TY - JOUR
T1 - Interpreting the clinical importance of group differences in chronic pain clinical trials
T2 - IMMPACT recommendations
AU - Dworkin, Robert H.
AU - Turk, Dennis C.
AU - McDermott, Michael P.
AU - Peirce-Sandner, Sarah
AU - Burke, Laurie B.
AU - Cowan, Penney
AU - Farrar, John T.
AU - Hertz, Sharon
AU - Raja, Srinivasa N.
AU - Rappaport, Bob A.
AU - Rauschkolb, Christine
AU - Sampaio, Cristina
N1 - Funding Information:
The views expressed in this article are those of the authors, none of whom have financial conflicts of interest related to the material presented in this manuscript. No official endorsement by the US Food and Drug Administration or the pharmaceutical companies that have provided unrestricted grants for the support of IMMPACT to the University of Rochester Office of Continuing Professional Education should be inferred.
PY - 2009/12/5
Y1 - 2009/12/5
N2 - An essential component of the interpretation of results of randomized clinical trials of treatments for chronic pain involves the determination of their clinical importance or meaningfulness. This involves two distinct processes-interpreting the clinical importance of individual patient improvements and the clinical importance of group differences-which are frequently misunderstood. In this article, we first describe the essential differences between the interpretation of the clinical importance of patient improvements and of group differences. We then discuss the factors to consider when evaluating the clinical importance of group differences, which include the results of responder analyses of the primary outcome measure, the treatment effect size compared to available therapies, analyses of secondary efficacy endpoints, the safety and tolerability of treatment, the rapidity of onset and durability of the treatment benefit, convenience, cost, limitations of existing treatments, and other factors. The clinical importance of individual patient improvements can be determined by assessing what patients themselves consider meaningful improvement using well-described methods. In contrast, the clinical meaningfulness of group differences must be determined by a multi-factorial evaluation of the benefits and risks of the treatment and of other available treatments for the condition in light of the primary goals of therapy. Such determinations must be conducted on a case-by-case basis, and are ideally informed by patients and their significant others, clinicians, researchers, statisticians, and representatives of society at large.
AB - An essential component of the interpretation of results of randomized clinical trials of treatments for chronic pain involves the determination of their clinical importance or meaningfulness. This involves two distinct processes-interpreting the clinical importance of individual patient improvements and the clinical importance of group differences-which are frequently misunderstood. In this article, we first describe the essential differences between the interpretation of the clinical importance of patient improvements and of group differences. We then discuss the factors to consider when evaluating the clinical importance of group differences, which include the results of responder analyses of the primary outcome measure, the treatment effect size compared to available therapies, analyses of secondary efficacy endpoints, the safety and tolerability of treatment, the rapidity of onset and durability of the treatment benefit, convenience, cost, limitations of existing treatments, and other factors. The clinical importance of individual patient improvements can be determined by assessing what patients themselves consider meaningful improvement using well-described methods. In contrast, the clinical meaningfulness of group differences must be determined by a multi-factorial evaluation of the benefits and risks of the treatment and of other available treatments for the condition in light of the primary goals of therapy. Such determinations must be conducted on a case-by-case basis, and are ideally informed by patients and their significant others, clinicians, researchers, statisticians, and representatives of society at large.
KW - Chronic pain
KW - Clinical importance
KW - Clinical meaningfulness
KW - Effect size
KW - Group differences
KW - Randomized clinical trials
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U2 - 10.1016/j.pain.2009.08.019
DO - 10.1016/j.pain.2009.08.019
M3 - Review article
C2 - 19836888
AN - SCOPUS:70350621483
SN - 0304-3959
VL - 146
SP - 238
EP - 244
JO - Pain
JF - Pain
IS - 3
ER -