When adult drugs are studied in kids

TRIALS

Pediatric Cancer Markets are too small, so companies are reluctant to do studies on kids. Companies are motivated to do studies in kids when:

there is a requirement

RACE PSP requirement

EMA PIP

there is an incentive

pediatric exclusivity incentive

voucher

first approval in kids so as to lower standards of approval for next, bigger indication

It’s not too much work and gets a faster accrual

they may include kids/teen on an adult trial

ACCESS OUTSIDE TRIALS

Companies are reluctant to provide access outside of trials, including

Compassionate Use

compassionate use cases

Off label access for expensive drugs, especially cell and gene therapies

(for premium priced new drugs, they may not agree to off label use because insurers might not pay and then they are stuck with a compassionate use request.