The notion that a quality clinical trial should be conducted in highly specialized and experienced study centers with tight oversight of patients is becoming obsolete. Decentralization means that clinical trials can now be conducted in routine treatment clinics supervised by medical teams practicing standard of care. Centricity means that data capture can be obtained without significantly interfering with the patient lifestyle. Decentralized centricity follows a simple digital paradigm: anywhere, anytime.
Background
“Virtual” or “decentralized” clinical studies are based on an organizational model that broadens the geographic location of study sites, expanding the ethnic, social and age diversity of study populations. This approach reflects more accurately real-world settings.
“Centricity” allows data collection at the patient’s home or local care facilities. This flexible delivery model means that clinical trials more accurately reflect standard of care practices. This real-world environment can also define more precisely the impact of a novel therapeutic intervention in the patient journey. The study procedures, even the informed consent process, are conducted electronically, online or through a tablet or smartphone application. These studies collect data either passively (e.g., fitness trackers, patches, wearables) or actively (e.g., electronic diaries for patient reported outcomes, such as quality of life).
Decentralized Clinical Trials (DCTs): Critical Steps
Once a preliminary list of potential decentralized sites has been created, a thorough assessment should address the following:
- Has the study site the essential technology and infrastructure, such as electronic health record systems, data management and pharmacy capabilities?
- Has the study site the essential personnel, including investigators, study coordinators and hospital pharmacists?
- Has the study site demonstrable operational experience in the therapeutic area and treatment modality focus of the clinical trial?
- Has the study site demonstrable good reputation with patients?
To ensure full engagement of the study site, the sponsor research team must develop:
- Effective and structured communication channels, including a rigorous project management approach.
- Internal processes to ensure a smooth planning project management. This is especially relevant for the initial interactions, such as site screening, site activation and initiation visits.
- Supportive documentation and tools to ensure the study site becomes familiar with the management of the target disease, prescription, and practice patterns.
- Capability to provide educational activities addressing any gaps at the study site.
- Combined ‘high tech and high touch’ Technology cannot replace direct interactions with study personnel.
Patient Centricity: Critical Steps
Once the study site has been thoroughly vetted for access to the patients to be enrolled in the clinical trial, the patients covered by the study site able to:
- Broadly meet the enrollment expectations of the clinical trial.
- Swiftly adopt virtual interactions between clinicians and patients, such as telemedicine, wearables.
- Readiness to accept home visits and direct delivery of study drugs.
- Motivation to provide feedback and be actively involved in the conduction of the study conduction.
- Willingness to receive educational support and materials covering:
- Ethical and administrative aspects of the clinical trial
- Logistics for study visits at the hospital or at home
- Safe drug storage, delivery and dosing diaries
- Essential safety, efficacy endpoints and study design elements, emphasizing patient reported outcomes
- Therapy’s mechanism of action
- Relevance of interactions of advocacy groups and participation in patient advisory boards or focus groups
Perspective
Although digitalization is massively impacting all aspects of clinical development, the key elements of planning, monitoring, data management and interpretation must be kept at the highest degree of rigor and quality. This level of excellence in clinical development will require that sponsors, study sites and patients incorporate the following 3 Ps into the study design and execution:
- Clinical trials must reflect the diversity of the target therapeutic profile and population. People are diverse, so is their personal health. Different genetic and phenotypic profiles result in variability of efficacy and safety results. The objective is to adapt new therapeutic modalities to the distinct healthcare systems.
- Clinical trials must integrate the patient with the healthcare and social support structure. All study participants should consider themselves stakeholders of the success and failures of the study objectives by providing transparent and timely access to information. Especially patients should take responsibility for self-care and able to take ownership of their health.
- Clinical trials must predict efficacy and safety endpoints based on pre-clinical and reference study data. The study protocol should consider the natural history of the target disease and the unique features of the study site and healthcare infrastructure.
Decentralized centricity must integrate personalization, participation, and prediction to ensure a steady progress of healthcare as a boon to society. Our role as clinical developers is to be keenly aware of the necessary support of study sites and to be empathetic with the needs and expectations of our patients as individuals, not just study subjects.
