Access to full data sets from clinical trials will benefit both large and small biopharmaceutical companies and researchers, according to an article published today in the New England Journal of Medicine by four members of the European Medicines Agency staff, including its Executive Director and Senior Medical Officer.   “A managed-release environment that allows sharing of patient-level data while ensuring patient privacy would create a level playing field for all stakeholders,” explain the authors.  In ‘Access to patient-level trial data – A boon to drug developersExternal link icon’, the authors argue that access to complete clinical-trial data, including patient-level data, will achieve the following:


Help increase the efficiency of drug development and improve cost effectiveness


Access to full data sets of completed studies will lead to improvements in the design and analysis of subsequent trials for which patient selection criteria will have been refined. Lessons learned from past trials about the heterogeneity of treatment effects will not only streamline drug development but may also enhance the value of a medicine in the marketplace by allowing the identification of patient populations with a high unmet need where the new medicine may be more cost-effective than other available treatment options.


Improve comparative-effectiveness analysis


Wider access to patient-level data will allow sponsors to present more robust comparative-effectiveness information about their product soon after licensing and at limited cost compared with head-to-head trials, which is essential information during reimbursement negotiations with payers.


Reduce duplication of effort among trial sponsors


One of the inherent inefficiencies of data secrecy is the repetition of unsuccessful trials and projects that cost millions of euros and unnecessarily expose trial subjects to potential risks, a drawback that would be avoided in the proposed alternative scenario.


In response to concerns related to the risk of misinterpretation of clinical trials, the authors argue that inappropriate secondary data analyses are likely to occur regardless of the nature of the data. To address this risk, they call for a two-way transparency principle by which any secondary analysis is also to be published and subject to critical review.


The European Medicines Agency recently published a draft policy on publication and access to clinical-trial data, including patient-level data. The Agency is currently reviewing the comments received on the draft policy as part of the three-month public consultation phase that ended on 30 September 2013.


Posted on the EMA website on 22 October 2013