Pandemic Sparks Promising Future for Clinical Trial Speed and Flexibility
Like wartime medicine, the pandemic inspires and challenges medical innovation.
The silver lining of the pandemic is the reinvigorated sense of urgency breaking down
cumbersome and expensive barriers to the FDA’s phased approval process. While the research- lab-to-patient-arm trials for the highly successful COVID-19 vaccines famously moved the traditional pace to warp speed, other critical and life-altering medicines, devices and therapies also broke through during this period.
To be clear, the current surge of medical innovation through clinical trials in immunology, cardiology, multiple sclerosis, oncology and more, is not the result of a rush-to-market panic. All necessary and appropriate testing protocols to ensure quality are still being achieved, but at a more expedient pace in many cases. This is the good news.
“In times of crisis, we can accelerate the development and review process,” explains Andrew Badley, MD, infectious disease specialist, Mayo Clinic. “Throughout the pandemic, many of these steps were accelerated. No steps were skipped. It was just the amount of effort that went into the development and the review that was increased.”
Of course, there is also some not-so-good news about clinical trials today. During the pandemic, the number of new studies launched dropped by as much as 57 percent, according to Trials Journal, and the overall completion rate of clinical trials decreased between 13 and 23 percent globally. Shifting research priorities (11 percent of studies shifted to pandemic-related trials in 2020) and initial challenges in recruiting and following up with volunteer patients during the global lockdown contributed to this decrease. Often, a clinical trial is tethered to an academic medical center with participants centered in one geographical area, limiting volunteer pools and access.
However, the future speed and flexibility of clinical trial protocols is very promising, reflecting the long-term viability of alterations made to the fabric of patient care and research during the pandemic. Some of the new flexibility that is being assessed and considered for permanent use includes:
- Telemedicine. While telemedicine has been available for years, the lockdown most certainly fast-tracked adoption among researchers, regulators, physicians and patients. Clinical trial investigators can now use telemedicine for many patient check-ins, saving time and broadening the geography of volunteer pools. Dr. Ray Dorsey, a neurologist at the University of Rochester, noted in a recent article that his virtual clinical study of
genetic predisposition to Parkinson’s disease moved forward more quickly amid the pandemic, spurred by a rising number of online enrollments.
- Delivery. Like specialty pharmacies and physicians during lockdown, clinical trial investigators are now allowed to deliver trial medicine to volunteers.
- Remote Access. Volunteer participants are able to use online platforms for completing consent forms, and they can often visit their local physician for basic assessments. Some
trials also require less frequent check-ins, which can be important in recruiting volunteer patients. The growing number of smartphone-enabled applications that provide measurement of critical physiologic variables means patients don’t need to continually return to the hospital or clinic for tests during the trial. For instance, an entirely remote study testing vitamin D for treating COVID-19 and preventing transmission is being conducted by Brigham and Women’s Hospital; participants obtain their own blood samples with a finger prick, dot the blood drop onto filter paper supplied to them and mail it back.
The Clinical Trial Explainer
In the United States, the Food and Drug Administration (FDA) directs and approves all prescribed medicines, diets, diagnostics, devices and therapies. Clinical trials are the part of research that determines whether a medical intervention should be moved, or “translated,” from the lab to routine patient care. At each phase along the way, the team must answer different questions about safety, efficacy (whether the intervention works as intended) and whether there might be better options available. The current clinical trial journey to FDA approval, shown below, can take years, a mountain of paperwork and millions of dollars – there is room for improvement.
- Preclinical phase establishes the pharmacological profile and determines toxicity on at least two animal species.
- Phase I, a short study of 20 to 80 healthy people to determine safe treatment and dosing.
- Phase II, a larger-scale study of targeted patients to determine treatment effectiveness and identify side effects; can take months to years to complete.
- Phase III compares the trial intervention with existing therapies; requires several years of multiple data collection check-ins and comparisons. About 1 in 15 won’t make it past phase III.
- FDA Review and Approval
- Phase IV follows patients after therapy approval to ensure the intervention is working and prove the long-term benefits outweigh any risks or side effects.