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The Need for Clinical Trial Registries

WIDESPREAD AGREEMENT THAT SOME FORM OF REGISTRY IS NEEDED

Clinical trial registries have four potential functions: (1) list and track the status of clinical trials, (2) provide information for patient recruitment, (3) provide a complete record of all trials to aid in doing systematic reviews of the evidence, and (4) report results of the trials. This information has many audiences: patients, health care providers, researchers, medical journal editors, pharmaceutical companies, health insurers, and regulators. These audiences have differing needs for data and for translation of information contained in the registry; however, they all have needs that are not adequately met by other sources.

Selective publication of clinical trial data does not provide a complete picture. Both advertently and inadvertently, there is an understandable bias toward disclosure of favorable results, commonly known as the “file drawer phenomenon.” The pharmaceutical industry does not have a compelling reason to publish the results of clinical trials that do not support the introduction of new products. Researchers and journal editors are generally more enthusiastic about publishing positive results than negative results. When the results are inconclusive, there is much less interest in peer-reviewed publication. For both safety and efficacy data, outcomes are more likely to be reported if there are statistically significant differences.

A recent empirical study found that 62 percent of 82 publicly and privately funded randomized controlled trials had major discrepancies between the primary outcomes specified in the protocols and those reported (Chan et al., 2004). Reports may also appear without acknowledgment that they are one of a larger corpus of evidence and that other



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Developing a National Registry of Pharmacologic and Biologic Clinical Trials: Workshop Summary 2 The Need for Clinical Trial Registries WIDESPREAD AGREEMENT THAT SOME FORM OF REGISTRY IS NEEDED Clinical trial registries have four potential functions: (1) list and track the status of clinical trials, (2) provide information for patient recruitment, (3) provide a complete record of all trials to aid in doing systematic reviews of the evidence, and (4) report results of the trials. This information has many audiences: patients, health care providers, researchers, medical journal editors, pharmaceutical companies, health insurers, and regulators. These audiences have differing needs for data and for translation of information contained in the registry; however, they all have needs that are not adequately met by other sources. Selective publication of clinical trial data does not provide a complete picture. Both advertently and inadvertently, there is an understandable bias toward disclosure of favorable results, commonly known as the “file drawer phenomenon.” The pharmaceutical industry does not have a compelling reason to publish the results of clinical trials that do not support the introduction of new products. Researchers and journal editors are generally more enthusiastic about publishing positive results than negative results. When the results are inconclusive, there is much less interest in peer-reviewed publication. For both safety and efficacy data, outcomes are more likely to be reported if there are statistically significant differences. A recent empirical study found that 62 percent of 82 publicly and privately funded randomized controlled trials had major discrepancies between the primary outcomes specified in the protocols and those reported (Chan et al., 2004). Reports may also appear without acknowledgment that they are one of a larger corpus of evidence and that other

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Developing a National Registry of Pharmacologic and Biologic Clinical Trials: Workshop Summary similarly designed trials with the same agents may have drawn different conclusions. Usually a body of evidence, rather than a single trial, is required to influence the thinking of clinicians, researchers, patients, and medical policy experts. This body of evidence, in fact, forms the basis of regulatory review and approval of pharmaceutical products. Responsible decision making requires awareness of both positive and negative clinical trial results, along with confidence that the findings are available and accessible. Accordingly, key stakeholders in the medical community have suggested that if clinical trials were registered in a systematic fashion at their inception, followed by the posting of summary results for the study, and both were easily accessible to interested parties, then the full range of clinical evidence surrounding an investigative therapy would become part of the public record. In addition, once a drug has been approved, the registry provides a complete record that can serve as input for decisions by guideline developers, insurers, and those who monitor quality of care in the United States. DIVERSE EXPECTATIONS AND PERCEIVED NEEDS The public and various entities within the medical community have different expectations and perceived needs regarding a public clinical trial registry: Individuals suffering from various diseases, and their family members, want to know that appropriate therapies are being offered and that patient safety is being secured. In addition, more and more patients today want the ability to search on their own for research that pertains to their disease and potentially to enroll in a clinical trial if suitable. Health care professionals need both unbiased summary information derived from all trials conducted on a drug or therapy, and the capacity to look at the clinical data from any single study. They do not want to confine their review to the approved drug labeling or articles published in medical journals.

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Developing a National Registry of Pharmacologic and Biologic Clinical Trials: Workshop Summary Researchers may generate new ideas for investigations or look for data trends by accessing all the trials conducted on a drug or therapy. Medical journals have an enormous impact on clinical practice and medical policy. When journal editors receive clinical trial manuscripts for publication, they are concerned that they fully understand the research. They want to know if clinical trials exist that may conflict with the submitted manuscript. Furthermore, they want to know if the authors failed to follow the original research plan because discrepancies may reflect serious defects in the research. Indeed, the integrity of the journal is at stake, as is the entire scientific enterprise, when research is published through the peer-review process. Regulators would find the information in a registry useful in developing policies regarding clinical research. Health insurers want to keep abreast of evidence-based results as the basis for insurance coverage policy. Sponsors of research who are developing a new therapy or drug incur great expense. Some of the information is highly proprietary and confidential to the parties who sponsor research. Industry is concerned that if all proprietary information were required to be made broadly available to the public at the outset of clinical trials, then they could not recoup their investment because competitors in the United States or abroad copied their innovations. At the same time, they recognize their responsibility to do everything possible to assure patient safety and to secure the public trust. In striking a balance among these competing interests regarding the scope of clinical trial registries, an overriding goal is to sustain public trust in the integrity of clinical research and in the process for translating research into new drugs and biologics.

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