Clinical trial design for the development of new therapies for nonmuscle-invasive bladder cancer: Report of a food and drug administration and american urological association public workshop

Jonathan P. Jarow, Seth P. Lerner, Paul G. Kluetz, Ke Liu, Rajeshwari Sridhara, Dean Bajorin, Sam Chang, Colin P N Dinney, Susan Groshen, Ronald A. Morton, Michael O'Donnell, Diane Zipursky Quale, Mark P. Schoenberg, John Seigne, Bhadrasain Vikram

Research output: Contribution to journalArticle

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Abstract

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.

Original languageEnglish (US)
Pages (from-to)262-264
Number of pages3
JournalUrology
Volume83
Issue number2
DOIs
StatePublished - Feb 2014
Externally publishedYes

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United States Food and Drug Administration
Urinary Bladder Neoplasms
Clinical Trials
Education
Therapeutics
Pharmacy and Therapeutics Committee
Intravesical Administration
Drug Approval
Radiation Oncology
Medical Oncology
Muscle Development
Papillary Carcinoma
Carcinoma in Situ
Urology
Advisory Committees
Mycobacterium bovis
Industry
Drug Therapy
Pharmaceutical Preparations

ASJC Scopus subject areas

  • Urology

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Clinical trial design for the development of new therapies for nonmuscle-invasive bladder cancer : Report of a food and drug administration and american urological association public workshop. / Jarow, Jonathan P.; Lerner, Seth P.; Kluetz, Paul G.; Liu, Ke; Sridhara, Rajeshwari; Bajorin, Dean; Chang, Sam; Dinney, Colin P N; Groshen, Susan; Morton, Ronald A.; O'Donnell, Michael; Quale, Diane Zipursky; Schoenberg, Mark P.; Seigne, John; Vikram, Bhadrasain.

In: Urology, Vol. 83, No. 2, 02.2014, p. 262-264.

Research output: Contribution to journalArticle

Jarow, JP, Lerner, SP, Kluetz, PG, Liu, K, Sridhara, R, Bajorin, D, Chang, S, Dinney, CPN, Groshen, S, Morton, RA, O'Donnell, M, Quale, DZ, Schoenberg, MP, Seigne, J & Vikram, B 2014, 'Clinical trial design for the development of new therapies for nonmuscle-invasive bladder cancer: Report of a food and drug administration and american urological association public workshop', Urology, vol. 83, no. 2, pp. 262-264. https://doi.org/10.1016/j.urology.2013.10.030
Jarow, Jonathan P. ; Lerner, Seth P. ; Kluetz, Paul G. ; Liu, Ke ; Sridhara, Rajeshwari ; Bajorin, Dean ; Chang, Sam ; Dinney, Colin P N ; Groshen, Susan ; Morton, Ronald A. ; O'Donnell, Michael ; Quale, Diane Zipursky ; Schoenberg, Mark P. ; Seigne, John ; Vikram, Bhadrasain. / Clinical trial design for the development of new therapies for nonmuscle-invasive bladder cancer : Report of a food and drug administration and american urological association public workshop. In: Urology. 2014 ; Vol. 83, No. 2. pp. 262-264.
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AU - Dinney, Colin P N

AU - Groshen, Susan

AU - Morton, Ronald A.

AU - O'Donnell, Michael

AU - Quale, Diane Zipursky

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AU - Seigne, John

AU - Vikram, Bhadrasain

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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.

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