DOI: 10.1097/01.sa.0000280262.95873.29
,
Issn Print: 0039-6206
Publication Date: 2007/10/01
Facebook
Twitter
LinkedIn
Email
 |
Checking for direct PDF access through Ovid | |
Excerpt
Jennifer S. Li,*†‡ Eric L. Eisenstein,† Henry G. Grabowski,§ Elizabeth D. Reid,† Barry Mangum,† Kevin A. Schulman,† Jon V. Goldsmith,∥ M. Dianne Murphy,‡ Robert M. Califf,† and Daniel K. Benjamin Jr*†‡
(JAMA, 207:480-488, 2007)
*Department of Pediatrics; †Duke Clinical Research Institute, Duke University, Durham, NC; ‡Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Rockville, MD; §Department of Economics, Duke University, Durham, NC; and ∥Office of Policy and Planning, Office of the Commissioner, US Food and Drug Administration, Rockville, MD.
Under the Pediatric Exclusivity Program, incentives for studies in children were created mainly because many of the drugs currently marketed are either used off-label or not at all because safety and efficacy studies were done only in adults. The pediatric exclusivity studies have initiated more than 100 product-labeling changes. However, critics contend these studies are only windfalls for the drug companies. This cohort study attempted to quantify the economic return to industry for completing pediatric exclusivity trials.
Clinical trials performed for pediatric exclusivity were identified. Key elements of the clinical trial design and study operations were determined, and the cost of performing each study was estimated; the net economic return to industry from participation in the trial was calculated as the net return-to-cost ratio. Three-year market sales were converted into estimates of after-tax cash inflows based on 6 months of additional market protection. Companies were contacted to confirm these estimates of costs based on the condition that the product and company not be identified.
For 2002-2004, data from 59 therapeutic agents were submitted to the Pediatric Exclusivity Program; 137 trials were completed and 22,991 children were enrolled in the trials. Median number of children per trial was 116, and median number of clinical trials per agent was 2. Annual sales of the 59 products totaled $181,254,000. Final study reports were obtained for 9 drugs; 8 of these had a labeling change resulting from the studies. Twenty-seven clinical trials were completed: 16 evaluated efficacy, 4 were multidose pharmacokinetic, 6 were single-dose pharmacokinetic, and 1 was a safety study. The median number of patients enrolled was 140 at 16 United States sites; 48% took more than 2 years to finish. The estimated costs of conducting each trial varied considerably. The median cost was $12.34 million; the median cost for a single-dose pharmacokinetic study was $894,941, for a multidose pharmacokinetic study, $2,297,250, and for an efficacy study, $6,464,921. Median cash inflows were $140,447,244, assuming 6 months of exclusivity; inflows decreased proportionately when the exclusivity period was only 3 months. Median cast outflows were $10,362,062. For 6-month and 3-month exclusivity, the median net economic benefits were $134,265,456 and $64,041,833, respectively. The net return-to-cost ratio ranged from −0.68 to 73.63. Drugs studied included those for asthma, tumors, attention deficit/hyperactivity disorder, hypertension, depression/generalized anxiety disorder, diabetes mellitus, gastroesophageal reflux, bacterial infection, and bone mineralization.
Although the program has been successful from the perspective of conducting trials for drug labeling for children, the economic return is variable. Pediatric exclusivity can create financial benefits or modest returns on investments. However, the benefits from the new information relevant and applicable to the care of children are many and should not be compromised.
