Clinical trials have long relied on the assumption that patients need to be present at the hospital or clinical facility to ensure safety, integrity and compliance. But what if that weren’t the case?
Adversity is the mother of invention, so goes the saying; and so it turns out also in healthcare. The COVID-19 pandemic brought new challenges with delivering medicines and treatments to patients on many levels, including the continuation of the ongoing clinical trials taking place across the globe. As lockdowns came into effect, patients and vulnerable populations were discouraged from traveling or visiting medical centres. How then, to ensure that enrolled patients could still receive their medication under the current clinical trial conditions?
In early 2020, Roche was among the companies that resolved to find a way to make sure that patients could still receive our investigational medicines. We would not let the COVID constraints of shelter in place, fear of trips to hospitals, and disrupted supply chains, weaken this commitment.
In May 2020, a survey of the effects of the pandemic on clinical development studies showed that only 45% of ongoing clinical trials were continuing with their original activity. A quarter of clinical trials across the 50 leading sponsor companies had been paused. However, the need to continue carrying out clinical trials has spurred quick action and innovation; an impressive 30% of trials had adapted to a remote or virtual basis in just two months.1
A decentralised or virtual protocol (procedural method) is a growing innovation in the conduct of clinical trials. It brings many benefits to patients and society, including less friction in accessing healthcare, more potential for health equity and inclusion in clinical trials, greater potential for the conduct of trials in low prevalence diseases, and ultimately, to lower healthcare costs to society.
A decentralised approach - also known as distributed, home-based or, siteless trials - is a means of getting the trial to the patient at home versus requiring the patient to come to the major clinical facility of the lead researcher. This is made possible through virtual collaboration between the lead researcher and local medical teams, enabled by digital technologies, and facilitated and monitored through home visits.
How was this possible? How did we manage this? Lada Leyens, Clinical Trial Innovation and Personalised Healthcare Digital Health in Regulatory explains the shift: “Primarily by moving onto a virtual basis, and using decentralised trial approaches, essentially taking the trial to the patient at home”. She explains that “The concept of decentralised trials has been around for a decade, and it’s an idea whose time has now come. COVID-19 has been a catalyst, and shows us what’s possible in the use of innovative technologies, combined with local health services, to meet the needs of patients at home”.
Clinical trials studying many Roche molecules across disease areas including cancer, immunology, infectious diseases, and opthamology proceeded on this basis, meeting the needs of enrolled patients in over 30 countries.
Given the exact context and demands of scientific clinical trials, the involvement of regulators at regional and country levels was integral to enabling and accommodating the changes. Through an intense back and forth on guidelines, adjustments, latitudes, and the art of the possible, the teams worked tirelessly to ensure that the ongoing trials kept integrity to the trial design and therapeutic outcome in question.
“We quickly realised that we needed to pivot and adapt in order to better meet the needs of patients wherever they are. That meant operating very differently, finding a way, in near real time, yet at the non-negotiable level of rigor, under conditions as if the patient were visiting the clinic”, says Karen Massey, Global Head of Clinical Development at Roche.
The new baseline established through the forced constraints of the pandemic was in no small part due to regulators offering flexibilities to apply the emergent approach of decentralised trials. In addition to needing openness and courage at regulatory levels, decentralised trials also call for changes in practice by the Principal Investigator, local physician practices, and for mobile/ home visit nurses.
Taken all together these are dynamic shifts in health ecosystems, and likely to build momentum with time. If anyone needed convincing of the art of the possible, the late phase trial of the COVID-19 vaccine of Johnson & Johnson, involving over 40,000 patients, was also conducted on a decentralised basis.
While early, it is also believed that the decentralised approach holds important potential benefits to society in reducing healthcare burden. Among them, making it easier for patients to join clinical trials, extending to communities not traditionally reached and included, and speedier recruitment for rare diseases.
Decentralised clinical trials have been pressure-tested through the pandemic, and can be seen as a silver lining from the crisis. Widespread adoption, supported by digitally-enabled healthcare, telemedicine, remote monitoring, and logistical tools that assure the integrity of data, holds great potential for health systems in improving outcomes and evolving healthcare as we know it.
Tufts CSDD data 2020. 50 largest sponsor companies.