Disclosures. Objectives 02/06/2017

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1 The 59 th annual meeting of the American Headache Society June 2017 Boston, Massachusetts Migraine Preventive Clinical Trial Data: Interpretation, Misinterpretation, and Integration into Future Study Designs and Clinical Practice Elizabeth Loder, MD, MPH Professor of Neurology Harvard Medical School Head of Research, The BMJ Disclosures No financial connections with any pharmaceutical or device companies I receive salary support from The British Medical Journal for work as the head of research Objectives Recognize common problems in the interpretation and presentation of clinical trial data, such as post-hoc analyses, subgroup analyses, and "spin Summarize relevant outcome measures when evaluating preventive treatment for migraine Describe 3 ways in which the design of preventive migraine trials can be improved to increase relevance to clinical practice and make the best use of trial data. 1

2 What is a clinical trial? The ICMJE defines a trial as any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions to evaluate the effects on health outcomes. The benefits of randomized trials The gold standard for determining treatment efficacy 2

3 Nonpublication or selective publication of trial results has been a big problem To deal with this, the ICME and journals that adhere to its recommendations require that all interventional clinical studies should be registered before the first participant is enrolled Tip: Check the trial registration website. Compare reported results to registered outcomes Primary and all secondary outcomes should be clearly identified and reported At the prespecified timepoints Any non-prespecified outcomes should be clearly identified as post-hoc analyses Beware of subgroup analyses 3

4 Tools to help with trial interpretation Several tools are available to help you interpret (and avoid misinterpretation) of clinical trial data. The CONSORT checklist is an evidence-based, minimum list of information that should be included in a trial report in order that readers will know how the study was designed, analyzed, and interpreted. (2) Tip: Print out a copy of the CONSORT checklist and systematically evaluate whether the recommended information is reported in the paper CONsolidated Standards of Reporting Trials The International Headache Society has published guidelines for clinical trials of migraine treatments. These guidelines were developed for drug trials, but many of the recommendations might reasonably be applied to trials of behavioral, surgical, or device treatments. (3) 4

5 Guidelines for controlled trials of drugs in migraine, 3d edition. The problem of spin Spin is defined as reporting that emphasizes the beneficial effects of the experimental (new) treatment Can distort findings and give patients and doctors unrealistic expectations Where does it occur? What are the effects? Misrepresentation of Randomized Controlled Trials in Press Releases and News Coverage: A Cohort Study Yavchitz A, Boutron I, Bafeta A, Marroun I, Charles P, et al. (2012) Misrepresentation of Randomized Controlled Trials in Press Releases and News Coverage: A Cohort Study. PLOS Medicine 9(9): e Examples include reporting positive effects that were not statistically significant, focusing on an outcome the trial was not designed to study, focusing on subgroups, or ignoring safety data. 5

6 How common is spin? Misrepresentation of Randomized Controlled Trials in Press Releases and News Coverage: A Cohort Study Yavchitz A, Boutron I, Bafeta A, Marroun I, Charles P, et al. (2012) Misrepresentation of Randomized Controlled Trials in Press Releases and News Coverage: A Cohort Study. PLOS Medicine 9(9): e What factors are associated? Misrepresentation of Randomized Controlled Trials in Press Releases and News Coverage: A Cohort Study Yavchitz A, Boutron I, Bafeta A, Marroun I, Charles P, et al. (2012) Misrepresentation of Randomized Controlled Trials in Press Releases and News Coverage: A Cohort Study. PLOS Medicine 9(9): e Tip: There are many good articles about how to identify spin in published research. HealthNewsReviewis an excellent resource for information about over-hyped medical research results. 6

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8 Integration of trial results into clinical practice and future trials Lessons learned help with design of future trials Placebo response rates in children Trials too short to identify long term adverse effects Clinical practice Could the patient in front of me have been in the trial? Thanks! 8