What did the FDA put forward to enhance Transparency?



On 16. January 2018, FDA Commissioner, Scott Gottlieb announced the FDA’s intention to evaluate how they could share Clinical Documents of Pivotal Phase III studies following a successful NDA, to enhance transparency of clinical trial information to support innovation and scientific inquiry related to new drugs. Later the same year, EMA Policy 0070 Part 1 was paused in August 2018, as part of the business continuity plan, in connection with the relocation of the agency to Amsterdam. Health Canada progressed their Policy and Guidance on Public Release of Clinical Information (PRCI), which became effective later on 1 April 2019. Last but not the least, GDPR became effective on 25. May 2018…

The FDA’s approach consisted in conducting pilots (nine planned) with sponsors who would be asked to volunteer as they were going through their NDA submissions. The FDA would then later proceed with a public consultation on their Clinical Data Summary Pilot Program.

Only one sponsor (Janssen Pharmaceutica / ERLEADA) participated in the Pilot and on 27 June 2019 (nearly 1.5 years after the initial announcement that also saw Scott Gottlieb resign from his position as FDA Commissioner), the FDA announced the start of the consultation period.

The consultation included reviews of both the FDA CSR Pilot Program solely based on the ERLEADA documents and of the new Integrated Review Template (Federal Register / Vol. 84, No. 124 / Thursday, June 27, 2019).


What did the ERLEADA documents contain and what did we learn from the Pilot Program?

The ERLEADA documents were made public on the FDA website. No login nor acceptance of Terms of Use (ToU) were required to access the documents.

As per the FOIA request approach, FDA conducts redactions of Confidential Commercial Information (CCI) and Personally Identifiable Information (PII), but demographics such as Age, Gender and Race are retained. It must be noted that under EMA Policy 0070 Part 1 or Health Canada PRCI, such personal data would be anonymised (generalisation, perturbation, redaction, etc.) to minimise risk of re-identification.

In a first version of the document (which has since been replaced), Subject IDs were also kept unchanged. This was corrected in the second and final version of the published redacted CSR.

Core dates (e.g. 12 July 2013) were redacted while relative dates (e.g. Study day 234) were retained.

Adverse events narratives were not shared and considered out-of-scope. Mini-narratives on Death are, however, present where PII is redacted: 

Mini-narratives from “ Deaths” section of ERLEADA CSR 


In addition, and unlike under EMA Policy 0070 Part 1 and Health Canada PRCI, staff names were released: 

Staff information released in ERLEADA CSR


In terms of process, the sponsor will not have an opportunity to review our redactions before the CSR is posted. If the sponsor is uncertain about whether we will redact certain types of information, the sponsor should ask us in advance (See question 6 of Pilot Program FAQ).


What did the reviewers say?

21 sets of comments were received by the 26. August 2019 deadline and published, from different organisations and individuals. A summary of those selected is provided below. The full sets of comments are available at the FDA Docket ID: FDA-2019-N-2012.

PHUSE Data Transparency Working Group: 

  • Acknowledge the possible secondary-purpose analyses that could be supported using FDA documents (Evaluation of Bias, Meta-analysis Using Methods and Results, etc.)
  • Highlight concerns over releasing quasi-identifiers that would otherwise be anonymised under EMA Policy 0070 and Health Canada PRCI
  • Highlight the loss of Data Utility in the ERLEADA CSR as Subject IDs are redacted and adverse event narratives are not in scope
  • Regret that no Terms of Use nor login process is in place to access the ERLEADA documents

COCHRANE, BMJ & PLOS, Peter Doshi:

  • Very supportive of the FDA Pilot Program and the release of CSRs
  • Believe individual reviews should not be replaced by the Integrated Review Report
  • Ask for listing of adverse events to be included (This must refer to the narratives)


  • Regret only one company participating in the Pilot and suggest continuing the Pilot Program and including more companies


  • Comments similar to PHUSE with regards to Data Privacy aspects of the FDA Pilot Program and reminder of GDPR’s considerations around anonymisation
  • Raise concerns about no longer having reviewers’ assessments of issues available anymore as this would provide clarity on how issues were resolved by the agency

LEO Pharma:

  • Suggest alignment with EMA Policy 0070 & Health Canada PRCI including process for sponsor to review reports
  • Also raise concerns with regards to meeting minutes and other internal documents no longer being released, resulting in diminishing transparency


  • Regret staff names are not redacted
  • Acknowledge that CSRs have greater value, but Integrated Review Reports are more “digestible”
  • Suggest alignment with the EMA and other agencies with regards to public release of documents

Flatiron, Karanges et al., Public Citizen:

  • Regret reviewers’ comments are not present


  • Stress risk of re-identification with demographics being retained and lack of alignment with other agencies
  • Regret no Terms of Use, preventing intention to re-identify patients and commercial use of the documents

What are the possible next steps?

It must be noted that the FDA approach outlined, as part of their Clinical Data Summary Pilot Program, significantly differs from EMA Policy 0070 Part 1 (effective since 1 January 2015) External Guidance and Health Canada PRCI (effective since 1 April 2019) Guidance. Differences range from processes and de-identification requirements to scope while EMA Policy 0070 Part 1 and Health Canada PRCI are fairly similar. Health Canada has even put in place a recognition process for documents published under EMA Policy 0070. It must be noted, however, that while other agencies consulted stakeholders during the elaboration of their policies, the FDA is the only agency that intended to conduct pilots with sponsors as a mean to review in practice the feasibility and benefits of their initiative.

There are different possible scenarios: 

  • The FDA adapt their approach to sharing of documents and CSRs, in particular. As suggested in some of the comments, an alignment with EMA Policy 0070 and Health Canada PRCI could be considered.
  • The FDA ignore Industry feedback and go ahead with their FOIA-like approach for public release of documents.
  • The FDA give up on sharing redacted CSRs and focus on the Integrated Review Reports instead to enhance transparency.
  • The FDA could also adjust the Integrated Review Reports, to add the reviewers’ opinions, as well as documentation of any issue resolutions.


Want to Learn More?

Please read our paper on synergies and differences across EMA, Health Canada, FDA and PMDA initiatives on the sharing of Clinical Documents.

About the Author

Jean-Marc Ferran

Jean-Marc Ferran


Jean-Marc Ferran is a Life Sciences Entrepreneur based in Copenhagen, with 15+ years of experience in the Biometrics arena.

He has also led the PHUSE Data Transparency Working Group since 2014 and is an appointed member of the European Medicines Agency’s Technical Anonymisation Group (EMA TAG) and Health Canada Stakeholder Reference Group (HCRG) on Public Disclosure of Clinical Information (PRCI).

Jean-Marc is a regular reviewer of Industry or regulatory guidance and policies and led a team of Industry experts from the PHUSE Data Transparency Working Group to answer the current FDA consultation on the Clinical Data Summary Pilot Program.


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