OnCo
ideasIdea

Restore weight-based dosing and vial sharing for immunotherapy in the label

Immunotherapy is given as one flat dose regardless of body size, which means smaller patients get more than they need. Dosing by weight would save a fifth of the drug at no cost to patients.

Pembrolizumab and nivolumab were developed with weight-based dosing but labelled at flat doses (200 mg, 240 or 480 mg) that exceed the weight-based equivalent for most patients; pharmacokinetic modelling and several observational studies suggest no loss of efficacy from weight-based or capped dosing, and Dutch and other groups estimate savings of 20-30%. The proposal is a regulator-endorsed label update permitting weight-based dosing with dose banding and multi-dose vial sharing, supported by a pragmatic non-inferiority trial where regulators require one.

Hypothesis
Weight-based, banded dosing reduces checkpoint inhibitor drug spend per patient by at least 20% with overall survival non-inferior to flat dosing (hazard ratio upper bound below 1.1).
Rationale
Exposure-response for PD-1 antibodies is flat across the approved range, so the flat doses were chosen for convenience and, arguably, revenue; the drug is the same either way.
What would test it
Pragmatic registry-based randomised trial in one health system comparing weight-based banded dosing with flat dosing across indications, with survival and cost endpoints; petition regulators for label change on the result.
Maturity
early clinical
Who has to act
clinic
Cost to try
Medium ($1M to $50M)
Years to first evidence
3
Bottlenecks it attacks
  • Prices and value · New cancer drugs routinely cost over $150,000 a year, often for months of benefit. Systems cannot afford them and patients go bankrupt.
  • Wrong doses · Most drug doses were chosen as the highest a person can tolerate, which is often more than they need.

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