After the recent webcast on “Best Practices for Boosting Enrollment in Oncology Clinical Trials,” we found a lot of interest in the use of patient-centric enrollment to foster participation in oncology trials. This would represent a shift from the more traditional enrollment model.
You may be wondering why such a shift is necessary, and what its advantages might be. We have to realize that the age of the “blockbuster drug” is over. Individual, personalized, and patient-centric medicine represents the future of clinical research, notably in oncology. We need to write protocols and plan recruitment strategies based upon individual patient needs and not on a disease class.
According to Dr. John L. Marshall, Chief, Division of Hematology/Oncology, Georgetown University Hospital, a key reason why cancer patients do not participate in clinical research is because many treatment studies do not provide enough of a benefit to these individuals. Dr. Matthew Wiener has written in Life Science Leader that as cancers and drugs become more personalized, the challenge for finding and enrolling patients becomes more daunting. However, as individually-tailored therapies become more prevalent, patients will have a greater incentive to participate in clinical trials.
The traditional enrollment model involves identifying and preparing research sites, as well as collecting all regulatory documentation, in advance of enrolling a single patient. Site start-up is time consuming and costly. Moreover, some sites may never enroll a single patient or take months after initiation to enroll their first patient. Additional training may be required, or deviations from protocol may occur. Data from Tufts Center for the Study of Drug Development shows that 11% of initiated sites never enroll a single patient, and 37% under-enroll and 39% of sites never meet enrollment targets.
On the other hand, the patient-centric enrollment model, which is similar to a cooperative group model, channels important resources to productive sites, and cuts development time and costs.
See below for our feedback to a related attendee question received during the webcast.
Question: In the patient centric model, we still have to keep sites ready with EC/IRB approvals. There is not much in the way of cost savings, time and effort, as far as start-up activities are concerned. So, what are some advantages of the patient-centric model when it comes to start-up activities? Also, what percentage of additional sites has to be considered, assuming that some sites would not be activated?
In the patient-centric approach as described by Dr. Wiener, although regulatory and documentation requirements still must be met, a sponsor will only initiate a trial site when an interested pre-identified patient is identified. This approach has a site identify individual cancer patients with specific disease profiles. With the patient-centric model, you can detect rare clinical events or diseases over a larger sample, e.g. more sites. You’re casting a broader net to find patients who qualify for your trial. Patients are identified across several study centers that all have potential to participate in a study. Remember that an investigative site is not activated until an eligible patient is identified, although regulatory approvals will have been obtained and contracts negotiated as with the traditional approach. When a site is activated, it generally enrolls within a few days.
An argument could be made that having trial sites open prior to patient enrollment draws patients to study centers. But the patient-centric approach funnels resources to productive sites. Patient-centric sites accrue patients faster than traditional enrollment sites, typically enrolling patients within a few days of being activated.
This blog post collaboratively written by Chris Mackay, Project Director, Early Phase Pharma, University of Kansas Cancer Center and Clinipace’s Barb Geiger.