Overview
Clinical trials are a vital research tool for advancing medical knowledge and, in turn, patient care. Following the COVID-19 pandemic and the phenomenal success of the RECOVERY Trial, the public profile of and emphasis around the need to deliver trials for the patient benefit has only heightened.
However, Clinical trials require a substantial amount of time, resources, and money to ensure that important research questions are answered. But what if we made the process more efficient and cost-effective? How could we improve the public outcome more effectively? Overcoming these challenges will require a streamlined and more efficient approach to trial processes, and our routinely collected health records may hold the key. In this case study, we will look at the challenge clinical trials face, how researchers have utilised routinely-collected data to salvage dormant trials, and how data-enabled clinical trials are now resulting in proof of concepts for future trials.
Challenge
Clinical trials are research studies conducted using real-life participants as a way of testing new treatments and approaches to help treat disease and illness. Clinical trials can; however, be expensive and time consuming, as they can require a substantial amount of money and resources to identify, recruit, and collect trial-specific data; leaving many trials dormant.
Currently, there is a significant overlap between the data that is needed for clinical trials and the data that is already being routinely collected electronic healthcare records (EHRs). From this, we know that opportunities exist for scientists and researchers access and utilise already collected data in ways to reduce trial costs, decrease duplication of effort, diversify research, and speed up trial recruitment, conduct and follow-up.
Approach
In a randomised trial carried out at English hospitals between August 1993 and October 2001, researchers set out to address whether infant formula modifications promoted long-term cognitive benefits when compared to standard infant formulas. The research team gathered data from 1,763 children, who were each randomly given enriched or standard formula milk. However, due long-term follow up needed for results, the trial was deemed inconclusive for cognitive ability, and the trials were referred to as dormant and were discontinued.
In 2018, researchers at UCL Great Ormond Street Institute of Child Health and the UCL Institute of Education, including Dr Maximiliane L Verfürden, Professor Ruth Gilbert, Professor Alan Lucas, Professor John Jerrim and Professor Mary Fewtrell, reopened the previously dormant trial; making it the first reactivated study of its kind in UK children.
In the reopening of the study, the research team were able to retain the previously unlinked data from seven of the historic trials and link 91.2% of it to routinely collected data from the National Pupil Database (NPD). NPD provides third-parties including schools, policy makers and researchers -with a lawful basis- access to de-identified pupil data for the purpose of improving the education standards and quality in UK schools.
Through linking trial data to the NPD data, the research team were able to analyse results and answer the long-standing question of whether the type of milk participants had been given as babies had a noticeable effect on their school performance.
Impact and Outcomes
The results from this study found that the infant formula modifications did not promote long-term cognitive benefit when compared with standard infant formulas, allowing researchers to finally answer the long-standing research question.
Thanks to access to a wealth of historical trial data and the ability to link this data to NPD, the team were able to maintain blinding of the outcome assessor, save time for participants, and increase rates of follow-up. Without their ability to link the historical data, the previously dormant trial would remain that still, dormant.
The research led by Dr Maximiliane L Verfürden and team is a significant demonstration of the how the data linkage approach can be used to maximise the use of trials – both dormant trials that require long-term follow up and new trials that require a depth of scientific insights. Being the first study of its kind in the UK, this study leads by a great example for future trials and cohorts looking to use data linkage to answer important research questions for the wider public benefit; and despite the study being specifically related to education data, similar linkages can be applied in health data research.
Patient and Public Involvement and Engagement
Six members of the public were involved in the conceptualisation stage of Dr Maximiliane L Verfürden, Professor Ruth Gilbert, Professor Alan Lucas, Professor John Jerrim and Professor Mary Fewtrell’s research.
Further reading:
Full paper – published in BMJ: Effect of nutritionally modified infant formula on academic performance: linkage of seven dormant randomised controlled trials to national education data | The BMJ
BMJ Editorial: https://www.bmj.com/content/375/bmj.n2661
UCL news article: https://www.ucl.ac.uk/news/2021/nov/modified-formula-milk-not-linked-better-academic-performance
