Emerging Methods to Engage Patients in Regulatory Review Process

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At a recent workshop convened by the Duke-Margolis Center for Health Policy, Dana DiBenedetti, PhD, Executive Director of Patient-Centered Outcomes Assessment at RTI Health Solutions presented information concerning the use of interviews conducted as part of clinical trials as an emerging method to understand clinically meaningful change.

The meeting was a cooperative effort co-led by the Duke-Margolis Center and the US Food and Drug Administration (FDA). Each workshop session was presented jointly by research experts and FDA representatives.

The overall aim of the meeting, Clinical Outcome Assessments: Establishing and Interpreting Meaningful Within-Patient Change, was to address challenges in collecting meaningful research across disease areas and patient populations (e.g., children, adults). 

During her session, Dr. DiBenedetti focused on the definition and potential value of the clinical trial interview, the types of data than can be generated from this method, potential applications, and operational aspects of the interview in clinical trial settings. She shared that data obtained using this method allows researchers to identify and further understand the relevance and clinical meaningfulness of specific treatment changes beyond clinical endpoints and side effects. Learn more about what Dr. Benedetti has to say about these issues by reviewing her slides.

Dr. DiBenedetti coauthored research based on exit interview data in carcinoid syndrome presented at the 2016 European Neuroendocrine Tumor Society (ENETS) Annual Meeting as well as the 20015 Symposium of the North American Neuroendocrine Tumor Society. Results from the exit interview study designed and conducted by Dr. DiBenedetti and her team established the level of symptom change, or reduction, considered meaningful by the patients interviewed.  Selected exit interview data were included in the submission documents of Xermelo and reviewed by the FDA as part of the approval process.

Further information about the workshop can be found at https://healthpolicy.duke.edu/events/clinical-outcome-assessments-establishing-and-interpreting-meaningful-within-patient-change

Research about patient interviews coauthored by RTI Health Solutions authors:

Pavel M, Horsch D, Anthony L, Ervin C, Kulke MH, Bergsland E, Caplin M, Oberg K, Warner R, Kunz P, Metz DC, Pasieka J, Pavlakis N, DiBenedetti D, Lapuerta P. Patient interviews in TELESTAR, a phase 3 study of telotristat etiprate, report meaningful improvement in carcinoid syndrome. Poster presented at the 2016 European Neuroendocrine Tumor Society (ENETS) Annual Meeting; March 2016. Barcelona, Spain. Neuroendocrinology. 2016; 103:89. 

Anthony L, Horsch D, Ervin C, Kulke MH, Pavel M, Bergsland E, Caplin M, Oberg K, Warner R, Kunz P, Metz DC, Pasieka J, Pavlakis N, DiBenedetti D, Haydysch E, Yang QM, Jackson S, Arnold K, Law L, Lapuerta P. Assessing treatment benefit of telotristat etiprate in patients with carcinoid syndrome: patient exit interviews. Poster presented at the 8th Symposium of the North American Neuroendocrine Tumor Society; October 15, 2015. Austin, TX

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