As the EMA management board faces up to a crunch meeting on Thursday this week at which it is due to finally decide its clinical trials data transparency rules, the U.S. Chamber of Commerce (CoC) has challenged its approach in a critical report that said the proposed policy calls into question the agency's attitudes to the "inherent confidentiality" of data submitted in marketing applications and its role as a custodian of the information.

While other drug regulators are facing calls for increased transparency, none plans to go as far as the EMA in publishing all clinical study reports once a product is approved. Rather, other agencies are showing they are "cognizant of the need to balance greater clinical trial transparency with the need to protect proprietary and confidential information," according to the report by the CoC's Global Intellectual Property Center.

The report said the EMA's transparency policy may have unintended consequences, resulting in commercially confidential information being public not only in the EU, but also internationally. The publication of clinical trial data submitted in a marketing authorization application to the EMA could be used by third parties as the basis of marketing applications in other jurisdictions, the report stated.

In addition, if information is already in the public domain, it may not be considered to be commercially confidential by regulators handling subsequent marketing applications in other countries.

Certainly, this is the fear of the European Federation of Pharmaceutical Industries and Associations (EFPIA), which has worked with its U.S. counterpart, the Pharmaceutical Research and Manufacturers of America, to implement a voluntary code in which data is disclosed in a controlled manner to authorized researchers.

EFPIA's opposition to a free-for-all in which clinical trial data is made available in public, searchable databases, has given the EMA pause for thought, prompting the agency to suggest study reports be available in a "view on screen" mode only. (See BioWorld Today, May 27, 2014.)

And while EMA's previous stance was that no data in a clinical study report could be considered confidential once a product was approved, the latest position is that study reports might be redacted to protect commercial confidentiality.

This represents a significant shift from the EMA's draft policy, published in July 2013, which promised open access.

The climb down in the face of industry pressure got the EMA into hot water with the European ombudsman, the EU's public interest watchdog, whose earlier rulings were the spur for the EMA's transparency policy. The ombudsman wrote to the EMA on May 13, questioning the change of direction, and telling the agency it could not set conditions around what use can be made of information once it is accessed.

Piling the pressure on the EMA, the EU's new directive on clinical trials, which was signed into law last week, creates a specific legal requirement to publish clinical trials data once a product is approved. This beefs up the requirement to disclose, which currently is based on freedom of information regulations.

Implementing the clinical trials directive requires the setting up of a new centralized pan-European portal for the submission and processing of applications to carry out clinical trials. This is unlikely to be in operation before 2016. Nevertheless, the EMA's transparency policy will need to accommodate the directive.

The policy was originally due to come into effect at the start of this year. Now it will go before the management board of the EMA for approval on Thursday, with publication promised as soon as possible after that, and implementation by the end of the year or the start of 2015.

EMA MAKES SELECTIONS FOR ADAPTIVE LICENSING PILOT

The EMA has selected the first two drugs to go into its adaptive licensing pilot after reviewing nine of 20 applications received to date, following the launch of the scheme on March 19.

Three applications have been rejected and the other four are seen as potential candidates. The remaining 11 applications are still under evaluation.

The two selected products were not named. The EMA is now talking to their owners to scope how adaptive licensing can best be applied. The agency plans to give an early approval in a limited patient population, to be followed by iterative phases of evidence gathering and the gradual extension of the marketing authorization across broader numbers of patients.

The discussions also will involve health technology assessment bodies and patients' bodies, in a confidential exploration of all options for development, licensing, reimbursement and monitoring, without any commitment on either side, but with concrete examples around which to work.

Once the best options are decided, the two products will move through the more formal stages of assessment by the EMA.

The scheme is still open and Hans-Georg Eichler, the EMA's senior medical officer, said more applications are on their way, adding, "We are very pleased to have received this number of applications in a short period of time."

The EMA has not specified how many products it will take into the adaptive licensing pilot, but has said it will include as many programs as necessary to gather knowledge and experience, and refine how the adaptive licensing pathway should be designed for different types of products and indications.

Criteria used in selecting products include the level of unmet medical need; that products are early stage – to allow for relevant inputs; the prospect that expansion from a restricted indication to broader populations is likely; and the potential to use real-world data from the initial, limited patient group to substitute for data from randomized trials, when expanding the label to broader indications.