Abstract
BackgroundWith millions of pounds spent annually on medical research in the UK, it is important that studies are spending funds wisely. Internal pilots offer the chance to stop a trial early if it becomes apparent that the study will not be able to recruit enough patients to show whether an intervention is clinically effective. This study aims to assess the use of internal pilots in individually randomised controlled trials funded by the Health Technology Assessment (HTA) programme and to summarise the progression criteria chosen in these trials.MethodsStudies were identified from reports of the HTA committees’ funding decisions from 2012 to 2016. In total, 242 trials were identified of which 134 were eligible to be included in the audit. Protocols for the eligible studies were located on the NIHR Journals website, and if protocols were not available online then study managers were contacted to provide information.ResultsOver two-thirds (72.4%) of studies said in their protocol that they would include an internal pilot phase for their study and 37.8% of studies without an internal pilot had done an external pilot study to assess the feasibility of the full study. A typical study with an internal pilot has a target sample size of 510 over 24 months and aims to recruit one-fifth of their total target sample size within the first one-third of their recruitment time.There has been an increase in studies adopting a three-tiered structure for their progression rules in recent years, with 61.5% (16/26) of studies using the system in 2016 compared to just 11.8% (2/17) in 2015. There was also a rise in the number of studies giving a target recruitment rate in their progression criteria: 42.3% (11/26) in 2016 compared to 35.3% (6/17) in 2015.ConclusionsProgression criteria for an internal pilot are usually well specified but targets vary widely. For the actual criteria, red/amber/green systems have increased in popularity in recent years. Trials should justify the targets they have set, especially where targets are low.
Highlights
With millions of pounds spent annually on medical research in the UK, it is important that studies are spending funds wisely
In the financial year 2015/16, the National Institute for Health Research (NIHR) spent £247.9 million on their research programmes and almost a third of this was spent on their Health Technology Assessment (HTA) programme [1]
With such large amounts of public money being spent on health research, it is important that the funds are used wisely and that money is not wasted on trials which are not likely to succeed
Summary
With millions of pounds spent annually on medical research in the UK, it is important that studies are spending funds wisely. In the financial year 2015/16, the National Institute for Health Research (NIHR) spent £247.9 million on their research programmes and almost a third of this was spent on their Health Technology Assessment (HTA) programme [1] With such large amounts of public money being spent on health research, it is important that the funds are used wisely and that money is not wasted on trials which are not likely to succeed. An internal pilot is a phase in a trial after which progress is assessed against pre-specified targets/criteria [2, 3] They are an opportunity to stop trials which are not likely to reach their recruitment, retention or site set-up targets (among others). Including an internal pilot in a study allows funders to take on more risky trials, such as trials where recruitment to time and target is uncertain due to a lack of previous research in the clinical area or a trial population
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