"Still, there’s often confusion about who is caring for the patient ... " Playing off of Jimmy the Greek's comment,…
Clinical Trials and the Data Diversity Problem
By Liz Beatty
Liz Beatty is chief strategy officer for Inato of Guilford, CT.
Minority and marginalized communities have historically been underrepresented in dozens of private sectors globally. This includes pharma, where there exists a steep chasm that drug makers have yet to bridge concerning research and trials – a chasm that directly impacts the quality, quantity, and diversity of data that determine the efficacy of a drug and its applicability to broad patient populations.
One needs to look no further than a retrospective review of 302 drug submissions to the FDA to find evidence of data problems. That review found that nearly 16% of submissions had insufficient data to determine safe dosages, more than 11% had inconsistent results between study sites, and about 13% failed to demonstrate statistically significant benefits. These findings suggest data limitations, including diversity, influence the FDA’s rejection of five out of six submissions.
The longer the trend of incomplete data exists, the harder it becomes to address. The feelings of mistrust, resentment, and disenfranchisement only get more complex to overcome with time. While there has been a concerted push by the pharmaceutical industry to improve the situation over the past decade, it’s not moving fast enough toward a solution.
The time has come for technologies to step in and innovate solutions for this situation once and for all.
Significant progress has been made in the public sector, particularly among studies funded by the National Institutes of Health (NIH) and the National Cancer Institute (NCI). However, the same cannot be said for industry-funded studies. For example, NIH requires grant applicants to include plans for recruiting women and members of minority groups, while the FDA released guidance focused on expanding eligibility criteria for such trials and discouraging unnecessary patient exclusions, as well as boosting the recruitment process in order to attract diverse patients. NCI also reported a 14% increase in minority participation among clinical trials it has funded over the past decade.
Among private-sector trials, however, little progress has been made with regards to inclusivity and diversity. The FDA reports that 75% of enrollees in trials for drugs it had recently approved were white, while just 8% were black and 6% were Asian. An NCI-funded study found that 9% of those participating in its SWOG Cancer Research Network trials were black, compared to fewer than 3% in trials sponsored by pharmaceutical companies.
Nor is the diversity problem limited to race. Under-representation also extends to gender and even disease. For example, just 8% of cancer patients enroll in cancer trials, and less than 2% of cardiovascular disease trials reported any female sex-specific cardiovascular risk factors.
Historically, one barrier has been a lack of medical facilities with the capacity to host clinical trials in underserved areas. One NCI study found that 75% of patients don’t participate in trials simply because there are none in their area. Additional barriers for underserved patient populations included distrust of clinical trials, insufficient information about the participation process, limited time and/or resources, and lack of awareness.
The resulting lack of diversity impacts sponsors and clinical trials in three key areas:
- Accurate, robust data. The scientific method is null and void when data sets are incomplete. Yet a multitude of clinical trials continue to enroll smaller, homogeneous groups of patients who predominantly reside within a short travel distance of major trial sites. This should ring alarm bells for any drug maker seeking the efficacy and side effects of the medications they intend to bring to market. Incomplete data is a risk to the patient and treating physician, and it’s a financial and reputational risk to the business.
- Trial efficiency. Including broader demographic and geographic groups can accelerate trial speed and boost efficacy, while testing on a narrow participant group can result in unanticipated results after approval. Additionally, pharma companies that run fully representative trials are likely to experience greater success in reaching FDA approval for their medications.
- Financial incentives. Greater patient participation enables speedier trials and reduces costs, while a wider prospective candidate pool can reduce recruiting time, which drives down opportunity costs associated with delays that can run from $600,000 to $8 million per day. Further, better detection of side effects that might otherwise be missed when enrollment is limited — resulting in an incomplete picture of the treatment’s actual impact on the broader population – can prevent post-approval FDA black box designations and millions in lost revenues.
Increasing diversity and minority recruitment requires more than simply making a greater number of trials available in underserved areas; it also involves increasing engagement by partnering with community sites, using digital tools to enhance accessibility, and employing a diverse staff to better translate trial information to broader patient populations, all of which can be accomplished by embracing community-based research centers.
Powered by advances in remote patient monitoring and telemedicine technologies, decentralized trials and distributed testing eliminate physical and geographic boundaries. When these technologies are combined with cloud-based marketplaces to connect trial sponsors with underutilized community sites and provide those smaller sites with additional enrollment support, the patient pool expands significantly. A larger patient pool allows investigators to recruit a more diverse and representative patient population, improving data quality, avoiding delays, lowering costs, and accelerating FDA submission and approval.
The benefits of trial diversity are apparent. And while steps have been taken in the right direction, there is always more to be done. The benefits of doing so, however, are clear. Prioritizing community sites and recruiting for and implementing equitable, inclusive clinical trials can have a significant impact from both a scientific and ameliorative standpoint.