Patient Engagement

Here’s what clinical trial utopia may look like

Between 2006 and 2016, the total number of registered clinical trials increased by 650 percent. To successfully recruit and run all of those studies today, something needs to change.

In theory, there shouldn’t be a problem. Patients by and large want to participate in clinical trials – for some their lives depend on it – and drug developers readily need participants to complete their studies on time.

Why then, is it so darn hard to recruit for many trials?It’s a complex problem, which not surprisingly has many in the industry looking for a technology-based solution.

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A Deep-dive Into Specialty Pharma

A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.

“The healthcare industry is trying to adopt a whole host of emerging technologies to accelerate and streamline its slow and expensive clinical trial processes,” said Debarati Sengupta, a senior research analyst at Frost & Sullivan, via email.

One such company is Science 37 whose aim is to build a truly patient-centric trial that optimizes recruitment and retention by leveraging mobile technology. If the Playa Vista, California-based startup succeeds, it may just be a win-win for patients and study sponsors and medicine at large.

“Right now it feels like the conversation is a lot about ‘when there’s no other option available this is where you go’ and it’s not about ‘this is the future of how we can make the world better for everyone,'” said Belinda Tan, cofounder and chief medical officer.

Open access 

To do that, one must look at the supply-demand problem of conventional clinical trials. On the demand side, the total number of registered studies jumped from approximately 36,000 in 2006 to 235,000 in 2016 — a 650 percent increase in a decade, per

But supply remains weighted down by traditional barriers to access. Most Americans are diagnosed and treated outside the major cancer centers where the trials sites are typically located. This means eligible patients and their physicians may not be aware of the study, it may be too far away, or they may not have the healthcare coverage needed to be treated at the trial site.

Science 37 groups these challenges into three buckets; geography, awareness, and trust. To tackle them, the company has built a direct-to-consumer clinical trial model, said Noah Craft, cofounder and CEO, and Tan in an interview at the company’s new headquarters.

It starts with that really buzzy word in healthcare circles these days: a patient-centric mindset. In practice, it means creating a clinical trial environment requiring no travel to a distant trial site, no hospitals, and …. wait for it, no waiting. This almost sounds clinical trial utopia.

The study comes to participants, capturing their attention through patient communities, social media, and word of mouth. For those enrolled, the company’s telehealth platform, dubbed NORA, channels everything to the patient’s home. The service is rounded out with the help of local health practitioners who can stop by to give infusions or to record important metrics.

It’s easy to participate and anyone can apply (though obviously, the normal inclusion/exclusion criteria apply). This allows a wider pool of interested applicants to come forward without the need for a physician’s referral, including groups that are woefully underrepresented.

Minorities account for less than 10 percent of participants in cancer clinical trials, but Craft declares it’s higher in their pool.

“In ours, it’s between 30-40 percent,” he said. “That changes the demographics, which makes your conclusions more generalizable. So instead of just studying it in Caucasians, you’re studying it in the general American population.”

This is especially important given data points like these: African-Americans and Puerto Ricans, two populations disproportionately affected by asthma in the United States, have a poor response to some of the most common drugs to control the disease. That should have been unearthed and addressed in the trial phase.

Beyond ethnicity, other biases continue to skew trial access and results. When sites are restricted to the top academic medical centers, socioeconomic and geographic factors come into play.

As a virtual meta-site, Science 37 can overcome those limitations to fill trials faster — something that is a major benefit for study sponsors.

“We basically cast the net to a much, much bigger denominator of participants,” Craft explains, which drives the cost of the overall trial down.

In May 2015, Science 37 started recruiting for a large Genentech clinical trial studying a rare autoimmune disease. It was one of 70 trial sites worldwide, but with the so-called meta-site design it was able to canvass seven U.S. states. The others were run out of a single physical location. Other sites are still enrolling, but Craft said his team is leagues ahead.

“If you average it out across the other 70 sites, our pace of recruitment was about 20-30 times faster than the normal,” he said. “So that’s 2,000-3,000 percent increase in speed.”

Craft declares the company has “probably saved about 50 percent of the time of recruitment.” 

 In biopharma terms, that’s millions – possibly tens of millions – of dollars saved. 

“If you have a big multi-site trial open, big pharma companies can be burning $3-5 million a month just to be open, not doing anything,” he said. 

And if the drug is destined to fail, it’s best to fail fast. For drugs that do have market potential: 

“Every day a blockbuster is not on the market is $2.5 million. Every day,” Craft stressed. “So [sponsors] take time very seriously.”

Building something bigger

Community building is central to this effort to fill trials faster, Tan said. At Science 37, it’s done through a division known as Patient Affairs.

“Part of what they do is to help recruit participants into these trials,” she said. “But there’s another part they’re working on, which is this world of building patient engagement, and advocacy, and communities, and awareness.”

Science 37 has several thousand people that have opted to join its communities, according to Tan. Many signed up through Science 37’s various landing pages, expressing a long term interest in participation for when a suitable trial rolls around.

“Especially with the shifting environment of healthcare, patients are going out to look for answers themselves,” Tan noted.

Patient Affairs keeps the engagement alive with information and education about the startup’s mode of operating. Further, it sends out surveys to gauge what kind of trials the individuals would be interested in participating in and what symptoms bothers them most.

“It’s also very powerful for us because when we’re talking to sponsors about studying certain conditions, we can actually go back into this community and find out what people are interested in, what they’re affected by, and if they have family members that might have a particular ailment,”  she said.

While it mostly does work with smaller companies for one-off trials, Craft said that the team has also signed five major pharma partners with long-term project goals.

“So they’re betting on long arc of work, like 3-5 year enterprise relationships with 5-10 big trials each,” Craft explained. “We asked them to make that commitment so we have a stable partnership and we can take more risks together.”

If the company grows and gains bigger contracts, those communities may be crucial for recruiting patients in challenging and/or competitive fields (think Alzheimer’s, oncology, or non-alcoholic fatty liver disease).

Staying ahead of the competition

To make that “if” a “when” Jay Trepanier has stepped in as Science 37’s chief operating officer. Trepanier’s mission is to install the kinds of systems and structure that will allow the company to scale. It’s a tricky role to play: He’s the hall monitor for a class of kids that have been successfully freestyling for several years. He is also refreshingly honest. 

“They’ve had success on a couple of trials, but now with the growth and the buzz in the company they need discipline, best practices, structure,” Trepanier said, whose aim is to build a system that can handle 15-20 trials at any one time, while also angling towards greater specialization. “Everyone’s drinking this Kool Aid and the Kool Aid’s here for sure, but it’s not all going to be this skyrocket success,” he said. You’re going to get some bumps in the road.”

To relieve some of the pressure and perhaps lower the risk, Trepanier recently convinced Craft to commercialize the NORA platform. Strategically, there may be several benefits. With a software-as-a-service (SaaS) product it could generate a revenue stream that’s separate from the trial management.

It could also help the company get an edge on any future competition. Other young CROs, such as Clinpal in the United Kingdom, are building their own versions of the virtual trial design. Many other sponsors and CROs are experimenting with different kinds of technology.

Sengupta, the Frost & Sullivan analyst, believes the industry is looking to integrate many digital solutions, “including cloud technology, artificial intelligence, big data, predictive and prescriptive analytics, Internet of Medical Things, and mobile technologies,” she wrote.

The goal of lower cost, faster trials remains the same. It’s just how the company gets to the Promised Land.

Photo: Science 37