re Rx: Dedicated to exploring, informing and reflecting on the world of repurposing research
Dr. Teresa McNally, October 3, 2013
Detailed clinical trial data are considered to be valuable information by both the pharmaceutical industry and the public sector. This information is critical for drug repurposing efforts where knowledge of the efficacy, off-target and side effect profiles of existing therapies are key to identifying new therapeutic opportunities. If detailed clinical data for a drug is publicly available, the time and effort required for a successful repurposing strategy are much reduced.
Issues regarding clinical data transparency, have been hotly debated in the medical literature recently. A summary of results of successful clinical trials are often published, but detailed information, and key negative data are rarely available despite existing legislation that commands data transparency. The European Medicines Agency (EMA), the International Committee of Medical Journal Editors (ICMJE) and the US Food and Drug Administration (FDA) and the declaration of Helsinki, the World Medical Association doctrine on ethical principles for medical research involving human subjects, all have mandates related to clinical trial transparency and data-sharing. Few, however, have a clear policy regarding precisely what information should be made publicly available, how it should be made accessible, who should have access and who is responsible for collating, depositing, updating and policing the information?
This year, the AllTrials campaign established a database and guidelines for consistent and centralized reporting of all clinical trial data, for both new and for existing medications. This is designed to address many of the issues of transparency. The campaign is a joint initiative of Bad Science, the British Medical Journal, the Center for Evidence-based Medicine, the Cochrane Collaboration, the James Lind Initiative, PLOS and Sense About Science and is being led in the USA by Dartmouth’s Geisel School of Medicine and the Dartmouth Institute for Health Policy & Clinical Practice. A petition supporting the organization and its goals has been signed by more than 250 research organizations worldwide. Most notably the pharmaceutical giant Glaxo SmithKline have stated their support for the campaign.
GSK has led the pharmaceutical industry in clinical data transparency. In 2004, they were the first pharmaceutical company to introduce a publicly available database of clinical data with more than 5000 summaries of their clinical studies. This year, they further committed to provide access to all clinical study reports (CSRs) for new medicines, whether approved or disapproved by regulators, going back to the formation of the company in 2000. Along with this summary data, GSK is also willing to share anonymized patient data with organizations who provide a scientific protocol on how they will use the data, and a commitment to publishing their findings.
James Shannon, the Chief Medical Officer of GSK stated “Our commitment to transparency of clinical trial data reflects our wish to help advance scientific understanding and enable the scientific community to learn from the research we have undertaken. It also allows us to acknowledge the great contribution made by the people who take part in our clinical research. When people volunteer for clinical trials they expect that the results from those trials will be used to help others. We have a responsibility to ensure that happens.”
More pharmaceutical companies and research organizations are expected to follow the GSK lead in clinical data transparency. This should lay the groundwork for more collaborative research opportunities. Broader and deeper investigation of this existing clinical data will help future drug repurposing efforts. The availability of negative clinical data will identify compounds that are safe in humans, but ineffective in the disease model tested. Successful repurposing will match these “orphan” drug candidates with areas of unmet medical needs. 2013 has been a year of great progress so far.