How Real World Data, Decentralized Trials, and Diversity Will Put the Patient First

By: Matt Walz – CEO – Trialbee

It has been nearly a year since the U.S. government introduced Operation Warp Speed (OWS). The public-private partnership has surely been imperfect during the quest to develop, manufacture, and distribute COVID-19 vaccines. But most agree we’ll long be pointing to this moment as a crucial one in the battle against the pandemic as well as a pivotal moment for the entire pharmaceutical industry. 

It is nothing short of amazing that such an unprecedented and urgent need has been met with the mass production of multiple vaccines and different types of vaccine technology.

A General Accountability Office report on OWS from February highlighted the fact that pharmaceutical companies adapted traditional practices to deal with the compressed timeline of vaccine development. 

Vaccine companies relied on data from other previously developed vaccines using the same platforms or conducted animal studies simultaneously with clinical trials. Government agencies rallied to assist participating companies with limited manufacturing capacity and disruptions to manufacturing supply chains. There was also greater prioritization of vaccine production supplies from manufacturing contractors.

What’s more is the general public has gained a front-row seat for one of the industry’s biggest successes, seeing in real-time its commitment, innovation, and potential in improving health outcomes. No amount of advertising could convey the importance of drug development better than the race for COVID-19 vaccines. More specifically, the value of and need for optimizing clinical trial operations and removing bottlenecks has been brought to the forefront because of the pandemic-related delay of hundreds of clinical trials. 

This represents an important opportunity for the pharmaceutical industry. With momentum from successful COVID-19 vaccines, companies are racing to speed up the clinical trial process for other drugs. This means developing new access points for patients, adopting AI to compress screening requirements, and expanding diversity as part of a broader effort to more effectively communicate participation benefits.

One theme that is emerging from the pandemic is an increasing appetite for developing direct relationships with patients into the pharma business model. This will fundamentally drive change across the clinical trial patient engagement spectrum. Outcome-based incentives will further draw pharma’s interest as the industry aims to play a role in telehealth’s pandemic-induced acceleration. Look no further than Amazon’s entry into retail pharmacy - its customer-centric approach will surely shape coming opportunities for cultivating long-term, meaningful relationships with patients that result in improved health outcomes 

OWS gave new hope for our abilities to speed vaccine development, while also putting renewed focus on the usual bottlenecks in the clinical trial framework. Here’s a look at how Real-World Data (RWD), decentralized clinical trials, and diversity are expected to play a significant role in shaping clinical trial patient engagement in the coming year.

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Everyone Is Strategizing About Real-World Data and Real-World Evidence

Last June, the FDA placed RWD squarely in the spotlight when it announced it was participating in the COVID-19 Diagnostics Evidence Accelerator, a project using RWD to help inform the agency’s overall response, drive more collaboration, and achieve faster innovation. It was truly a shining example of how broadening RWD availability to more communities of data holders and researchers can accelerate learning cycles, advance discovery, and pinpoint treatments.

Ultimately this points to better patient outcomes because traditional drug development programs can utilize RWD to shrink timelines and clinical trial costs while increasing the likelihood of success.

What’s more, the accelerator’s success signals the transition from evidence-based discovery to the era of big data. RWD simply enables improved protocol design and site selection strategy, which results in improved patient enrollment and a better chance of improving patient outcomes primarily through the real-world evidence (RWE) it produces. 

More than 80% of organizations surveyed in a Deloitte report said they were creating strategic partnerships to access new RWD sources. Expanded access to RWD in China, Germany, and Japan is especially a priority given their RWE strategies beyond the U.S.

These RWE strategies are surely a big driver of the overall trends. Deloitte found the areas of RWE’s greatest impact center on use cases with a long history of leveraging RWE, including understanding the burden of disease, monitoring patient safety, and comparative effectiveness research. Deloitte calls out how these applications are decades old and include well-defined methods and data sources that have been vital to crafting the evidence story for treatments. The study points to RWE’s future use (94% surveyed believe RWE will be important or very important to their organization) for more nuanced use cases like supporting regulatory submissions, building synthetic control arms, and informing the design of value-based contracts. 

RWE enables the optimization of clinical study design by using collections of pragmatic studies to determine trial feasibilities and initiation. RWE is challenged by data access, quality, and governance, all of which can threaten trial validity, but when those factors are favorable, RWE can be powerful, especially when coupled with real-time analytics.

In early discovery and Phase I, RWE prioritizes efforts on unmet medical needs, illness history and burden, and risk factors for disease prevention, captured in real-world provider settings. In fact, one recent study based clinical trial simulations on Randomized Controlled Trials (RCTs) and Real-World Evidence to demonstrate that sample sizes for phase III studies could be reduced by 40%. In phase IV, RWE can help determine additional safety signals or rare adverse effects.

Digital advertising will be even more useful and effective as clinical trials aim to expand their pools of potential participants by using multiple sources of patients from large RWD repositories. This additional focus and availability of RWD will drive more study-ready patients to clinical trials and keep and greatly improve ROI of digital advertising spend.

Decentralized Clinical Trials will have Profound Impact on Patient Engagement

The pandemic’s impact on clinical trials cannot be overstated. Concerns over virus spread, staffing levels, and supply chain issues have created a backlog of 1,000 clinical trials placed on hold since the pandemic’s start.

Since December, some 100 life sciences and healthcare organizations joined forces to form the Decentralized Trials and Research Alliance, which aims to unite stakeholders across the industry to promote policies, research practices, and technology innovation for decentralized clinical trials. It’s exactly the kind of effort needed to help more organizations and patients realize the many benefits of decentralized trials.

Prior to COVID-19, adoption of decentralized trials was slow due to industry culture and a lack of collaboration among stakeholders. The pandemic quickly changed that, with 76% of companies surveyed by Pharma Intelligence reporting increased use of remote trials, and more than 90% expect those increases to be maintained going forward.

There are many benefits to conducting trials remotely. They allow for a far more patient-centric approach by reducing the amount of clinic visits required of patients and caregivers, improving access, convenience, retention, and outcomes.

Decentralized trials provide opportunities for using a range of technologies and locations that create more participation options. According to the Pharma Intelligence survey, mobile technologies like eConsent and EHRs (77%), wearables (60%) and in-home devices (54%) are the most used in current decentralized trials. Reliability (49%) and ease of use (29%) were the most important factors in deciding on decentralized trial technologies. 

Not surprisingly, collaboration is driving change here as well. The Open Wearables Initiative was founded in late 2019 to promote use of bio-metrics-driven wearables and other remote trial tech. In May 2020 the group began indexing open data sets and benchmarking algorithms.

Patient Convenience and Engagement are Incredibly Critical Parts of Clinical Trial Success

As the regulatory environment continues to warm for decentralized trials and companies mature their data protection and privacy and technology functionality, it’s not hard to imagine how they could become the new standard operating procedure for modern drug development.

As decentralized trials increase, and data analysis needs increase, it is expected that AI will play an increasingly important role in efficiently processing data.

Long Overdue, Focus on Diversity Will Increase Access, Positive Outcomes

Diversity in clinical trials has long been an area needing drastic improvement. In the U.S. between 2007-2017, African Americans and Hispanics/Latinx were underrepresented and Asian-Americans were overrepresented compared to their population in pivotal trials fornew drug applications.

When patients are unable to access clinical trials, they lose the opportunity to receive the newest potential treatment for their illness. This further enforces health disparities for groups of people who are most likely left on the outside looking in. 

There have been a number of obstacles in recent history for increased diversity in clinical trials. Most important, there is a wholly justifiable mistrust of clinical trials among minority populations due to trials conducted on prison inmates or the Tuskegee Institute syphilis study. Other obstacles include a lack of information and logistical challenges including time and out-of-pocket expenses.

It is clear that both the global pandemic and demonstrations for social justice across the U.S. last spring and summer have reinvigorated attention and efforts on the need for increased diversity in clinical trials. The industry produced two major commitments aimed at making clinical trials more equitable. 

In November, the FDA helped diversity take a full step forward by introducing new guidelines for making clinical trials more equitable. The guidance broadens eligibility criteria for clinical trials in an effort to increase participation of underrepresented groups. The FDA document, titled Enhancing the Diversity of Clinical Trial Populations — Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry, is intended to improve trial recruitment so enrolled trial participants will better reflect the population most likely to use the drug.

The document aims to improve clinical trial diversity by focusing on logistical and other participant-related factors that might limit participation, high-level considerations related to inclusion of important groups, and the broadening of eligibility criteria for clinical trials and improving enrollment and retention of participants with rare diseases.

Last month, PhRMA demonstrated across the industry its commitment to enhancing diversity in clinical trial participation by putting into effect a lengthy set of principles, the first-ever industry-wide directives on clinical trial diversity. The focus here is more on the systemic issues that cause black and brown communities to participate less in clinical trials, including a strong push to build trust and restore confidence. 

For example, one recommendation is for biopharmaceutical companies to go beyond voluntary adoption of principles and make public its efforts and materials to increase underrepresented populations’ participation in clinical trials and to broaden eligibility criteria. Another section advises how to take into account the needs of diverse populations in clinical trial design with a patient-centered approach, including an adaptive clinical trial design that allows for pre-specified trial design changes during the trial when data becomes available (including altering the trial population).

Those operating remote trials can drive change for underrepresented or hard-to-reach populations by linking clinical data and RWD to bring down access barriers, and linking trial data to RWD can give researchers the data they seek and at the same time reduce the need for regular in-person site visits. RWD can also be instrumental in uncovering inherent biases in previous research and ensuring they do not creep back to the surface of current research. This will also improve the overall quality of studies and a therapy’s benefit-risk profile in later stage drug development.

While the past year has been a blur for many of us, it has also been transformational in a way that offers hope after much uncertainty and struggle. The lessons learned from the pandemic will likely underscore one of the largest shifts pharma has seen, and it is clear that clinical trials provide ample opportunity for innovation and improving patients’ lives.

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