Balancing transparency and confidentiality: orphan drug producer fails to prevent disclosure of clinical trial report

New Jersey-based biotech company Amicus Therapeutics focuses on treatments for rare metabolic diseases. Its lead product, Galafold (migalastat), received European marketing approval for the treatment of Fabry disease in May 2016. The drug benefits from an orphan drug designation, which brings with it advantages like protocol assistance and ten years of market exclusivity. 

Later in 2016 Amicus learned that the European Medicines Agency (the EMA) had been asked for documents underlying the marketing authorisation, including the clinical study report, under the Transparency Regulations. These regulations allow public access to EU institution documents.

Is the clinical study report confidential?

Amicus proposed limited redactions to the report. The EMA accepted these and informed Amicus of its decision to disclose the redacted report. Shortly afterwards, Amicus changed its mind and told the EMA it considered the whole report to be confidential. Amicus argued that the report should not be disclosed based on the exception to the right of access in Article 4(2) of the Transparency Regulations:

“The institutions shall refuse access to a document where disclosure would undermine the protection of commercial interests of a natural or legal person, including intellectual property…unless there is an overriding public interest in disclosure.”

The EMA disagreed with Amicus. It relied on Article 4(6) - any parts of a document that are not covered by an exception should be disclosed. So access to the document could only be refused if the entire document was shown to be confidential. Amicus asked the European court to overrule this decision.

The European court’s decision 

Amicus argued that clinical study reports should benefit from a general presumption of confidentiality. But the court was very clear that the right to access in the Transparency Regulations applies to all documents, and clinical study reports do not fall into any special category. A party opposing access must show specifically how its commercial interests would be undermined, and “cannot merely plead the existence of inherent confidentiality, or merely allege infringement of fundamental rights in abstract terms.”

Amicus’s second argument turned on the proper balance between disclosure and publication. Disclosure would harm their interests by:

• allowing competitors to take advantage and obtain an authorisation for a competing product both within and outside the EU
• undermining their ability to obtain an authorisation outside of the EU and
• compromising their ability to license know-how in the report to partner companies around the world.

The court disagreed. Amicus had followed the legal requirements and the protocol as set out in EMA guidelines and had not shown any novelty in their models, analyses or methodologies. Disclosure of the report would not reveal any “roadmap” to competitors. It did not include information on the composition or manufacturing of Galafold, and competitors would have to conduct their own trials in order to successfully develop a medicinal product. And Galafold benefited from the market exclusivity granted to orphan drugs.

Amicus had not identified which parts of the report were commercially confidential, despite having been asked by the EMA to do so.

What can we learn from this ruling?

The EMA is increasingly focused on transparency and its approach has repeatedly received support from the European court. In February this year, it supported the EMA’s decision to release documents in three landmark rulings, in which pharmaceutical companies argued commercial confidentiality.  In those cases, as here, the court focused on the failure to produce any concrete evidence as to how release of the documents would undermine commercial interests.

The EMA is committed to transparency and companies will have to do more than plead commercial interest to successfully argue an exception to the Transparency Regulations. Identifying specifically how disclosure will undermine its commercial interest, and showing that this is a real possibility rather than merely hypothetical, is essential. To argue an entire document is confidential will no doubt be difficult, and a more pragmatic approach of redaction is likely to prove more fruitful.