Transparency on trial: How to navigate FDAAA 801 final rule implementation and compliance

By Melissa Fassbender

- Last updated on GMT

(Image: Getty/ipopba)
(Image: Getty/ipopba)
Regulations are changing across the globe as patients, the scientific community, and other stakeholders call for increased clinical trial data transparency.

In the US, the Final Rule went into effect in January 2017; however, the requirement to share clinical trial results came into effect in less than two months ago​. As per the new rule, the US Food and Drug Administration (FDA) is able to levy fines of more than $10,000 a day against clinical trial sponsors who fail to publish trial results in

According to the FDAAA Trials Tracker​, the US Government could have imposed fines of at least $33.96m as of April 2, 2018.

To help bring clarity to the changing requirements, recently caught up with TrialScope VP of Global Transparency, Francine Lane and Chief Strategy Officer, Thomas Wicks.

What are the requirements of FDAAA 801?

As Lane explained, Section 801 of the FDA Amendments Act (FDAAA) of 2007​ is the baseline regulation defining clinical trial disclosure in the US. 

The regulation was refined in 2016 with the issuance of 42 CFR Part 11 for Clinical Trials Registration and Results Information Submission​ – commonly referred to as the “Final Rule.”

While the regulation is complex, it generally means that phase 2-4 interventional studies that are under FDA jurisdiction must be registered on within 21 days of enrolling the first patient, and summary results must be disclosed within a year of the trial’s Primary Completion Date,”​ Lane told us.

“One of the key changes introduced in the Final Rule is the requirement to disclose results for unapproved products. Under the FDAAA 801, the results were only required for approved products and indications.”

What are the best procedural practices for registration and submission?

Lane explained the effect of disclosure is experienced across many areas of an organization beyond clinical operations providing protocol and results information.

“Coordination with regulatory affairs around submissions and approvals is necessary, as is working with publications on the content and timing of articles,”​ she said.

Additionally, Lane said corporate communications, investor relations, and other groups – such as patent attorneys – need to understand the scope and timing of disclosures.

“Because disclosure is wide-reaching, there are a few high-impact best practices that organizations can implement,”​ she explained – the first of which is to publish a transparency policy.

As Lane explained, the transparency policy informs both internal and external stakeholders on expected transparency commitments and should be specific, measurable, and generous.

Lane also recommends that an internal procures be implemented to formalize information flows between disclosure teams and other stakeholders. “While informal practices are still common, these are often insufficient to manage tight timelines, accurate data dissemination, and adequate internal communication,” ​she said.

Additionally, Lane said organizations should establish a cross-functional transparency committee to oversee disclosure and transparency across the organization.

“Lastly, and perhaps most importantly, simplify policies and procedures,”​ she added. “Since disclosure requirements can be complex and nuanced, they can lead to significant internal confusion and the external appearance of selective or incomplete disclosure.”

Global regulatory changes

The requirement to disclose results for unapproved products and indications on is the primary regulatory change this year in the US, said Lane.

Correspondingly, the National Institute of Health (NIH) has implemented policy changes that effect NIH-funded studies and may require clinical trial disclosure beyond the regulatory requirements in the US. 

The FDA also recently announced a pilot to share Clinical Study Reports (CSRs) for the studies supporting up to nine recently approved drugs.

Outside the US, Health Canada has issued a draft regulation around data sharing that is expected to be finalized in 2018.

Additionally, the International Committee of Medical Journal Editors (ICMJE) updated its data sharing policy in 2017​. The policy – lauded by US Senator Elizabeth Warren​ – requires manuscripts to contain a data sharing statement if submitted to ICMJE journals on or after July 1, 2018. Also, clinical trials that begin on or after January 1, 2019 must contain the data sharing plan when the trial is registered, explained Lane.

The World Health Organization (WHO) also recently updated the list of minimum trial information​ that must appear in a register to include a description of the data sharing plan, Lane added.

“While global disclosure regulations are steadily expanding the scope of clinical data and documents that must be made publicly available, leading organizations are reviewing their transparency policies to deliver on the promise to patients, trial participants, and the scientific community,”​ added Wicks.

“There is a clear shift from focusing only on compliance to considering how best to provide information that will actually be used by patients and other researchers,” ​he said.

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