CancerGuide: Clinical Trials and Experimental Treatments
Clinical trials are conducted within a complex system with fairly well defined rules. A good understanding of the system gives you a framework for making good decisions. It also gives you an essential framework for understanding clinical trial results reported in the medical literature. There are can find brief superficial explanations of the sytem in many sites. My approach is to give you a detailed and intensely patient oriented tour specifically designed to help you find the best treatment.
Understanding the details will help you:
In this article I set the stage with a brief introduction to the system, and then link you to CancerGuide's detailed Phase by Phase Guides. If you're already somewhat familiar with clinical trial phases you may want to skip directly to the detailed guides.
A Few Important Principles
As you go through CancerGuide's clinical trial articles, there are a few general principles which you'll want to keep in mind which will help you put it all together.
Promise and Uncertainty
I talk much about things being more or less promising based on the data, but often you just can't tell. When considering previous results of new treatments there is almost always great uncertainty. Often the data simply do not exist to suggest a new approach is truly promising and you may be unable to determine whether a particular trial is promising or not. Don't expect to be able to come to a definite conclusion about each and every trial - or even most of them. But when there are data suggesting there is real promise, and when you find that data, understand it, and act upon what you find, you may change your odds by getting the best treatment!
Each Option Must Be Compared to All Others
Although I describe strategies for investigating and thinking about each phase of trial, in reality, to find the best, any treatment option must be evaluated in the context of all other options. The alternative to a Phase I trial is not just another Phase I trial, but any other trials you are qualified for as well as any standard treatments which might be of value. It is impossible to know if something is promising unless you know what results can be expected with standard therapies.
Variations on a Theme
Trials can vary from the standard or classic model, and a surprisingly large number of trials are non-standard in one way or another. I describe the standard and some of the variations for each phase of trial, but I can't hope to cover all of the possiblities. But understanding the standard cases will help you figure out how to understand and evaluate trials even when they don't quite fit the classic mold.
The System In a Nutshell
Phase I Trials
Phase II Trials
Phase III Trials
Regulatory and Institutional Aspects in Brief
Human Subject Protection
Clinical trials are governed by a complex bureaucracy and process designed to protect people from unethical research. CancerGuide does not dwell on regulatory protections because I believe that your best protection is to become deeply informed! But you should know that all reputable clinical trials are approved by a local Institutional Review Board to ensure that they meet minimum nationally established ethical standards. This includes ensuring that trials do not seriously harm patients solely for the purpose of gathering research data, that the risks are appropriate considering the situation, and that patients who participate in a trial give their informed consent through a process which culminates in signing an informed consent document. For advice on how to get the most out of informed consent, see my article, A Guide to Informed Consent for The Active Patient
Regulation of Experimental Drugs and Devices
In the US, to use any drug or device that is not approved for general marketing (e.g. "experimental") researchers must obtain a permit called an IND. An IND is approval to use an experimental drug usually in a clinical trial, but it does not mean the FDA has approved the treatment for general use or even that it thinks the treatment has merit. Occasionally I see drugs or nutritional supplements touted as "FDA approved" when the only approval is an IND for use in clinical trials.
A notable and important feature of the clinical trial system is the strict and complex set of eligiblity criteria for each trial. Understanding this aspect of the system will make it much easier to figure out which trials you qualify for. The eligiblity criteria are include relatively standard requirements for all trials as a whole, some requirements which are typical of each phase trial, as well as many requirements which are specific to a particular trial. I cover the standard phase specific requirements in my detailed article on each phase of trial.
Steve's Strategic Clinical Trial Guides: Phase by Phase
Additional Information on the Clinical Trial System
The US National Cancer Institute's Clinical Trials Section is a "must see"! It has loads of patient oriented information on clinical trials and the clinical trial system. If you probe deep enough you'll also find a great deal of technical information for researchers about the conduct of trials.
Cyclist Lance Armstrong, who came back from widely metastatic testicular cancer to win the Tour De France six times in a row, has devoted a section of his Cycle of Hope site to Cancer Clinical Trials . What you'll find there is pretty basic, but it's friendly with a definite patient oriented edge.
This CancerGuide Page By Steve Dunn. © Steve Dunn
Page Created: March 3, 2002, Last Updated: August 7, 2004