Decentralised trials, patient insight boards, meaningful diversity in research and the patient voice

The success of clinical trials hinges on clear research objectives and quality data.

But underlying the ideal of clinical research lies a diverse cohort of patients, whose lived experiences are increasingly recognised by the medical community as integral to drug discovery and long-term outcomes.

Decentralised clinical trial (DCTs) design and patient insight boards are two key methods that, when used in tandem, can help ensure clinical data retains the nuance, humanity, and relevance to all stakeholders engaged in clinical research.

If decentralised trials provide a breadth of raw patient information, patient insight boards bring the depth of first-hand knowledge of lived experiences into clear focus, providing the basis for true empathetic technical solutions.

The clinical trial ecosystem, historically, has not drawn from a genuinely diverse research pool. Study design prioritised the optimisation of resources, safety, control and consistency.

In both the US and Britain, in-person clinical trials are primarily held in major metropolitan areas for this reason.

Knowing travel may incur significant cost and time for patients already experiencing a health struggle, some patients and clinical teams felt it was not in their best interest to take part. Practical decisions such as these filter rural, low-income and elderly individuals out of the participant pool.

When efforts to widen participation were made, they were often thwarted by insufficient diversity measurement benchmarks and lack of understanding of cultural and community needs.

Recruitment can be challenging – getting enough participants was the focus over getting a truly representative population sample.

We now have a much greater understanding and awareness of how inflexible study structures can inadvertently create exclusivity. Fortunately, we also have the technology required to make meaningful changes.

Decentralising aspects of clinical trials is becoming the norm in medical research, to the benefit of patients and researchers alike. This decentralisation opens up possibilities to ensure that studies more accurately reflect the age, race, ethnicity and geography of the end users.

While the conversation around DCTs has, to this point, treated them as a stand-alone study design, Medidata has found it far more useful to think of decentralisation as a delivery mechanism through which we apply technology to existing programmes to better meet the needs of patients and study teams.

The degree of decentralisation and elements targeted will change study to study. As we look to broaden the use of decentralisation, we should seek out individual trials with the best eligibility profile, rather than trying to target broad therapeutic areas.
In vaccine or pain studies, where patients are generally well, decentralisation offers a low-burden way of fitting research into daily life.

On the other end of the spectrum, DCTs reduce onsite exposure risk in clinically vulnerable oncology patients.

The indications that would currently benefit most from DCTs sit on a similar continuum. Rare diseases that affect only a handful of people dispersed around the world demand decentralisation technology to maximise engagement and recruitment.

Common chronic conditions such as diabetes and cardiac issues, which appear across all segments of all populations, can use DCTs to illuminate the nuances of disease across demographics.

The COVID-19 vaccine trials provided the ultimate proof-of-concept, because we had no other choice but to adapt to travel restrictions.

It showed doctors, clinical teams and patients that technology was available and capable to allow for a seamless clinical trial experience anytime, anywhere.

Meanwhile, regulators in the US, UK and EU responded to the need quickly, coaxing the industry to become comfortable with the delivery mechanism’s novel approach to clinical trial delivery. There are countless conditions and potential therapies that could be the ‘next big thing’

in decentralised clinical trials. Effective change management within clinical research will realise their potential to produce meaningfully diverse and scientifically impactful data.

We cannot support genuinely inclusive clinical trials without understanding the variety of challenges facing patients participating in research.

We rely on patient advocacy groups and insight boards to provide that context and help develop a deeper understanding on how to support and engage patients through the clinical trial life cycle.

Recently, a member of Medidata’s patient insight board passed away after dealing with metastatic breast cancer for over 16 years.

She participated in numerous clinical trials as part of her care journey and was a fierce advocate for expanding access to clinical research for communities of colour.
Her contributions were critical to our understanding of the Black experience of clinical research in the American South.

She taught us what it meant to reach out and build relationships with populations historically under-represented in trials when accurate information was not reaching these communities.

Patient insight boards, like DCTs, require long-term investment. A one-off engagement is not going to provide a complete picture.

We need committed facilitators and moderators to foster bi-directional relationships to achieve an in-depth, comprehensive understanding of patient burdens and needs. We need to create the proper infrastructure to action the feedback emerging from these sessions. Only then can these exercises generate goodwill, good information, and good answers to complex questions and serve as a bridge to communities in need.

We are confident that, with time, trust and effort, patient insight boards will become a standard feature in the industry’s approach to clinical research. And insight boards themselves are undergoing a process of decentralisation. Before the pandemic, in-person meetings were the standard.

Medidata uses its own DCT technology to support more conversations in more places. In turn, we engage patient advocacy groups to get their feedback as we develop our patient-facing services.

We don’t think about DCTs and patient insight boards as two distinct diversification projects; they are complementary elements of an inclusive, representative clinical research infrastructure. It’s all about opening up communication channels.

If we design patient advocacy to be ingrained in research, we recognise where we can apply decentralised elements to studies. Patients, ultimately, are the best judge and advocates for their own therapeutic experience and care pathways.

Decentralised technology is already here, and it is already the norm. If anything, we hope to see a move away from labelling trials as decentralised.

Trials, if designed in the best interest of the patient, will build in appropriate decentralised delivery methods as standard.

Patient insight boards, if engaged with proper intent, will make clear where those methods and improvements should be directed. Ironically, that may be up to and including inviting patients in for site visits, if that is their preference.

The medical research industry is increasingly aware of the human element at the core of drug development. While the targets remain the same – finding better treatments and lengthening life – we must not lose sight of the individuals driving scientific discovery forward.

DCTs inject research with refined, nuanced data best applicable to the end user. Patient insight boards ensure those patients’ vital contributions are recognised and respected.

Paul O’Donohoe and Alicia Staley are from Medidata. Go to medidata.com