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Figure.  Public Disclosure of Application Submissions in Press Releases (PRs), Regulatory Filings From the US Securities and Exchange Commission (SEC), and Any Other Documents
Public Disclosure of Application Submissions in Press Releases (PRs), Regulatory Filings From the US Securities and Exchange Commission (SEC), and Any Other Documents

Data from a subscription-based database (secondary outcome) are not included.

aThe “other” documents were only searched when a PR or SEC filing was not found (n = 32). The following documents were found: a financial highlights document (n = 1), a notice of patent extension (n = 1), a stockholder update document (n = 1), and miscellaneous press articles (n = 2).

Table.  General Characteristics of All Applications and Disclosuresa
General Characteristics of All Applications and Disclosuresa
1.
US Food and Drug Administration.  FDA Transparency Initiative: Draft Proposals for Public Comment Regarding Disclosure Policies of the U.S. Food And Drug Administration. Washington, DC: US Dept of Health and Human Services; 2010.
2.
US Securities and Exchange Commission (SEC) and Electronic Data Gathering, Analysis, and Retrieval System (EDGAR). 2017. http://www.sec.gov/edgar/searchedgar/companysearch.html. Accessed July 1, 2017.
3.
Informa  PLC. Pharmaprojects: Complete drug development database. 2017. https://pharmaintelligence.informa.com/products-and-services/data-and-analysis/pharmaprojects. Accessed July 1, 2017.
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    Research Letter
    June 3, 2019

    Public Disclosure of the Filing of New Drug and Therapeutic Biologics Applications With the US Food and Drug Administration

    Author Affiliations
    • 1Office of Public Health Strategy and Analysis, Office of the Commissioner, US Food and Drug Administration, Silver Spring, Maryland
    • 2Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, New Brunswick
    • 3Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland
    • 4Center for Science in the Public Interest, Washington, DC
    JAMA Intern Med. 2019;179(8):1144-1146. doi:10.1001/jamainternmed.2019.1213

    Current regulations prohibit the US Food and Drug Administration (FDA) from publicly disclosing the existence of pending New Drug Applications (NDAs) or Biologics License Applications (BLAs) unless previously publicly disclosed or acknowledged.1 Applicants may disclose applications in press releases, Securities and Exchange Commission (SEC) filings, or in other media; how frequently they do so is not known.

    Methods

    We conducted a cross-sectional study of all NDAs for new molecular entities and BLAs for new and biosimilar biological products submitted to the FDA between January 1, 2010, and December 31, 2016. Data were collected in July 2017. Because this was a cross-sectional study based on nonclinical data, we did not submit this study for human subjects ethics review.

    Primary outcomes included disclosures of applications in press releases, SEC filings, and other media. For press releases, we searched applicants’ websites and websites where press releases are disseminated. For SEC disclosures, we searched a public SEC database for filings from firms publicly traded in the United States.2 If disclosures were not found, we conducted Google searches to identify any disclosures in other media (eg, investor materials or annual reports). We calculated the percentage of submissions disclosed and the median time to disclosure (days with interquartile range [IQR]).

    We also searched Pharmaprojects, a subscription-based drug development database, and determined the percentage of submissions disclosed; such a listing was not considered to be public disclosure.3

    Results

    Of the 249 applications (188 NDAs and 61 BLAs), 222 (89.2%) were disclosed in at least 1 public medium (Figure), including in a press release for 196 applications (78.7%) (Table). Of the press releases, 196 (86.2%) were found on applicants’ websites. Of the applications, 157 (63.1%) were disclosed in SEC filings, including 136 (54.6%) in press releases and SEC filings. The overall rates of public disclosures increased from 87.5% in 2010 to 97.6% in 2016. Publicly traded companies were more likely to disclose applications in any medium compared with nonpublic companies (relative risk, 1.43; 95% CI, 1.09-1.90); private companies were less likely to disclose applications (relative risk, 0.70; 95% CI, 0.53-0.92) (Table). The median time to disclosure was 6 days (IQR, 1-63 days) for press releases, 55 days (IQR, 9-86 days) for SEC filings, and 6 days (IQR, 1-63 days) for any public media.

    The Pharmaprojects database listed 246 of the 249 study drugs (98.8%). We found a reference to FDA submission for only 162 applications (65.9%), including 11 not disclosed in public media. For 43 of the 162 applications, the listing date preceded the applications’ receipt by the FDA (median, 59 days before date of FDA receipt; IQR, 13-195 days).

    Discussion

    We found that information that the FDA treats as confidential with regard to applications for NDAs and BLAs was in most cases already available to the public and that the disclosure rate in 2016 was 97.6%. Most disclosures took place in press releases, SEC filings, or both and occurred within 1 week of application submission.

    A 2010 FDA report recommended that these applications be publicly disclosed at filing.1 Implementing such a recommendation would mark a significant departure from longstanding FDA practice that may necessitate changes to statutes or regulations.1 Other transparency proposals in the 2010 report, such as the recommendation to disclose the FDA’s complete response letters, which list deficiencies in applications that preclude approval, would reveal that an NDA or BLA had been filed and thus necessarily include eliminating the prohibition against disclosing the existence of unapproved applications. We also found that subscription-based databases would not obviate the need for FDA disclosure because they are not public databases, and the database that we reviewed contained incomplete and, in some instances, inaccurate data.

    The limitations of our study include underestimated disclosure of older applications because materials may no longer be Internet accessible and more recent applications in some disclosure media (eg, annual reports) may not yet be disclosed. We also did not review foreign securities filings or other subscription-based databases that may provide different information. Finally, findings cannot be generalized to other types of FDA applications, which may have different disclosure rates.

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    Article Information

    Accepted for Publication: March 17, 2019.

    Corresponding Author: Harinder Singh Chahal, PharmD, MSc, Office of Public Health Strategy and Analysis, Office of the Commissioner, US Food and Drug Administration, 10903 New Hampshire Ave, Bldg 32, Room 4220, Silver Spring, MD 20993 (harinder.chahal@fda.hhs.gov).

    Published Online: June 3, 2019. doi:10.1001/jamainternmed.2019.1213

    Author Contributions: Dr Chahal had full access to all the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis.

    Concept and design: Chahal, Lurie.

    Acquisition, analysis, or interpretation of data: All authors.

    Drafting of the manuscript: Chahal, Szeto, Kim, Sigelman.

    Critical revision of the manuscript for important intellectual content: Chahal, Szeto, Chaudhry, Sigelman, Lurie.

    Statistical analysis: Chahal, Chaudhry.

    Administrative, technical, or material support: Szeto, Chaudhry, Lurie.

    Supervision: Chahal, Szeto, Lurie.

    Conflict of Interest Disclosures: Drs Szeto and Lurie were employed by the US Food and Drug Administration (FDA) when the manuscript was conceived, researched, and drafted. No other disclosures were reported.

    Funding/Support: This research did not receive any funding from any source; the work was conducted during the authors’ duties with the FDA. This project was supported in part by an appointment to the Research Participation Program at the Office of Public Health Strategy & Analysis, FDA, administered by the Oak Ridge Institute for Science and Education (ORISE) through an interagency agreement between the US Department of Energy and FDA. Ms Chaudhry and Dr Kim were ORISE fellows during this research.

    Disclaimer: This article reflects the views of the authors and should not be construed to represent the FDA’s views or policies. Drs Szeto and Lurie are no longer with the FDA. Dr Szeto was with the FDA from July to August 2017; Dr Lurie was with the FDA from 2009 to 2017. When the study was conducted, all authors were staff, fellows, or interns in the FDA’s Office of Public Health Strategy and Analysis.

    Additional Contributions: Nancy Sager, Robert Temple, MD, and Karen Marcus Giardiello, JD, reviewed earlier versions of the manuscript and provided valuable feedback.

    Additional Information: This research used data from Data Analysis Search Host, an internal FDA database that provided application-specific attributes.

    References
    1.
    US Food and Drug Administration.  FDA Transparency Initiative: Draft Proposals for Public Comment Regarding Disclosure Policies of the U.S. Food And Drug Administration. Washington, DC: US Dept of Health and Human Services; 2010.
    2.
    US Securities and Exchange Commission (SEC) and Electronic Data Gathering, Analysis, and Retrieval System (EDGAR). 2017. http://www.sec.gov/edgar/searchedgar/companysearch.html. Accessed July 1, 2017.
    3.
    Informa  PLC. Pharmaprojects: Complete drug development database. 2017. https://pharmaintelligence.informa.com/products-and-services/data-and-analysis/pharmaprojects. Accessed July 1, 2017.
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