Tolerability as a co-primary endpoint with its own label claim
New cancer drugs should have to prove not only that they extend life but how they affect how people feel and function, with that result printed in the label like efficacy.
Trials are powered on progression or survival; tolerability is described from clinician-graded adverse events and rarely tested as a hypothesis. The proposal is a regulatory framework in which a pre-specified patient-reported tolerability endpoint (e.g., PRO-CTCAE composite or physical function decline) can be co-primary or a formal secondary endpoint eligible for a label claim, with the analysis methods (time to deterioration, area under the curve, cumulative burden) standardised by regulators and academic groups such as SISAQOL-IMI.
- 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.
- 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.
Pages like this
not linked directly; found by shared links- IdeaDefine tolerability endpoints as rigorously as efficacy endpoints
Shares Immune-related adverse events (irAEs), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaReport time toxicity, the days spent in healthcare, as a standard outcome for older patients
Shares Progression-free survival (PFS), Trial design, endpoints and cost, 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), Progression-free survival (PFS), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaTime to treatment failure and quality of life as co-primary endpoints in non-curative trials
Shares Progression-free survival (PFS), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaSeamless phase 2/3 with pre-registered go rules as the default for new agents
Shares Progression-free survival (PFS), Trial design, endpoints and cost.
- IdeaA permanent platform trial for supportive-care interventions inside cooperative groups
Shares Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaReport time toxicity, the days a treatment consumes, in every trial and decision aid
Shares Progression-free survival (PFS), Toxicity and quality of life are undervalued.
- IdeaRequire a randomised phase 2 before any phase 3
Shares Progression-free survival (PFS), Trial design, endpoints and cost.