LEADER 04481nam 2200685 a 450 001 9910966753603321 005 20251017110111.0 010 $a9780309171144 010 $a0309171148 010 $a9780309513456 010 $a0309513456 035 $a(CKB)111069351122094 035 $a(EBL)3375259 035 $a(SSID)ssj0000246882 035 $a(PQKBManifestationID)11186331 035 $a(PQKBTitleCode)TC0000246882 035 $a(PQKBWorkID)10194596 035 $a(PQKB)10704404 035 $a(Au-PeEL)EBL3375259 035 $a(CaPaEBR)ebr10032465 035 $a(OCoLC)923255502 035 $a(MiAaPQ)EBC3375259 035 $a(Perlego)4735412 035 $a(DNLM)1127615 035 $a(BIP)53854110 035 $a(BIP)7054093 035 $a(EXLCZ)99111069351122094 100 $a20010823d2001 uy 0 101 0 $aeng 135 $aur|n|---||||| 181 $ctxt 182 $cc 183 $acr 200 00$aSmall clinical trials $eissues and challenges /$fCharles H. Evans, Jr. and Suzanne T. Ildstad, editors ; Committee on Strategies for Small-Number-Participant Clinical Research Trials, Board on Health Sciences Policy, Institute of Medicine 205 $a1st ed. 210 $aWashington, D.C. $cNational Academy Press$dc2001 215 $a1 online resource (221 p.) 225 1 $aCompass series 300 $aDescription based upon print version of record. 311 08$a9780309073332 311 08$a0309073332 320 $aIncludes bibliographical references (p. 177-200). 327 $a""Front Matter""; ""Reviewers""; ""Preface""; ""Contents""; ""Executive Summary""; ""1 Introduction""; ""2 Design of Small Clinical Trials""; ""3 Statistical Approaches to Analysis of Small Clinical Trials""; ""4 General Guidelines""; ""References""; ""Appendix A Study Methods""; ""Appendix B Glossary of Statistical and Clinical Trials Terms""; ""Appendix C Selected Bibliography on Small Clinical Trials""; ""Appendix D Committee and Staff Biographies"" 330 $aClinical trials are used to elucidate the most appropriate preventive, diagnostic, or treatment options for individuals with a given medical condition. Perhaps the most essential feature of a clinical trial is that it aims to use results based on a limited sample of research participants to see if the intervention is safe and effective or if it is comparable to a comparison treatment. Sample size is a crucial component of any clinical trial. A trial with a small number of research participants is more prone to variability and carries a considerable risk of failing to demonstrate the effectiveness of a given intervention when one really is present. This may occur in phase I (safety and pharmacologic profiles), II (pilot efficacy evaluation), and III (extensive assessment of safety and efficacy) trials. Although phase I and II studies may have smaller sample sizes, they usually have adequate statistical power, which is the committee's definition of a "large" trial. Sometimes a trial with eight participants may have adequate statistical power, statistical power being the probability of rejecting the null hypothesis when the hypothesis is false. Small Clinical Trials assesses the current methodologies and the appropriate situations for the conduct of clinical trials with small sample sizes. This report assesses the published literature on various strategies such as (1) meta-analysis to combine disparate information from several studies including Bayesian techniques as in the confidence profile method and (2) other alternatives such as assessing therapeutic results in a single treated population (e.g., astronauts) by sequentially measuring whether the intervention is falling above or below a preestablished probability outcome range and meeting predesigned specifications as opposed to incremental improvement. 410 0$aCompass series. 606 $aClinical trials 606 $aClinical trials$zUnited States 615 0$aClinical trials. 615 0$aClinical trials 676 $a610/.72/4 701 $aEvans$b Charles H$g(Charles Hawes),$f1940-$01804041 701 $aIldstad$b Suzanne T$01804042 712 02$aInstitute of Medicine (U.S.).$bCommittee on Strategies for Small-Number-Participant Clinical Research Trials. 801 0$bMiAaPQ 801 1$bMiAaPQ 801 2$bMiAaPQ 906 $aBOOK 912 $a9910966753603321 996 $aSmall clinical trials$94351885 997 $aUNINA