Findings from individual subjects can establish drug efficacy de facto. For this to happen, the variability of the crucial endpoint in the untreated population needs to be actuarially or theoretically negligible. Under such circumstances positive findings from minimalist designs can be more persuasive than statistically significant findings from large samples. Background knowledge affects the logic of selecting sample sizes that enable sound inductive inference.
The impact of randomized clinical trials on health policy and medical practice: Background paper OTA-BP-H-22. U.S. Congress: Office of Technology Assessment. Washington D.C., August, 1983.
2.
Guideline for the format and content of the clinical and statistical sections of new drug applications. U.S. Department of Health and Human Services, Public Health Administration, Food and Drug Administration, July, 1988.
3.
ThagardPNisbettRE. Variability and confirmation. Philosoph Stud.1982;42: 379–394.
4.
SimpsonRJGriggsTR. Case reports and medical progress. Perspect Biol Med.1985;28: 403–406.
5.
HunterKM. “There was this one guy …”: The uses of anecdotes in medicine. Persp Biol Med.1986;29: 619–630.
6.
HunterKM. An N of 1: Syndrome letters in the New England Journal of Medicine. Persp Biol Med.1990;33: 237–251.
7.
KazdinAE. Single-case Research Designs: Methods for Clinical and Applied Settings.New York: Oxford University Press, 1982.
GuyattGSackettDTaylorDWChongJRobertsRPugsleyS.Determining optimal therapy—randomized trials in individual patients. N Engl J Med.1986;314: 889–892.
10.
CohenMRNagelE.An Introduction to Logic and Scientific Method.New York: Harcourt Brace, 1934.
11.
HollandJHHolyoakKJNisbettREThagardPR. Induction: Processes of Inference, Learning, and Discovery.Cambridge, Massachusetts: The MIT Press, 1986.
12.
CohenLJ. An Introduction to the Philosophy of Induction and Probability.Oxford: Clarendon Press, 1989.
13.
CampbellMAndersonLTSmallAMLocascioJJLynchNSChorocoMC. Naltrexone in autistic children: A double-blind and placebo-controlled study. Psychopharm Bull.1990;26: 130–135.
14.
SafrinSRushJDMillsJ.Influenza in patients with human immunodeficiency virus infection. Chest.1990;98: 33–37.
15.
De VriesJBaarsmaGSZaalMJBoen–TanTNRothovaABuitenhuisHJCyclosporin in the treatment of severe idiopathic uveitis. Br J Ophthal.1990;74: 344–349.
TaylorP.Agents acting at the neuromuscular junction and autonomic ganglia. In: GilmanAGRallTWMuradF, eds. Goodman and Gilman's The Pharmacological Basis of Therapeutics, eighth ed.New York: MacMillan Publishing Co., Inc.; 1990: 166–186.
18.
SmithSMBrownHOTomanJEPGoodmanLS. The lack of cerebral effects of d-tubocurarine. Anesthesiology.1947;8: 1–14.
19.
KitlerME. The changing face of clinical trials. J Hypertension.1988;6 (Suppl 1) s73–s80.
KennedySKLongneckerDE. History and principles of anesthesiology. In: GilmanAGRallTWMuradF, eds. Goodman and Gilman's The Pharmacological Basis of Therapeutics, eighth ed.New York: MacMillan Publishing Co., Inc.; 1990: 269–284.
22.
LarnerJ.Insulin and oral hypoglycemic drugs; glucagon. In: GilmanAGRallTWMuradF, eds. Goodman and Gilman's The Pharmacological Basis of Therapeutics, eighth ed.New York: MacMillan Publishing Co., Inc.; 1985: 1490–1516.
23.
HofmannA.Psychotomimetic agents. In: BurgerA, ed. Drugs Affecting the Central Nervous System, Vol. 2. New York: Marcel Dekker; 1968: 169–235.
24.
MazurRH. In: SteginkLDFilerLFJr, eds. Aspartame: Advances in Physiology and Biochemistry.New York: Marcel Dekker Inc.; 1984: 3–9.
25.
CoxJSG. Cromolyn sodium. In: GoldbergME, ed. Pharmacological and Biochemical Properties of Drug Substances. Washington, D.C.: American Pharmaceutical Association Academy of Pharmaceutical Sciences; 1977: 277–310.
26.
SchwabRSEnglandACJr.PoskanzerDCYoungRR. Amantadine in the treatment of Parkinson's disease. J Am Med Assoc.1969;208: 1168–1170.
27.
WardellWMSheckLE. Is pharmaceutical innovation declining?: Interpreting measures of pharmaceutical innovation and regulatory impact in the USA, 1950–1980. Rational Drug Ther.1983;17;1–5.
28.
FriedmanWFHirschklauMJPrintzMPPitlickPTKirkpatrickSE. Pharmacologic closure of patent ductus arteriosus in the premature infant. N Engl J Med.1976;295: 526–529.
