Lisa V. Hampson

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Bayesian decision procedures have already been proposed for and implemented in Phase I dose-escalation studies in healthy volunteers. The procedures have been based on pharmacokinetic responses reflecting the concentration of the drug in blood plasma and are conducted to learn about the dose-response relationship while avoiding excessive concentrations.(More)
OBJECTIVES Chronic musculoskeletal pain and osteoarthritis can significantly limit the functional independence of individuals, and given that 25% of the population experience these problems, the socioeconomic impact is immense. Exercise and self-management have proven benefits for these conditions, but most trials tailor interventions for specific joints.(More)
This paper considers the design and interpretation of clinical trials comparing treatments for conditions so rare that worldwide recruitment efforts are likely to yield total sample sizes of 50 or fewer, even when patients are recruited over several years. For such studies, the sample size needed to meet a conventional frequentist power requirement is(More)
BACKGROUND Many randomized controlled trials (RCTs) collect cost-effectiveness data. Without appropriate sample size calculations, patient recruitment may cease before the cost-effectiveness of the intervention can be established or continue after the cost-effectiveness of the intervention is established beyond doubt. PURPOSE We determined the frequency(More)
In randomised controlled trials, the effect of treatment on those who comply with allocation to active treatment can be estimated by comparing their outcome to those in the comparison group who would have complied with active treatment had they been allocated to it. We compare three estimators of the causal effect of treatment on compliers when this is a(More)
OBJECTIVES Definitive sample sizes for clinical trials in rare diseases are usually infeasible. Bayesian methodology can be used to maximise what is learnt from clinical trials in these circumstances. We elicited expert prior opinion for a future Bayesian randomised controlled trial for a rare inflammatory paediatric disease, polyarteritis nodosa (MYPAN,(More)
Extrapolating from information available on one patient group to support conclusions about another is common in clinical research. For example, the findings of clinical trials, often conducted in highly selective patient cohorts, are routinely extrapolated to wider populations by policy makers. Meanwhile, the results of adult trials may be used to support(More)
Background Bayesian statistics are an appealing alternative to the traditional frequentist approach to designing, analysing, and reporting of clinical trials, especially in rare diseases. Time-to-event endpoints are widely used in many medical fields. There are additional complexities to designing Bayesian survival trials which arise from the need to(More)
In phase II platform trials, 'many-to-one' comparisons are performed when K experimental treatments are compared with a common control to identify the most promising treatment(s) to be selected for Phase III trials. However, when sample sizes are limited, such as when the disease of interest is rare, only a single Phase II/III trial addressing both(More)