Cutting-edge treatments for rare disease patients are often only available through trials. Recruitment rates in these trials are notoriously low and many sites fail to recruit a single patient. A ‘just-in-time’ (JIT) model has been successful elsewhere. This model allows sites to open only when a patient presents. It provides patients with access to rare disease trials while mitigating the risk of non-recruiting sites.
SKYHOOK will adopt a JIT model once the lead site is open. It is currently being developed by a consortium of paediatric oncology sites. The consortium will adopt several streamlining initiatives for trial set-up to ensure participants are treated within a clinically acceptable timeframe - days rather than months.
The National Clinical Trial Governance Framework encourages organisations to have, “a system-wide commitment to continuous improvement to minimise pre-approval delays...”. SKYHOOK is an example of the Consortium’s commitment to continuous improvement in the trial set-up process, which, if successful, will enable greater access to trials for a patient population that is currently underserved by clinical trials.
This session will describe the work to date to develop the SKYHOOK model and the initiatives planned.

N-of-1 trials are individualised randomised controlled trials where patients serve as their own control. They represent a powerful method in clinical research and practice because they provide information about how an individual patient responds to a drug or treatment, where traditional clinical trials often cannot. Research shows that ‘pooled’ N-of-1 studies (where data from a series of N-of-1 studies are statistically aggregated) can be used to estimate population-based effects of drugs/treatments with fewer participants than randomised controlled trials. This reduces the time, cost and recruitment issues associated with conventional clinical trials. The aim of this presentation is to discuss the role of pooled N-of-1 trials in providing innovation in protocol and trial design. Key opportunities associated with conducting pooled N-of-1 trials will be outlined including flexibility, efficiency, precision, patient-centredness, and cost-effectiveness. Limited awareness about the potential of N-of-1 trials to obtain individual and population estimates of effectiveness is a challenge to their wider adoption. Rapid advances in digital technology facilitate use of this powerful, personalised treatment methodology. Pooled N-of-1 trials play an important role in the movement towards personalised medicine, digital health, shared clinical decision-making and patient-centred healthcare.