Why amplifying the patient voice is crucial to clinical trial success

Participants are vital to the success of any clinical trial. Without the data they provide, our research system would collapse. But being a clinical trial participant is not easy. Financial barriers, transport challenges, and a lack of engagement and support can leave patients feeling marginalised and disconnected.

Historic failures to address the burden of research participation have come at a huge cost to our industry. More than two-thirds of clinical trials fail to meet original patient enrolment targets, 85% fail to retain enough patients, and the average dropout rate across all trials is 30%.¹ Yet the evidence suggests most reasons for recruitment failure, including high patient burden, are preventable.²

In recent years, there has been increasing recognition of the value of working with patients and their care givers.³ Yet, there is still work to be done to ensure diverse patients from around the world can engage with studies and remain engaged from screening until study completion, regardless of their personal circumstances.

To meet this challenge, our traditionally cautious industry must embrace new technologies and recognise patients and their representatives as true partners in clinical trials.

Engaging with clinical trial participants

To understand patient experience, we need to engage with participants and their caregivers in a meaningful way. However, definitions of patient engagement vary throughout the literature.

To help overcome this, in 2020, the Professional Society for Health Economics and Outcomes Research (ISPOR) patient-centred interest group proposed the following definition for patient engagement in research: “The active, meaningful, and collaborative interaction between patients and researchers across all stages of the research process, where research decision-making is guided by patients’ contributions as partners, recognising their specific experiences, values, and expertise.”⁴

Crucially, this definition identifies patients as equal partners throughout a clinical trial.

So, how can we engage with patients across all stages of the process? Technology holds some of the answers. For example, mobile apps can be used to allow participants to give direct feedback on their trial experience. This can give researchers insight into participant satisfaction in real time, identify those at risk, and take proactive steps to change their experience before they dropout.

In-app message functionalities also offer new opportunities for patient engagement. Regular communication between site staff and participants that uses tools like secure two-way messaging can facilitate enrolment and reduce dropouts.⁵ In-app messaging functions also provide sites with a centralised way to analyse interactions and participant satisfaction, while one-way notifications via SMS, email, and push notifications can be used to decrease missed appointments that could compromise data.

Amplifying diverse patient voices

Every participant has the potential to provide unique insights into the clinical trial experience. Understanding and utilising these insights leads to more relevant research outcomes⁶ and considerable financial value⁷ for site sponsors.

But, to truly reap the benefits of listening to the patient voice, we need to ensure everyone is empowered to share their experience. Distrust in research organisations has been identified as a barrier to clinical trial participation, particularly among minority ethnic groups.⁸ Other barriers include attitudes and beliefs, including a lack of social approval,⁹ and a lack of knowledge about clinical trials.¹⁰

Patient testimonials¹¹ and a personalised approach¹² have been identified as key strategies for improving trust in, and understanding of, clinical trials. To broaden the reach and impact of these approaches, we need to give participants and their care givers from all backgrounds a platform to share their stories.

One example of such an initiative is The Portrait Project. This collection of impactful stories, shared by trial participants and care givers in their own words, offers insight into the people behind groundbreaking medical innovations. By offering an insight into the highs – and lows – of trial participation, it can help prepare participants and highlight areas where we as an industry still need to improve.

Overcoming practical barriers

In addition to the informational and societal barriers outlined above, there are practical challenges that prevent patients from enrolling in, or remaining on, a clinical trial. They include a lack of transport,¹³ time,¹⁴ and indirect costs associated with trial participation.¹⁵ These logistical challenges tend to disproportionately effect under-served groups.¹⁶

Again, technology offers solutions, with digitisation allowing instantaneous stipend payments and faster study start-up. Modern payment technologies give participants a choice in how they receive funds, whether that is via online payment portals, direct to their account or cheque, and ensure patients are not out-of-pocket. Integrated platforms can also pay for things like travel directly, rather than requiring participants to claim expenses.

Technology is also helping to reduce the time and travel burden on participants by enabling data collection through electronic participant-reported outcomes and monitoring via digital access to electronic health records.

However, more still needs to be done to ensure all participants are supported to engage in clinical trials – no matter what their background or where they live in the world. In 2022, the number of trials registered in high-income countries was more than 90 times greater than in low-income countries.¹⁷ A higher median household income is associated with a greater likelihood of participation in a clinical trial.¹⁸

Value for the clinical trial industry

As discussed throughout this article, amplifying participant voices benefits not just patients themselves, but the wider clinical trial industry. A comprehensive review of the value of patient engagement identified 18 key benefits.¹⁹ They included more relevant research topics and priorities, more appropriate resource allocation, improved recruitment and retention, increased diversity of study participants, faster study completion, and increased knowledge and public awareness of products.

There is also a clear financial incentive to amplify the patient voice. Patient engagement activity that avoids one protocol amendment, and improves enrolment, adherence, and retention, leads to an increase in expected net present value of $35 million in pre-phase II projects.²⁰ This is the equivalent of accelerating a pre-phase II product launch by two-and-a-half years.

The case for amplifying the participant voice is clear. We need to make clinical trial participation a seamless experience for patients, build relationships between patients and site staff, and remove logistical barriers to participation for the good of the wider health research landscape.

The only question that remains is, is the industry willing to harness new technologies and seize the opportunities on offer? We sincerely hope the answer is yes.

About the authors

Jason Dong is co-CEO and co-founder of Mural Health. He was previously an investor in pharma technology businesses at Advent International, where had an off-the-cuff conversation with co-founder Sam Whitaker about what it would look like to modernise the patient experience. Dong grew up in New Zealand and went to the US to attend Harvard. Previously, he was a management consultant at McKinsey & Co.

Sam Whitaker is co-CEO and co-founder of Mural Health. Previously, he founded Greenphire in 2008. Whitaker is from suburban Philadelphia and is a graduate of the University of Pennsylvania.