The objectives of this presentation were to give an overview of the OCTGT, to provide context to the discussion of the regulatory basis for endpoint selection. Looking to the past, Dr Shannon picks out the lessons learnt from traditional endpoints. Looking to the future, he discusses critical clinical endpoints in cancer vaccine trials.
Laws and Regulations govern drug approval.
Â· 1938 FD&C Act (drugs must be safe)
Â· 1944 PHS Act (biologics must be safe, pure, potent, and licensed)
Â· 1962 Kefauver-Harris Amendment (drugs must work, too!)
Regulation protects both users and producers of vaccines.
Â· Reasonable expectation the biological product will have a clinically significant function in the diagnosis, cure, mitigation, treatment, or prevention of disease in man (clinical benefit)
Â· Proof of effectiveness shall consist of adequate and well-controlled investigations â€¦ that allow valid comparison to a control and provide quantitative assessment of effect (good trial design)
Â· 1997 FDAMA – Single trial for approval in certain circumstances (one may be enough!)
In summary, Kevin Shannon places emphasis on the need to consider patient population: disease burden, homogeneous vs heterogeneous and time to develop immune response.
Why not download the full presentation and find out more about:
Â· Early trial endpoints: consider biomarkers, immune responses
Â· Randomized P2 trials: demonstrate activity
Â· Consider EOP2 meeting with FDA
Â· P3 trial endpoints: must demonstrate clinical benefit
o OS may be best
o PFS may not be successful
Â· Pre-specify all necessary trial conduct and analyses
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