From a Nurse's Perspective |
Clinical Trials
An ESUN Article
Editor's Note: There is a small glossary at the end of this article that might prove useful to some readers.
New treatment options are identified through clinical trial research. There are several different ways in which clinical trials are developed. One way is the development of a clinical trial concept by a pharmaceutical company. A company will have a product that they believe from their testing may be helpful to patients. They develop a trial and seek physicians to participate by enrolling patients. Another way is investigator initiated clinical trials. The investigator will develop an idea, write a protocol and seek financial support to conduct the clinical trial. The cost of doing clinical research is dependent upon the complexity of a trial and number patients to be enrolled. It may cost several million dollars to complete one clinical trial.
Trials require a specified number of patients to answer the question being asked. Statisticians work with the investigators to define the appropriate number of patients needed so that the results can be analyzed and be considered in terms of statistical significance. Prior to opening a clinical trial to patients, the trial must be reviewed and approved by institutional review boards (IRBs) and at some centers scientific review boards. The scientific board includes experts who evaluate the scientific merit of a study. The institutional board is primarily concerned about protecting patients. Before a drug can be provided to patients not on a clinical trial, it must be reviewed and approved by government regulatory agencies. In the United States the Food and Drug Administration (FDA) approves drugs and in Europe the European Medicines Agency (EMEA) approves drugs. Each of these agencies have rigorous review processes to ensure that new drugs approved have been proven through clinical trials to be safe and effective.
Given that sarcoma is a relatively uncommon type of cancer, investigators may collaborate to do clinical trial research. For some clinical trials a single center may not be able to enroll enough patients to answer a clinical trial question in a timely fashion. Therefore, centers may work together with several centers opening the same trial. Cooperative groups work together to pool their patients “resources” and often times will collaborate on the trial concept, design and ultimate goal or endpoint to be studied in essence pooling their intellectual “resources”. The National Cancer Institute (NCI) is a United States government agency that funds cooperative group clinical research in a variety of cancers through its Clinical Trials Cooperative Group Program. At this time, the Children’s Oncology Group (COG) is an NCI funded cooperative group that conducts sarcoma clinical research.
Your Questions about Clinical Trials are Important The Sarcoma Alliance for Research through Collaboration (SARC) is a non-for-profit consortium of sarcoma experts who collaborate together to do clinical research. A goal of this organization is to design and conduct clinical research that will result in new and better treatment options for patients with sarcoma.
For more on SARC, see Dr. Robert Maki's Op Ed piece, Sarcoma Alliance for Research through Collaboration (SARC): An effort to perform clinical trials efficiently and effectively for people with sarcoma, which appeared in the December 2007 issue of ESUN. |
This column includes two articles about clinical research from distinct perspectives. One is by Kelly Scheu, MS, NP-C who is a nurse practitioner in a private hematology oncology practice in Jackson, Michigan. Prior to completing her NP training, Kelly most recently worked as a research nurse at Memorial Sloan Kettering with the sarcoma team. She provides insights to help better understand clinical trials. The second article is a view of clinical research from the patient perspective. While navigating “the peaks and potholes”, Marcia gives a view of the ultimate benefit that she received by participation in a clinical trial. Finally, a set of "Additional Considerations" that appear on the Liddy Shriver Sarcoma Initiative's website are given. Together nurse and patient help us better understand sarcoma clinical research.
Sarcoma Clinical Trials Sarcomas are an uncommon type of cancer. Over the past forty years, sarcomas have been treated with chemotherapy. Standard chemotherapy unfortunately has not been effective for all patients. New treatment options are needed. Sarcoma clinical trials are one way to identify new therapies. Sarcoma clinical trials provide an opportunity to have access to new drugs which have the potential to improve outcomes and hopefully cure sarcoma. The words “clinical trial” may evoke uneasiness for some people. The thought of having a new drug that is still in the process of being studied may be concerning. Understanding the different phases of clinical trials and as well as having strategies to navigate the clinical trial system may help alleviate concerns. There are 4 phases to clinical trials.
Many drugs used in treatment today, were initially evaluated in phase 1, 2 or 3 clinical trials prior to approval by the FDA. Clinical trials have rigorous regulations and guidelines to ensure the safety of all volunteers. There are several important things to remember when considering a clinical trial.
Clinical trials provide valuable new options for treatment in the fight for a cure. The best way to determine effective new treatments is through systematic evaluation done within clinical trials. |
| Clinical Trials from a Participant's Perspective Living inside a clinical trial is a journey of peaks and potholes. Peaks are easy. The highest is waking up alive. Potholes litter the path too and are usually annoyances or inconveniences. When they occur, I try to remember what G. K. Chesterton said, “An inconvenience is an adventure wrongly considered.” The busy ER provided one such recent adventure. Sick babies, strokes, motor vehicle accidents, and traumas from the nearby tornado hit to a motor home park filled the nooks and crannies of a small town in southwestern Missouri in January. When CODE BLUE was announced overhead, my first impulse was to rush to Room 214 and save the day. Then I remembered I hadn’t saved anyone’s day for eighteen months when metastatic leiomyosarcoma caused me to leave my beloved internal medicine practice. I was in the ER not as a physician but as a cancer patient whose oncologist had referred for evaluation of a suspected deep vein thrombosis (DVT). My port used for chemo was inflamed and tender. After twelve hours of waiting for the true emergencies to be seen, I was told the Doppler was negative and released. The deepest pothole in my journey came with the news that without chemo I had less than a year to live. My options included several unappealing traditional chemo regimen and ET-743, a promising drug available only via clinical trial in limited supply in only a few sites across the country. Through the intervention of my oncology team, I was referred to another center for consideration of an expanded access clinical trial. Five months into my clinical trial, I am filled with hope. My CT scans have demonstrated no progression of metastatic disease. Stable disease is considered a response. Gratitude continues to be a constant companion that I have been given this chance to participate in this drug trial. Every day I live breeds hope that I will survive to benefit from advances in research that may bring more definitive treatment. In between the peaks of gratitude and hope exists the reality of traveling six hundred miles round trip for a 24-hour infusion of chemo every twenty-one days. Although my physical side effects are limited to annoying potholes of nausea, gastroesophageal reflux, constipation, anorexia, and fatigue, my mind and soul suffer more limiting blows. A life-suffocating fatigue breeds a mental paralysis that dulls my ability to experience pleasure, animation, or hope. Fortunately, this paralyzing inertia lasts for only a week or so of the cycle. This shadow self is transient and precedes a rebirth of desire, joy, and hope. During these peaks I have enjoyed traveling to the Rocky Mountains, Greece, Grand Cayman, and spending more time with my family. There is nothing like the loss of vitality to make its return a cause for uninhibited celebration. Life is a gift. It is precious beyond description. I will be forever grateful for those who continue to do cancer research and champion the cause of those like me who depend on the kindness of strangers for our very life. |
Mini Glossary
Taken from NCI Dictionary of Cancer Terms
Clinical trial
A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called a clinical study.
Endpoint
In clinical trials, an event or outcome that can be measured objectively to determine whether the intervention being studied is beneficial. The endpoints of a clinical trial are usually included in the study objectives. Some examples of endpoints are survival, improvements in quality of life, relief of symptoms, and disappearance of the tumor.
Expanded access trial
A way to provide an investigational therapy to a patient who is not eligible to receive that therapy in a clinical trial, but who has a serious or life-threatening illness for which other treatments are not available. Expanded access allows a patient to receive promising but not yet fully studied or approved cancer therapies when no other treatment option exists. Also called compassionate use trial.
FDA
The Food and Drug Administration (FDA) is an agency in the U.S. federal government whose mission is to protect public health by making sure that food, cosmetics, and nutritional supplements are safe to use and truthfully labeled. The FDA also makes sure that drugs, medical devices, and equipment are safe and effective, and that blood for transfusions and transplant tissue are safe.
Investigator
A researcher in a clinical trial or clinical study.
IRB
A group of scientists, doctors, clergy, and consumers that reviews and approves the action plan for every clinical trial. There is an IRB at every health care facility that does clinical research. IRBs are designed to protect the people who take part in a clinical trial. They check to see that the trial is well designed, legal, ethical, does not involve unnecessary risks, and provides safeguards for patients. Also called Institutional Review Board.
Placebo
An inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo. Placebo therapy An inactive treatment or procedure that is intended to mimic as closely as possible a therapy in a clinical trial. Also called sham therapy.
Placebo-controlled
Refers to a clinical study in which the control patients receive a placebo.
Protocol
An action plan for a clinical trial. The plan states what the study will do, how, and why. It explains how many people will be in it, who is eligible to participate, what study agents or other interventions they will be given, what tests they will receive and how often, and what information will be gathered.
Statistically Significant
Describes a mathematical measure of difference between groups. The difference is said to be statistically significant if it is greater than what might be expected to happen by chance alone. Also called significant. [Author's addition: Statistical significance is achieved when the results of a trial are deemed sufficiently reliable (not having occurred by chance) to support a given hypothesis.]
Additional Terms
EMEA
The European Medicines Agency (EMEA) is a decentralized body of the European Union with headquarters in London. Its main responsibility is the protection and promotion of public health, through the evaluation and supervision of medicines for human use.
Sponsor
Pharmaceutical companies, nonprofit organizations, or other institutes, the National Cancer Institute for example, may sponsor clinical trials. Pharmaceutical companies fund studies because they must prove their new medication or treatment is safe and effective via success in a clinical trial in order for it to become available to the public.
Reference
Drug Development: Innovative Drug Development Approaches, Final Report of the EMEA/CHMP Think-Tank Group on Innovative Drug Development, March 2007
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