed. The main aspects of such an early phase regulatory strategy will be discussed and illustrated with a number of case studies such as the development of a regulatory strategy for a bacteriophage and discussions with Health Authorities. Contact Us: clinicalresearch@sgs.com Visit our Website: http://www.sgs.com/cro Follow Us on LinkedIn: http://bit.ly/SGSLifeSciences

1. REGULATORY DUE DILIGENCE, USING
REGULATORY AFFAIRS TO MAXIMIZE
THE VALUE OF A COMPOUND WITH
INVESTORS IN EARLY PHASE
Bruno Speder
Head Clinical Regulatory Affairs
SGS Life Science Services
SAFETY & EFFICACY CLINICAL TRIAL SOLUTIONS
SGS Life Science Services Biopharm Day Seminar – Antwerp, October 29, 2015

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EVOLUTION OF DRUG APPROVALS
Reuters
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FDA approved drugs per year

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EVOLUTION OF DRUG DEVELOPMENT COST
Scannell et al. Nature Drug Discovery Review 11, 191-200 (2012)

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PHARMA COMPANIES
 ‘Big Pharma’ traditional approach
 Own funding
 Full development till MAA and commercialisation
 Pipelines get ‘empty’
 Since 2000’s
 Increased collaboration with biotech companies
• License deals
• Partnership
• Acquisition

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SGS LIFE SCIENCE SERVICES BIOPHARMA DAY – OCTOBER, 29 2015
BIOTECH COMPANIES
 Innovative drugs / therapies
 Clinical and regulatory challenges
 High drug development cost
 Funding to phase II
 External investors
 After phase II
 Sell compound to pharma partner
 License compound to pharma partner

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DRUG DEVELOPMENT PATHWAY
Dickson et al., Nature Drug Discovery Review 3, 417 – 429 (2004)

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ROLE OF REGULATORY IN EARLY PHASE
 Regulatory Affairs has a twofold role:
 Tactical role
• Ensure drug development is conducted in accordance with the
applicable regulations
 Strategic role
• Develop and execute a regulatory strategy to ensure that the
collective efforts of the drug development team results in a
product that is approvable

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TAILORED STRATEGIC REGULATORY
APPROACH
 Compound development
 Biotech / Chemical
 Indication
 Target markets
• Europe / US ?
• Emerging markets ?
 Special status
• Orphan / ATMP
 Funding development
 External investors?
 Large pharma company?
 Till where in the development does the company want to go?

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SGS LIFE SCIENCE SERVICES BIOPHARMA DAY – OCTOBER, 29 2015
TAILORED STRATEGIC REGULATORY
APPROACH
 Non-Clinical Development plan
 Non-clinical Requirements Phase I ≠ Marketing Authorization
 Sufficient to support the phase I study
 Long term toxicology studies
 Clinical Development
 Studies
• Phase I / II
• Proof of concept
 Similar compounds

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TAILORED STRATEGIC REGULATORY
APPROACH
 CMC strategy
 Depending on region
 Stability testing
 GMP batches
 Formulation
 Interactions with Health Agencies
 Validate strategy
 De-risk development

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BELGIAN COMPANY
 Compound
 Developing novel LNG intrauterine anti-conceptive device
 Number of academic studies performed
 Next steps for development
 Currently under discussion with large pharma company for
license
 Approach
 Clinical Development Plan
 Validate with Reference Member State (RMS)
1

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FRENCH BIOTECH
COMPANY
 Compound
 Developing a novel insuline formulation
 Regulatory CMC issue
• Insulin owned by third party
• Formulation owned by sponsor
 Company willing to sell drug after proof of concept (POC)
 Approach
 Discuss with EMA acceptability of DMF for insulin
 Adapt RA strategy to maximalise value in regions where
biotech DMF accepted
2

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PORTUGUESE BIOTECH
COMPANY
 Compound
 Developing bacteriophages
 Nove product with several regulatory uncertainties
• CMC issues
• Unclear message from EMA / FDA
 How to convince pharmaceutical partner to co-fund
 Approach
 Discussion Innovation Task Force of EMA
 Propose CMC strategy
3

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US BIOTECH COMPANY
 Compound
 H1N1 influenza vaccine
 Succesfull phase I
 Efficacy data needed to receive NIH funding
 Approach
 Viral Challenge POC study proposed
 Discuss acceptability of preliminary data phase I data for
POC
4

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CONCLUSION
 Strategic Regulatory planning
 Optimalise studies to be done
 Optimalise budget / cash flow
 Tackle potential regulatory issues early on
 Reassure external investors with validate regulatory strategy
 Long term development

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SGS LIFE SCIENCE SERVICES BIOPHARMA DAY – OCTOBER, 29 2015
THANK YOU FOR YOUR ATTENTION
+ 41 22 739 9548
+ 1 866 SGS 5003
+ 65 637 90 111
+ 33 1 53 78 18 79
+ 1 877 677 2667
+ 33 1 41 24 87 87
Life Science Services Bruno Speder
Head Clinical Regulatory Affairs
SGS Belgium NV Phone: + 32 15 440 116
Life Science Services, Fax: +32 473 26 11 73
Generaal De Wittelaan 19a bus 5 E-mail : bruno.speder@sgs.com
2800 Mechelen
Belgium Web : www.sgs.com/lifescience

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QUESTIONS ?

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