Economic Return of Clinical Trials Performed Under the Pediatric Exclusivity Program

Department of Pediatrics, Duke Clinical Research Institute, Duke University, Durham, NC 27705, USA.
JAMA The Journal of the American Medical Association (Impact Factor: 35.29). 03/2007; 297(5):480-8. DOI: 10.1001/jama.297.5.480
Source: PubMed


In 1997, Congress authorized the US Food and Drug Administration (FDA) to grant 6-month extensions of marketing rights through the Pediatric Exclusivity Program if industry sponsors complete FDA-requested pediatric trials. The program has been praised for creating incentives for studies in children and has been criticized as a "windfall" to the innovator drug industry. This critique has been a substantial part of congressional debate on the program, which is due to expire in 2007.
To quantify the economic return to industry for completing pediatric exclusivity trials.
A cohort study of programs conducted for pediatric exclusivity. Nine drugs that were granted pediatric exclusivity were selected. From the final study reports submitted to the FDA (2002-2004), key elements of the clinical trial design and study operations were obtained, and the cost of performing each study was estimated and converted into estimates of after-tax cash outflows. Three-year market sales were obtained and converted into estimates of after-tax cash inflows based on 6 months of additional market protection. Net economic return (cash inflows minus outflows) and net return-to-costs ratio (net economic return divided by cash outflows) for each product were then calculated.
Net economic return and net return-to-cost ratio.
The indications studied reflect a broad representation of the program: asthma, tumors, attention-deficit/hyperactivity disorder, hypertension, depression/generalized anxiety disorder, diabetes mellitus, gastroesophageal reflux, bacterial infection, and bone mineralization. The distribution of net economic return for 6 months of exclusivity varied substantially among products (net economic return ranged from -$8.9 million to $507.9 million and net return-to-cost ratio ranged from -0.68 to 73.63).
The economic return for pediatric exclusivity is variable. As an incentive to complete much-needed clinical trials in children, pediatric exclusivity can generate lucrative returns or produce more modest returns on investment.

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Available from: Eric Eisenstein, Oct 05, 2015
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    • "Several reasons explain why clinical studies are performed less often in pediatric age groups than in adults. The specific ethical, methodological, and technical obstacles to pediatric trials are well recognized, as is the lack of financial rewards for the pharmaceutical industry [3], [6]–[10]. To address the paucity of pediatric research and to encourage investment by pharmaceutical companies, the United States and Europe have enacted new legislation about efficacy and safety drug trials in children [11]–[18]. "
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    ABSTRACT: The need for encouraging pediatric drug research is widely recognized. However, hospital-based clinical trials of drug treatments are extremely time-consuming, and delays in trial implementation are common. The objective of this qualitative study was to collect information on the perceptions and experience of health professionals involved in hospital-based pediatric drug trials. Two independent researchers conducted in-depth semi-structured interviews with principal investigators (n = 17), pharmacists (n = 7), sponsor representatives (n = 4), and drug regulatory agency representatives (n = 3) who participated in institutionally sponsored clinical trials of experimental drugs in pediatric patients between 2002 and 2008. Dissatisfaction was reported by 67% (16/24) of principal investigators and pharmacists: all 7 pharmacists felt they were involved too late in the trial implementation process, whereas 11 (65%) principal investigators complained of an excessive regulatory burden and felt they were insufficiently involved in the basic research questions. Both groups perceived clinical trial implementation as burdensome and time-consuming. The sponsor and regulatory agency representatives reported a number of difficulties but were not dissatisfied. The heavy burden related to regulatory requirements, and suboptimal communication across disciplines involved, seem to be the main reasons for the major delays in pediatric drug trial implementation. The pharmaceutical aspects are intrinsically tied to trial methodology and implementation and must therefore be examined, in particular by involving Clinical Research Pharmacists at early stages of study conception.
    PLoS ONE 05/2013; 8(5):e64516. DOI:10.1371/journal.pone.0064516 · 3.23 Impact Factor
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    • "Components—Economic models are frequently used to evaluate problems for which it is not feasible to collect empirical data and have previously been used in studies of clinical research methods and practices [3] [6] [7] [8]. We developed three clinical research network economic models to estimate the data management costs for conducting clinical research studies using one centralized and two decentralized EDC data management strategies, as described above. "
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    ABSTRACT: New data management models are emerging in multi-center clinical studies. We evaluated the incremental costs associated with decentralized vs. centralized models. We developed clinical research network economic models to evaluate three data management models: centralized, decentralized with local software, and decentralized with shared database. Descriptive information from three clinical research studies served as inputs for these models. The primary outcome was total data management costs. Secondary outcomes included: data management costs for sites, local data centers, and central coordinating centers. Both decentralized models were more costly than the centralized model for each clinical research study: the decentralized with local software model was the most expensive. Decreasing the number of local data centers and case book pages reduced cost differentials between models. Decentralized vs. centralized data management in multi-center clinical research studies is associated with increases in data management costs.
    Studies in health technology and informatics 01/2011; 164:82-8. DOI:10.3233/978-1-60750-709-3-82
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    • "One possible explanation is the impact of previously mentioned legislation in the US and subsequently the EU that brought in more tangible incentives and repercussions for drug manufacturers with regard to testing their products on children. The goal of this legislation was to encourage pediatric study; however, it has been shown that pediatric exclusivity provisions can generate lucrative returns on investment [25], [26], [27]. It would also be interesting to see if this legislation is related at all to the trend we noted of a decrease in child-only studies over time, as manufacturers may have moved to including children in larger studies in order to prolong market exclusivity of drugs with both pediatric and adult indications but proportionately a much larger adult market share. "
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    ABSTRACT: The objective of this study was to describe randomized controlled trials (RCTs) and controlled clinical trials (CCTs) in child health published between 1948 and 2006, in terms of quantity, methodological quality, and publication and trial characteristics. We used the Trials Register of the Cochrane Child Health Field for overall trends and a sample from this to explore trial characteristics in more detail. We extracted descriptive data on a random sample of 578 trials. Ninety-six percent of the trials were published in English; the percentage of child-only trials was 90.5%. The most frequent diagnostic categories were infectious diseases (13.2%), behavioural and psychiatric disorders (11.6%), neonatal critical care (11.4%), respiratory disorders (8.9%), non-critical neonatology (7.9%), and anaesthesia (6.5%). There were significantly fewer child-only studies (i.e., more mixed child and adult studies) over time (P = 0.0460). The proportion of RCTs to CCTs increased significantly over time (P<0.0001), as did the proportion of multicentre trials (P = 0.002). Significant increases over time were found in methodological quality (Jadad score) (P<0.0001), the proportion of double-blind studies (P<0.0001), and studies with adequate allocation concealment (P<0.0001). Additionally, we found an improvement in reporting over time: adequate description of withdrawals and losses to follow-up (P<0.0001), sample size calculations (P<0.0001), and intention-to-treat analysis (P<0.0001). However, many trials still do not describe their level of blinding, and allocation concealment was inadequately reported in the majority of studies across the entire time period. The proportion of studies with industry funding decreased slightly over time (P = 0.003), and these studies were more likely to report positive conclusions (P = 0.028). The quantity and quality of pediatric controlled trials has increased over time; however, much work remains to be done, particularly in improving methodological issues around conduct and reporting of trials.
    PLoS ONE 09/2010; 5(9). DOI:10.1371/journal.pone.0013106 · 3.23 Impact Factor
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