European Court of Justice rules on the transparency of clinical data for new drug approvals.

RB – 02/2020

On 22 January, the European Court of Justice confirmed the right of access to documents contained in the file of an application for marketing authorisation for medicinal products. This ruling confirms that the European Medicines Agency (EMA) has the right to operate in a transparent manner and publish the results of clinical trials of medicinal products submitted as part of the marketing authorisation application.

In July 2014, the manufacturer PTC Therapeutics International Ltd. was granted marketing authorisation for Translarna, a medicinal product for treating the rare disease Duchenne muscular dystrophy (DMD). In October 2015, PTC Therapeutics was notified by the EMA that a competing manufacturer had been granted access to a clinical trial report on the drug from the marketing authorisation application. PTC Therapeutics was of the opinion that the clinical trial contained in the report is essential to the granting of marketing authorisation and is subject to confidentiality. In November 2015, the EMA rejected the request for confidentiality of the report and took the decision to grant third parties access to the entire disputed report, apart from their own redaction.

PTC Therapeutics filed the case on the decision to publish the clinical report in December 2015. The case went through the stages of appeal to the European Court of Justice and was ruled in January 2020.

The judges based their ruling on the requirements of Regulation 1049/2001 to ensure transparent public access to European Parliament, Council and Commission documents as well as Regulation 536/2014 in conjunction with the ruling made in 2018 on access to clinical trial documents.

The ECJ confirmed that the general presumption of confidentiality is optional rather than mandatory. The judgment clarified that a holder of a marketing authorisation must demonstrate, in a reasonably foreseeable and concrete manner, how access to documentation would undermine its commercial interests. In this case, PTC Therapeutics failed to demonstrate and identify specifically and precisely how disclosure of the passages of the report at issue could harm its commercial interests. As a result, the EMA was within its rights to disclose the information solely on the basis of the non-confidential nature of the report without the need to weigh up the interests involved. The mere unsubstantiated allegation of a risk of abuse by competing companies was not sufficient in the eyes of the court, especially since PTC Therapeutics had the opportunity to comment on the confidentiality of the documents but did not do so.