By Aiden Flynn. Exploristics CEO
Successful clinical trials depend on people participating in them. For participants this means, opting in and staying the course even while in some cases being very ill. However, for those that sign up, they often do so because they want to make a difference and help clinical researchers identify new or improved treatments, perhaps in part for their own sake but also importantly for the sake of others.
Difficulties with recruitment and dropout
Despite the benefits of joining a clinical trial, recruitment issues and dropout rates remain common pain points for developers of new medicines. They can heavily impact development timelines, costs and success rates and are often closely linked to patient burden. People taking part in clinical trials commonly must overcome burdens such as:
- Lost days from work for site visits
- Travel costs
- Physical and psychological strain involved in participation.
So, anything that can be done to alleviate burden, avoid waste and put the needs of patients first should be a top priority to encourage and support participation, and avoid dropout.
Maximising the impact of patients’ involvement
While several strategies are now being utilised to improve outreach and reduce burden such as increased patient involvement in protocol development, mobile data collection devices and digital or decentralised trials, one that is often overlooked is clinical trial design.
De-risking design upfront through methods like modelling and simulation can play a key role in helping to reducing patient burden. It can ensure that clinical trials are designed more effectively to collect the right data, in the right patients, in the right way. This means that:
- suitable numbers of eligible patients will be recruited to the right trial.
- the data collected will be able to help deliver meaningful results.
- the clinical question posed can be answered.
In short, those patients that participate in a de-risked clinical trial are more likely to contribute to a successful one and so make the difference they seek.
Getting better at designing the right trials for patients
It is often cited that the failure rate in clinical development still exceeds 90%, despite growing R&D spending. While many issues can impact trial success, one that really shouldn’t still need to be included is poor study design. It is easily avoidable. Yet, experience tells us that many trials are still destined to fail before the first patient has been recruited due to inappropriate design. Each of these failed trials represents people who were prepared to overcome the burden involved in the hope of making a difference. Unfortunately, in these cases the odds were stacked against this from the start.
As an industry, we need to ask ourselves if we can do better. Can we design better studies that don’t fail due to design flaws? Can we utilise insights better from the historical data we already have to help with this? Can we get better at capturing meaningful clinical insights from the clinical studies we design given the ballooning costs to industry and the patient burden involved?
Thinking differently will make the difference.
The good news is that we can, and the people and the tools that can help with this are already available. To make headway, however, we need to think differently about de-risking clinical trials to ensure that the people who make it possible, the patients, get a chance to make the difference they hoped. Currently, scant time and money are spent on de-risking design upfront as a proportion of the cost of running a clinical trial. It can often amount to as little as $1000 spent which represents less than 0.05% of the budget and can be limited to asking a statistician for a sample size calculation. This represents a missed opportunity in terms of building in a much greater likelihood of study success. Sample size and power calculations alone are not sufficient to ensure success. They simply do not provide the holistic approach required to design a successful study.
Statisticians and data scientists now offer a staggering array of methods and technologies that can support better design and analysis to extensively de-risk a clinical trial from the outset. They just need to be asked. Reaching out to them earlier in the design process will enable these impactful approaches to be harnessed. As an industry, we owe it to the many stakeholders involved in the development process to ensure that studies no longer fail due to inappropriate design. Most importantly, however, as healthcare innovators we owe it to the people that make the whole process possible, the patients.