Re: Docket No. FDA-2016-D-1174
The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on the “Special Protocol Assessment; Draft Guidance for Industry.”
BIO is the world's largest trade association representing 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.
BIO is supportive of FDA’s efforts to clarify the special protocol assessment (SPA) process for sponsors through the updated draft Special Protocol Assessment guidance, which provides helpful clarification on which protocols are eligible for SPA, as well as other updates and details.
Notwithstanding that the draft guidance is helpful in describing FDA’s processes, generally, BIO believes that the final guidance should place more emphasis on, and describe opportunities for, less formal modes of communication (e.g., by telephone or email to allow for timely resolution of any area(s) of disagreement) throughout the SPA process (e.g., during the initial 45-day review period, following receipt of a non-agreement letter, when an amendment may or may not warrant re-submission, during review of a re-submission). Such open and less formal interactions between sponsors and FDA would facilitate reaching agreement in an efficient and effective manner.
Similarly, BIO believes that implementation and consistent application of the final version of the guidance offers an opportunity to focus on the importance of cross-division and within-division consistency in SPA processes. Moreover, the draft guidance and process, in general, would benefit from development of assessment process tools (e.g., checklists, Manual of Policies and Procedures [MaPPs], Standard Operating Policies and Procedures [SOPPs]) which are likely to promote predictability of outcomes.
To provide clarity to sponsors looking to determine the value of pursuing a SPA in the context of complex design questions, further description of the concepts of “substantial scientific issue” and “essential to determining the safety or efficacy” are crucial. Although some general scenarios are provided in Section IX. “Changes in Or Rescission of Special Protocol Assessment Agreements,” they cover a broad range of possibilities that may be open to interpretation, potentially resulting in concerns about reliance on SPA agreements as mitigation against uncertainty in late-stage development as well as potentially dissuading sponsors from pursuing SPA discussions with the agency.
Likewise, it would be helpful for the final version of the guidance to include more procedural details, such as further description of the process for meetings on agreement and rescission of a SPA (e.g., Type A).
Lastly, we note that use of the terms “trial” and “study” is not consistent throughout the draft guidance; likewise, the term “animal trial” is not commonly used in industry. Consistency in utilization of these terms in the final version of the guidance would be helpful.
We provide additional, more specific comments on the draft guidance in the table following this text. We would be pleased to provide further input or clarification of our comments, as needed.