Clinical trial data transparency: the EU inches closer to legal certainty


On 1 March 2017, the Vice-President of the Court of justice issued a ruling that sets up the opportunity for a much needed final adjudication of the parameters of commercially confidential information within clinical trial data[1].


To recap briefly on the facts of this case, PTC Therapeutics obtained a conditional orphan marketing authorisation (“MA”) for Translarna for the treatment of muscular dystrophy. Translarna was granted a conditional MA in May 2014 as an orphan product. Following the grant of this conditional MA, in October 2015 the EMA received an access to documents request pursuant to Regulation 1049/2001/EC (the “Transparency Regulation”). When this was submitted to PTC, the company objected to the scope of the request for disclosure on the basis that the entire clinical study report should be treated as commercially confidential. In November 2015, the EMA refused PTC’s request and issued a decision to grant the third party applicant access to the entire report at issue, subject to certain redactions made of its own initiative. Thereafter, PTC filed an annulment action combined with a request for interim orders to suspend the enforcement of the EMA’s decision, pending the outcome in the main proceedings.

On 20 July 2016, PTC was successful in obtaining interim orders, which was subsequently appealed by the EMA in September 2016. The 1st of March 2017 Order (the “Order”) dismissed the EMA’s appeal. Whilst limited to the ruling on the interim orders, the Court has provided a lengthy and reasoned opinion and reserved the key legal issues for determination on the merits.

Legal arguments

In brief, the EMA sought to argue that the entire CSR at issue could not be treated as confidential unless the applicant could demonstrate that each and every element of the report was confidential. Access to an entire document may only be refused if one or more of the provisions of Article 4 of the Transparency Regulation applies to the whole document. If confidentiality attaches to only one part then the remaining parts of the document must be disclosable.

In response, PTC raised the policy argument that disclosure would give a ‘road map’ to product development and any commercial advantage of orphan exclusivity would be lost if valuable know-how was disclosable to competitors. Moreover, competitors could use that data outside the EU i.e. in jurisdictions not protected by 10 years market exclusivity. PTC also submitted that clinical trial summaries are short and of a very different nature to the reports at issue in the present case.

The Order

The Court held that the General Court, in granting interim orders, did not err in law in considering that the argument advanced by PTC that there is a general presumption of confidentiality covering the report at issue is not manifestly unfounded. The Court also ruled that the dismissal of the application for interim measures and the subsequent disclosure of the report at issue by the EMA would clearly deprive any judgment annulling the decision at issue of any practical effect. On that basis, it was held that the dismissal of the application for interim measures would have de facto prejudged the future decision on the merits.

So, the Court has not directly said that there is a general presumption of confidentiality under the Transparency Regulation. Indeed, to do so would place the Court in direct conflict with the policy objectives of the EMA since 2010.  Nevertheless, whilst this issue is left open, it creates an anxious wait for both the EMA and industry on where the balance lies.


Before drawing general comparisons between this case and the wider debate on commercially confidential information, it should be emphasised the Translarna was granted a conditional MA. In order to obtain a definitive MA for that medicinal product, PTC would need to submit new clinical data to the EMA, complete ongoing studies or carry out new studies in order to confirm that there is a positive benefit/risk ratio for the product. In this respect, the CSR for Translarna can be regarded as a ‘living document’ and as such, a higher threshold should apply to the consideration of disclosable material within the CSR for Translarna compared to CSR’s submitted for finalised MAs.

Moreover, the fact that the product was an orphan was significant in this case; orphan products benefit from 10 years market exclusivity meaning that an MA will not be granted for a similar product for a similar therapeutic indication. Unless a competitor can establish that their product was safer, more effective or clinically superior (Art 8(3)). If a competitor can access comprehensive data of the innovator, it gives a ‘road map’ to product development and allow that competitor a step up / shortcut in product development. A second applicant would not rely upon the innovator’s data but it would be able to sift through comprehensive regulatory strategy, technical data and clinical trial design. This knowledge would enable it to streamline its own dossier and thereby potentially short circuit its regulatory approval timeframe.

From a general policy perspective, disclosure would create a platform for competitors to gain an accelerated pathway leading to MA grant. Any commercial advantage benefitting the innovator (i.e. orphan exclusivity) would be lost if valuable know-how was disclosable to competitors. Moreover, competitors could use that data outside the EU i.e. in jurisdictions not protected by 10 years market exclusivity.

The General Court’s ruling on the merits of the case, namely whether the contested decision should be annulled, is eagerly anticipated by the industry as a whole. The EMA has previously stated its desire for a ruling on the merits in order to enable a workable approach by the EMA to the redaction and disclosure of clinical data. There is no doubt that a judgment from the Court of Justice would be welcome by the entire pharmaceutical industry as it will assist greatly in advancing legal certainty in providing guidance on the interpretation on the boundaries of CCI.

[1] Case T-718/15 Order of the Vice-President of the Court of 1 March 2017 European Medicines Agency (EMA) v PTC Therapeutics International Ltd

Grant Strachan