Factors That Cause Different Treatment Responses Are Not Well Understood

NPC/SOP Study Published in Journal of Comparative Effectiveness Research Looks at Stage IV Prostate Cancer

By Andrea Hofelich
November 5, 2013

Although researchers and health care providers widely recognize that individuals with stage IV prostate cancer react differently to treatments, the factors that impact treatment outcomes, including biological factors such as race, age, or co-morbidities, and nonbiological factors such as geography, diet or social issues, are not well understood or studied. A study examining this issue, led by researchers from the University of Maryland School of Pharmacy and the National Pharmaceutical Council, was published today in the Journal of Comparative Effectiveness Research.

The study, “The Emerging Relevance of Heterogeneity of Treatment Effect in Clinical Care: A Study Using Stage IV Prostate Cancer as a Model,” examined the impact of individual treatment effects, or heterogeneity, on survival, adverse events or health-related quality of life in stage IV prostate cancer patients. As the study explains, “individual treatment effects can make it challenging for the patient and the provider to select appropriate patient-centered treatments that provide maximum clinical benefit to specific patients and subgroups.”

“At a time when comparative effectiveness research is shifting into high gear in the United States, we are only just beginning to fully consider how individual patient differences and treatment preferences impact health outcomes,” said C. Daniel Mullins, PhD, professor and interim chair of the Department of Pharmaceutical Health Services Research at the University of Maryland School of Pharmacy. “We understand how treatments work on average, but getting the right treatment to the right patient at the right time is paramount.”

The authors chose to study stage IV prostate cancer because researchers have found considerable differences among patients with respect to optimal treatment strategies, as well as survival across various subgroups. Study authors reviewed existing literature and other evidence on the topic, which showed that “factors related to the clinical signs/symptoms, laboratory tests and disease severity have been extensively studied,” but “few articles studied the role of nonbiologic factors in treatment outcomes.”

“It’s clear that more work needs to be done so that we can better target treatments to patients,” said Robert Dubois, MD, PhD, chief science officer, National Pharmaceutical Council. “Some organizations that are funding comparative effectiveness research—like the Patient-Centered Outcomes Institute—are beginning to fund studies that more closely examine the individual impact, but we need to encourage the practice more broadly.”

The study also highlights the many challenges faced by health care decision-makers because of the lack of evidence regarding individual treatment effects. To address these challenges, the study authors suggest that individual treatment effects should be taken into account by physicians, when applying treatment decisions to diverse populations; by researchers, when designing clinical trials and interpreting the results; by regulatory bodies that approve drugs and might not recognize the positive or negative effects on specific populations; and by payers, to ensure that a wide range of treatment options are available to patients, especially for conditions with diverse treatment effects.

NPC has been examining and funding research on the challenge of individual treatment effects for some time. Most recently, NPC hosted a conference and a user-friendly booklet, “The Myth of Average: Why Individual Treatment Differences Matter,” and sponsored a webinar series to help decision-makers understand and consider individual treatment effects.

About the University of Maryland School of Pharmacy

The University of Maryland School of Pharmacy, the fourth oldest school of pharmacy in the United States, leads pharmacy education, scientific discovery, patient care, and community engagement in the state of Maryland and beyond. With an enrollment of nearly 700 doctor of pharmacy and graduate students, the highly ranked school conducts cutting edge research in drug delivery mechanisms, cost impact studies, basic drug discovery and development, and disease management, and engages in a wide variety of professional practice activities, partnering with over 200 community pharmacies, hospitals, assisted living facilities, and other agencies to provide services to citizens and practitioners around the State and across the nation.

About the National Pharmaceutical Council

The National Pharmaceutical Council is a health policy research organization dedicated to the advancement of good evidence and science, and to fostering an environment in the United States that supports medical innovation. Founded in 1953 and supported by the nation’s major research-based pharmaceutical companies, NPC focuses on research development, information dissemination, and education on the critical issues of evidence, innovation and the value of medicines for patients. For more information, visit www.npcnow.org and follow NPC on Twitter @npcnow.

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