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PRESIDENT’S INVITED COLUMN
The FDA has a variety of issues to deal with. For many years, the primary focus was on examining the efficacy and safety of products in clinical
trials. In the last few years, there has been increasing emphasis on post-marketing safety evaluation. This is different from pre-marketing evalu-
ation in that with usage, more adverse events and a greater variety of those events occur. One is not easily able to conduct significance tests on
outcomes that are not pre-specified. We also hear a great deal of de-emphasis on hypothesis testing with many suggestions for alternatives. However,
the ultimate decision on product approval or non-approval is a dichotomous one. The risk to the public is essentially the type I error, and the risk
to the sponsor is the type II error.
~ Tony Lachenbruch, ASA President
Statisticians at the
Food and Drug Administration
Robert O’Neill, Greg Campbell, and Henry Hsu
T
his is an exciting time for team is usually composed of physicians and other scientists
statisticians at the Food needed to evaluate the submission, such as pharmacologists,
and Drug Administration. toxicologists, chemists, engineers, biologists, bacteriologists,
A big thank you to Tony virologists, physicists, geneticists, epidemiologists, and safe-
Lachenbruch—who used to be ty experts. Members of the team meet with the sponsor dur-
one of us—for giving us the ing the process, and the FDA statistician represents the agency
opportunity to report on interest- in terms of the statistical aspects of the evidence presented in
ing challenges for statisticians the submission.
from the perspective of the three It is not uncommon for extensive statistical discussion to
biostatistical groups we head: occur at these meetings and for the sponsor to invite academic
Center for Drug Evaluation and and other expert statistical consultants along with their own stat-
Research (CDER), Center for istician to aid in the discussion. What distinguishes the work
Devices and Radiological Health (CDRH), and Center for Biologics at FDA from work as an academic biostatistician or a statisti-
Evaluation and Research (CBER). The FDA employs more than 200 cian at the National Institutes of Health (NIH) is the need for
PhD and master's-level statisticians who have a range of backgrounds the agency to make a decision about the quality and strength of
and skills and substantially contribute on a daily basis to the decisions evidence presented by a particular submission, to do so within
FDA makes. There are many new opportunities for statisticians who a timeframe dictated by regulations, and to document that deci-
want to contribute to FDA's mission of promoting and protecting the sion in writing. When the evidence is lacking, the agency needs
health of those living in the United States. to provide feedback to the sponsor to indicate what is needed to
bring it into a form where the claim is supported and the product
What Do Statisticians at FDA Do?
is able to be marketed.
Most of our work is concentrated on the evaluation of applica-
tions submitted to the agency prior to marketing. This pre-market
What Skills Are Most Helpful at the FDA?
activity includes both the evaluation of trial plans (protocols) and Almost all FDA statistical reviewers have a master’s or, more like-
the final reports (including statistical analyses) for concluded stud- ly, a doctoral degree in statistics or biostatistics. This is helpful
ies. The best kind of evidence a sponsor can submit is based on because the kinds of methodological challenges faced are rarely
adequate and well-controlled clinical trials. simple textbook applications. Some reviewers have other valuable
Clinical trials are randomized experiments performed on humans training in areas such as pharmacology, microbiology, and engi-
to investigate unproven therapies or diagnostics. As most of the neering. While familiarity with biology and medicine is helpful,
products we evaluate pose a significant risk, the FDA is charged it is not always required.
with safeguarding trial participants from exposure to such products The ability to communicate both orally and in writing is
if there is little likelihood of success. Statisticians contribute to this important. Usually, a statistician gains experience with the regula-
charge through the review of study designs, sample size calculations, tory nature of the job and the laws under which FDA functions
and analysis plans. and incorporates it into their statistical decisionmaking. What is
It is important to note that the sponsors are fundamentally important is the desire to continually learn, for there are many
responsible for the analysis of their studies, which they submit new products to evaluate, new statistical techniques, and new
to the FDA as evidence of safety and effectiveness. The FDA sta- medical ideas.
tistical reviewer works as a member of an internal interdisciplin- To occupy a permanent government position, it is required
ary team to evaluate the evidence in the submission to support that the applicant be a U.S. citizen, but there are several hiring
a marketing claim consisting of studies of different designs. This mechanisms for permanent residents and other noncitizens. Many
2 AMSTAT NEWS OCTOBER 2008
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