The European Medicine Agency’s (EMA) Policy on Publication of Clinical Data for Medicinal Products for Human Use (“EMA Policy”)is now effective as of January 1, 2015. Across the Atlantic, the U.S. Institute of Medicine (IOM) released a report, Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk (“IOM Report”) on January 14, 2015. These documents serve as only the most recent efforts in the movement to promote greater transparency in life sciences research and development.

Both documents offer a similar rationale regarding the importance of releasing clinical data in connection with clinical trials and product development and emphasize the multitude of advantages that data sharing provides. Data sharing improves trust in manufacturers who develop new products and in the governmental agencies that approve marketing applications for new products. Data sharing also increases the efficiency of clinical research and strengthens safety in clinical trials by preventing unnecessary duplication of trials, which is especially critical for trials involving products found to be unsafe or ineffective. Above all, data sharing ensures that clinical trial participants’ contributions to research have the most impact. With the ability to validate and advance clinical trial results, researchers are able to accelerate efforts to prevent, detect, and treat disease.

Both documents also address the risks and challenges that come with releasing clinical trial data. Data sharing must occur in a manner that incentivizes manufacturers and researchers to develop new clinical trials. However, the timing of data sharing must happen in a way that allows researchers to retain data until they have sufficient time to analyze and publish the data they collect with a mechanism in place to guard against secondary analyses that erroneously invalidate the research. Most importantly, the privacy of clinical trial participants must be maintained.

While the EMA Policy and IOM Report share similarities, an important distinction exists between the two. The EMA Policy mandates that those seeking approval in the European Union (EU) release their clinical data with limited exceptions. On the other hand, the IOM Report, is just that, a report that merely encourages the publishing of clinical data. As a result, companies seeking regulatory approval in the United States still have the choice to release their clinical trial data. There are, however, additional rules contemplated in the United States that could require release if adopted (see below).

Transparency Requirements of the EMA Policy
The EMA Policy requires publication and transparency of clinical data submitted in conjunction with the submission of various regulatory filings with the EMA, such as a marketing authorisation application.[1] The scope of the releasable clinical data generally includes clinical reports, such as clinical study reports and the protocol, and individual patient data (without violating patient privacy principles). However, pharmacovigilance data from individuals or clinical data that the EMA does not maintain, such as data from a clinical trial on an approved product conducted by independent investigators, does not need to be released.

Likewise, the EMA Policy does not require the release of commercially confidential information (CCI). CCI is defined as “… any information contained in the clinical reports submitted to the Agency by the applicant/Marketing Authorisation Holder that is not in the public domain or publicly available and where disclosure may undermine the legitimate economic interest of the applicant/MAH.”[2] The EMA recognizes that CCI is critical information and could allow competitors, in the EU or elsewhere, to find and patent new uses for drugs before the company who originated the research.

In order to protect the CCI, applicants may utilize the EMA Policy’s redaction principles.[3] To use the redaction principles, the applicant needs to substantiate the basis for the redaction, including that the CCI is: (1) critical information that could impact the applicant’s economic interest and (2) confidential. If the EMA finds that redacted information is not CCI, the EMA requires the applicant to provide the EMA with further support for the redactions during what the EMA Policy refers to as “a consultation with all relevant stakeholders.”[4] This consultation is the last chance to persuade the EMA that information is CCI and warrants redaction. If unsuccessful, the applicant must choose between appealing the EMA decision through litigation or assessing the risks associated with releasing the information.

Another critical consideration in the release of clinical data is the protection of patient privacy through the individual clinical patient data. The EMA continues to determine the best way to protect this information, either through a plan of anonymization (such as de-identification) or pseudonymization (such as coding). Once the EMA determines the best way forward, the individual patient data will be released with the rest of the clinical data.  Until then, only clinical data contained in clinical reports must be released when submitted with the marketing authorisation applications to the EMA. For now, sponsors should consider their current policies and procedures for reporting patient data and reevaluate their adequacy with respect to the submission of clinical data to the EMA if it is to become public information. 

More on the Way
Whether sharing clinical trial data will realize the benefits contemplated by the EMA Policy and IOM Report remains unknown. Nonetheless, the U.S. Department of Health and Human Services and National Institutes of Health are in the process of following suit with proposed rule 42 CFR Part 11 (Clinical Trials Registration and Results Submission)[5] and the proposed policy titled, Dissemination of NIH-Funded Clinical Trial Information.[6] As the release of clinical trial data becomes more commonplace around the globe, understanding the scope of releasable information and how to protect confidential and proprietary information about the product becomes ever more important.

Notes:

[1] EMA Policy on Publication of Clinical Data for Medicinal Products for Human Use, 2014, Section 2.

[2] Id. at Section 3.

[3] Id. at sec. 4.2.2.1

[4] Id.

[5] https://www.federalregister.gov/articles/2014/11/21/2014-26197/clinical-trials-registration-and-results-submission

[6] https://www.federalregister.gov/articles/2015/02/13/2015-02994/announcement-of-a-draft-nih-policy-on-dissemination-of-nih-funded-clinical-trial-information#h-8