Jonathan Cotliar, MD, Chief Medical Officer, Science 37
The pandemic-necessitated paradigm shift toward decentralizing certain clinical trial activities is a definitive, long-overdue push toward a more patient-focused clinical research ecosystem that leverages technologies and expertise to reduce the burden of clinical trial participation for patients and investigators. While this shift toward home-based clinical trials is certainly a welcome trend for patients with any condition across all therapeutic areas, it is perhaps most welcome for those with rare diseases. For these patients, participating in clinical trials can be an enormously taxing endeavor — not just for patients, but for their families, their caregivers, and their doctors as well.
It’s fortuitous then, that the rapid and requisite growth in decentralized clinical trial (DCT) applications over the past year has given us a glimpse of an emergent, patient-friendly hybrid model of clinical research that reduces or eliminates prohibitive travel time and other logistical barriers that typically limit clinical research participation among rare disease patients. This hybrid model — in which some functions are conducted remotely through DCT methods and others conducted through traditional site or clinic visits — is particularly appropriate for rare disease studies where the unique manifestations and characteristics of each disorder already necessitate a customized approach. And according to a survey of rare disease patients and families by Xperiome, this type of study design is preferred; with the vast majority surveyed suggesting they would prefer to participate in a clinical trial through a mix of clinic- and home-based activities (for both monitoring and medication dispensing).
This finding also helps to illustrate a critical, often mis-understood point about decentralized clinical trials: implementing DCT technologies and methods into clinical trials does not need to be an all-or-nothing approach. While a long-term, low-touch, non-interventional study might be appropriate for a fully virtual study design, the majority of studies — and certainly those for rare diseases — will require a hybrid design of some sorts. A study utilizing remote coordinators, for example, could potentially include additional DCT elements such as eConsent and mobile nurse management to optimize clinical workflow, while also including scheduled site visits for medication dispensing and specialized assessments more conducive to in-person examination.
Further suggesting a broad consensus for a hybrid development model, an Industry Standard Research (ISR) survey of biopharmaceutical executives commissioned by Science 37 found that while 80% expect their company to conduct a clinical trial using at least some DCT elements in the coming year, only one-in-six of these are expected to be completely virtual — all others will be designed with a unique configuration of technology, processes, and resources.
This survey also illustrated an area of concern, however, in that nearly 60% of biopharma executives say that their organization does not have the internal capabilities to operationalize any components of a decentralized clinical trial. In lacking internal DCT capabilities but planning for their increased adoption, biopharma sponsors will soon be facing a familiar problem of how to coordinate and integrate DCT into clinical trial operations, and whether to do so by building infrastructure or by outsourcing these activities to any number of service providers. In either case, these data help illuminate the contours of a future, network-based clinical research ecosystem in which DCT components are integrated into trial protocols from the onset — easing the burden on investigators and patients while providing biopharma developers with data at the requisite level of quality.
For rare disease patients and their families, the emergent, patient-focused hybrid model of clinical research should be welcome and encouraged as it continues the critical trend of including the patient voice in clinical development plans. From endpoint identification to pragmatic, patient-important protocol design, early consultation with patient advocacy groups and other disease specific organizations will continue to help dictate how best to incorporate DCT technologies and methods into rare disease studies; in doing so, biopharma can demonstrate a commitment to easing the burden of clinical trial participation while also accelerating the development timeline and generating quality evidence.