It was a routine IND meeting at the FDA. Just a few low-level agency staffers were expected to attend. Instead, some of the FDA's most prominent, senior scientific and enforcement leaders filled the space to standing room only. These are the sorts of headline-making officials who can strike abject dread into the hearts of all but the most seasoned industry professionals.
In this case, the FDA brass showed up to learn, not criticize. What held their interest was a genuinely new approach to patient recruitment. Mytrus, a San Francisco startup until recently in stealth mode, would later convince the agency that it would handle patient recruitment and find 600 patients with overactive bladders using only the web.
The company has developed a new way to find patients online. Crucially, it can verify patients' identities. Mytrus is using commercial databases—in concert with a series of biographical questions—to be completely certain a hypothetical 47-year-old patient who claims to be Maryellen Johnson of Hannibal, Missouri must be Maryellen, not her teenage son and his mischievous pals. Mytrus has also convinced the FDA that its web-based informed consent process is equivalent to what occurs in a face-to-face setting.
Like the weather, patient recruitment woes have been discussed for decades. But they are seldom attacked in novel ways. Mytrus believes it can address the most intractable, chronic, billion-dollar problem in clinical research. It expects that its projects will have fewer patient recruitment difficulties and less attrition for patients who do enroll.
In essence, Mytrus wants to tear up the current blueprint for finding patients, shred the scraps and burn them in a steel drum. So readers can put aside old-fashioned notions about every clinical trial needing large numbers of scientist-physicians in white coats. Or every patient being slowly examined and screened in a medical setting. Forget all that.
In a Mytrus project, patients will volunteer online. Some will come to the Mytrus website after typing "inflammatory bowel disease" into Facebook or Bing. Others will learn about a Mytrus trial from ads or editorial content on websites for patients with the same disease. Mytrus's social-media marketing sophistication is probably equal to its tools for verifying patients' identities.
Patients will answer screening questions about their health. Legitimate patients will sail through the process. Anyone trying to game the Mytrus system just to get medication will encounter ingenious obstacles.
Eligible research subjects may get a confirmatory phone call from a Mytrus study nurse. Once a patient's eligibility is determined, a study drug is sent by overnight courier. There's no need to travel to a doctor or a pharmacy, wait around, miss work, get stuck in traffic or be exposed to the time-sucking eternity of a physician waiting room.
Anthony Costello, chief operating officer at Mytrus, releases a good-natured sigh at the numerous hurdles the small, privately-backed firm has overcome in its quest to rewrite the script for patient recruitment. With a green light (and a shipping-related waiver) from the FDA, Mytrus was able to enlist a small, fledgling Manhattan firm called Pfizer as its first customer for a Phase IV trial. Two other real trials are under way, as are discussions with a variety of big pharma firms.
Costello says the Mytrus approach could work in 15 percent of clinical trials, or thousands of projects that amount to $8 billion in annual research spending. In his view, any phase of research or therapeutic area would be appropriate. The crux of the matter is that a Mytrus-ready protocol has no hospital- or clinic-based procedures. The approach is vaguely similar to that of CRN, which recruits for clinical trials by having nurses visit patients at home, and MediGuard, a Quintiles post-marketing database that offers personalized drug safety bulletins in exchange for detailed health information from its 2.5 million members. (A ClinPage article on MediGuard, formerly known as "iGuard," can be found here).
Costello is a mellow northern Californian who co-founded Nextrials, an electronic data capture (EDC) firm. But he realizes that the Mytrus conceptual framework will be radical for many in the industry. He's prepared to elevate his rhetoric to help industry insiders see that it's not necessary to continue digging the same dry well. "The model is obviously broken in many, many ways," says Costello.
Costello mocks the notion that the loss of the existing system of finding patients should be mourned. "This model has yielded us incredibly slow recruitment, incredibly inefficient recruitment, late data, shoddy data, queries that take months and months to resolve," he says.
"Patients have been pawns in the clinical research game for a long time," Costello says. He notes that they seldom know what happens to their data. "We are in the middle of a revolution of patients using the internet to do their own health care."
"Patients have been seen as numbers," he continues. "There is an opportunity for a company like Mytrus to find patients who want to move the drugs along and take the responsibility. What we can do to give back to those patients is make it clear we are concerned about safety. They own their data. They agree to share it for research purposes." Patients will receive summaries of the trial and can deposit such data in their own patient health record as they see fit.
What if a patient in the study develops a rash? Mytrus will serve as the clinical site, taking patients' calls. "We are the site in terms of offering a place the patient can call," Costello says. "That will all be done through us." A central institutional review board (IRB) will simplify the trial's progress as well.
The Mytrus approach is placeless. That will widen the geographical target territory for every condition the company is working on. The company can envision an end to tightly delimited circles around a particular physician, with the resulting puny numbers of potential patients in each territory.
So a series of funnels resulting in twelve patients (or a single patient or zero patients) need not be a fact of life in every clinical trial. Instead, the entire U.S. becomes the de facto recruitment region for every Mytrus project.
Mytrus does have physicians on its staff. The company is replicating the functions of both a boutique contract research organization (CRO) and a clinical trial technology firm. To a degree that will make some in the industry uncomfortable, Mytrus assumes its patients are adults of sound mind. It is not infantilizing research subjects. Costello and his colleagues are assuming research subjects have the capacity to make intelligent decisions and accept the terms of a new bargain.
The proposition comes down to approximately this: For patients willing to help report scientific data, we'll make it as easy as possible to participate in the research process, remove as many annoyances as we can, and ship you your medicine to get started. Depending on the study design, patients may even get a bit of money for their trouble, thanks to the rechargeable Greenphire debit card system ClinPage wrote about here.
"We're trying to appeal to patients on the grounds that they can be part of a new way of doing trials," says Costello. "They can take control of their own condition by trying to learn more about it." For readers who want technical jargon to describe what Mytrus is doing, the term might be "direct to patient" research or "direct to participant clinical setting."
Costello seems adamant on the point that the relationship between the pharmaceutical industry and the public can be rebuilt. All of the fines, lawsuits, investigations, scandals and consent degrees the drug industry has faced can be, well—perhaps not forgotten, but subordinated to the more immediate concerns of a suffering human being. That human being, Mytrus assumes, will have an urgent personal or social interest in pushing the frontier of medical knowledge. The industry's challenge is to make such participation as convenient as possible.
Part of the appeal of Mytrus's solution is a set of pleasantly clear computer screens. In visual terms, the system is light years beyond the tiny, indecipherable rows and numbers of most clinical trial systems that industry purchases today. Mytrus has built a consummately consumer-facing website with a 21 CFR Part 11 infrastructure behind the scenes.
The system adheres to all existing clinical trial regulations. It combines elements of an EDC system, a patient diary system, a drug safety reporting system, an electronic health record and a clinical trial management system. It's one database with an unusually attractive user interface.
Could patients falsify their experiences? Deliberately submit bad data? Costello says the firm will use algorithms to detect suspicious patterns.d9A2t49mkex
But he pushes back against the notion that the Mytrus approach might yield lower data quality than a conventional trial. Says Costello: "Fraud has been a huge problem on brick and mortar trials since the beginning of time. All of those things will continue to exist."
It all amounts to bringing the clinical trial into the internet age. In monitoring what Pfizer and Mytrus are doing, the FDA has offered succinct but gentle encouragement. "As part of our ongoing efforts in clinical trial modernization, the FDA is very interested in exploring the potential utility—both the benefits and the limitations—of direct to participant research and acknowledges that the internet may be a useful tool in reaching, educating and engaging patients who participate," says Jeff Ventura, a media official at FDA.d9A2t49mkex