The European Medicines Agency and Publication of Clinical Study Reports

A Challenge for the US FDA

Anna L. Davis, JD, MPH¹; James Dabney Miller, JD, MPH¹

The US Food and Drug Administration (FDA) has been the standard of drug regulation worldwide for decades. In 1962, in response to the thalidomide tragedy, Congress directed the FDA to evaluate the effectiveness of drugs based on “adequate and well-controlled investigations, including clinical investigations” conducted by qualified experts.

Backed by this congressional enactment, the FDA crafted groundbreaking administrative regulations (eg, the requirements of a prespecified protocol, placebo or active controls, the phasing of clinical investigations, and informed consent from patients) that have guided the global clinical trial enterprise ever since.¹ The FDA’s regulations revolutionized the evaluation of drugs in the United States and abroad.

However, the European Medicines Agency (EMA), the counterpart to the FDA in the European Union, is poised to leapfrog ahead of the FDA in an equally important drug regulatory space of transparency. In October 2016, the EMA published on its website the clinical study reports for 2 drugs (carfilzomib and lesinurad) it recently approved. In so doing, the EMA enabled access to approximately 260 000 pages of detailed clinical trial information, including the protocol, statistical analysis, and detailed clinical data. Although some information was redacted to protect patient privacy, the EMA considered only 2 pages to contain “confidential commercial information.”

he EMA adopted policy No. 0070 to achieve the goals of “better informed use of medicines” and “to make medicine development more efficient” by allowing researchers to “learn from past successes and failures.” The EMA concluded that disclosure of detailed clinical data would enable the development of “new knowledge in the interest of public health.” Whether policy No. 0070 will achieve these goals is an unproven hypothesis, but, by publishing clinical study reports, the EMA has arranged a form of natural experiment that should allow an estimate of the actual benefit, if any, from its new policy.

However, policy No. 0070 faces legal uncertainty. In July 2016, the EU General Court issued an interim injunction that has the potential to undermine or reverse that policy.⁷ In that case, PTC Therapeutics submitted to the EMA a clinical study report of a phase 2 controlled efficacy study of ataluren for the treatment of Duchenne muscular dystrophy. Based on that clinical study report, the EMA conditionally approved ataluren in July 2014. Another unidentified pharmaceutical company requested a copy of the ataluren clinical study report. The EMA offered to redact several portions of the clinical study report, but PTC Therapeutics took the position that the entire clinical study report was confidential commercial information.The challenge the FDA must confront is that the clinical study reports submitted in support of drug marketing applications in the United States are basically the same as in the European Union.⁸ The FDA currently considers clinical data to be confidential commercial information, whereas the EMA does not. The EMA’s policy No. 0070—if it survives the current legal case with PTC Therapeutics—could lead to the anomaly that the EMA proactively publishes clinical study reports online (after decisions are made regarding marketing authorization applications), whereas the FDA withholds the same or similar clinical study reports. Despite the importance of this issue to public health, in neither the United States nor the European Union is there yet clear legal authority on whether clinical study reports should be made public and, if so, under what conditions.

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