Qualify a physical function endpoint so anti-wasting drugs can be approved
Regulators are unsure what to accept as proof that an anti-wasting drug helps. Agreeing on a simple measure such as stair climbing would unblock the whole field.
Cachexia drug development has repeatedly stalled on endpoint uncertainty: weight is easy but not clearly meaningful, and composite quality-of-life measures are noisy. Candidate function measures — stair climb power, six-minute walk, handgrip strength, wearable-derived daily step count — are simple and reproducible but not formally qualified. A qualification programme with pre-competitive data pooling would create a defined path.
- Cachexia, toxicity and the limits of the patient · Patients often die of wasting or cannot tolerate the doses that would work. Treating the patient, not just the tumour, lags far behind.
- 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.
- Patients lack understanding, navigation and agency · Most patients cannot understand their options, find trials, or push back, so decisions are made for them.
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