Clinical Trials: What You Need to Know

Clinical trials are vitally important when it comes to the development of new drugs and treatments for illness and disease, but as with anything related to health and medicine, there are pros and cons to consider before deciding to participate or not to participate. It takes an average of twelve years for a drug to go from development in the lab to regulatory approval, and much of this time is spent in clinical trials determining the drug’s efficacy and proper dosage. When studying a new treatment method, the researcher’s goal is to determine if the new drug (or combination of drugs) is superior to the current standard of care for an illness or disease.

There are many compelling reasons to take part in a clinical trial. Chief among these is the possibility of receiving a drug that improves one’s health, extends one’s life – or even results in a cure. This could be life changing; if the new drug is only available in a clinical setting, than those in the trials will be the first to receive it and its potential benefits. Patients in trials will have the advantage of being closely monitored, and may have some of their expenses covered. It is highly likely that the people who are cured first of an illness or disease will be those who participated in a clinical trial.

A great number of trials are conducted specifically for cancer research. These trials typically compare the standard regimen with a new or different protocol to see if the current treatment can be improved upon. Fear of being in the control arm or receiving placebo may cause reluctance on the parts of some cancer patients to participate in a clinical trial, but it should be noted that, because of ethical reasons, these patients are generally not deprived of treatment – whether it’s the standard care or the investigational drug.

The negatives of participating in a trial are just as numerous. First of all, there is no way to predict the effectiveness of a new treatment. There may be negative side effects or unexpected risks that are not known until the trial is already underway. If you are in a randomized study, you won’t have a say in the treatment that you are given. Additional requirements (including doctors visits and tests) may not be covered by health insurance, and there’s even the possibility of having a worse outcome as a result of taking the experimental drug.

Here is a general description of each clinical trial phase a drug must go through before a doctor can prescribe it to patients.

Phase I: Safety, potential side effects and dosage

The purpose of a Phase I study is to determine how a potential new drug reacts in the human body and how it causes the human body to react. Generally speaking, a small number of people (10-80) are recruited for this phase of the study. These subjects are relatively healthy, but have a specific disease or a range of diseases or conditions. Overall safety is studied and dosages are considered.

Phase II: Efficacy and ideal dosage

Phase II studies focus on short-term side effects and safety risks associated with the drug that is being studied. Researchers will also narrow the focus of the treatment to a specific type of cancer or disease. 100-200 new subjects will be recruited to participate. Doctors and drug companies will use this time to decide if more studies should be done and to consider who might benefit from them.

Phase III: Compare investigational drug to the clinical standard

Phase III involves larger numbers of patients (possibly in the thousands). At this point in the research, it is critical to determine how the investigational drug compares to the gold standard of care. If the results are positive, then an application is made to regulatory authorities to request a license for clinical use.

Phase IV: Post-marketing studies

After the drug is approved, patients who are taking it are monitored for long-term safety and efficacy.

Deciding to participate in a clinical trial is a personal choice, and there are many things to consider before signing up for one. The seriousness of the disease is an important factor. If you have a non life-threatening disease, and are receiving treatment that is working, then foregoing that treatment (on the off chance that something new may work better) might not be the appropriate choice. On the other hand, if the prognosis is bad (or even marginally bad) then pursuing an alternative to the standard protocol may be a reasonable choice.
Research is critical when it comes to making an informed decision. For example: If there is some indication or awareness that a new, potentially successful treatment is on the horizon, a clinical trial which studies that drug may be a prospect well worth investigating. Having a physician’s assistance in navigating the possibilities is a definite plus. An expert opinion is always helpful when it comes to translating medical language into layman’s terms. It is also essential when it comes to managing expectations.

If you are interested in participating in a clinical trial, go to clinicaltrials.gov. You can punch in keywords that apply to your specific situation as well as your city (or a major city nearby) to narrow the results. Do not be afraid to call the investigator or nurse to get more information about the trial. They can answer any questions and address any concerns you have about participating.