Future Research Needs for Comparative Effectiveness of Treatments of Localized Prostate Cancer: Future Research Needs Paper Number 4

Publisher Marketing: An estimated 1.8 million men living in the United States have a diagnosis of prostate cancer, with about 218,890 newly diagnosed men each year. Approximately 90 percent of men with prostate cancer have disease considered confined to the prostate gland (i.e., clinically localized disease). If left untreated, frequently men die with, rather than from, prostate cancer. Largely because of widespread prostate-specific antigen (PSA) testing, the lifetime risk of prostate cancer diagnosis in the United States has nearly doubled to 20 percent, while the risk of dying of prostate cancer has remained at approximately 3 percent. Therefore, considerable over detection and treatment may exist. Moreover, the treatment of localized prostate cancer is associated with substantial adverse effects. The primary goal of treatment is to target those men most likely to need intervention to prevent prostate cancer death and disability, while minimizing intervention-related complications. Common treatments include watchful waiting (active surveillance), surgery to remove the prostate gland (i.e., radical prostatectomy), radiotherapy (e.g., external-beam radiation or brachytherapy), freezing the prostate (i.e., cryotherapy), and androgen-deprivation therapy (ADT). All treatments for prostate cancer have risks of complications, although their frequency and severity may vary. Common adverse events include urinary, bowel, and sexual dysfunction. The vast majority of prostate cancers currently detected in the United States are asymptomatic, clinically localized, and found on routine PSA testing. PSA testing detects more tumors, at an earlier stage, with a smaller volume within each stage, and at an earlier period in a man's life than nonscreen-detected tumors. The clinical significance, natural history, and comparative effectiveness of treatments in PSA-detected cancers are not known but likely differ from those detected and treated in the pre-PSA era (before the late 1980s to early 1990s). The objective of this project is to pilot an approach for developing future research priorities and suggesting specific projects to address evidence gaps. From the results of this and comparable pilot projects conducted by other Agency for Healthcare Research and Quality (AHRQ) Evidence-based Practice Centers (EPCs), AHRQ will identify generalizable strategies and lessons learned. The topic of this pilot project, the comparative effectiveness of treatments for localized prostate cancer, was selected because of its importance. The Minnesota EPC completed a comparative effectiveness review (CER) on this topic in 2008 for AHRQ. This pilot project amends the list of recommendations from that report and creates prioritized lists of research gaps and proposed research studies. Subsequently, management strategies for local prostate cancer were in the first quartile of the Institute of Medicine's 100 initial priority topics for comparative effectiveness research: Compare the effectiveness of management strategies for localized prostate cancer (e.g., active surveillance, radical prostatectomy [conventional, robotic, and laparoscopic], and radiotherapy [conformal, brachytherapy, proton beam, and intensity-modulated radiotherapy]) on survival, recurrence, side effects, quality of life, and costs.