Speeding Clinical Development With Real-World Evidence
The development of new medicines is expensive, time-consuming, and often plagued with risk.
According to the industry trade group PhRMA, it takes 10 to 15 years and costs $2.6 billion to take a new compound through clinical development to regulatory approval. And with a high failure rate, only a few of those compounds will ever make it to patients. Of all the potential medicines that enter clinical trials, only about 12% are eventually approved by the U.S. Food & Drug Administration.
One of the biggest bottlenecks in the development of new medicines is patient recruitment for large-scale clinical trials. The failure to recruit and retain clinical trial participants efficiently can slow development times and add to overall costs.
Access to real-world evidence, however, offers pharmaceutical companies an opportunity to overcome this hurdle. Data can provide a clear understanding of where real patients with a specific disease type are actually living, the physicians and hospitals they are visiting, and where they are in their diagnosis or treatment journey. With this information, clinical development teams can select study sites with access to a large pool of potential study participants and target recruits at the right point in their patient journey, accelerating the recruitment phase. By carefully and intentionally setting up the recruitment process, researchers have a better chance of holding on to patients throughout the trial as well.
Dr. Aswin Chandrakantan, Chief Medical Officer at Komodo, recently shared his insights into how real-world evidence has the power to radically transform the clinical trial process. The following is an edited transcript of that exchange:
Challenges Facing the Industry
Aswin Chandrakantan: There are tremendous challenges and costs for the industry around study feasibility and study site selection. Traditionally, the tools used for patient recruitment often lack a complete view of eligible populations. They rely on individual recruiters and networks of professionals to help with recruitment. Because of this, they don’t always capture every potential participant.
In fact, 20–40 percent of the eligible patients already at contracted sites are never even screened for participation. In addition, many eligible patients are in remote pockets of the U.S. and hard to reach – even though they’d consider commuting for a chance to participate in a clinical trial.
But through patient-level data, clinical study teams can reach and recruit more patients to optimize a trial.
There are three ways real-world evidence can accelerate clinical trials;
1. Identifying the Real Disease Burden
The first concerns feasibility and site selection. Traditionally, biotech and pharmaceutical companies partner with providers and institutions they already know. However, those are often the same providers and institutions that every life sciences company in that disease area or with that indication routinely contracts.
This can make recruitment challenging. For example, when a study organizer reaches out to a well-known provider or institution with a selection survey, they might be told that the site has “15 patients a week,” making it an attractive study center. However, when the study team later tries to recruit those 15 patients, they might find that none is eligible for their clinical study because they’ve already been tapped by competitors.
Real-world evidence, however, can help the study team identify the real disease burden at different potential study sites – and which locations may already be over-tapped.
2. Targeting Recruitment
The second way real-world evidence can accelerate clinical trials is by helping clinical teams identify when to reach out to recruit patients for a trial by accessing and analyzing real-time data.
Komodo streams 15 million patient encounters each day into its Healthcare Map™. By tapping into this data, we’re able to identify specific patient cohorts of individuals who might consider enrolling in a clinical trial because they are at a stage in their journey in which they’re screening-eligible.
At this point in time, study teams can reach out to the relevant providers about the compound in development, communicating information about the mechanism of action as well as information about the trial itself.
3. The “Crown Jewel”
The “crown jewel” of real-world data and clinical trials is the external control arm and long-term outcomes studies.
What's powerful about external control arms and long-term outcome studies is that you can reduce the number of participants in a clinical trial by hundreds or thousands of patients. By studying past clinical studies, teams can make informed choices about how many patients are needed in a clinical trial and whether that data will stand up to regulatory scrutiny. In fact, by creating a control arm with retrospective data, clinical studies may only need a single study arm.
The next frontier would be to use real-world data and not even run a trial. This is especially relevant when studying a label expansion for an already-approved product.
These approaches promise to save billions of dollars and years of research to bring better therapeutics to patients more efficiently and faster.