Abstract
For the approval of any new medicinal product quality, safety and efficacy are essential requirements. This manuscript focusses on the clinical development programme. For the investigation of antiepileptic drugs some international guidelines are of special importance. They are based on the knowledge of many experts and can be seen as a consensus on minimal requirements; deviations must be thoroughly justified. In phases II and III, usually randomised, double‐blind add‐on studies versus placebo in patients with therapy-resistant seizures are used to get an impression of the efficacy and certain safety issues. A clear dose-response relationship may be a good indication for efficacy. However, assessment of safety of the new product in add‐on studies is difficult. Therefore comparative phase III monotherapy studies versus established antiepileptic drugs are essential to confirm the results obtained in add‐on studies and are needed for a proper judgement of the efficacy/safety balance. The percentage of reduction of seizure frequency has played a dominating role as efficacy criterium. Nowadays preference is being given to the percentage responders. Which parameter is the most relevant for the given group of patients and what change is considered clinically relevant must be thoroughly argued. The definition of responder should focus on major benefit for the patients involved.
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References
European Commission. Medicinal products for the treatment of Epileptic Disorders, the Rules governing medicinal products in the European Union, volume III, part 1, 1996: 537–44.
International League against Epilepsy. Guidelines for clinical evaluation of antiepileptic drugs. Epilepsia 1989; 30: 400–8.
European Commission, The extent of population exposure to assess clinical safety for medicines intended for long-term treatment of non-life-threatening conditions, the Rules governing medicinal products in the European Union, volume III, part 1, 1996: 445–6
European Commission. Clinical Investigation of medicinal products for long-term use, the Rules governing medicinal products in the European Union, volume III, part 1, 1996: 447–50
European Commission. Clinical investigation of medicinal products in children, the Rules governing medicinal products in the European Union, volume III, part 1, 1996: 469–74.
European Commission. Good clinical practice for clinical trials on medicinal products in the European Community, the Rules governing medicinal products in the European Union, volume III, part 1, 1996: 377–96.
International Conference on Harmonization Steering Committee. Guideline for good clinical practice, 1996.
European Commission. Biostatistical methodology in clinical trials, the Rules governing medicinal products in the European Union, volume III, part 1, 1996: 451–68.
European Medicines Evaluation Agency. European Public Assessment Report on Felbamate, 1995.
Elferink AJA, van Zwieten-Boot BJ, Analysis based on number needed to treat shows differences between drugs studied. BMJ 1997;314: 603.
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Schobben, F., Hekster, Y. & van Zwieten‐Boot, B. Evaluation of drug treatment outcome in epilepsy: a clinical perspectiv. Pharm World Sci 19, 223–226 (1997). https://doi.org/10.1023/A:1008650925277
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DOI: https://doi.org/10.1023/A:1008650925277