Cataplexy is easily overlooked and frequently undiagnosed in patients with narcolepsy, according to Cathy Gendreau, senior product manager, Xyrem, at Orphan Medical, Minnetonka, Minn. Sleep Review spoke with Gendreau about how the company is helping to alter that trend through increased educational efforts and a new diagnostic tool.
How has the perception of narcolepsy changed since Orphan Medical entered the market?
Prior to Orphan’s entering the market, there was minimal marketing and/or medical education activity regarding narcolepsy and specifically cataplexy. Orphan has spent quite a bit of time educating physicians and office staff about the disease state. Narcolepsy affects one in 2,000 people in the United States, and that’s more than cystic fibrosis. The literature suggests that 60% to 90% of patients with narcolepsy have cataplexy. We’ve focused on educating physicians that narcolepsy is not only excessive daytime sleepiness, but also cataplexy, and it should be treated.Patients’ quality of life is so significantly impacted that it typically has profound effects on their socioeconomic circumstances. They have decreased performance at schools and workplaces. They have difficulties with interpersonal relationships and social interaction; increased incidence of accident, injury, and depression; and lower self-esteem. Due to the embarrassing symptoms of narcolepsy and the fact that cataplexy is triggered by emotions, people with narcolepsy tend to be socially isolated. Orphan has helped broaden the awareness of narcolepsy, specifically the symptom of cataplexy, to allow those underserved patients to empower themselves to get the appropriate treatment.

How has the restricted distribution of Orphan Medical’s cataplexy drug Xyrem affected the company?
Initially, we were concerned. But we’ve been very pleased with our restricted distribution program, called the Success Program, which offers comprehensive and interactive support to physicians and patients who use Xyrem. It offers prescription tracking, prescribing use information, and 24/7 patient support. The central pharmacy offers significant support for insurance reimbursement and has secured 75% reimbursement for Xyrem. Our patient and physician registration enables physicians who prescribe this medication to have more confidence because they can better monitor the treatment.

Orphan Medical provided an unrestricted grant to develop the Cataplexy Assessment for Treatment to Augment Life—Your Standardized Toolkit (CATALYST). Of what importance is this diagnostic screener?
The one validated tool for diagnosing cataplexy is the Stanford Cataplexy Questionnaire, but it’s long and not commonly used in practice. Orphan Medical asked several sleep specialists, including Jed Black, MD, and Emmanuel Mignot, MD, PhD, from Stanford, to be part of a steering committee that would develop a tool that people could use in practice to help make the diagnosis of cataplexy easier and to help facilitate dialogue between the patient and their caregiver. We hope to start a validation process for CATALYST at the beginning of 2005. We feel it will help in the early diagnosis and appropriate management of cataplexy to improve the lives of patients with narcolepsy.