Statistics and Medical ExperimentationAuthor(s): James A. Hagans and Paul MeierSource: Biometrics, Vol. 31, No. 4 (Dec., 1975), p. 996Published by: International Biometric SocietyStable URL: http://www.jstor.org/stable/2529829 .

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996 BIOMETRICS, DECEMBER 1975

From James A. Hagans VACO Cooperative Studies Program Medical Research Service

Washington, D.C. To the Editor of Biometrics:

The article on "Statistics and Medical Experimentation" by Paul Meier [Biometrics 31:511-29, June, 1975] was both thoughtful and provocative. I should appreciate the opportunity to express the opinion that decisions made during the course of a clinical trial should be based on valid conclusions substantiated by the experimental data. On the surface, any other course of action seems pure folly, and decisions made sooner than that would of course abort and in general destroy the ability to ever attain a valid conclusion. It is certainly time that the professions (medical research and medical biostatistical) unite and support some such rational position, and remove this area once and for all from the political arena (and other arenas perhaps equally undesirable or worse such as that of the economic, personal glory, financial reward, etc.) Just as there can be no therapy without risk, there can be no medical experimentation without risk. It is highly desirable that society recognize this (I personally think most do) and that individuals participate with this in mind. Medical experimentation is serious business, should not exist (exclusive of pilot trials) except on a sound scientific base, and should be stopped when valid con- clusions are obtained-but not before-certainly not out of fear, or political considerations or based on hunches. It should be recognized that any therapeutic trial carried to the point of valid conclusions demonstrating one therapy superior to another at once becomes "unethical" in hindsight. It is perhaps a greater digression to stop a trial short of valid conclusions on the presumption that one therapy might be better than the other (i.e., at the earliest appearance of a trend).

Occasionally, a trial must be stopped short of conclusions where, for example, it becomes clear that prior planning was in gross error, or that sample sizes beyond the resources available would be necessary for valid conclusions, or that no hope exists within the frame of the trial (i.e., poor execution of a trial, defectively planned trial, or an unanticipated major event occurs not planned for and not able to be properly evaluated in the frame of the present trial, etc.)-situations perhaps not far removed from that of the well known UGDP effort-but when this is done, it should be so reported and with an urgent statement that an appropriate trial be reinstituted to provide conclusions to the important questions left unanswered in the aborted trial. Rarely have I see such a report in the literature, yet such situations in real life are not all that uncommon. This less than candid, if not less than honest, predicament seems to be endorsed not only by the medical researcher and medical biostatistician, but perhaps also by the granting agencies and controllers of medical research publications, etc. (It is rumored that it is difficult to have "negative results" published.) Admittedly it takes courage, but presumably not beyond the call of duty, to publicly acknowledge the expenditure of large sums of money that failed to provide answers to the questions addressed. The pressures are great to make such situations count for something. The medical biostatistician can help this situation by assuming his proper role as a collaborating health-scientist professional rather than the more common role analogous to a shoe clerk, so aptly described recently by IDJ Bross in the November '74 issue of The American Statistician. (Parenthetically, this would be greatly enhanced by proper education, training and certification of the medical biostatistician-a far cry from the present graduate who at least grasps something of the theory behind statistical practice, but preciously little of anything else.) I suggest a joint committee of medical biostatisticians and medical researchers could be appointed to consider this most serious problem and offer a rational solution, else the door to the barn may be closed after the horse is gone.

I further urge the Editor of Biometrics to actively solicit and publish additional views and opinions on the vitally important issues Paul Meier has so aptly raised in his landmark article. Constructive dialogue is sorely needed, and long overdue.

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