According to at least one highly respected mind, if one does the same thing over and over again and expects different results, they are in fact insane. Now, with the cost to develop drugs skyrocketing and many of the underlying causal factors understood yet not addressed—after decades—we could extend this classical observation to the clinical trials process and its sponsors. And then ask the question: Are pharma, CROs, and research centers insane?
Afterall, with the cost of developing a new drug totaling $2.6 billion—and over two-thirds of this allocated to the clinical research process—the sponsors, their CROs, and research centers keep trying to develop drugs in the same way—over and over. And, within the clinical research process, patient recruitment was and has been the number one bottleneck contributing to countless clinical trials delays. These delays can result in costs of up to $8 million per day in lost future revenues to the sponsor paying for the research.
Making matters worse, when sponsors and sites actually find patients, 40% of the time they drop out—leading to more delays. This author has been involved with patient recruitment and clinical trials since the 1990s and many of the exact same problems that existed then still exist now. Moreover, new technologies aside, many of the processes are pretty much the same—as is the company politics and, of course, the egos. But could pharma actually be insane? Or are they merely victims operating in what is actually an insane system?
Technological Disruption can Help Pivot the Course
According to some bright minds serving the drug development sector, technology disruption (made possible by the introduction of telehealth/telemedicine platforms, powerful ubiquitous cloud computing, Big Data and smartphone and intelligent device revolution), infused with intelligent process change, will shake things up enough to pivot the clinical research agenda on a more sound course. After all, $2.6 billion for just one drug is a lot—too much actually. And of course, it is passed right on to the consumer, especially if they happen to reside in the U.S. where pricing can be outright astronomical.
The logic goes if patient recruitment, enrollment and retention is the biggest bottleneck—and primary cost generator—than there is a technological fix: the virtual clinical trial that is designed to keep patients at home. With the assumption that this could positively impact recruitment and enrollment rates while reducing dropouts, industry sponsors (pharma), CROs and research centers (sites) will increasingly embrace virtual clinical research based on remote models to conduct studies as recently discussed in “Virtual Clinical Trials: Perspectives in Dermatology” in Physicians Weekly.
What does Virtual Clinical Trials offer in Dermatology?
Now with enough mature technology in place (apps, monitoring devices, telehealth platforms, etc.) and inclusion of web platforms (portals for study start up, IRB review, etc.), studies can be designed, in many cases, to keep and treat the patient at home at nearly all stages of the clinical trial. In fact, this is the point of the Swiss publisher Karger: that the time is now for a paramount shift in the clinical trials paradigm away from the site and toward the patient’s home. The benefit: recruitment rates will go up, retention rates will improve, (e.g. lower drop out rates) while the vast majority of people that never consider participating in a clinical trial in the first place may actually start to consider doing so.
And at least with dermatological studies, the visual nature of dermatological conditions, not to mention the relative ease in evaluating skin diseases virtually—coupled with the fact that most skin diseases are not life threatening and rarely requiring complex examinations, suggest the virtual clinical trials represent an important direction for dermatological research. Moreover, the publisher points out in Physician’s Weekly that the dermatologist practice routine supports the move to virtual research as doctors are well used to making correct diagnoses based on photographs and patient symptomatology.
Impediments to Virtual Clinical Trials
Biopharmaceutical sponsors should investigate virtual clinical research models and determine where and when it makes sense to implement change to impact the growing cost, complexity, and risks associated with clinical trials. To not do so would be insane at this point. A number of challenges must be overcome of course, from regulatory and legal considerations to technology/device selection to process and data quality and governance considerations. An example on this latter point involves how a sponsor must ensure that the data collected fits into the ALCOA and other GcP frameworks. There are a thousand details to consider, and we don’t believe the virtual clinical trial is a panacea for the entire drug development system but certainly they should be considered as one model that is employed, when justified, to conduct studies that may have better patient participation rates.
Example of the New Model: Powered by Vendor Science37
AOBiome Therapeutics, a Cambridge, MA-based clinical-stage therapeutics company working on the science of Ammonia Oxidizing Bacteria (AOB) and human health, conducts primary research, collaborates with others on research, and develops applications to positively impact skin conditions and reduce systematic inflammation. FastCompany reported that the sponsor designed one of its studies to be 100% remote—a virtual clinical trial. This Phase II/III, 12-week study involved an experimental acne drug called B244. In the randomized, double-blind and placebo-controlled trial, the sponsor showed that the topical spray containing good bacteria was evidenced to be safe and effective in reducing the severity and number of acne lesions.
Virtual Clinical Trial Elements
Apparently, the entire study was conducted via the virtual clinical trial paradigm—made possible by the vendor called Science37 (TrialSite News has profiled them). For prospective patient screening, the sponsor screened 8,000 of them by employing Science37’s virtual clinical trial platform to support the site-less study (although technically there were still of course physician-investigators involved but the patients didn’t have to travel to a site.) According to the FastCompany piece by Barbara Mantel-Undark, the actual 372 participants that ultimately signed up for the study received the drug or placebo in the mail and used company-issued iPhones to take selfies of their acne. An iPhone app was utilized for transmission of the photos to the participating investigators for evaluation; patient to investigator or coordinator and/or investigator to sponsor communication and collaboration was achieved via video web conferencing technology.
This whole process differed from the typical clinical trial, reported Dawn Anderson, managing director in Deloitte Consulting LLPs life sciences practice. Ms. Anderson, advising clinical trials sponsors for over a decade, noted for FastCompany on the impact that such a virtual trial can have on patients where in the standard model the patient must often take time from work or family; spend hours in a car, representing “quite a burden of time and effort” while in many cases the patient can’t even participate as the research center is simply too far from the patent’s home.
Pharma sponsors, CROs, and research centers must change how clinical research is conducted today. With a $2.6 billion average spend for a successful drug, the costs are unsustainably high and are therefore passed down to consumers in a model that cannot be sustainable. New technologies and process disruption won’t necessarily fix underlying systemic problems in healthcare and research (which can often be driven by political, socio-economic and psycho-social factors and forces); however, where and when it makes sense, moving trials toward virtual, patient-centric models seems to make sense. To not do so, knowing the dynamics out there, appears pretty crazy.