December 16, 2013
Dockets Management Branch (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852
Re: Docket No. FDA-2013-N-1276: Meta-Analyses of Randomized Controlled Clinical Trials (RCTs) for the Evaluation of Risk To Support Regulatory Decisions; Notice of Public Meeting; Request for Comment
The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on Meta-Analyses of Randomized Controlled Clinical Trials (RCTs) for the Evaluation of Risk To Support Regulatory Decisions.
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.
BIO fully supports FDA’s initiative under PDUFA V to engage industry, academia, researchers, providers, and the patient community in a public dialogue regarding best practices for the use of meta-analysis in regulatory decision-making. BIO believes that the November 25th Public Meeting was an important first step towards the development and adoption of rigorous common standards for third party conduct of meta-analysis. Further, this program can help to provide FDA with the tools and capacity to evaluate and conduct meta-analyses, and educate the public on the strengths and potential biases of these studies.
When skillfully conducted, meta-analysis is one way researchers can combine and evaluate different bodies of research to determine the answers to important questions regarding the safety and efficacy of medical products. However, as use of meta-analysis grows, it is imperative that the proper methods are used in order to draw meaningful conclusions from these analyses. This is particularly important when these studies inform FDA regulatory decisions that can BIO Comments on Meta-Analysis Docket: FDA-2013-N-1276, December 16, 2013, Page 2 of 2 significantly affect the public health or impact patient access to potentially beneficial drugs and biologic products. Meta-analysis can be a powerful tool in evaluating health care treatments and interventions, but there are many potential pitfalls and problems that are yet to be resolved. While the idea behind combining studies to improve precision and power is straightforward, the actual implementation of the process is difficult. Those who act or react based on metaanalysis - including regulators, industry, providers, caregivers, and patients - should understand the various biases that could be incorporated into a review.
With these challenges and potential opportunities in mind, BIO commissioned the following white paper on Methodological Challenges Associated with Meta-Analyses in Health Care and Behavioral Research (Shinogle, 2012). The paper presents an overview of meta-analysis technique, provides a description of a sound metaanalysis methodology, and discusses challenges and issues involved with developing a meta-analysis study. The paper concludes with a discussion of the use and interpretation of the technique as well as questions that should be asked of a meta-analysis study.
BIO hopes that FDA finds this paper as a useful and informative resource as the Agency develops guidance on this topic.
BIO appreciates this opportunity to comment on Meta-Analyses of Randomized Controlled Clinical Trials (RCTs) for the Evaluation of Risk To Support Regulatory Decisions. We would be pleased to provide further input or clarification of our comments, as needed...
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