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Forensic dissection of a clinical trial: lessons learned in understanding and managing interstitial cystitis.

ABSTRACT: A pharmaceutical company-sponsored post-registration clinical trial designed to determine the most effective dose of pentosan polysulfate for the treatment of interstitial cystitis (IC) reported negative results. However, because of a priori trial design features, important posthoc analyses were able to answer many of the important clinical issues on the epidemiology, diagnosis, and treatment of IC that to date had remained unanswered. Seven published follow-up reports based on data and outcomes from the original study evaluated the clinical significance of a positive Potassium Sensitivity Test, confirmed the O'Leary-Sant Interstitial Cystitis Symptom Index as a valid and sensitive outcome measure, and determined that doses of pentosan polysulfate higher than the standard US Food and Drug Administration-approved dose of 300 mg/d did not increase efficacy although increased duration of therapy increases the chance of symptom amelioration. Further analyses determined that sexual dysfunction is an important parameter to assess in IC and that successful therapy can improve sexual functioning. Finally, the data showed that symptom severity, quality of life (QoL), and sleep function are interrelated. Consequently, symptom improvement with therapy correlates with improvement in both sleep function and QoL. This post hoc forensic dissection of a clinical trial initially undertaken for simple regulatory reasons has significantly improved our understanding and management of the enigmatic condition we call IC.

SUBMITTER: Nickel JC

PROVIDER: S-EPMC2931285 | biostudies-literature | 2010

REPOSITORIES: biostudies-literature

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Forensic dissection of a clinical trial: lessons learned in understanding and managing interstitial cystitis.

Nickel J Curtis JC

Reviews in urology 20100101 2-3

A pharmaceutical company-sponsored post-registration clinical trial designed to determine the most effective dose of pentosan polysulfate for the treatment of interstitial cystitis (IC) reported negative results. However, because of a priori trial design features, important posthoc analyses were able to answer many of the important clinical issues on the epidemiology, diagnosis, and treatment of IC that to date had remained unanswered. Seven published follow-up reports based on data and outcomes ...[more]

PMID: 20811556

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Project description:BackgroundDesign and implementation of multi-country clinical trials for multidrug-resistant tuberculosis (MDR-TB) are complex for several reasons, including trial duration, varying levels of experience and infrastructure across settings, and different regulatory requirements. STREAM was an MDR-TB clinical trial that recruited over 1000 participants. We documented challenges and best practices/lessons learned from the site perspective to improve implementation of future trials.MethodsWe conducted a voluntary survey of trial staff at all sites to obtain information on challenges encountered and best practices/lessons learned from implementation of the STREAM trial. Respondents were asked to identify substantive aspects of trial implementation from a list that included: trial administration, laboratory strengthening/infrastructure, pharmacy and supply chain management, community engagement, regulatory and ethics requirements, health economics, and other (respondent designated) about which a practical guide would be useful to improve future trial implementation. For each aspect of trial implementation selected, respondents were asked to report challenges and best practices/lessons learned during STREAM. Lastly, respondents were asked to list up to three things they would do differently when implementing future trials. Summary statistics were generated for quantitative data and thematic analysis was undertaken for qualitative data.ResultsOf 67 responses received from 13 of 15 sites, 47 (70%) were included in the analyses, after excluding duplicate or incomplete responses. Approximately half the respondents were investigators or trial coordinators. The top three aspects of trial implementation identified for a best practices/lessons learned practical guide to improve future trial implementation were: trial administration, community engagement, and laboratory strengthening/infrastructure. For both challenges and best practices/lessons learned, three common themes were identified across different aspects of trial implementation. Investment in capacity building and ongoing monitoring; investment in infrastructure and well-designed trial processes; and communication and coordination between staff and meaningful engagement of stakeholders were all thought to be critical to successful trial implementation.ConclusionsExisting practices for clinical trial implementation should be reevaluated. Sponsors should consider the local context and the need to increase upfront investment in the cross-cutting thematic areas identified to improve trial implementation.

Dataset Information

Forensic dissection of a clinical trial: lessons learned in understanding and managing interstitial cystitis.

Publications

Forensic dissection of a clinical trial: lessons learned in understanding and managing interstitial cystitis.

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