Transparency: BIO Comments on Availability of Masked and De-identified Non-Summary Safety and Efficacy Data
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.</p>
- 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.