Epilepsy – which poses a serious burden for patients, with effects on quality of life, work productivity, healthcare needs and risk of premature death – is diagnosed in some 2.4 million people globally each year, and currently affects around 300,000 American children under the age of 14. Pediatric epilepsies present particular challenges, including correct diagnoses, comorbidities and interactions with developmental processes in the brain.
While epilepsy is usually controlled with available medication, either as monotherapy or add-on anti-epileptic drug combinations, more than 30% of people with epilepsy do not have adequate seizure control, even with the best available medications. Epilepsy is therefore an active area for R&D, with more than 20 potential therapies in various stages of development. Pediatric trials will be required for many of these products; considerations such as rational study design, careful attention to the specifics of the protocol, and appropriate investigator and site choices are key to successful planning and execution. This paper provides an overview of the 36 industry sponsored, interventional trials involving approximately 6,600 pediatric patients that are currently underway.
The authors examine the regulatory and competitive landscape in the United States and Europe. They also describe lessons learned from Quintiles’ experience with 49 epilepsy studies since 2000, recruiting more than 9,300 subjects across 1,200 global sites in 35 countries.