Life Science Compliance Update

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June 18, 2013

FDA Proposes Making Product Approval Applications More Transparent

The Food and Drug Administration (FDA) is seeking public comments from interested persons on the proposed availability of de-identified and masked data derived from medical product applications, according to a recent posting in the Federal Register.

"Improving the efficiency and effectiveness of medical product development is a main priority for FDA. The ability to make available de-identified and masked clinical and preclinical data derived from marketing applications could make an important contribution to that goal by providing scientific data that may be of value in the generation of new knowledge to facilitate innovation in the development and evaluation of critically needed medical products. The contribution of patients who participate in clinical trials should be maximized for the benefit of society."

Thus, FDA invites comments on the issues to be considered with regard to such availability and on any limitations that should be placed on the availability of these data. Comments are due August 5th, 2013.


The notice and request from comments for FDA begins by maintaining that FDA has a responsibility to "promote the public health." In carrying out this duty, FDA, in collaboration with the National Institutes of Health (NIH), launched the Regulatory Science Initiative, a call to action to enhance the science and knowledge critical to improving the development, manufacture, evaluation, and safe use of critically needed new therapies.

In addition, the Food and Drug Administration Safety and Innovation Act (FDASIA), enacted on July 9, 2012, contains important new authorities that will enhance the Agency's ability to promote innovation across industry, research and clinical care settings, including new provisions that require the development of a plan for advancing regulatory science for medical products in order to promote the public health and advance innovation in regulatory decision making. (See, e.g., section 1124 of FDASIA).

Based on this background, FDA asserted that the "development of new knowledge and insights from clinical and preclinical study data is an important regulatory science opportunity" because such "data have a tremendous potential to help address critical challenges and provide new opportunities for innovation in medical product development, including for human drugs, medical devices, and biological products."

For example, "safety and effectiveness data from multiple studies have been used in the past to address key hurdles in drug development." Additionally, "analysis of data from multiple clinical and preclinical studies has been used to identify potentially valid endpoints for clinical trials, understand the predictive value of preclinical models, clarify how medical products work in different diseases, and inform development of novel clinical designs and endpoints to the benefit of patients." FDA pointed to trials and analysis regarding drugs used to treat chronic hepatitis C.

In addition to identification of additional endpoints for clinical studies, pooled data (both preclinical and clinical) have also been applied to the analysis of safety issues. An analysis of 199 clinical trials of 11 antiepileptic drugs by FDA helped quantify the increased risk of suicidal behavior or ideation for patients and prescribers.

Also, an independent analysis of data on 5 potential biomarkers of kidney injury by the Predictive Safety Testing Consortium led to their qualification for inclusion in pre-clinical safety data submissions. Based on these examples, FDA maintained that advances in regulatory science "can arise from analysis of diverse data submitted as part of marketing applications, including, for example data related to clinical outcomes, safety, biomarker status, drug disposition, drug action, or patient reported outcomes."

While FDA has the expertise in analyzing individual patient level and aggregated clinical trial data, the agency recognized the need to use outside experts to become "actively engaged in the research." Thus, FDA is considering approaches to providing access by non-FDA experts and other interested parties to data that have research value in a way that would both safeguard the privacy interests of patients enrolled in clinical trials, and appropriately protect the commercial investments of sponsors.

Definition of Terms

FDA used the term "masked data" in this notice to refer to data with information removed that could link it to a specific product or application. FDA will consider different strategies to minimize the ability to identify specific products and the impact of any such strategies. Such strategies might include:

  • Making available certain data from a random sample or appropriately chosen subset of subjects,
  • Restricting the data fields made available or
  • Pooling data where possible from studies of multiple members of a product class, without identifying the specific product.

FDA next clarified that "de-identified data" refers to data that does not identify an individual and with respect to which there is no reasonable basis to believe that the information can be used to identify an individual. The agency reiterated its "unwavering commitment" to protecting the privacy of research subjects' identities. As such, consistent with FDA's regulations at 21 CFR 20.63(a), any data that might be made available under this proposal would be stripped of any information which could identify patients or research subjects, either directly or through combination with other publicly available information. Outside parties accessing such data would also be required to remove personal identifiers.

Next, FDA noted that de-identified and masked data could be used to develop a model of disease progression in control arms of future studies based on pooled control group data from past studies of the same disease or indication and would not require identification of a product or even product class nor would there be personal identifiers associated with the data. Similarly, characterization of risk factors might only involve control group data. On the other hand, validating a biomarker as a surrogate for a clinical outcome or as a predictive classifier of potential treatment response might require identification of products by class or analysis across a class to show consistency.

FDA further explained that this proposal does not pertain to unmasked safety and effectiveness data, (i.e., data that can be linked to a specific, identified application) including full study reports. Further, FDA will not make available business-related confidential commercial information contained in product applications, including but not limited to information concerning licensing agreements and information identifying suppliers, unless such information has already been publicly disclosed by the sponsor. Nor will the Agency make available trade secret information under this proposal.

Requested Comments

Based on this background and explanations, FDA is asking stakeholders to submit comments on the following topics:

  1. What factors should be considered in masking study data (e.g., data fields from regulatory submissions to remove or modify, number of different products to pool within a product class)?
  2. What limitations, if any, should there be on the Agency's ability to make available the masked data as described previously?
  1. Are there any additional factors FDA should consider in de-identifying data in addition to FDA's requirement to remove any names and other information (e.g., birth date, death date, local geographic information, contact information) which would identify patients or research subjects before disclosing information?
  1. Would regulatory changes facilitate implementation of such a proposal, and if so, what changes would be most useful? and
  1. Which situations do you believe disclosing masked data would be most useful to advance public health?

Submit electronic comments to:!submitComment;D=FDA-2013-N-0271-0001

Submit written comments to: the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852.

All comments should be identified with: Availability of Masked and De-identified Non-Summary Safety and Efficacy Data; Request for Comments (Document ID FDA-2013-N-0271-0001)

For Questions Contact:

Nancy B. Sager, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., HILL-3110, Silver Spring, MD 20993, 301-796-3603, FAX: 301-431-6351,; or

Stephen Ripley, Center for Biologics Evaluation and Research (HFM-17), Food and Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-827-6210;

or Aaliyah Eaves-Leanos, Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 66, rm. 5435, 301-796-2948, FAX: 301-847-8510.


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