The last five years mark the beginning of a revolution in clinical research: data collected on patients treated in clinical trials to support approval of new drugs and devices, which for a long time were considered confidential, will from now on be shared with a view to enhancing public health and informing future research. In 2013, the European Medicines Agency set a new standard for clinical trial data transparency by enabling interested parties to request data from clinical trials submitted for marketing authorization of medicinal products, including patient-level data (European Medicines Agency 2013). This followed a previous policy of this agency on access to clinical trial documents (Bonini et al, 2014).
In this webinar, first the principles, opportunities, complexities, and risks of data sharing will be reviewed. Documents from the Pharmaceutical Research and Manufacturers of America and European Federation of Pharmaceutical Industries and Associations (PhRMA and EFPIA 2011) will be referred to, as well as a detailed report from the Institute of Medicine (2015) which discusses the principles underlying data sharing and methods aimed at maximizing benefits and minimizing the risks of data sharing. Essentially, data sharing should protect patient privacy for research participants, preserve the integrity of regulatory systems, and protect intellectual property to maintain incentives for investments in biomedical research. In this context, methods of data de-identification, data standardization (e.g. CDISC), and data coding (i.e., medication procedures, adverse events) will be discussed. Also, the various types of analysis that can be conducted with patient-level data will be outlined.
In parallel with the above efforts some members of the pharmaceutical industry have created websites that provide access to patient-level clinical trial data for qualified investigators who submit a proposal that respects the principles of responsible research. GlaxoSmithKline put in place an independently managed website (clinicalstudydatarequest.com), and an Independent Review Panel to assess the research proposals submitted to access patient-level data (Nisen and Rockhold 2013). Eleven further companies joined this effort since (Astellas, Bayer, Boehringer-Ingelheim, Eisai, Lilly, Novartis, Roche, Sanofi, Takeda, UCB and ViiV Healthcare). As of January 2016, the Wellcome Trust will take over the responsibility of managing the website and Independent Review Panel on behalf of these companies. Yale University has set up a similar system, YODA (Yale Open Data Access at yoda.yale.edu), which is currently used by Johnson and Johnson and Medtronic. Another initiative, Project Data Sphere (projectdatasphere.org), provides access only to the control groups of clinical trials. As such it does not meet the basic principles of data transparency and will not be discussed here.
The first year of experience with sharing of pharmaceutical trial data has been previously reported (Strom et al 2014). Here, an update of this report will be done and review the 100 first proposals submitted to clinicalstudydatarequest.com, in terms of the number of studies with relevant data, disease areas, study designs and statistical methods. It will be shown, that these proposals largely fulfill the promise of data sharing through further analyses of the data from individual trials in meta-analyses, with a view to establishing better prognostic models, identifying as yet unknown predictive factors, confirming and optimizing treatment effects, developing and validating new statistical approaches, and facilitating the design of future clinical trials.
The webinar will be concluded by discussing future perspectives, including full access to all data, including biomarker and –omic data, access to analysis code for reproducible research, involvement of principal investigators in the research process, and access to data from academic trials.
Bonini S, Eichler HG, Wathion N, and Rasi G. Transparency and the European Medicines Agency – Sharing of clinical trial data. N Engl J Med 2014; 371: 2452-5.
European Medicines Agency. Publication and access to clinical-trial data, 2013. www.ema.europa.eu/ema/pages/includes/document/open_document.jsp?webContentId=WC500144730
Institute of Medicine. Report: Sharing clinical trial data: maximizing benefits, minimizing risk, January 2015. http://iom.nationalacademies.org/Reports/2015/Sharing-Clinical-Trial-Data.aspx?_ga=1.42879139.740695931.1448773346
Nisen P, Rockhold F. Access to patient-level data from GlaxoSmithKline clinical trials. N Engl J Med 2013; 369: 475-478.
PhRMA and EFPIA. Principles for responsible clinical trial data sharing. Our commitment to patients and researchers, 2011. http://www.phrma.org/sites/default/files/pdf/PhRMAPrinciplesForResponsibleClinicalTrialDataSharing.pdf
Strom BL, Buyse M, Hughes J, Knoppers BM. Data sharing, year 1 – Access to data from industry-sponsored clinical trials. N Engl J Med 2014; 371: 2052-4.
Marc Buyse, ScD, Chief Scientific Officer, International Drug Development Institute (IDDI)
Marc Buyse holds a ScD in biostatistics from the Harvard School of Public Health (Boston, MA). He is the founder of the International Drug Development Institute (IDDI) and of CluePoints, two biostatistical service organizations based in the US and Europe. He is interested in clinical trial design, meta-analysis, validation of biomarkers and surrogate endpoints, statistical methods in oncology, statistical detection of errors and fraud, statistical monitoring of clinical trials, and medical data sharing (http://publicationslist.org/marc.buyse). He served on the first Independent Review Panel for clinicalstudydatarequest.com
Frank Rockhold, PhD, Senior Vice President, Safety and Pharmacovigilance, GlaxoSmithKline
Frank Rockhold is Senior Vice President, Safety and Pharmacovigilance, GlaxoSmithKline and Professor of Biostatistics and Bioinformatics, Duke University. He holds a PhD in Biostatistics from the Medical College of Virginia. He has had diverse leadership experience in clinical trials, data standards, benefit/risk, clinical research, epidemiology, cardiovascular development, and most recently pharmacovigilance. He has served on the board of directors of the non-profit Clinical Data Interchange Standards Consortium and is past president of the Society for Clinical Trials. He currently serves on the board of directors of the nonprofit Frontier Science and Technology and Research Foundation and is also a member of the nonprofit Patient Centered Outcomes Research Institute Advisory Panel on Clinical Trials. He has been a leader in promoting data disclosure and transparency in clinical research. He has chaired the GSK operations and governance board for data disclosure, which covers disclosure of protocols, clinical trials summaries, study reports as well as individual patient level data.
Who Should Attend?
- Clinical Trialists
- Clinical Development
- Medical Experts
International Drug Development Institute (IDDI) is an expert center in biostatistical and integrated eClinical services for Pharmaceutical and biotechnology companies in several disease areas, including oncology and ophthalmology.
IDDI optimizes the clinical development of drugs, biologics and devices thanks to proven statistical expertise and operational excellence. Founded in 1991, IDDI has offices in Belgium, Boston (MA), Raleigh (NC) and San Francisco (CA).