Case Study One

Financial Comparison of a Patient Centric (Just-In-Time) versus a Traditional Enrollment Model on a small Phase II Pancreatic Cancer Trial

Synopsis:

A patient centric model (Just-In-Time) enrolls 36% more patients per month with 52% fewer sites and with an estimated site-cost savings of $109,500 when compared to traditional enrollment models.

Case Study:

Background: A start up Biotechnology Company based in Boston, MA, approached Pharmatech Oncology to support enroll patients in a difficult protocol Phase II Stage IV pancreatic cancer trial. At the time Pharmatech was approached the study had been open for 13 months at 20 institutional sites and had fallen behind its enrollment objective.

Just-in-Time: Just-In-Time (JIT) modifies the traditional project management methodology for enrolling patients in a clinical trial. Instead of opening a site and waiting for the patient, JIT has many sites looking for patients with the available standard of care information (SOC). Once a patient is pre-identified the investigator and the site is submitted to the IRB. Using a central IRB (Pharmatech has all the essentials documents for the project), a site can be opened within 48 hours and patients are dosed in 10 business days.

Results: A large grouping of sites enter a “pre-identification” stage without incurring the cost of opening and maintaining the site. Once a patient is identified, IRB approval is obtained – a process which requires on average only 2 business days - and the site is opened and patients are dosed. Therefore, every open site shall produce a patient, which eliminates the risk, time and cost of non-enrolling sites. On average, 15% to 25% of all traditional sites that are opened never enroll a patient, though the cost of opening and maintaining those sites is still incurred.

Upon being hired 13 months into this project, the Just-In-Time methodology enrolled its first patient within 4 weeks, thereafter 19 subjects were enrolled within 6 months – an enrollment rate of 2.86 patients per month. Pharmatech Oncology had selected 38 sites to pre-identify, and 8 sites were actually opened. No sites were non-enrolling.

In comparison, the traditional approach enrolled 42 patients over a course of 20 months – an enrollment rate of 2.1 patients per month by opening 13 sites, of which 2 (15%) were closed for non-enrollment. The cost of a non-enrolling site consists of opening costs, maintenance costs and closing costs. For this particular study, the non-enrolling sites cost the sponsor an estimated $41,000, not accounting for the missed opportunity cost of offering the study to two other sites that may have enrolled patients. In addition, open sites require monthly maintenance costs prior to enrolling a patient – these costs include ongoing IRB, IND Safety filings, etc., where as Just-In-Time avoids pre-patient maintenance costs altogether.

In a patient-per-month-per-site comparison, the Just-In-Time model yielded a 121% improvement when compared to the traditional approach. In the traditional model, 42 patients were enrolled at 13 sites over a course of 20 months, while with Just-In-Time, 20 patients were enrolled at 8 sites over a course of 7 months

Discussion: Lastly, this study does not factor in the macro costs related to study efficiency. Just-In-Time improves patient enrollment efficiency, thus reducing the overall cost of the project, improves speed to market and permits a longer patent-protected earning period. For earlier stage biotech companies, enrollment efficiency reduces annual burn rate and may help expedite research milestone requirements. Each of these factors could cost several millions of dollars per month or year for biotech and pharmaceutical companies. A 36% improvement in patients-per-month and a 121% improvement in patients-per-month-per-site can have a profound impact on the cost of bringing products to market.

Conclusion: Just-In-Time is a cost effective method to enroll patients in certain oncology trials from a project management perspective. It also has economic value to society by getting products to market quicker.