March 2024 • PharmaTimes Magazine • 10-12
// DIVERSITY //
Eclectic mix
Diversity in clinical trials – not just ‘nice to have’ but instrumental
The success of running and managing a clinical trial to assess the efficacy of a new treatment or device can largely be determined by how effectively it mirrors real-world outcomes.
In order to mirror the real world, it is vital that trial populations accurately reflect the real-world patient populations.
The diversity of trial participants remains an issue within the industry and having a diverse patient pool is key to the success of trials.
There has been growing pressure on companies to improve the diversity of their trial populations for decades, but work still needs to be done, including by regulators and ethics groups, who are increasingly taking action to bring about further progress in this area.
In the key of life
The aim of a clinical trial is to test the efficacy and safety of experimental products in patients.
In order to produce results that are as accurate as possible, the patient population receiving an experimental treatment in a trial – or a placebo – should mirror the real-world patient population.
More representative patient groups enable a much better understanding of how new treatments and medical devices impact different patients.
This means that factors including gender, race, age, disability and geography must be considered in recruiting patients for clinical trials.
Increasingly, the general population is becoming more aware of the importance of this issue and of the consequences of failing to ensure that diversity and inclusion are a foremost consideration within clinical research.
It is vital that investigators expand access to clinical trials and encourage trial recruitment through greater patient engagement so that patients feel comfortable taking part in research.
A recent national study carried out by the UK’s Health Research Authority (HRA) in collaboration with YouGov found that the majority of those surveyed have more confidence in trial findings when the trial population was more diverse.
Additionally, nearly three-quarters of respondents said that diversity in trial populations was important even if it results in greater costs and longer timelines.
This highlights the broader consequences of a lack of diversity in trials and how it can undermine confidence in trial outcomes amongst the general population.
Trust and confidence are vital in the relationship between regulators, researchers and, most importantly, patients.
Trials that lack a diverse population can also cause regulatory consequences – in some cases regulators will mandate investigators to carry out post-approval studies to validate the results of a trial in a more diverse population.
This can take time, however, and result in significantly higher costs and can therefore have an impact on the industry’s ability to continue to bring new treatments to patients.
A lack of diversity can therefore have a considerable impact on how both regulators and patients view trial results.
Tuning into diversity
In order to tackle this ongoing issue, authorities around the world are starting to take action and are legislating to bring about change in the industry to make trials more inclusive.
This includes 2023 US Food and Drug Administration (FDA) guidance on post-marketing approaches to broaden research in underrepresented populations.
In 2022, however, the US regulator also noted the importance of integrating diversity into trial designs as early as possible by introducing recommendations around the inclusion of diversity plans in draft guidance.
In order to accelerate progress in this area and specifically the implementation of diversity plans, regulators are moving from voluntary guidance towards mandated action.
At the end of 2022, the use of diversity action plans was made law by US Congress with clinical trials used by the FDA for safety and efficacy requiring the submissions of such plans.
According to the law passed by Congress, these plans should include goals for enrolment, the reasoning behind this goal and setting out a route to achieving the targets.
Mandating these action plans is an important step in increasing diversity within trials. It is vital now that we see action from the industry to implement these changes.
Simultaneously, UK regulators such as the Health Research Authority (HRA) and the Medicines and Healthcare products Regulatory Agency (MHRA) are developing non-mandatory guidance for the clinical research industry to improve inclusion and diversity in trials.
A first version of this guidance is expected later this year to provide recommendations for the industry and ensure that trials are designed to be accessible to more diverse patient populations.
Importantly, these guidelines are being developed in a collaborative way with input from a variety of different stakeholders, including the public, researchers and pharmaceutical companies.
Pump up the volume
Improving access to trials requires this collaborative approach. A 2023 review of clinical trial activity in the UK by former government minister Lord O’Shaughnessy highlighted the importance of public outreach around research, primarily with the widespread aim of increasing participation.
The O’Shaughnessy Review of the commercial clinical trials sector in the UK was commissioned by the UK government to arrest a decline in research activity in the UK and address some of the key challenges that face the sector.
Significantly, the review notes the current difficulties of patient recruitment in UK clinical trials, challenging the UK commercial clinical trials sector to double recruitment within two years following the publication of the report in 2023, and double it again by 2027.
Recommendation 17 of the review specifically highlighted the importance of increasing diversity in trials and also introducing specific targets around gender, geography, ethnicity and age.
The review also recommends increased monitoring of patient populations within trials to ensure that access to research is equal and that this can be measured.
Lord O’Shaughnessy notes in the review that the UK’s diverse population means that researchers have the opportunity to demonstrate safety and efficacy across a broad patient population.
UK chart positions
The UK is an attractive location for clinical researchers, but the industry must widen access to ensure that diversity is reflected within clinical trials.
O’Shaughnessy also notes the potential value of decentralisation in the UK clinical trial sector and the significant role technology can play in boosting the sector.
Digital health technologies can support trial diversity in a number of ways. Notably, AI can help in the search for patients suitable for trials, and using ethical and trustworthy AI systems to reduce bias can ensure that more diverse patient populations are recruited.
Additionally, the use of trial decentralisation technologies can broaden access to trials to wider populations.
Researchers are able to reach patients who would not have previously participated in trials as decentralisation can reduce travel time, costs and time commitment, which ensures that more people are able to sign up to clinical trials.
This integration of technology into trials presents a real opportunity in this sector throughout the UK.
Indeed, it has been further accompanied by some supportive action from the government, including in its response to the O’Shaughnessy Review, which included a commitment to increase the adoption of trial decentralisation.
Tracks of our years
The importance of widening access to clinical trials cannot be overstated.
It is a crucial tool to increase confidence in the clinical research process for both regulators and patients.
While regulators are increasingly mandating action in this space, the opportunity presented to improve both clinical research insights and eventually treatment outcomes for patients must not be forgotten.
As the industry faces a series of headwinds – notably, challenges in recruiting patients for trials – broadening access to trials not only ensures more accurate trials but can also ensure patients feel more confident participating in research.
Technology can be an important tool in this process as researchers can reach more patients and also reduce the burden for those who wish to participate but otherwise may not have been able to.
Although issues remain, recent technological and regulatory developments have led to significant progress in improving clinical trial diversity.
Make no mistake, however, it is vital that industry and regulators continue to collaborate with a view to igniting change for the benefit of patients – all patients.
Fiona Maini is Senior Director Global Compliance and Strategy at Medidata.
Go to medidata.com
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