Wednesday, June 10, 2015

Patient-Centered Trial Design: A Real-Life Example

By: Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

The increasing complexity of protocols can cause several inefficiencies in the clinical trials process, such as longer study cycle times, inadequate recruitment and retention rates, and more protocol amendments. Many see the need to simplify such costly and time-consuming trials, but how do sponsors actually achieve this?

At the 2015 Partnerships in Clinical Trials conference, Pablo Lapuerta, Executive Vice President, Clinical Development and Chief Medical Office of Lexicon Pharmaceuticals, presented a session titled, “How Lexicon Achieved Efficiency in Phase 3 with a Patient-Centric Study Design.” In this session, Lapuerta discussed how his company successfully implemented a patient-centric study design for its Phase 3 trial. This oncology study was for a serotonin synthesis inhibitor developed for the treatment of Carcinoid Syndrome, a rare tumor that secretes chemicals into the bloodstream, most often in the gastrointestinal tract or lungs.

Like most rare diseases studies, enrollment was a challenge. In the past, it has taken more than five years to enroll 400 patients with metastatic carcinoid tumors for Phase 3 trials (even with more than 500 sites participating). Lexicon decided to do something different for this study. Patients with carcinoid syndrome who screen failed the Phase 2 protocol would have a new opportunity to be eligible for the Phase 3 study that was designed to focus on the patient. It included the following features to support recruitment and retention:

·         Less frequent visits
·         Home health option
·         Support for travel
·         Short placebo-control period
·         Long open-label phase
·         Less procedures
·         Flexibility on treatment during open-label extension patient focus

In addition to a protocol design that reduced inconveniences for the patient, Lexicon also had an effective CRO partnership. Representatives from both companies visited sites repeatedly in a coordinated fashion, attended scientific congresses together, updated sites on study progress, obtained feedback from investigators on the protocol and patients, and shared best practices in study conduct and patient management.

By maintaining a focus on the patient throughout the trial, this unique Phase 3 study was a success. Lexicon saw both positive site feedback and significant patient interest. Though it still took two years to complete enrollment, there were over 200 total patients in the Phase 3 program. Taking a patient-centric approach means committing to making the patient the priority in choices related to clinical trial design. This includes keeping things simple, holding back on the “wish list” of procedures and measures, and appreciating the value of safety data collected in a setting that reflects actual practice.

Have you been part of a study that incorporated a patient-centric trial design (from protocol design to study conduct)? How did it go?

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina served as the guest blogger covering Partnerships in Clinical Trials 2015. 

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