ideasIdea
Aggregated single-patient crossover trials for symptom and supportive treatments
For treatments of symptoms like nausea, fatigue or neuropathy, each patient can try the drug and a placebo in alternating periods and see what works for them; pooling many such personal trials gives a population answer too.
N-of-1 trials (multiple randomised crossover periods within one patient) with ePRO outcomes are aggregated across patients using Bayesian hierarchical models to estimate both population and individual effects. Suitable for chronic, stable symptoms and reversible treatments (antiemetics, neuropathic pain agents, appetite stimulants, sleep aids) where conventional RCTs are rare because there is little commercial incentive.
Hypothesis
Aggregated N-of-1 designs will answer supportive-care questions with a fifth of the patients required by parallel-group trials and will give each participant a personal answer they act on.
Rationale
Supportive care is under-researched relative to its impact on QoL. N-of-1 designs are statistically efficient for stable conditions and are inherently patient-centred.
What would test it
Run a platform of N-of-1 trials for three common symptoms in a survivorship clinic with a smartphone ePRO app and compare precision per patient with the literature.
Maturity
speculative
Who has to act
research
Cost to try
Small (under $1M)
Years to first evidence
2
Bottlenecks it attacks
- 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.
- 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.