Role at ICON:

I joined ICON in 2007 to oversee the integration of Health Economics and Health Outcomes (HEOR) into a single global function within the Lifecycle Sciences Group. HEOR studies involve innovative prospective research to support the value of our clients' products. These studies involve patient-reported outcomes, economic endpoints, economic models, literature reviews and comparative effectiveness research carried out in prospective studies or retrospective data analyses.

Background:

I was involved in health services research in academic medical centers for more than 20 years, including positions at Stanford University and the University of California, San Francisco. After completing a doctorate in economics at the University of California, I joined the Department of Immunology/Rheumatology at Stanford University and became involved in the design and analysis of disease registries in rheumatoid arthritis, osteoarthritis, HIV, and aging community populations. At the University of California, I continued with longitudinal research studies, focusing on oncology. For several years prior to joining ICON, I was a member of the HEOR groups at Amgen and Genentech. My observational research has resulted in over 160 peer-reviewed publications in clinical and health services research journals.

Career highlights:

At the University of California, I was involved in the design, execution and analysis of a large longitudinal database of prostate cancer patients as Co-Principal Investigator. This registry has grown to over 13,000 patients who were followed for clinical, quality of life, and health economic outcomes. It has resulted in more than 80 peer reviewed articles, including innovative studies of predictors of disease recurrence in men receiving a variety of initial treatments, development and validation of a patient-reported comorbidity index (now used in other therapeutic indications), a number of studies evaluating the impact of prostate cancer treatment on patient quality of life, and several pooled analyses with other prostate cancer registries. The continuing value of this registry 15 years after its initiation highlights the importance of these studies to industry, physicians, and patients.

What are the most challenging factors in conducting observational studies?

One challenge is that these studies are often conducted in sites with limited clinical research experience, and as a result, often require considerable education on the processes involved. Proper training includes detail on study design, the importance of adhering to the protocol, enrollment procedures, and data reporting processes. In addition these sites are unfamiliar with the effort necessary to participate. Convincing sites that participating in an observational study will not interfere with the patient care and not be too burdensome is often a daunting task. However, this challenge can be minimized by creating recruitment materials that clearly outline the responsibilities of the site and detail the time commitment as well as the support that will be provided.

Finally, managing expectations regarding the value of the registry is challenging. Considerable time and effort are put forth to develop and implement an observational study. Patient enrollment may take many months and follow-up may last years. With so much effort expended, there may be an expectation that the study goals should be met within the fi rst year or two. However, data from an observational study may take multiple years to mature. Proactively setting expectations regarding the type and timing of publication or achievement of study goals is crucial to the success of the study.

How will observational studies help answer safety demands in today's evolving drug safety environment?

New medical innovations have inherent risks associated with their use. Many of these risks are noted during clinical trials and addressed. However, many other side effects are only observed after the intervention is used in a larger population or for a longer duration. Observational studies provide continuing evaluation of outcomes in large numbers of patients who have comorbidities, who may take other medications, or are members of patient populations that may be excluded in randomized clinical trials (such as the elderly, pregnant women, or certain high risk patients). Observational studies, therefore, allow for important safety data to be collected that otherwise would not be available. New approaches to data collection facilitate the implementation of these types of studies globally and over many years in a very cost-effective manner.