Device, Pharmaceutical Industries Say FDA Proposal to Release Trial Data Illegal

| 30 October 2013 |  By 

Within the regulatory affairs space, few topics have received as much attention and debate in recent years as the topic of clinical data transparency. In the EU, regulatory officials have launched an all-out attempt to grant wider public access to clinical data, arguing that it will ensure that products are safer and that companies do not need to duplicate failed research.

But in the US, the tone and tenor of the debate has been decidedly different, with few advocacy groups calling for open access to trials data and regulators pushing for measures that are decidedly more modest in scope compared to those put forth by the European Medicines Agency (EMA). But as two recent public comments submitted by prominent national trade groups show, even these modest measures are encountering some fierce pushback from industry.


You'd be forgiven if you had no idea FDA was even looking at its own transparency policy. It quietly announced the policy in June 2013 in a Federal Register notice entitled, "Masked and De-Identified Non-Summary Safety and Efficacy Data," and offered no further explanations to the public by way of press releases or other postings on its website. Outside of Regulatory Focusand a few trade publications, seemingly no one noticed its release.

In the Federal Register posting, FDA proposed allowing limited clinical trials transparency by permitting it to share de-identified data with selected experts, allowing them to identify common safety issues as well as "key hurdles in drug development."

Regulators provided the example of a hepatitis C treatment, which had previously relied upon disease detection at 24 weeks. An analysis of data across 15 trials allowed FDA to determine that patient response at 12 weeks was just as indicative of the ultimate response, allowing companies to cut the trial time in half.

The problem, FDA said, is that it lacks some of the expertise it wants and needs to analyze these types of data in all cases. FDA explained that if it brought in outside experts, they would not be given the full data. Instead, they might be given pooled data from multiple trials (de-identified), data with certain fields masked, or random samples or subsets of data.

FDA added in its statement that it has an "unwavering commitment" to patient privacy and "will not make available business-related confidential commercial information contained in product applications" such as licensing agreements or information identifying suppliers.

Pharmaceutical Industry Responds

But even with those commitments and its general framework for releasing data, FDA's proposal was panned by one of the pharmaceutical industry's more influential advocacy groups, the Pharmaceutical Research and Manufacturers of America (PhRMA).

The group, fresh off the launch of its own transparency initiative intended to facilitate the release of clinical trials data to select experts, said FDA's proposed policy raised "significant concerns" and could "distract FDA from its primary mission to review marketing applications to bring new medical products to patients expeditiously."

"Focusing on processing these data for outside analysis risks distraction and additional burdens on FDA's valuable resources," it added.

PhRMA highlighted other potential faults in the plan, including its concern that data could end up falling into the hands of competitors, end up being misinterpreted by non-experts, and that the release of data was simply illegal under the Federal Trade Secrets Act, the Federal Food, Drug and Cosmetic Act and the Fifth Amendment of the US Constitution.

Those comments, as well as those of others in industry, were apparently enough to cause FDA to extend its deadline on the proposal, which it did on 30 September. The deadline for comments is now 31 October 2013.

Medical Device Industry Responds

But the pharmaceutical industry isn't the only one with concerns about the plan. In a comment posted to FDA's Docket on 29 October 2013, the device trade group AdvaMed weighed in, calling the plan "vague and poorly defined" and likely illegal.

"FDA does not have legal authority to proceed with this proposal with respect to medical devices," the group said in its comments. It said the legal provisions cited by FDA as being the source of its authority-Section 1124 of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012-referred to regulatory decision-making, yet had no mention of masked or de-identified data.

This distinction could explain why FDA endeavored to link its plan to regulatory outcomes in its June 2013 announcement, but still opens the door to a likely legal challenge by both PhRMA and AdvaMed if it proceeds.

AdvaMed also cited many of the same statutory challenges as PhRMA-- the Federal Trade Secrets Act, the Federal Food, Drug and Cosmetic Act and the Fifth Amendment of the US Constitution-as being problematic for FDA's efforts.

Voluntary Efforts and Other Issues

And like PhRMA, AdvaMed also said that its members groups are still permitted to make data available on a voluntary basis, which some already do. Many of the benefits cited by FDA are already made available using private sector mechanisms, AdvaMed observed, adding that these efforts "to a large degree obviate the need for FDA to expend significant resources on its own masked and/or de-identified data initiative that will challenge FDA's already limited resources."

Instead, FDA should reach out to industry on an as-needed basis when it believes voluntary sponsor sharing would be of value to either the public or to industry, AdvaMed said.

The group said it was also concerned about being able to identify the device being tested from the data, particularly "where test methods are unique to a given technology" and where "test method summaries are provided in the scientific literature."

"Given the proprietary nature of device development, including clinical trial designs and data collection or test methods, making clinical and pre-clinical data available to the public could effectively disclose the sponsor's device design and would provide unfair advantages to competitors," AdvaMed wrote.

And as stated earlier, AdvaMed said there was a lack of detail in FDA's proposal that made it difficult to comment event more extensively. "The proposal fails to adequately and fully describe the situations when FDA would seek to share masked and/or de-identified sponsor data, who FDA will share sponsor data with, what will be done with the data, and what restrictions or controls (other than masking or de-identifying the proprietary data) will be placed upon those who receive the sponsor data," it wrote.

AdvaMed Comments to FDA


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