Designing Clinical Trials to Study Rare Disease Treatment
- PDF / 313,926 Bytes
- 4 Pages / 504 x 719.759 pts Page_size
- 81 Downloads / 239 Views
Drug Information Journal. Vol. 32. pp. 957-960. I Y Y X
Printed in the USA. All rights reserved.
DESIGNING CLINICAL TRIALS TO STUDY RARE DISEASE TREATMENT MARLENEE. HAFFNER,MD, MPH Director, Office of Orphan Products Development, United States Food and Drug Administration, Rockville. Maryland
This paper provides an overview of the design of clinical trials for rare diseases and the impact of the Orphan Drug Act on the successful study of diseases found in small, widely dispersed patient populations. Orphan drugs are defined. Randomized, placebo-control trials may not always be an option for studying orphan drugs; designs which may work better include: open protocol, open label, historical control, crossover trials, withdrawal design trials, or trials with surrogate endpoints. Each of these is discussed. The benefits of studying orphan drugs and the procedure for approval of trial design by the Food and Drug Administration are outlined. Key Words: Orphan drugs; Clinical trial design; Orphan Drug Act; Office of Orphan Products Development; Rare diseases
SINCE 1983, THE Orphan Drug Act has encouraged clinical research for the treatment of rare diseases and disorders, and has played a major role in bringing treatment and diagnostic products for those disorders from the laboratory bench to the patient. Rare disease research calls for clinical trial design that accommodates the study of diseases found among small, widely dispersed patient populations. Prior to the Orphan Drug Act, researchers and drug sponsors often felt that clinical trials targeting minuscule patient populations could not possibly derive results sufficient for Food and Drug Administration (FDA) market approval. Over the past 15 years, this legislation has proven that in many many instances, products for rare diseases Presented at the DIA Workshop “Improving Clinical
Trials: Contemporary Design Solutions,” October 2728, 1997, Philadelphia, Pennsylvania. Reprint address: Marlene E. Haffner. MD, MPH. Director, Office of Orphan Products Development, US Food and Drug Administration (HF-35). 5600 Fishers Lane, Rm. 8-73, Rockville, MD 20878.
can successfully be studied, and ultimately achieve FDA approval. Due to the small number of patients affected by most rare,“orphan” diseases, an appropriately designed trial is imperative; but methodologies for studying orphan drugs are, in general, not significantly different from the methodologies for studying any other drug. Exactly what are the barriers to orphan drug development? They are not terribly surprising: the small number of patients affected by the disease; the small market for the product; the wide dispersement of patients throughout the United States, andfrequently-throughout the world; and products that frequently are not patentable, or require a lengthy patent process. These barriers all influence the development of drugs for rare diseases. In the 10 years before the Orphan Drug Act was enacted, it is estimated that 10 products were approved for rare disorders. Because of the high cost of drug dev
Data Loading...
