chizophrenia is a mental disease that affects approx-imately 1% of the population with distressing long-termconsequences for the patient and society. There is con-sistent evidence that the principal etiology of schizo-phrenia involves predisposing genetic factors.However, the search for the susceptibility genes with aview to any form of gene therapy has proved elusive.Furthermore, it is not clear whether the genes of familialschizophrenia are also involved in sporadic cases, whichrepresent the overwhelming majority of patients withschizophrenia. For sporadic cases, genetic associationstudies comparing the distribution of allelic frequenciesof candidate genes in patients with schizophrenia andcontrols have been performed, but the outcome of suchC l i n i c a l r e s e a r c h4 4 9Development of neuroleptic agents:pharmacogenetics and current safety issues of regulatory concernRashmi Shah, MD, FRCP, FFPMKeywords: pharmacogenetics; CYP2D6; dose schedule; cardiotoxicity; QT inter-val; torsade de pointes; drug interactions; polypharmacy; prescribing label; pres-cribing patternsAuthor affiliations: Senior Medical Officer, Medicines Control Agency,London, UKAddress for correspondence: Rashmi Shah, MD, FRCP, FFPM, Senior MedicalOfficer, Medicines Control Agency, Market Towers, 1 Nine Elms Lane,London, SW8 5NQ, UK (e-mail: rashmi.shah@mca.gsi.gov.uk)SThe development of safe and effective new drug treatments for schizophrenia poses a challenging task. This class of drugsis known to be associated with a wide range of serious and troublesome safety problems that include neurological, car-diac, endocrine, and metabolic side effects. Many of these drugs have a narrow therapeutic index and generate metabo-lites that often have their own unique pharmacological profile different from the parent compound. These features makeit imperative that the optimal dose schedules for neuroleptic drugs are carefully characterized. Many of these drugs aremetabolized by cytochrome P450 enzymes, which show genetic polymorphism and a bimodal distribution within the pop-ulation. A significant subset of the population cannot eliminate these drugs as effectively as the majority. This brings anadded dimension of complexity in characterizing the dose and individualizing therapy. Many neuroleptic agents are proar-rhythmic with an adverse effect on cardiac repolarization. They are prone to prolonging the QT interval and inducing tor-sade de pointes. Given the potentially fatal outcome of this ventricular tachyarrhythmia, drug development programsneed to ensure that the proarrhythmic potential of any new neuroleptic agent is thoroughly explored and its proar-rhythmic risk characterized. The clinical use of many of these drugs is further troubled by their high potential for drugdruginteractions. These too need to be adequately investigated during development. The approval and the labeling of a newneuroleptic agent require a careful regulatory assessment of its risk/benefit ratio in comparison with the available alter-natives. Their safe and effective use in routine clinical practice depends on careful attention to prescribing information,especially the contraindications, precautions, and patient-monitoring requirements.Dialogues Clin Neurosci. 2002;4:449-462.