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Patient Safety in Clinical Trials:
A Q&A with James T. Gourzis, M.D., Ph.D.
Introduction
Clinical investigators are responsible for protecting the rights, safety, and welfare of subjects under their care during a clinical trial (21 CFR 312.60 and 812.100). In many trials, the sponsor delegates some decisions regarding trial design and conduct to an entity such as a steering committee or contract research organization (CRO).
Patient safety has always been the industry’s focus during clinical trials. However, a recent spate of well-publicized patient safety issues has increased public scrutiny and the industry’s desire to improve study quality, resulting in larger, longer, more expensive trials.
In this issue of Peer Perspectives in Oncology, James T. Gourzis, M.D., Ph.D., talks with Medelis about issues affecting patient safety:
- Factors that have launched patient safety to the forefront of public scrutiny, including the effects of increased reporting requirements
- What to look for when evaluating CRO excellence and commitment to patient safety
- Unique safety considerations in oncology trials and the ramifications of the rare toxicity
- Optimizing the role of the Data Monitoring Committee
- Budget decisions that support patient safety
- The evolution and future of FDA regulations
About Dr. Gourzis
James T. Gourzis, M.D., Ph.D. is a member of Medelis' Medical Advisory Board and has more than 30 years of experience in designing and leading clinical trials, implementing regulatory strategies and negotiating licensing transactions. Dr. Gourzis currently provides consulting services to a wide range of biotechnology and medical device companies with respect to scientific, strategic and regulatory considerations associated with drug and biologic development.
Previously, Dr. Gourzis was an executive with PAREXEL International Corp., a contract research organization that provides a range of services to pharmaceutical and biotechnology companies. He began his career in the clinical research groups of McNeil Laboratories, Inc. and Schering Corp. and is experienced in a broad range of therapeutic areas, including cardiology, immunology and infectious disease.
Dr. Gourzis received his bachelor's degree in biology from Harvard University, his master's degree in pharmacology from Boston University, and his M.D. from the University of Manitoba, Winnipeg, Canada. Dr. Gourzis also received a doctorate in pharmacology from the University of Manitoba.
Patient Safety in Clinical Trials:
A Q&A with James T. Gourzis, M.D., Ph.D.
Medelis: In clinical trials, patient safety has always been a cause for concern. But today it is under increased scrutiny, and CROs are bearing that scrutiny – rightly or wrongly – alongside sponsors. What forces have brought patient safety to the forefront of our industry?
Dr. Gourzis: There are two factors that have brought safety issues so prominently into the public radar. The first is the sheer volume of data required to conduct a trial and win FDA approval.
I started out in the pharmaceutical industry shortly after the Kefauver-Harris Amendments came into law in the 1960s. Before that time — the pre-CRO days — the medical staff of the pharmaceutical houses oversaw investigators. Until the late 70s, it was very difficult to go into an investigator’s office and scour case report forms and patient records for safety issues. CRFs, as we know them today, didn’t exist. Investigators weren’t as meticulous with data capture as they have to be today.
With new regulations and increased FDA oversight, sponsors are now required to generate exponentially more data for each trial, bringing more safety issues to light. In fact, Institutional Review Boards (IRBs) now complain that the increasingly large volume of individual adverse event reports — often lacking in context and detail — are actually inhibiting rather than enhancing their ability to adequately protect human subjects.
The second factor driving awareness of safety concerns is the fact that our industry is conducting more clinical trials now than ever before. Many trials relate to disease states that concern so many of us, such as oncology. Thus, the general public is much more aware of clinical trials, and primary care physicians are more likely to refer patients to investigators conducting trials for investigational drugs that may be of interest.
Medelis: In your mind, is there one patient safety tragedy that stands out as an early catalyst for publicizing clinical trial safety concerns?
Dr. Gourzis: Yes … around 2000, the University of Pennsylvania reported on the death of a subject in an ill-fated gene trial. The 18-year-old patient died after he was injected with an adenovirus carrying a gene to treat his liver disorder.
That incident sent shock waves through the field of study, and similar trials were quickly halted. After that fatality, individual cases suddenly came to light in the literature and the general media.
Medelis: On the topic of the media, a recent article in The New England Journal of Medicine (October 4, 2007) described a “CRO Boom” in the last decade. It explained that CROs have absorbed much of academia’s traditional role in drug development by offering greater speed and efficiency in conducting clinical trials. Has this “boom” affected patient safety?
Dr. Gourzis: By using independent CROs, pharmaceutical and biotech sponsors are bringing an objective third party into the study, which ideally increases visibility into problems so they can be remedied as quickly and safely as possible.
When a pharmaceutical or biotech sponsor contracts with a CRO, it’s the CRO’s responsibility to ensure that each study site adheres to good clinical practices and safety guidances. The CRO collects the data from the sites and reports back to the sponsor.
It’s also critical for the CRO to illuminate data that may be prejudicial or suspicious, protecting the sponsor from getting blindsided by problems such as false data, improper study procedures, or failure to report adverse reactions at the study site.
Once that data is submitted to the sponsor, the CRO has discharged its responsibility. It is now the sponsor’s responsibility to clarify any safety issues with the FDA and to ensure that the health status of the patient or trial subject is protected.
In 99% of the cases, this system works. But the few times it doesn’t, it’s like a shark attack. Forty million people swim in the ocean and four get attacked by sharks. What do you read about? The four attacks.
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