regulatory due diligence, using regulatory affairs to maximize the value of a compound with...

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

2 SGS LIFE SCIENCE SERVICES BIOPHARMA DAY – OCTOBER, 29 2015

EVOLUTION OF DRUG APPROVALS

Reuters

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1990

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FDA approved drugs per year


EVOLUTION OF DRUG DEVELOPMENT COST

Scannell et al. Nature Drug Discovery Review 11, 191-200 (2012)


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


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


DRUG DEVELOPMENT PATHWAY

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


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


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?



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



APPROACH

CMC strategy

Depending on region

Stability testing

GMP batches

Formulation

Interactions with Health Agencies

Validate strategy

De-risk development


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


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


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


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


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


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 : [email protected]

2800 Mechelen

Belgium Web : www.sgs.com/lifescience


QUESTIONS ?

regulatory due diligence, using regulatory affairs to maximize the value of a compound with...

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