Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses

J.H. Rex; T.J. Walsh; M. Nettleman; E.J. Anaissie; J.E. Bennett; E.J. Bow; A.J. Carillo-Munoz; P. Chavanet; G.A. Cloud; D.W. Denning; B.E. De Pauw; J.E. Edwards Jr; J.W. Hiemenz; C.A. Kauffman; G. Lopez-Berestein; P. Martino; J.D. Sobel; D.A. Stevens; R. Sylvester; J. Tollemar; C. Viscoli; M.A. Viviani; T. Wu

doi:10.1086/320876

Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses

J.H. Rex^*, T.J. Walsh, M. Nettleman, E.J. Anaissie, J.E. Bennett, E.J. Bow, A.J. Carillo-Munoz, P. Chavanet, G.A. Cloud, D.W. Denning, B.E. De Pauw, J.E. Edwards Jr, J.W. Hiemenz, C.A. Kauffman, G. Lopez-Berestein, P. Martino, J.D. Sobel, D.A. Stevens, R. Sylvester, J. TollemarC. Viscoli, M.A. Viviani, T. Wu

^*Corresponding author for this work

Division of Evolution, Infection and Genomics

Research output: Contribution to journal › Article › peer-review

Abstract

Studies of invasive fungal infections have been and remain difficult to implement. Randomized clinical trials of fungal infections are especially slow and expensive to perform because it is difficult to identify eligible patients in a timely fashion, to prove the presence of the fungal infection in an unequivocal fashion, and to evaluate outcome in a convincing fashion. Because of these challenges, licensing decisions for antifungal agents have to date depended heavily on historical control comparisons and secondary advantages of the new agent. Although the availability of newer and potentially more effective agents makes these approaches less desirable, the fundamental difficulties of trials of invasive fungal infections have not changed. Therefore, there is a need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses, and this article summarizes the possible strategies in this area.

Original language	English
Pages (from-to)	95–106
Number of pages	12
Journal	Clinical infectious diseases
Volume	33
Issue number	1
DOIs	https://doi.org/10.1086/320876
Publication status	Published - 2001

Access to Document

10.1086/320876

Cite this

Rex, J. H., Walsh, T. J., Nettleman, M., Anaissie, E. J., Bennett, J. E., Bow, E. J., Carillo-Munoz, A. J., Chavanet, P., Cloud, G. A., Denning, D. W., De Pauw, B. E., Edwards Jr, J. E., Hiemenz, J. W., Kauffman, C. A., Lopez-Berestein, G., Martino, P., Sobel, J. D., Stevens, D. A., Sylvester, R., ... Wu, T. (2001). Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses. Clinical infectious diseases , 33(1), 95–106. https://doi.org/10.1086/320876

@article{069d9029740a49f5933f98410d1e1f5a,

title = "Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses",

abstract = "Studies of invasive fungal infections have been and remain difficult to implement. Randomized clinical trials of fungal infections are especially slow and expensive to perform because it is difficult to identify eligible patients in a timely fashion, to prove the presence of the fungal infection in an unequivocal fashion, and to evaluate outcome in a convincing fashion. Because of these challenges, licensing decisions for antifungal agents have to date depended heavily on historical control comparisons and secondary advantages of the new agent. Although the availability of newer and potentially more effective agents makes these approaches less desirable, the fundamental difficulties of trials of invasive fungal infections have not changed. Therefore, there is a need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses, and this article summarizes the possible strategies in this area.",

author = "J.H. Rex and T.J. Walsh and M. Nettleman and E.J. Anaissie and J.E. Bennett and E.J. Bow and A.J. Carillo-Munoz and P. Chavanet and G.A. Cloud and D.W. Denning and {De Pauw}, B.E. and {Edwards Jr}, J.E. and J.W. Hiemenz and C.A. Kauffman and G. Lopez-Berestein and P. Martino and J.D. Sobel and D.A. Stevens and R. Sylvester and J. Tollemar and C. Viscoli and M.A. Viviani and T. Wu",

year = "2001",

doi = "10.1086/320876",

language = "English",

volume = "33",

pages = "95–106",

journal = "Clinical infectious diseases ",

issn = "1058-4838",

publisher = "Oxford University Press",

number = "1",

}

Rex, JH, Walsh, TJ, Nettleman, M, Anaissie, EJ, Bennett, JE, Bow, EJ, Carillo-Munoz, AJ, Chavanet, P, Cloud, GA, Denning, DW, De Pauw, BE, Edwards Jr, JE, Hiemenz, JW, Kauffman, CA, Lopez-Berestein, G, Martino, P, Sobel, JD, Stevens, DA, Sylvester, R, Tollemar, J, Viscoli, C, Viviani, MA & Wu, T 2001, 'Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses', Clinical infectious diseases , vol. 33, no. 1, pp. 95–106. https://doi.org/10.1086/320876

TY - JOUR

T1 - Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses

AU - Rex, J.H.

AU - Walsh, T.J.

AU - Nettleman, M.

AU - Anaissie, E.J.

AU - Bennett, J.E.

AU - Bow, E.J.

AU - Carillo-Munoz, A.J.

AU - Chavanet, P.

AU - Cloud, G.A.

AU - Denning, D.W.

AU - De Pauw, B.E.

AU - Edwards Jr, J.E.

AU - Hiemenz, J.W.

AU - Kauffman, C.A.

AU - Lopez-Berestein, G.

AU - Martino, P.

AU - Sobel, J.D.

AU - Stevens, D.A.

AU - Sylvester, R.

AU - Tollemar, J.

AU - Viscoli, C.

AU - Viviani, M.A.

AU - Wu, T.

PY - 2001

Y1 - 2001

N2 - Studies of invasive fungal infections have been and remain difficult to implement. Randomized clinical trials of fungal infections are especially slow and expensive to perform because it is difficult to identify eligible patients in a timely fashion, to prove the presence of the fungal infection in an unequivocal fashion, and to evaluate outcome in a convincing fashion. Because of these challenges, licensing decisions for antifungal agents have to date depended heavily on historical control comparisons and secondary advantages of the new agent. Although the availability of newer and potentially more effective agents makes these approaches less desirable, the fundamental difficulties of trials of invasive fungal infections have not changed. Therefore, there is a need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses, and this article summarizes the possible strategies in this area.

AB - Studies of invasive fungal infections have been and remain difficult to implement. Randomized clinical trials of fungal infections are especially slow and expensive to perform because it is difficult to identify eligible patients in a timely fashion, to prove the presence of the fungal infection in an unequivocal fashion, and to evaluate outcome in a convincing fashion. Because of these challenges, licensing decisions for antifungal agents have to date depended heavily on historical control comparisons and secondary advantages of the new agent. Although the availability of newer and potentially more effective agents makes these approaches less desirable, the fundamental difficulties of trials of invasive fungal infections have not changed. Therefore, there is a need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses, and this article summarizes the possible strategies in this area.

UR - http://www.scopus.com/inward/record.url?eid=2-s2.0-0034983131&partnerID=MN8TOARS

U2 - 10.1086/320876

DO - 10.1086/320876

M3 - Article

SN - 1058-4838

VL - 33

SP - 95

EP - 106

JO - Clinical infectious diseases

JF - Clinical infectious diseases

IS - 1

ER -

Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses

Abstract

Access to Document

Other files and links

Fingerprint

Cite this