In future, virtually all data from the relevant clinical trials should be disclosed once a new medicine is cleared for marketing, believes the European Federation of Pharmaceutical Industries and Associations (EFPIA).  

“For several years my members have shared summary results for all clinical trials involving patients,” said EFPIA director general Richard Bergström. “Now is the time to take another step towards full transparency.”

There will be “details on patients and individuals that need to be protected, but beyond that I think all clinical test results should be disclosed as soon as a new medicine is approved”, Bergström added.

Draft guidance

The industry association’s position is set out in its response to a draft guidance document put out for consultation by the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) at the beginning of June.

As part of a general drive towards greater transparency in the EU and member state regulatory systems for medicines, the draft guidance outlined the types of information in applications for marketing authorisation (MA) that could be released following a request for access to documents, once the MA had been granted.

The EMA and the HMA were particularly interested in comments on proposed criteria for the release or protection of personal data; whether contractual arrangements between companies needed to be maintained as confidential; and how to address the personal security of individuals involved in studies using animals.  

In its response to the draft guidance document on the identification of commercially confidential information and protection of personal data within the structure of the marketing-authorisation dossier – release of information after granting of a marketing authorisation, EFPIA says it “welcomes and supports” the agencies’ efforts to increase transparency within the European regulatory framework.

Not only does enhanced transparency accord with the wider goal of promoting and protecting public health but it is “essential in maintaining overall public trust and confidence in the system for the regulation of medicines in Europe”, the association suggests.

Significant challenges

At the same time, EFPIA agrees with the EMA/HMA that there are “significant challenges to address in identifying and separating the information that can be released, from the information which should be withheld”.

This is partly “due to the size and complexity of MA dossiers, and the fact that much of the ‘commercially confidential’ and ‘personal data’ is intermingled with large amounts of other data throughout the dossiers”.

The association believes three key issues need to be addressed before marketin authorisation dossiers can be routinely disclosed to the general public:

1) Publishing clinical study reports at the time of MA authorisation “may be regarded as ‘prior publication’, such that scientific journals will not accept or put on hold journal manuscripts”, EFPIA warns.

It suggests that HMA/EMA “should seek an understanding with editors that disclosure by authorities, to meet their obligations regarding public access to documents held by European and Member States Institutions, does not constitute ‘prior publication’”.

Alternatively, EFPIA proposes, studies identified as ‘awaiting publication’ should not be disclosed until the journal manuscript has been published, “provided this happens in a reasonable time-frame”.

HMA/EMA and industry together “might consider discussing these options with journal editors to explore how their way of working can be adjusted to the new reality of full transparency”, it comments.

2) EFPIA is concerned that publishing “certain CMC [chemistry, manufacturing and controls] and non-clinical data following EU approval could “enhance the ability of non-innovators to obtain approval outside the EU based on the originator’s data”.

This would undermine the incentives that drive innovation and deliver medicines to patients, EFPIA argues. It is therefore “necessary to ensure that key CMC data is classified as CCI [commercially confidential information]”, the association says.

It proposes that non-clinical study reports should be classified as CCI and not disclosed. EFPIA does, however, support the release of non-clinical, summaries “with appropriate redactions”.

3) Another concern is that releasing “a vast amount” of clinical data in this way raises the bar for regulatory agencies to make sure current versions of the data are made public.

For example, EFPIA notes, “the suggested pharmacovigilance measures suggested in the original application may have been revised during the regulatory review. Similarly, post-approval communications or measures may be important to mention when information from the original MAA is requested”.

In a similar vein, the association believes, EMA/HMA should take a cautious view on how clinical data can or cannot be re-analysed by third parties.

It is important, for example, that the same standards should apply to any subgroup analysis, regardless of who is conducting the analysis, EFPIA contends, suggesting that the agencies could monitor how data are used and re-analysed over time.   

Practical steps

Accepting that the bulk of clinical data for medicines approved in Europe should be made available to the public, the “real challenge” now is “find practical ways to make this data useful”, Bergström commented.

“No one will benefit from a ‘data dump’,” he added. “Industry and regulators must work with patient groups to make sure that data is provided in a way that is helpful for patients and relatives. New tools are needed to help users navigate and understand the vast amount of data that will be released.”

In particular, Bergström stressed, the concept of a benefit–risk equation needs to be clearly explained “so that everyone who is interested can understand the reasons for approving a particular medicine”.