ideasIdea
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.
Hypothesis
A qualified function endpoint, anchored to patient-reported meaningfulness, lets a cachexia trial of about 300 patients demonstrate benefit convincingly, and at least three sponsors initiate registrational trials within three years of qualification.
Rationale
Endpoint qualification unlocked development in Duchenne muscular dystrophy, idiopathic pulmonary fibrosis and heart failure. Cachexia has more patients than all of those and one approved drug in most of the world.
What would test it
Pool individual-patient data from completed cachexia trials to establish the test-retest reliability and minimal clinically important difference of candidate measures; submit a qualification package.
Maturity
speculative
Who has to act
regulator
Cost to try
Small (under $1M)
Years to first evidence
5
Bottlenecks it attacks
- 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.