Case Study Two

Financial Comparison of a Patient-First (Just-In-Time) Clinical Trial Enrollment Methodology Versus Traditional Enrollment in a US-based, Phase 3, Leukemia (CML) Study.

Synopsis:

A patient-first clinical trial enrollment model, Just-In-Time (JIT), used in community practices, was compared to traditional enrollment, used in large hospitals. This study demonstrates that overall enrollment in community sites can be 52% more efficient than in large hospitals, when the JIT model is applied. Costs were favorable for JIT.

Case study:

Background: A major pharmaceutical company contracted with Pharmatech Oncology to support US enrollment in an ongoing phase 3 clinical trial in CML. The sponsor was in the process of qualifying 23 large hospitals and institutions to enroll patients to the study. Of these 14 were opened to enrollment. Pharmatech Oncology proposed a patient-first enrollment acceleration system (Just-In-Time) to be used in the Pharmatech Oncology Research Network, comprised of primarily community based cancer clinics. 42 Network practices participated in the pre-identification stage of the study using standard of care (SOC) information to pre-identify CML patients. Of the JIT pre-identifying sites, 14 identified patients to enroll into the study.

Just-In-Time System: Just-In-Time (JIT) modifies the traditional management method for enrolling patients in a clinical trial. In the traditional model, 25-30% of all sites opened in a clinical trial never enroll a patient, though the cost of opening and maintaining these sites is incurred. In the JIT system, instead of opening a site and waiting for patients to present, JIT has many sites looking for patients using available SOC information and protocol selection criteria, without incurring the cost of opening sites.

Initially the study protocol and core documents are approved by the IRB. Only when a qualifying patient is pre-identified and interested in clinical trial participation, the site is submitted to the IRB for approval which takes only 48 hours. Then study drug and research materials are shipped to the site, and training/initiation of the site research staff is conducted. Within 10 business days the patient is dosed.

Results: The sponsor commenced its rollout of hospitals 7 months prior to implementing JIT in the Pharmatech Research Network. Over the course of 23 months, the 14 large hospitals have enrolled 17 patients, while JIT enrolled 18 patients through 14 community sites over the course of just 17 months. This represents a 52% increase in patient enrollment efficiency.

In addition, the first patient was enrolled after 3 months using the JIT model, while the first enrollment by a traditional site required 9 months. Early enrollment is an important milestone for trial sponsors as well as for maintaining investigator attention on the study.

Economics: To open a large institution or hospital is time intensive and costly. The pre-study qualification process is $2,500 per site, and the net cost of opening the site is in the range of $12,000-$15,000, plus the cost of delays and ongoing monitoring prior to enrollment. Conservatively, the traditionally managed sites in this study incurred opening costs of 23 x $2,500 = $57,000 plus 14 x $13,000 = $182,000, all together $239,000. 17 patients were enrolled which means that distributing the opening costs over 17 patients gives an added cost of $239,000/17 = $14,100/enrolled patient in addition to per patient clinical research costs.

For JIT, the sponsor paid a JIT start up fee of $50,000, each site that participated in the pre-identification received a stipend of $1,000 = $42,000. The cost of opening open a community sites in this study was $9,000. 14 sites were opened which gives a total of 14 x $9,000 = $126,000. Opening cost and stipend for pre-identification is together $218,000 or $12,100/enrolled patient – a cost savings of 14%.

Discussion: This study demonstrates the great potential of utilizing community based research practices for clinical oncology trials. The private owned community oncology practices are unique to the US, and together they treat 85% of cancer patients. More and more community practices are participating in clinical research with dedicated research personnel. Physicians have interest in offering their patients alternative treatments, which they obtain through clinical research. JIT offers community oncology investigators a means to efficiently offer clinical trials for their rare cancer patients.

Conclusion: The study with rare CML patients demonstrates that clinical research in community based oncology practices can be conducted with efficiency to challenge traditional large site model. Combining a 14% cost savings and 52% greater enrollment efficiency, Just-In-Time has a profound impact on the macro project costs. In addition, this case study demonstrates that JIT offers clinical trial sponsors an efficient alternative to the traditional site centered model for more rapid enrollment at lower cost.