If you’re like most Americans, you’ve probably never participated in a clinical trial, or even thought about it. Fewer than 5% have. Maybe you remember seeing that ad on the subway train one time. They offered a sizable payment, but it was for a treatment you didn’t need for a condition you didn’t have. Just the other day, you found out there are clinical trials even for healthy adults and folks with not so rare conditions. You’d like to sign up, so how would you even go about seeking relevant trials?
That’s the same question researchers have been asking themselves. In the past, recruitment methods were limited to crude forms of advertising. Until recently, the biggest bottleneck for research has always been simply finding and retaining enough participants, especially those that meet the right criteria. The average drop out rate is 30% and a fifth of trials are forced to shut down prematurely due to lack of participation. Many take three times as long to complete as planned due to struggles with compliance. Compounding these issues is the need to maintain a cross-section of participants across a broad geographical area. Expenses quickly get out of hand. No wonder it takes over 10 years and more than a couple of billion dollars to introduce a new drug.
Virtual clinical trials present a way forward. Virtual clinical trials, like conventional clinical trials, produce observational data and contribute to the body of Real-World Evidence (RWE). The difference is that virtual clinical trials leverage the power of modern communication technologies. If data can be collected and measured at home and reported through an app on a phone or smart device, the researcher no longer needs to rent and manage research sites, and the patient no longer needs to make appointments and drive to them, perhaps a dozen times or more. According to the Center for Information and Study on Clinical Research Participation, all five of the top five most commonly reported reasons for quitting a clinical trial have to do with the inconvenience of having to physically go to the research site. Virtual clinical trials bring the trial to you instead of you having to drive hours to get to a research site, find a parking spot, and then wait your turn in the waiting room reading irrelevant, dogeared, outdated magazines to see the clinician.
Of more than a hundred thousand current registered clinical trials, very few are virtual. It’s still a new concept. Traditional researchers set in their ways are slow to adopt, and technologies like social media, apps, and wearable devices, often from small startups, have only just begun to open the door. Among many early successes are trials which have used a smartphone’s gyroscopes to track and measure arthritis severity, and a smartphone’s camera to analyze progress from a bacteria-based topical acne treatment. There are still challenges. User experience must be optimized to be simple and streamlined to reach and maximize usability for the elderly, the unwell, and populations with low technical literacy. Many wearable devices generate enormous amounts of data from round-the-clock sensors. Great care is needed to ensure integrity, accuracy and reliability, especially in the virtual environment. Technology considerations also play a big role in ensuring privacy and verifying provenance of data collected from wearables and home medical equipment. Trust is paramount. Health Wizz and others are embracing blockchain cryptography for this reason.
Your personal health records are a valuable commodity, and increasingly include Patient Reported Outcomes (PRO). PRO refers to any self-reported outcomes such as adverse events, generally that which is experienced by the patient rather than observed by a doctor or a clinician. A common method for obtaining PROs is through questionnaire. Apps for health tracking and reporting enable PROs to be solicited easily. No need for another doctor appointment and more form filing. A patient can report on the spot, and a highly personalized data set can be kept current. As demand for more personalized treatment rises, these qualities add a lot of value for researchers. With proper checks on privacy, PROs can be shared, with patient permission, or perhaps traded at patient’s request, in a dynamic marketplace, either anonymously or with identity intact. Once a major hurdle, gathering of data becomes a trivial part of the equation.
But what is the purpose of all this data? For some time, industry professionals complained of data overabundance. There are many large-scale epidemiological studies to analyze, and the challenge has been to tease out useful information. Big studies make the rounds in media, with headlines touting ever smaller relative risk associations. Virtual clinical trials stand to improve upon this, paradoxically, with more data, data on demand. Easier access to patients gives researchers the power to take a deeper, more individualized look, to anachronistically return to a form of patient centered medicine, rather than population centered medicine. Doctors no longer make house calls, but everyone can have medical technology in their pocket. It’s the ultimate expansion of anecdotal evidence into a true gestalt. So many limitations on data are gone. Reaching patients in distant geographical regions or those who were of limited mobility or otherwise impractical as research subjects means greater quantity and quality of data.
Evidence obtained through unbiased and systematic analysis of this type of data, called Real World Evidence (RWE), is a hot topic at professional medical conferences. If you’ve ever wondered, why are there so many different types of diets and why do they seem to have so many different effects on different people? Or, how do we know if a trial ran long enough to assess possible long-term effects, for vulnerable populations? RWE improves decision making on an individual basis and brings us a step closer to answering these sorts of questions. RWE is a new horizon. It comes into play when a population-based approach doesn’t have the resolution necessary to account for patient diversity.
Much has changed in the past 25 years – mobile phones have replaced land lines, the encyclopedia has been replaced by Wikipedia, and we have an app for everything. It is time for the clinical trial industry to rethink how clinical trials are designed and conducted. Of course, ensuring patient care and effective study oversight must remain primary priorities with a virtual study. The patient has never been more important, nor their input more valuable. Product and treatment development depend on it. PROs play a key role in forming the basis of RWE, and a more individualized approach to better reflect patients’ needs and values. Prospective participants from all demographics have armed themselves with smartphones. They are willing to participate, they are reachable, and they are keen to interact and further science. There is no reason to leave anyone behind. The key is that researchers be open to the possibility of using a virtual approach for clinical trials prior to finalizing the protocol. It may be that with a few minor alterations to a protocol, a study can be managed virtually, rather than via sites. If you think you are the one to lead this revolution click here.