Define tolerability endpoints as rigorously as efficacy endpoints
Trials report side effects as a table of percentages that hides how long they lasted, how bad they felt and whether people stopped treatment. Tolerability should be measured with defined endpoints and a decision rule, like efficacy.
Protocols pre-specify tolerability estimands under ICH E9(R1): time to first dose modification, cumulative patient-reported symptom burden (PRO-CTCAE), treatment discontinuation for toxicity, and a toxicity-over-time summary, with hypotheses and analysis plans. Regulators include a tolerability summary in the label, and dose-optimisation decisions reference it.
- Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.
- Toxicity and quality of life are undervalued · Trials measure how long people live, not how they live. Side-effects are under-reported and under-treated.
- Wrong doses · Most drug doses were chosen as the highest a person can tolerate, which is often more than they need.
Pages like this
not linked directly; found by shared links- IdeaTolerability as a co-primary endpoint with its own label claim
Shares Immune-related adverse events (irAEs), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaA permanent platform trial for supportive-care interventions inside cooperative groups
Shares Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaDose adjustments driven by patients' own symptom reports, tested against clinician judgement
Shares Wrong doses, Toxicity and quality of life are undervalued.
- IdeaReal-world dose intensity and toxicity monitoring to revise labelled doses
Shares Wrong doses, Toxicity and quality of life are undervalued.
- IdeaAnalyse and dose by sex: women get more toxicity from many cancer drugs at the same dose
Shares Wrong doses, Toxicity and quality of life are undervalued.
- IdeaModel-informed individual dosing with drug-level monitoring for every oral cancer drug
Shares Wrong doses, Toxicity and quality of life are undervalued.
- IdeaMeasure blood levels of oral targeted drugs and adjust doses to a target range
Shares Wrong doses, Toxicity and quality of life are undervalued.
- IdeaWin-ratio endpoints that weigh survival, toxicity and quality of life together
Shares Immune-related adverse events (irAEs), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.