Precision medicine is an evolving field focused on bringing more precisely targeted medicine to more precisely targeted patients. The goal is to identify specific subgroups of patients, and match them to a customized treatment that potentially could be transformative. Advances in genetic analysis, the use of AI to analyze patient data, and new drug discovery techniques are making precision treatments much more possible to develop and deliver.

But there’s a huge hurdle between research lab and pharmacy shelf: Clinical trials for precision medicines require precisely targeted participants — patients who match the treatment’s target profile.

The existing clinical trial system was painstakingly developed to try to ensure that drugs work on real people in the real world, without unexpected side effects. But for several reasons, the system is unintentionally skewed in terms of the patients who actually participate. Trials tend to be held at a limited set of medical sites. The principal investigators tend to be a limited set of doctors affiliated with these sites. And patients tend to be a limited set of respondents who learn about the trials and will take the time to participate.

Brendan O’Neill, Senior Director of Patient Recruitment at Pfizer, says, “It used to be that if you had an oncology compound, you were looking for patients with breast cancer. Now, it’s so targeted that not only do you need patients with a specific type of breast cancer, you need to have a specific gene mutation that needs to be measured, analyzed, archived, and ready to go before you even talk to an investigator. It makes identifying the right patients and conducting clinical trials that much harder.” A gene mutation is an area of one’s DNA code that is different from that of most people.

“In addition,” he says, “the FDA has been critical of a lot of the registration submissions over the past ten to fifteen years due to the lack of diversity in trial participants. And rightly so. It’s an unintended consequence  of the way companies select investigator sites and recruit patients. We need to enroll a more diverse patient population in our clinical trials — not only to demonstrate safety and efficacy, but because sometimes the drug works differently in different populations.”

To test precision medicines on the people for whom they are intended, the pharmaceutical industry is taking several steps to recruit patients from outside the usual pool.

Sometimes it’s as simple as getting the word to potential participants. The National Institutes of Health’s ClinicalTrials.gov website doesn’t actively recruit patients. “Person-to-person online marketing is one promising potential solution,” O’Neill says. “Patients do research online now, so let’s meet them there with information,” which includes proactively suggesting trial participation rather than simply listing available trials.

Identifying target patients is also getting easier. Some patients have already had relevant parts of their DNA sequenced. So long as strict privacy guidelines are followed, these patients can be told if there’s a trial for which they are one of the rare candidates, and if they choose to participate they can.

The industry is also finding ways to make participating in a trial easier. The traditional procedure relied on repeated visits for patients to take tests and receive their next round of study drug. Today, drugs can sometimes be shipped to patients or their treating physicians. Measurements may be taken at their home or office, using inexpensive equipment or even wearable devices.

More fundamentally, as precision medicine evolves to provide more effective treatment for specific groups of patients, the pharmaceutical industry hopes to educate patients and their doctors that participating in a trial is a worthwhile effort. 

“Many physicians don’t see trials as a treatment option, but as a last resort,” O’Neill says. “Doctors may not feel it is worth suggesting a trial that may not help. They may even worry that they’ll lose the patient to another practice.” 

O’Neill says drug makers hope to get doctors and patients to think of trials not as a last resort, but as another option. To that end, Pfizer and other companies foster online communities focused on clinical trial participants. O’Neill mentions one network of trial participants who had been vaccinated against HPV (human papillomavirus, which can cause cervical cancer in women). This young, mobile group from all across the globe stayed in touch online for years, providing emotional support for one another and helpful information for researchers.

Today, a concerned woman searching online  for information on her diagnosis of breast cancer will see results or even ads from sites that list clinical trials seeking participants. Trial descriptions and criteria are laid out in more patient-friendly language. Consent forms may be less intimidating. And she can find others who share her situation.

As medicine becomes more precisely targeted to specific groups of patients, one participant summed up the importance of her role this way:  “Without us, there can’t be any research. And without research, there can’t be any new methods, there can’t be any new medicines, there can’t be any cures.”