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The EMA is committed not just to greater accessibility of data, but proactive publication.
Everyone in Europe, it seems, is looking at transparency. It isn't just the revelations about US espionage of EU institutions that have provoked the frenzy—although tensions have certainly been intensified as a result of the new attentions to who is hiding what from whom. But as this column noted last month, transparency had already become one of the dominant themes of the current debates over new clinical trial rules for the European Union. Confusingly, transparency is also the focus of the EU directive on pricing and reimbursement of medicines, which is also under review at present (the so-called Transparency Directive governs how much information national authorities have to provide over how they reach their pricing and reimbursement decisions). Even more confusingly, controversy has flared over the last few weeks about the merits of the EU Transparency Register—the list of EU lobbyists, including numerous drug firms; this is under new attack from campaigners demanding tighter controls over big business contacts with EU officials and politicians. Berlin-based Transparency International concluded in its annual corruption index, released in July, that medical and health services are subject to corruption across most of eastern Europe. And most recently, the European Medicines Agency (EMA) has released its draft policy on the publication and access to clinical trial data.
For the clinical trial community, this is likely to be among the most crucial transparency developments for a long time. EMA has been under repeated attack for years for sitting on data that other researchers—often those critical of EMA decisions—claimed rights of access to in the name of good science. When Guido Rasi took over as the new boss of EMA, one of his first public promises was to open up some of the information vaults. The policy paper that emerged in late June bears the hallmark of those promises, and is explicitly driven by a belief that the release of data is important in establishing trust and confidence in the system. Avid students of EU policy on medicines are advised to take a look at it. There is a limited window for submitting comments, which closes on September 30, 2013, and EMA expects the policy to come into force on January 1, 2014.
The nub of the EMA commitment is not just greater accessibility of data, but proactive publication. Data will be released automatically, unless there is a compelling case for withholding. The default, in other words, becomes automatic publication. In this draft, what will be published is data from clinical trials submitted in support of a marketing-authorization application, once the decision-making process has ended. EMA describes its approach as "considered"— in that it claims to respect the views and concerns emitted so far on the subject. In reality, this boils down to a search for balance between the competing claims of access to data for independent scrutiny, the need to protect personal data, and the sensitivity of "legitimate commercially confidential information."
The draft policy offers three categories of clinical trial data. "Commercially confidential information" would cover details of an investigational medicinal product, some in vitro studies or bioanalytical data characterizing the product, suggests EMA. An "open access" category, covering any clinical trial data, information or documents that do not contain patients' personal data, would be downloadable from the EMA website once a European public assessment report had been generated on the product concerned. And 'controlled access' would cover information containing patients' personal data, such as individual patient data sets, patient line-listings, or case report forms, as well as documentation explaining the structure and content of data sets. For this category, to prevent retroactive patient identification, de-identification of data and a data-sharing agreement would also be required.
EMA's ambitions are, it admits, subject to some external complications—not unrelated to the fact that everyone in Europe seems to be looking at transparency.
One, of course, is "the ongoing legislative process to replace the current European directive on clinical trials," the draft EMA policy acknowledges with an almost perceptible sigh. As it goes on to point out, the position agreed in May by the European Parliament's health committee states that "in general the data included in clinical trial study reports should not be considered commercially confidential once a marketing authorization has been granted or the decision-making process on an application for marketing authorization has been completed." The committee's decision is not final. The Parliament as a whole still has to reach its formal position, which will probably take until the end of this year—and maybe longer. But the sentiment in the Parliament is strongly in favor of maximum transparency, inflamed by anger over PRISM, and resentment at the alleged readiness of Internet giants to comply with state demands for access to data.
Another of the potential impediments to the EMA plan lies with the European Union's courts. As EMA points out, with studied neutrality, "a number of court cases are currently ongoing that challenge the agency's 2010 access-to-documents policy. These cases will bring the opportunity for legal clarification of the concept of commercially confidential information." The reality is less neutral. AbbVie has led a frontal attack on EMA policy on information release, principally to defend its rheumatoid arthritis drug Humira (adalimumab). In April, AbbVie won provisional support from the EU court against the EMA's plan to prevent publishing data. Similar temporary protection was provided by the court to US biotech firm InterMune, which is likewise fighting EMA policy because it fears release of data on its idiopathic pulmonary fibrosis medicine, Esbriet (pirfenidone) will give advantages to competitors.
These interim injunctions have enraged campaigners for access to trial data. The European consumers organization, BEUC, has lined up alongside EMA in fighting the case, and "hopes to lend additional support in EMA's effort to continue its policy of openness." BEUC urges the merits of publication of clinical data reports to "help restore trust in public regulatory authorities; trust which has suffered because of medicines scandals such as Mediator in France." In addition, "public access to clinical data can lead to additional scrutiny of decisions about the approval of medicines," counteracting the trend under which "regulators often rely solely on the pharmaceutical company's own analysis of their products' benefit-harm balance." The tone demonstrates how febrile the atmosphere is becoming.
Meanwhile, the European pharmaceutical industry's main trade body, the European Federation of Pharmaceutical Industries and Associations is finalizing its own approach to making clinical data available on drugs. This is likely to focus on separating information useful to researchers from information for the general public. One of the principal aims is to prevent competitors' access to unlimited data on such sensitive issues as production methods. But according to Christopher Viehbacher, Sanofi's CEO, the EFPIA President, sharing data between companies could also speed up research.
Against that tempestuous background, it is a relief to be able to comment briefly on an attempt to bring transparency to clinical trials in a less contentious manner. The British BioIndustry Association has just released a guide to running trials in the United Kingdom with a claim that "effectively navigating clinical infrastructure can streamline research and de-risk investment." It highlights the role of the National Office for Clinical Research Infrastructure (NOCRI) as a single point of contact offering a bespoke service to clinical research sponsors.
The guide takes the reader by the hand, from addressing study requirements and obtaining expert guidance through to late-state research. Along the way it explains the new service available since June to help life sciences companies with a single online application gateway for all studies, customized assistance including early feedback on study deliverability at protocol development stage, site intelligence, and a streamlined process for applications offering turn around in 15 working days. And it outlines the model Industry Collaborative Research Agreement designed to support clinical research collaborations involving industry, academia, and public health organizations across the United Kingdom. Its template clauses can be brought together to form a contract, offering a range of options for handling the ownership of any intellectual property rights resulting from the collaboration. It is an interesting example of the competitive sophistication that these days seems almost as vigorous as drug discovery itself, as countries and institutions fight for a share of the clinical trials market.
Finally, good news this month from the European Union (a rare occurrence in itself these days) with the adoption of plans for Horizon 2020, the seven year multi-billion research project that should start next year—on the assumption some last-minute squabbles over funding are resolved in time. For the clinical trials community, this could mean some big new money in support of research projects. Watch out for IMI-2, the second phase of the Innovative Medicines Initiative that has backed public-private partnerships in early-state research over the last seven years. Part of Horizon 2020, IMI-2 promises to be bigger and better than its predecessor, with a budget of more than $3 billion—and a focus on target identification and biomarker research, innovative clinical trial design, and innovative medicines. That's the sort of transparency about the future that everyone likes.
Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium.