TY - JOUR
T1 - Clinical trial design for the development of new therapies for nonmuscle-invasive bladder cancer
T2 - Report of a food and drug administration and american urological association public workshop
AU - Jarow, Jonathan P.
AU - Lerner, Seth P.
AU - Kluetz, Paul G.
AU - Liu, Ke
AU - Sridhara, Rajeshwari
AU - Bajorin, Dean
AU - Chang, Sam
AU - Dinney, Colin P.N.
AU - Groshen, Susan
AU - Morton, Ronald A.
AU - O'Donnell, Michael
AU - Quale, Diane Zipursky
AU - Schoenberg, Mark
AU - Seigne, John
AU - Vikram, Bhadrasain
PY - 2014/2
Y1 - 2014/2
N2 - Objective To summarize the discussion at a public workshop, cosponsored by the U.S. Food and Drug Administration (FDA) and the American Urological Association, reviewing potential trial designs for the development of new therapies for non-muscle-invasive bladder cancer (NMIBC). There have been only 3 drug approvals for NMIBC in the last 30 years, and product development for this disease has been stymied by difficulties in trial design and patient accrual. Methods A workshop evaluating potential trial design for the development of therapies for NMIBC was held in San Diego, CA, in May 2013. Invited experts representing all stakeholders, including urology, medical oncology, radiation oncology, industry, and patient advocates, discussed development of products for all risk strata of NMIBC. Results The panel responded to specific questions from the FDA, discussing eligibility criteria, efficacy endpoints, and trial design for patients with a mix of high-grade papillary disease and carcinoma in situ, Bacillus Calmette-Guerin (BCG)-refractory disease, and intermediate-risk disease. Panel members also addressed the magnitude of response that would be clinically meaningful for various disease strata and trial design options for perioperative intravesical chemotherapy instillation at the time of resection of bladder tumors. Conclusion Expert commentary provided by panel members will inform a planned FDA guidance on pathways for drug and biologic development for NMIBC and will be discussed at meetings of the FDA's Oncologic Drugs Advisory Committee. FDA intends to develop a set of principles that can be used to promote the development of new products for this disease.
AB - Objective To summarize the discussion at a public workshop, cosponsored by the U.S. Food and Drug Administration (FDA) and the American Urological Association, reviewing potential trial designs for the development of new therapies for non-muscle-invasive bladder cancer (NMIBC). There have been only 3 drug approvals for NMIBC in the last 30 years, and product development for this disease has been stymied by difficulties in trial design and patient accrual. Methods A workshop evaluating potential trial design for the development of therapies for NMIBC was held in San Diego, CA, in May 2013. Invited experts representing all stakeholders, including urology, medical oncology, radiation oncology, industry, and patient advocates, discussed development of products for all risk strata of NMIBC. Results The panel responded to specific questions from the FDA, discussing eligibility criteria, efficacy endpoints, and trial design for patients with a mix of high-grade papillary disease and carcinoma in situ, Bacillus Calmette-Guerin (BCG)-refractory disease, and intermediate-risk disease. Panel members also addressed the magnitude of response that would be clinically meaningful for various disease strata and trial design options for perioperative intravesical chemotherapy instillation at the time of resection of bladder tumors. Conclusion Expert commentary provided by panel members will inform a planned FDA guidance on pathways for drug and biologic development for NMIBC and will be discussed at meetings of the FDA's Oncologic Drugs Advisory Committee. FDA intends to develop a set of principles that can be used to promote the development of new products for this disease.
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U2 - 10.1016/j.urology.2013.10.030
DO - 10.1016/j.urology.2013.10.030
M3 - Article
C2 - 24332121
AN - SCOPUS:84895069903
SN - 0090-4295
VL - 83
SP - 262
EP - 265
JO - Urology
JF - Urology
IS - 2
ER -