First Quantitative Assessment of Treatments for Non-muscle-invasive Bladder Cancer After Bacillus Calmette-Guerin (BCG) Therapy Published in European Urology Oncology

Date:May 6, 2020
Location:Boston, MA
Contact:Eric Seymour
617 425 8103

BOSTON, MA – May 06, 2020 – Analysis Group, a global leader in health economics and outcomes research (HEOR), announced the publication of the first study to quantitatively assess the efficacy and safety evidence of current and emerging treatments for patients with non-muscle-invasive bladder cancer (NMIBC) whose cancer recurred following treatment with Bacillus Calmette-Guerin (BCG) therapy. The meta-analysis, published in European Urology Oncology, is the first to quantitatively assess and compare current and emerging treatments for NMIBC after BCG, and sheds light on the challenges of future NMIBC trials and the evolving treatment landscape of this disease.

Currently, there is no standard of care for patients whose cancer recurs after BCG therapy. Radical cystectomy, which removes the bladder entirely, is typically recommended. Yet, many patients decline to undergo, or are ineligible to receive, this procedure. Multiple therapies are being investigated for use in this patient population. However, the variability in efficacy and safety outcomes as well as differences in patient population definitions make proper evaluation of these new treatments difficult. This study aims to provide a clear baseline for future clinical trials to overcome these challenges.

“After a thorough systematic literature review, it was clear to us that the first challenge faced was the need for consistent endpoint reporting and patient population definitions. Variability to the degree we saw creates a variety of challenges when trying to understand critical trial results for new treatments. We believe this to be a landmark study for its clinical relevance and timeliness,” said lead author Dr. Ashish Kamat of The University of Texas MD Anderson Cancer Center. The study has already been the focus of clinical video lectures, such as one given to the UroToday Journal Club.

Analysis Group Vice President Min Yang and Associate Mihaela Georgieva supported a systematic literature review of MEDLINE, Embase, and the Cochrane Central Register of Controlled Trials (CENTRAL) from January 2007 through June 2019, and abstracts and presentations from major conference proceedings. Data for single-arm trials were pooled using a random-effect meta-analysis with the proportions approach. Trials were grouped based on the minimum number of prior BCG courses required before enrollment and further stratified based on the proportion of patients with carcinoma in situ (CIS).

“While both the FDA and the International Bladder Cancer Group have put together guidance documents on clinical trial design for NMIBC, we found considerable variation, particularly across those studies published before the 2018 guidance was issued. This sharp divide in study design was particularly challenging when comparing results,” said Dr. Yang. “An important finding from our meta-analysis confirmed that a higher number of previous BCG courses, BCG-refractory/unresponsive status, CIS, and high-grade tumors are associated with lower treatment response. This understanding is key when interpreting results from studies that may have included less severe patients, such as some observational studies on intravesical chemotherapy agents.”

The study, “Evidence-based Assessment of Current and Emerging Bladder-sparing Therapies for Non-muscle-invasive Bladder Cancer After Bacillus Calmette-Guerin Therapy: A Systematic Review and Meta-analysis” was published in the March 2020 issue of European Urology Oncology. Coauthors include leading researchers: Drs. Ashish M. Kamat, University of Texas MD Anderson Cancer Center; Seth P. Lerner, Baylor College of Medicine; Michael O’Donnell, University of Iowa; Brant A. Inman, Duke University; Wassim Kassouf, McGill University Health Center; Stephen A. Boorjian, Mayo Clinic; Mark D. Tyson, Mayo Clinic; Girish S. Kulkarni, University of Toronto; Sam S. Chang, Vanderbilt University; Badrinath R. Konety, University of Minnesota; Robert S. Svatek, University of Texas Health Science Center; Arjun Balar, NYU Langone Health; and J. Alfred Witjes, Radboud University Nijmegen Medical Centre. Funding was provided by FerGene.

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Founded in 1981, Analysis Group is one of the largest international economics consulting firms, with more than 1,000 professionals across 14 offices. Analysis Group’s health care experts apply analytical expertise to health economics and outcomes research, clinical research, market access and commercial strategy, and health care policy engagements, as well as drug safety-related engagements in epidemiology. Analysis Group’s internal experts, together with its network of affiliated experts from academia, industry, and government, provide our clients with exceptional breadth and depth of expertise and end-to-end consulting services globally.