{"entity":{"id":"idea-bio2-cachexia-function-endpoint","kind":"idea","name":"Qualify a physical function endpoint so anti-wasting drugs can be approved","aka":[],"tldr":"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.","summary":"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.","asOf":"2026-09-08","links":[{"label":"FDA Oncology Center of Excellence (page moved; nearest live section)","url":"https://www.fda.gov/about-fda/"}],"tags":[],"related":[],"cancers":[],"sections":[],"technologies":["exercise-oncology","geriatric-assessment"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":["b-cachexia-supportive","b-trial-design","b-patient-voice"],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"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.","test":"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","actor":"regulator","cost":"small","horizonYears":5},"route":"/ideas/idea-bio2-cachexia-function-endpoint/","neighbours":{"technology":[{"id":"cachexia-appetite-pharmacotherapy","kind":"technology","name":"Cachexia pharmacotherapy: GDF-15 blockade, anamorelin, olanzapine","route":"/technologies/cachexia-appetite-pharmacotherapy/"},{"id":"exercise-oncology","kind":"technology","name":"Exercise & lifestyle oncology","route":"/technologies/exercise-oncology/"},{"id":"geriatric-assessment","kind":"technology","name":"Geriatric assessment","route":"/technologies/geriatric-assessment/"}],"bottleneck":[{"id":"b-cachexia-supportive","kind":"bottleneck","name":"Cachexia, toxicity and the limits of the patient","route":"/bottlenecks/b-cachexia-supportive/"},{"id":"b-patient-voice","kind":"bottleneck","name":"Patients lack understanding, navigation and agency","route":"/bottlenecks/b-patient-voice/"},{"id":"b-trial-design","kind":"bottleneck","name":"Trial design, endpoints and cost","route":"/bottlenecks/b-trial-design/"}],"trial":[{"id":"romana-1-2","kind":"trial","name":"ROMANA 1 and ROMANA 2","route":"/trials/romana-1-2/"}],"roadmap":[{"id":"survivorship-roadmap","kind":"roadmap","name":"Supportive care and survivorship roadmap: making treatment bearable → proving it extends life → caring for tens of millions afterwards","route":"/roadmaps/survivorship-roadmap/"}]}}