Transparency: BIO Comments on Availability of Masked and De-identified Non-Summary Safety and Efficacy Data

Re: Docket No. FDA–2013–N–0271: Availability of Masked and De-identified Non-Summary Safety and Efficacy Data; Request for Comments
 
Dear Sir/Madam:
 
The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on the “Availability of Masked and De-identified Non-Summary Safety and Efficacy Data.”
 
BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products, thereby expanding the boundaries of science to benefit humanity by providing better healthcare, enhanced agriculture, and a cleaner and safer environment.
 
GENERAL COMMENTS:
 
BIO and its member companies appreciate FDA’s commitment to modernizing regulatory science and are committed to enhancing public health through advancements that fill knowledge gaps in drug development. To that end, BIO supports voluntary, responsible sharing of appropriately masked and de-identified safety and efficacy data as one potential way to develop new knowledge or new scientific insights. BIO member companies routinely publish clinical research results and collaborate with academic researchers, while both adhering to applicable privacy laws and regulations and protecting confidential commercial information.
 
It is important to recognize that issues of transparency and disclosure of regulatory data are extremely complex and currently being debated and evaluated globally in various venues. We urge FDA to consider these issues in the context of other transparency initiatives, including the Agency’s own Transparency Initiative, as well as the Expansion of the ClinicalTrials.gov database as directed by the Food and Drug Administration Amendments Act (FDAAA) enacted in 2007. As stated in BIO’s April 2009 testimony on the ClinicalTrials.gov expansion, it is critical to ensure that data to be interpreted outside of FDA’s expert review process have scientific merit and can enhance the treatment and safety of patients. Accordingly, the purpose of disclosure of such data, as well as appropriate recipients of such data, must be clearly and transparently identified.
 
In addition, it is important to strike a balance in achieving dual public health goals, both recognized in the Agency’s Notice: the goal of enhancing scientific knowledge to advance public health and patient care, as well as the goal of protecting confidential and trade secret information, in order to maintain the incentives necessary for further investment in and development of new treatments for patients. With regard to the latter, the trade secret and commercially confidential nature of clinical data may remain even when such data are masked or pooled with data from other products.
 
As FDA contemplates mechanisms to responsibly release data from approved marketing applications, BIO recommends that FDA first conduct a formalized review of voluntary data sharing initiatives, such as the Cardiac Safety Research Consortium, to share lessons learned, to evaluate if these initiatives have been productive and efficient in addressing scientific questions, and to identify the gaps FDA’s own initiative could address. BIO strongly recommends that the Agency then consider a pilot program (e.g., data sharing in indications where there is focused research, such as an indication where there have been multiple trial failures, so that research may be conducted to develop a more reliable study design) as a next step.
 
As FDA develops its plan, it is essential for the Agency to publish for comment a complete, detailed, and transparent proposal for data sharing prior to its implementation. FDA’s plan, at minimum, should:
 
  • Clarify the purpose of its data-sharing proposal (e.g., is the purpose to advance regulatory science/fill knowledge gaps through qualified third party research, to assist the FDA in pursuing its own research, or both?). It is imperative that data sharing arrangements not be used as a means to evaluate data to influence regulatory decisions under the FDA’s purview or as a way to shortcut regulatory exclusivity and intellectual property protections.
  • Clarify the scope of its data-sharing proposal (e.g., applicable only to confirmatory trials for marketed drug, biologic, or device products, regardless of whether results are considered “positive” or “negative” ).
  • Clarify effective date for potential availability of applicable data sets (e.g., data sharing arrangements are applicable to data sets held by FDA upon effective date of any future Guidance issued).
  • Define the criteria that constitute qualified research, including development of a list of important research categories that are within scope and specification of criteria used to select a product class or indication.
  • Define “de-identified data” and parameters to ensure prevention of re-identification and fully protect the privacy of patients. The provision of de-identification must be consistent with informed consent and applicable privacy laws and regulations.
  • Define “masked data” and safeguards that will ensure the maintenance of masking efforts, thereby protecting commercial confidential information. Sponsors of approved applications should have an opportunity to address with FDA which particular data would be protected by trade secret/CCI provisions even if masked.
  • Ensure reciprocal transparency for Sponsors whose data are being disclosed by:
    • Defining “non FDA expert” qualified researchers who could be granted access to data (e.g., academic researchers, government scientists, etc).
    • Defining the process for evaluating qualified research requests, including processes for evaluating whether the available data can support the research objective, specifying the duration of time that data will be accessible (i.e., limited duration), and requiring research requestors to submit a research proposal for review by FDA and the Sponsor company. Requestors should be required to enter into agreements with Sponsors outlining the scope of research, as well as other parameters and safeguards, prior to being granted access to data sets.
    • Defining a notification process that will ensure Sponsors are notified before their data are released.
  • Identify appropriate financial and staff resources necessary to mask, de-identify, and redact data for potential disclosure. The aggregation, standardization and electronic conversion of clinical data for products submitted in paper or legacy formats alone represents a considerable added workload burden on the Agency. In this era of budgetary austerity, it is important that FDA prioritize its limited resources towards its core mission activities.