Advances in clinical trial approach: home-based-studies!
Home-Based Clinical Studies – A Paradigm Shift?
Steve Sisk, MSc Pharmacy, Director, Clinical Operations, CytoSorbents Incorporated, examines the opportunities offered by remote-based trials.
While patient recruitment and retention are central to the success of clinical trials, achieving this can be difficult. These challenges, along with ensuring protocol compliance, directly influence overall study costs, results, and timelines.
Patient participation in a clinical study can confer significant difficulties to study patients due to the frequency of visits, travel to the study sites, long visit times, disruption in their schedules and activities, and finally, geographic exclusion from distant sites.
The end result of imposing hardship upon unwell or busy patients often has dire consequences upon patient enrollment and retention in the clinical trial, with dropout rates approaching 60 percent for complex multi-visit studies.
Do Home-based Studies Offer a Solution?
One answer to this is in the form of home-based or remote clinical trials in which all or the majority of the patient’s clinical experience is obtained from the comfort of their own home. Although there has been some hesitation on the part of the pharmaceutical industry to embrace this concept, an industry-wide emphasis on cost savings has prompted us to look more closely at the model. To support this study mode, a number of specialty CROs have made the commitment to provide specialized clinical trial support services.
There are a number of real advantages offered to patients in home-based studies that move the trial beyond being of chief benefit to the sponsor. These include elimination of enhanced health care, reduction in patient travel time, convenient home visit times, reduction of financial constraints associated with travel and missed work, and even chaperoning services for the occasional clinical visit. This is well in keeping with the FDA’s desire for greater patient centricity in clinical trials.
As a result of not being tied to a physical study site location, remote access to clinical trials for the patient classes listed below can substantially improve rates of enrollment and retention for the following patient reasons:
Remote pediatric participation reduces impact on parent to transport patient
Patients who do not drive or are too sick to drive
Patients with a rare disease and no nearby study center
Patients who dislike or are afraid of physician’s offices or hospitals
Patients who move annually (“snowbirds”) and are in long trials
Patients that cannot afford to miss work to attend multiple office visits
Patients with busy schedules and multiple responsibilities
Remote-based Trials are More Convenient
Some of the clinical trial services being offered by CROs working in this field span a wide range of services and procedures formerly only offered at clinical research sites. These are now being routinely provided to home-based study participants and include the following:
Study-drug transportation, preparation and administration, and accountability
Patient/caregiver training and education (drug self-administration, and supervision)
Long-term and post-study drug observation
ECGs, pulmonary testing, accurate blood pressure monitoring, etc.
Patient Informed Consent
Laboratory sample collection, preparation, packing, and shipping
Adverse Event collection and reporting
Enhanced observation and training of patient reported outcomes measures and administration of questionnaires
Certain classes of studies that are particularly well suited to this approach are the following:
Rare and Orphan diseases
Congestive heart failure
Pediatric and elderly
CNS and Alzheimer’s or dementia trials
Mega and longitudinal relying on patient retention (such as cardiovascular outcome or vaccine trials)
Long-term trials and/or those that require frequent site visits
It should be understood that studies that are related to acute care, in-patient procedures, or measurements that have to be made on very short notice would not be appropriate for this approach. It is important to consider that efficient planning and excellent logistical support are required for home-based clinical studies.
Just as the move away from paper to eCRFs a decade ago resulted in enhanced clinical trial efficiencies, the trend toward remote-based trials may ultimately change how we view the industry 10 years from now. Sponsors should be able to improve patient enrollment and retention, achieve greater patient compliance, and work with a more clinically representative patient population. Study patients will have greater access to clinical studies, fewer hardships, and better home care. This does not mean this approach should be considered a panacea for poorly enrolling studies or will always be the best approach. Rather, it should be considered as part of a well-planned clinical development strategy.