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CRO APPROACHES ABOUT HOW
REGULATORS PERCEIVE THE RISK
TODAY FOR A NEW COMPOUND
Bruno Speder, ir.
Head Clinical Regulatory Affairs
2 SGS BIOPHARMA DAY – OCTOBER 25, 2016
HISTORY
“All substances are poisons; there is none which is not a poison.
The right dose differentiates a poison from a remedy.”
Philippus Aureolus Theophrastus Bombastus von Hohenheim-Paracelsus (1493-1541)
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WHAT IS RISK?
Hazard
Something that will cause harm
Risk
The possibility that a hazard may cause harm
Benefit
Something that is advantageous or good
Risk Assessment
The identification, evaluation and estimation of the levels of
risks involved in a situation,
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BENEFIT - RISK ASSESSMENT (RB)
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POINTS CONSIDERED BY REGULATORS
Regulators assess data presented to them
Points to be considered
Risk
Benefit
Analysis of the condition
Current treatment options
Currently available knowledge
Interpretation of the assessment
Evidence
Uncertainties
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DRUG DEVELOPMENT PATHWAY
Dickson et al., Nature Drug Discovery Review 3, 417 – 429 (2004)
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ASSESSMENT PHASE I HEALTHY VOLUNTEER
STUDY
Non-clinical
Safety Pharmacology
Proof of efficacy
Appropriate animal model
Existing data
Compound
Similar compounds
Study design
Dose calculation
Safety measures
8 SGS BIOPHARMA DAY – OCTOBER 25, 2016
RENNES INCIDENT – GAME CHANGER?
Temporary Specialist Scientific Committee (TSSC)
recommendations:
1. Therapeutic effectiveness – predictive efficacy in humans
2. CNS drugs – neuropsychological assessment
3. FIH – doses to be adapted according to data collected
already having been exposed
4. FIH - safety volunteer should take precedence
5. Dose escalation strategies should be based on common
sense
6. Access to data of previous / ongoing FIH studies
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RISK / BENEFIT EVOLUTION
New evidence becomes available during development
Clinical data
Non Clinical data
Competitive landscape change
Risk / Benefit is ‘work in progress’
Indication specific
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MANAGEMENT OF RISK ASSESSMENT
Ther Adv Drug Saf (2010) 1(1) 2138
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MANAGEMENT OF RISK ASSESSMENT
Map out of risks at every stage
Non-Clinical
Clinical
CMC
Make informed decisions
Make your homework
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MANAGING THE REGULATORS EXPECTATIONS
Aim of compound
What do you want to achieve?
Added value of compound
Take advice
Scientific Advisory Board
Discuss with Regulators where appropriate
Scientific Advice meetings with Agency
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ASSESSMENT OF A MARKETING
AUTHORISATION APPLICATION
Risk / Benefit balance
Added value of compound
Indication
Target population
Comparison to existing therapies
Usage of drug
How will the drug actually be used?
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CONCLUSION
Risk Assessment depends
On the situation
Where in the development you are
Regulators
Look at the data presented
Look at ‘bigger picture’
Clinical Trial Application vs Marketing Authorisation
Make your own risk assessment
15 SGS BIOPHARMA DAY – OCTOBER 25, 2016
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 Sciences Bruno Speder
Head Clinical Regulatory Affairs
SGS Phone: + 32 15 440 116
Life Sciences Fax: +32 473 26 11 73
Generaal De Wittelaan 19a bus 5 E-mail : [email protected]
2800 Mechelen
Belgium Web : www.sgs.com/lifescience
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QUESTIONS ?
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