Clinical Trial Basics

Participating in a Clinical Trial

Clinical trials make up a large proportion of advanced breast cancer research that is currently ongoing. Clinical trials also provide support for the development and mass production of new and effective treatment options. Clinical trials may also provide support for using already approved treatments in new ways and for different conditions. They can assess drugs, different procedures, and more, but require human subject participation. However, it is important to note that the journey from a treatment idea to its use on humans is a very long and structured one.

It can often take 10-15 years or more to get a drug down the “pipeline” and into consumers’ hands, with human subjects only entering near the middle or end of the process. Of course, before humans are involved, many qualifications and safety tests need to be completed.

What are the phases of a clinical trial?

A clinical trial needs to follow a specific set of phases before human subjects are included or a new treatment receives FDA approval. The typical steps of a clinical trial are as follows:

• Phase 0: Not everyone agrees that this phase is a true “step” in the process; however, it represents the exploratory beginnings of a new clinical trial. This phase typically includes a very small number of people and involves minor interventions or doses of a drug to see how it affects the body.
• Phase I: Typically, in this phase, varying doses of a drug are administered or procedures are performed. A very small number of human volunteers may be recruited at this step to assess a new drug’s basic safety and tolerability. In phase I trials the goal is to find the best dose of a drug, with the least amount of side effects. It includes a small number of patients. If the drug is found safe enough, it moves to Phase II.
• Phase II: Once phase I is successfully completed, more human subjects are recruited, and the focus of study generally changes from assessing a treatment’s basic safety to investigating how effective it is at doing its intended job. Usually, these trials include no more than 200 individuals from the treatment’s target population. The focus of phase II trials is to continue to assess safety, as well as making sure the drug works, using the dose found in phase I. In a cancer trial, it may mean does the tumor shrink or not grow larger. Phase II trials enroll more patients than in phase I.
• Phase III: This phase represents when a trial is at its largest, and often includes thousands of individuals. At this point, researchers can identify any additional side effects, make further conclusions on a treatment’s effectiveness, and make a definitive judgment on whether or not the treatment should be made available to the rest of the target population. In phase III trials the drug is compared to the current standard of care to see if it is as safe and works as well.
• Licensing and Approval: This step is essential for new drugs making it through this clinical trial pipeline, as in the United States, a treatment generally needs FDA (Food and Drug Administration) approval in order for physicians to prescribe it and pharmacies to carry it. For experimental drugs to make it this far in the process is an incredible feat, as it has been estimated that only 1 or 2 experimental drugs out of every 10,000 that begin the process will even be presented to the FDA or other regulatory agencies. Similarly, proposed new procedures or adjustments to the standard of care that reach this point can also be presented to associations that create the general guidelines for the treatment of a condition, for potential approval and implementation.

A clinical trial’s path through this process can be halted at any point if a treatment is found to be unsafe or unsatisfactory (or for any other reason).

Will I be safe?

Clinical trials are highly regulated, and the rights and safety of their participants are protected. For a clinical trial to be approved, even at its earliest stages without human participants, it needs to receive IRB (Institutional Review Board) approval and follow a strict protocol. The IRB reviews, approves, and monitors all studies surrounding new biological products, medical device, drugs, and other new treatment options. Protocols presented to the IRB include who is eligible to participate, the number of individuals needed, why the trial is being conducted, a schedule of procedures or the frequency and dose of drugs given, the estimated length of the study, and what information will be collected about those participating.

This protocol is carried out by the study’s Principle Investigator (PI) and has specific inclusion and exclusion criteria to make sure that the individuals participating meet a very specific demographic. Factors that contribute to these criteria include age, previous treatment history, other health conditions, gender, activity level, and severity of the condition, among other things. By controlling as many factors as possible about those included, the researchers can carry out the safest protocol possible and not expose any participants to unnecessary risks. Additionally, clinical trial participants are closely monitored by a healthcare team throughout the entire process, and even beyond.¹

Are there any other questions I should ask or things to consider?

Participating in a clinical trial is voluntary, and you can withdraw yourself at any time. However, there are positives and negatives to participating that an individual should consider before they commit. Although an individual will be adding to the knowledge base around a condition, and will potentially be receiving helpful, new treatment options before they’re released to the public, they are still putting themselves at risk for any unknown risks or side effects of the experimental treatment. Additionally, the new treatment may not be as effective as the current standard of care.

Further, not all clinical trials cover all costs for participants, and some insurance companies will not cover clinical trial participation. It is important to talk with your provider and the study team to determine what the potential risks of participating are, as well as any additional support they can provide you throughout this time. It is important to ask as many questions as you need to before you enroll in a trial, as well as throughout the process. Some example questions might include:

• Are there any serious risks to participating in this study?
• How will you protect or help me if something goes wrong?
• Will I be reimbursed for my expenses, such as travel and lodging?
• Will I be paid to participate?
• Will I or my insurance company need to pay for any part of this?
• What kinds of factors would exclude me from participating in this trial?
• Can I withdraw at any time?
• How long will the trial last?
• Will you provide follow-up care afterwards?

Where can I find clinical trials that I’m eligible for?

Clinical trials can take place across multiple sites and facilities, and may even be taking place near where you are currently. You can ask your provider if they are aware of any clinical trials that you may be eligible for, as well as check organizations dedicated to publicizing and reporting on clinical trials such as ClinicalTrials.gov, and the National Cancer Institute.¹