Post on 21-Mar-2017
Why adaptive clinical trials?
I Human subjects not like light bulbs.
I Have to proceed cautiously.
I Want to treat subjects effectively.
Why adaptive clinical trials?
I Human subjects not like light bulbs.
I Have to proceed cautiously.
I Want to treat subjects effectively.
Why adaptive clinical trials?
I Human subjects not like light bulbs.
I Have to proceed cautiously.
I Want to treat subjects effectively.
Objectives
I Maximize the probability/expectation of a good outcome
I Mimimize the probability/expectation of a bad outcome
I Stop early because things are looking bad
I Stop early because things are looking good (less common)
Objectives
I Maximize the probability/expectation of a good outcome
I Mimimize the probability/expectation of a bad outcome
I Stop early because things are looking bad
I Stop early because things are looking good (less common)
Objectives
I Maximize the probability/expectation of a good outcome
I Mimimize the probability/expectation of a bad outcome
I Stop early because things are looking bad
I Stop early because things are looking good (less common)
Objectives
I Maximize the probability/expectation of a good outcome
I Mimimize the probability/expectation of a bad outcome
I Stop early because things are looking bad
I Stop early because things are looking good (less common)
Opportunities for improvement
I Use continuous outcomes
I Monitor frequently or continuously
I Use patient characteristics
I Use multiple outcome events
Opportunities for improvement
I Use continuous outcomes
I Monitor frequently or continuously
I Use patient characteristics
I Use multiple outcome events
Opportunities for improvement
I Use continuous outcomes
I Monitor frequently or continuously
I Use patient characteristics
I Use multiple outcome events
Opportunities for improvement
I Use continuous outcomes
I Monitor frequently or continuously
I Use patient characteristics
I Use multiple outcome events
Dangers
I Fitting complex models with little data
I A priori overfittingI Exploring high dimensional design/outcome space:
I Arbitrary simulation scenariosI May have blind spotI Temptation to cherry pick scenarios
Dangers
I Fitting complex models with little data
I A priori overfitting
I Exploring high dimensional design/outcome space:I Arbitrary simulation scenariosI May have blind spotI Temptation to cherry pick scenarios
Dangers
I Fitting complex models with little data
I A priori overfittingI Exploring high dimensional design/outcome space:
I Arbitrary simulation scenariosI May have blind spotI Temptation to cherry pick scenarios
Dangers
I Fitting complex models with little data
I A priori overfittingI Exploring high dimensional design/outcome space:
I Arbitrary simulation scenarios
I May have blind spotI Temptation to cherry pick scenarios
Dangers
I Fitting complex models with little data
I A priori overfittingI Exploring high dimensional design/outcome space:
I Arbitrary simulation scenariosI May have blind spot
I Temptation to cherry pick scenarios
Dangers
I Fitting complex models with little data
I A priori overfittingI Exploring high dimensional design/outcome space:
I Arbitrary simulation scenariosI May have blind spotI Temptation to cherry pick scenarios