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  • Want data on drugs? Look for clinical study reports

    Oct 24, 13 Clinical Updates

    For information on how drugs work and affect people, a new study says regulatory reports that are rarely published are better sources than the typical documents that are publicly available.

    German researchers found that so-called clinical study reports produced by pharmaceutical companies had complete information on 86 percent of study outcomes that are relevant to patients. In contrast, only about 39 percent of those outcomes were found in other sources, such as studies published in academic journals.

    “For the first time, we know how much information we can gain from the types of documents we investigated,” lead author Beate Wieseler said.

    Wieseler is the head of drug assessment at the Institute for Quality and Efficiency in Health Care (IQWiG). IQWiG is a German agency that examines the risks and benefits of medical interventions.

    Extra information is needed for various reasons, including the creation of medical guidelines and treatment decisions between doctors and patients, she said.

    But clinical study reports, which can be hundreds or thousands of pages long, are not usually available to the public.

    “I don’t know when it was decided or how it was decided, but the (U.S. Food and Drug Administration) considers this information proprietary and doesn’t give it to anyone else,” Dr. Joseph Ross said.

    Ross, who was not involved in the new study, has researched the availability of study results at the Yale School of Medicine in New Haven, Connecticut.

    For the new analysis, published in PLOS Medicine, Wieseler and her colleagues analyzed trials on 16 drugs that IQWiG examined between 2006 and 2011.

    They compared the information in the private clinical study results to publicly available sources, such as academic journals, the U.S. government-run website clinicaltrials.gov and a registry managed by the World Health Organization, to see which included more information on patient-relevant outcomes.

    Those outcomes include events such as the number of study participants who died or experienced serious side effects, how patients responded to the drug and effects it had on their quality of life.

    “The thing that’s interesting about this report is that clinical study results are not systematically available so people have not been able to study it,” Ross said.

    Overall, the researchers identified 1,080 patient-relevant outcomes for the drugs. Of those, the clinical study reports had complete information on almost nine out of 10. The publicly available sources, however, only had complete information on about four of 10.

    “It’s not that surprising that unpublished clinical study reports have more data and results than published study results,” Ross said.

    Wieseler said that the U.S. Food and Drug Administration (FDA) and its European counterpart the European Medicines Agency could start publishing clinical study reports on their websites to give people access to the data.

    A FDA spokesperson, however, told Reuters Health in an email that federal law prohibits the agency from “making a fair amount of clinical data publicly available.”

    “The issue of clinical trial data sharing - for a lack of better word - is a hot topic at the moment,” Jeff Francer, vice president and senior counsel at the Pharmaceutical Research and Manufacturers of America (PhRMA), said.

    PhRMA represents biopharmaceutical researchers and biotechnology companies.

    “In many cases, companies and researchers have been sharing data for quite some time really under the radar,” he said. “And I think companies are comfortable sharing clinical data with qualified researchers” as long as they protect patient privacy and use the data in an appropriate way.

    In July, PhRMA and the European Federation of Pharmaceutical Industries and Associations adopted Principles for Responsible Clinical Trial Data Sharing (onphr.ma/1ie4SYy).

    Under those principles, which take effect January 1, companies will make synopses of the clinical study reports that are submitted to government approval organizations available to the public.

    “Each company is going to be going about it a different way,” Francer said. “The key is that all of our members will have information on their websites about where (the public) can find this information.”

    As part of the adopted principles, researchers and scientists can also request clinical trial data from companies for medicines and indications approved in the U.S. and the European Union.

    Preparing to share patient-level data takes the most work, according to Francer.

    For example, companies need to address privacy commitments that are made to trial participants, create data request applications and put together boards to review those requests.

    Implementing the principles will be an evolutionary process, Francer said.

    “We also hope the academic community and government researchers will be just as open as industry researchers are going to be starting now,” he added.

    Ross said making clinical trial data available will help patients better understand the risks and benefits of drugs and medications.

    “It needs to be in a place where people can access it and understand it to make informed decisions on what to use,” he said.

    SOURCE: PLOS Medicine, online October 8, 2013

    Completeness of Reporting of Patient-Relevant Clinical Trial Outcomes: Comparison of Unpublished Clinical Study Reports with Publicly Available Data

    Access to unpublished clinical study reports (CSRs) is currently being discussed as a means to allow unbiased evaluation of clinical research. The Institute for Quality and Efficiency in Health Care (IQWiG) routinely requests CSRs from manufacturers for its drug assessments.

    Our objective was to determine the information gain from CSRs compared to publicly available sources (journal publications and registry reports) for patient-relevant outcomes included in IQWiG health technology assessments (HTAs) of drugs.
    Methods and Findings

    We used a sample of 101 trials with full CSRs received for 16 HTAs of drugs completed by IQWiG between 15 January 2006 and 14 February 2011, and analyzed the CSRs and the publicly available sources of these trials. For each document type we assessed the completeness of information on all patient-relevant outcomes included in the HTAs (benefit outcomes, e.g., mortality, symptoms, and health-related quality of life; harm outcomes, e.g., adverse events). We dichotomized the outcomes as “completely reported” or “incompletely reported.” For each document type, we calculated the proportion of outcomes with complete information per outcome category and overall.

    We analyzed 101 trials with CSRs; 86 had at least one publicly available source, 65 at least one journal publication, and 50 a registry report. The trials included 1,080 patient-relevant outcomes. The CSRs provided complete information on a considerably higher proportion of outcomes (86%) than the combined publicly available sources (39%). With the exception of health-related quality of life (57%), CSRs provided complete information on 78% to 100% of the various benefit outcomes (combined publicly available sources: 20% to 53%). CSRs also provided considerably more information on harms. The differences in completeness of information for patient-relevant outcomes between CSRs and journal publications or registry reports (or a combination of both) were statistically significant for all types of outcomes.


    Beate Wieseler,
    Natalia Wolfram,
    Natalie McGauran,
    Michaela F. Kerekes,
    Volker Vervölgyi,
    Petra Kohlepp,
    Marloes Kamphuis,
    Ulrich Grouven

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