The European Medicines Agency’s release of documents that provide unparalleled information on the effectiveness and safety of medicines has come under legal attack, threatening a return to greater opacity.
The documents, called Clinical Study Reports (CSRs), provide a highly detailed picture of the design, conduct, analysis and findings of a trial, including data on the positive and negative effects experienced by patients. CSRs thus allow far deeper insight into a trial’s methodology and findings than other information sources do.
For example, a German team of experts found that CSRs provided over twice as much information on patient relevant outcomes than all other publicly available sources combined.
Several large-scale incidents of harm to patients in the recent past could have been averted if data contained in Clinical Study Reports had been accessible at the time.
Update: A paper by Jean-Marc Ferran and Sarah Nevitt on the data utility of CSRs published in November 2019 provides a good overview of research done to date using CSRs, including assessments of bias, detailed evaluations of drug harms, and evidence syntheses).
In 2016, the European Medicines Agency drew applause from numerous patient and transparency advocates by making a radical break with regulators’ traditional opacity and proactively releasing CSRs for newly approved drugs, as well as continuing to providing access to older CSRs on request.
This victory for patients is now under threat. Recently, in the context of an ongoing court case, an Advocate General issued a legal opinion warning that the previous court judgements might have been flawed, and that CSRs should no longer be released.
In response, the Management Board of the European Medicines Agency warned that:
‘’If the Court of Justice were to follow the recommendations set out in these opinions, EMA’s existing transparency policies would be impaired and have to be revised. Whilst waiting for the decisions of the Court of Justice, EMA Management Board members reiterate that making [data] available to the public… generates trust in the EU regulatory network, broadens the scientific knowledge base, fosters the development of medicines and ultimately benefits public health.”
Yannis Natsis, patient representative on the EMA board, told the BMJ that:
“This will mean that data secrecy will over-ride the public interest of disclosure which is now the default option. It will be a major blow to patient safety, scientific progress, and society at large. Pharmaceutical companies—one of the most secretive business sectors—have long fought against clinical trial transparency, harming patients. The public interest needs to prevail over narrow commercial interests.”
IQWIG, the institute that assesses the effectiveness of new medicines in Germany, stated that:
“High quality health care requires full information on study methods and results – information from clinical studies cannot be confidential.”
Professor Peter Goetzsche, Director of the Institute for Scientific Freedom, said:
"Lack of access to clinical study reports at drug agencies is immoral. It will result in thousands of unnecessary deaths because clinicians will be kept in the dark about how dangerous many drugs are. (...) But some drug companies try to wind the clock back to the dark ages."
Ancel.la Santos, senior health policy officer at consumer group BEUC and a long-time observer of the issue, told Politico that the legal opinion “threatens the public’s right to know” about the safety and efficacy of the medicines they take.
Legal experts noted that the case could undermine citizens' access to information from European Union institutions across the board.
By challenging the balance currently being struck between commercial interests and public interest in disclosure, it would likely impact the disclosure of other clinical trial data under the incoming Clinical Trials Regulation, and in general “would make it more difficult for EU citizens to access documents from all other EU institutions”, according to reporting by Pink Sheet.
Pharmaceutical companies had taken the European Medicines Agency to court to prevent the release of the documents, citing concerns about commercial confidentiality, despite the fact that such information is redacted prior to release.
In early 2018, the European General Court ruled in favour of continuing the release of CSRs. The Advocate General’s opinion comes in the context of an appeal against one of these rulings. The 2018 rulings are crucial because they form the legal backbone of the European Medicines Agency's current approach to transparency.
The Court of Justice, which is handling the appeal, is expected to reach a decision in late 2019 or early 2020.
Cochrane, Transparency International and TranspariMED in 2017 recommended that regulators worldwide should provide access to all Clinical Study Reports they hold.
Health Canada has recently started proactively releasing CSRs. To date, the U.S. Food and Drug Administration still refuses to release CSRs.
The study below explains in more detail what CSRs are and why they are so important.