title: Data from regulatory studies: What do they tell? What don't they tell?
abstract: Phase III studies of antiepileptic drugs (AEDs) are specifically designed to satisfy strict regulatory criteria. As they are conducted in protocol-restricted patient populations over short treatment periods and employ fixed study designs and dosing schedules, they are not fully representative of 'real-life' clinical practice. Therefore, in order to provide an overall assessment of clinical performance, regulatory studies must be backed up by post-marketing clinical experience. Phase IV studies provide information on a drug's performance in a setting more closely representing real clinical practice, with broader patient populations and a more flexible approach to individual treatment. Prospective long-term studies allow the determination of efficacy and safety (and cost-effectiveness) over extended treatment periods; these studies and audit data provide a means of assessing idiosyncratic side effects, unusual interactions and the effects of an AED in rare patient groups. By complementing regulatory evidence with real-life clinical experience, a comprehensive assessment of the risks and benefits of an AED can be made.
authors.affiliation: Department of Clinical Neuroscience, Sahlgrenska Academy, Göteborg University, Göteborg, Sweden. elinor.ben-menachem@neuro.gu.se
authors.forename: E
authors.initials: E
authors.lastname: Ben-Menachem
mesh_terms: D016907:Adverse Drug Reaction Reporting Systems; D000927:Anticonvulsants / Q000009:adverse effects / Q000627:therapeutic use*; D017326:Clinical Trials, Phase III as Topic / Q000331:legislation & jurisprudence*; D017327:Clinical Trials, Phase IV as Topic / Q000331:legislation & jurisprudence*; D017277:Drug Approval / Q000331:legislation & jurisprudence; D004347:Drug Interactions; D004827:Epilepsy / Q000188:drug therapy*; D005500:Follow-Up Studies; D006801:Humans; D011358:Product Surveillance, Postmarketing; D011446:Prospective Studies; D018570:Risk Assessment; D016896:Treatment Outcome; D014481:United States
chemical_list: D000927:Anticonvulsants