What Type of Pivotal Study Design is Appropriate for an Investigational New Drug to Manage Chronic Angle Closure Glaucom
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Drug Informorion Journal. Vol. 36, pp. 499-500. 2002 Printed in the USA. All rights reserved.
WHAT TYPE OF PIVOTAL STUDY DESIGN IS APPROPRIATE FOR AN INVESTIGATIONAL NEW DRUG TO MANAGE CHRONIC ANGLE CLOSURE GLAUCOMA: ACTIVE CONTROL OR PLACEBO? YEONG-LIANG LIN, MD, BEY-LIINGMAU,MD, HERNG-DER CHERN,MD, AND MONG-LING CHU,MD Medical Reviewers, Center for Drug Evaluation, Taipei, Taiwan
In Asia, there is a need for agents to treat chronic angle closure glaucoma. This article highlights factors in determining whether to conduct an active or placebo control study and special circumstances that increase the complexity of this decision in clinical trials for chronic angle glaucoma trials. The authors advise the use of a placebo control design in pivotal clinical trials dealing with chronic angle closure glaucoma. Once a medication is approved for use in chronic angle closure glaucoma, subsequent pivotal trials of similar drugs can use an active control design. Key Words: Angle closure glaucoma; Pivotal study; Active control; Placebo control
CHRONIC ANGLE CLOSURE glaucoma is more common in Asia than in the west (1). Many agents have been approved for, and used in treating, open-angle glaucoma, but few, if any, agents have been approved to treat chronic angle closure glaucoma. By noting the problems that can arise during drug development for products to treat chronic angle closure glaucoma, the authors present a possible approach that best complies with regulatory agency concepts. In assessing the efficacy of an investigational new drug to manage a chronic disease, determining whether to use an active or placebo control in a pivotal clinical trial usually depends upon whether an efficacious, proven Reprint address: Yeong-Liang Lin, MD, Center for Drug Evaluation, IF, No 15-1, Sec 1, Hangjou S Road, Taipei, Taiwan (e-mail: [email protected]).
therapeutic agent exists. A placebo control study design could be favorable because it may allow a stronger interpretation of data to be reached, and if an active control is used without placebo, there will be no statistical test to demonstrate that the active drug is effective, because any change observed may be a placebo effect (2). There are, however, reasons to exclude the use of placebo in clinical trials, such as ethical concerns and the safety of patients who do not receive active treatment. Several factors must be carefully considered in weighing statistical significance against the safety of the trial subjects: 1. Whether patients have received standard treatment, 2. Whether the disease is mild and lack of treatment is not considered medically important, at least during the trial period, and
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Y-L Lin, B-L Mau,H-D Chern, and M-L Chu
3. Whether allowed concomitant treatment, as needed, can lead to effective management of the disease.
sponse study compared various doses of an angle closure glaucoma pressure lowering medication. Another study used an extremely low dose of a pressure lowering medication
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