ideasIdea
Kidney and liver impairment dosing studies completed before approval, not years after
Dosing advice for people with weak kidneys or liver is often missing at approval and added years later, if ever. Requiring those studies before approval would protect a large group of real-world patients from day one.
Regulators require dedicated or population-PK-based organ impairment dosing recommendations (mild, moderate and, where feasible, severe renal and hepatic impairment) as part of the initial marketing application for oncology drugs, rather than as post-marketing commitments, using sparse PK from broadened-eligibility trials and small dedicated cohorts. Ties to relaxing organ-function eligibility criteria.
Hypothesis
Drugs approved with organ-impairment dosing will show lower toxicity-related hospitalisation among patients with impairment in real-world data than drugs approved without, and post-marketing commitments for these studies will fall.
Rationale
Organ impairment is common in cancer patients and clinicians either extrapolate or withhold drugs; post-marketing studies are frequently delayed or never completed.
What would test it
Audit completion times of organ-impairment post-marketing commitments for the last decade of oncology approvals; then require them pre-approval for three years and compare real-world outcomes in impaired patients.
Maturity
speculative
Who has to act
regulator
Cost to try
Small (under $1M)
Years to first evidence
2
Bottlenecks it attacks
- Wrong doses · Most drug doses were chosen as the highest a person can tolerate, which is often more than they need.
- Older and multimorbid patients are excluded and undertreated · Most people with cancer are over 65 but most trial patients are younger and fitter. We guess how to treat the majority.
- Trials enrol too few, too slowly · Fewer than one in ten adults with cancer joins a trial. Trials close for lack of patients, not lack of ideas.