Lighting the Way

A practical guide to clinical trials

By Patient Advocate Foundation

The future of medi­cine has never been so bright. Many advances in treatments are being announced every day that can help eliminate disease or improve the life of patients suffering from illness. Even more drugs and treatment options that have the potential to improve the health of individuals, communities, and populations are currently in devel­opment in medical facilities and laboratories across the country. As part of the development and approval process, it is crucial that these new treatments are studied to make sure that they are safe and as effective, or more effective, than current standard treatment options.

When a person is diagnosed with a life-threatening, chronic, or debilitating illness, his or her medical team will present options to consider for treatment; these might include options known as current standard-of-care treatment or, in some situations, options within clinical trials. The National Cancer Institute (NCI) defines standard of care as: “Treatment that is accepted by medical experts as a proper treatment for a certain type of disease and that is widely used by healthcare professionals.”1 Healthcare providers are obligated to provide patients with access to standard-of-care treatment and best practices associated with the diagnosis.

Care that goes beyond or pro­vides alternative options from what is considered the standard-of-care treatment can be delivered through clinical trials. Participat­ing in a clinical trial may be excep­tionally beneficial for a patient who is not finding success using the standard treatment protocol and is ready to consider alternative options.

Making the decision to enroll in a clinical trial is a personal one. There are a lot of potential bene­fits, such as expanded access to the newest medical therapies and cut­ting-edge technology. Because tri­als are studying previously untried combinations of therapies or new drugs coming to the marketplace, however, successful results are not guaranteed and both the risk fac­tors and the side effects must be considered carefully.

Benefits And Risks Associated With A Trial

There are many benefits of partici­pating in a clinical trial:

  • You have access to promising new treatment approaches that are often not available outside a clinical trial setting.
  • The approach being studied may be more effective than the standard approach.
  • You receive regular and care­ful medical attention from a research team that includes doctors and other health pro­fessionals.
  • You may be the first to benefit from the new method under study.
  • Results from the study may help others in the future.

There are also risks associated with participating in a clinical trial:

  • New drugs or procedures under study are not always better than the standard care with which they are being compared.
  • There may be unpleasant, seri­ous, or even life-threatening side effects from treatment.
  • Treatment may not be effective for some individuals.
  • Health insurance may not cover all study costs.
  • The study may require a lot of time for traveling to the study site, receiving treatments, or hospital stays. This may incur additional costs associated with transportation.

What Defines A Clinical Trial, And How Is It Structured?

The most common clinical trial involves studying new drugs, com­binations of drugs (some already approved by the US Food and Drug Administration [FDA] for other purposes), and treatments to see how well they work—especially when compared with current standard-of-care treatment. Clini­cal trials have protocols, or action plans, that describe how the trial will be conducted. The protocol helps the participant understand what will be done, how it will be done, and why each part is neces­sary.

All clinical trials are overseen by an independent committee of physicians, statisticians, and community members, who must approve and monitor the protocol; this group is referred to as an Insti­tutional Review Board (IRB). The IRB makes sure that the risks as outlined in the protocol are small and are justified by the potential benefits.

Depending on the study, clini­cal trials have different eligibility requirements. Some studies need volunteers with a certain disease, whereas other studies require healthy people; some trials want all female participants, whereas others want all male participants. The sponsor of the study writes the protocol, which describes what the trial will do, how the trial will be conducted, the location of the study, eligibility criteria, and how and when the participants will be evaluated.

Clinical trials are sponsored by a variety of sources, including physicians, medical institutions, the National Cancer Institute, the Department of Defense, the Depart­ment of Veterans Affairs, and bio­pharmaceutical manufacturers.

The nature and the purpose of the trial define how it will be con­ducted.2 There are several different types of trials:

  • Treatment trials test experi­mental treatments, new com­binations of drugs, and new approaches to surgery or radia­tion therapy.
  • Prevention trials look for bet­ter ways to ward off disease in people who have never had a spe­cific disease or to prevent a dis­ease from returning. Approaches may include medicines, vaccines, vitamins, minerals, and lifestyle changes.
  • Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular dis­ease or condition.
  • Screening trials test the best way to detect certain diseases or health conditions.
  • Quality-of-life trials (or sup­portive care trials) explore ways to improve comfort and quality of life for cancer patients and survivors.

What Are The Phases Of Clinical Trials?

In the United States, the FDA requires that all new drugs and treatments complete multiple layers of testing before becoming avail­able to the general public. Clinical trial phases are stages designed to test the new treatments and care standards being proposed. Once all phases are complete, evidence prov­ing that the drug is safe and that the benefits of using the drug outweigh the risks must be presented before the drug can gain FDA approval for use as standard of care.

PHASE I. When a drug or treat­ment is in a Phase I trial, the researchers are trying to determine how the drug or treatment will be administered (orally, intravenously). The researchers are looking for the proper dose and monitoring side effects. The study focuses on a small group of participants.

PHASE II. When a drug or treat­ment advances to a Phase II trial, the researchers are studying the results to determine the effective­ness of the drug or treatment. Phase II trials collect information on the safety and benefits of the treatment. The study expands to 100 or more participants.

PHASE III. When a drug or treat­ment advances to a Phase III trial, the researchers are determining if one treatment is better than another. The trial agent is stud­ied in comparison with current standard-of-care treatment. Phase III trials expand in size from several hundred to thousands of partici­pants.

PHASE IV. When a drug or treat­ment advances to a Phase IV trial, the researchers are monitoring long-term safety and effectiveness of the treatment. These are also known as post-marketing surveil­lance studies.3

As a drug or treatment advances in each phase of a clinical trial, the number of institutions or facilities offering the trial increases. Clinical trials can be conducted in medi­cal centers, community clinics, or doctors’ offices, so location is an important aspect to consider as you examine whether a trial is right for your situation.

Trial participants are assigned to random groups, with each group receiving the specific treatment being tested or a placebo (“sugar pill,” or dummy medical treatment). It is important to know, however, that in cancer clinical trials, par­ticipants always receive either the current standard-of-treatment protocol or the new drug treatment protocol and do not go without treatment.

To be sure that trial results are free of any outside influence, neither the doctors, the person administering the treatment, nor the study participant know to which treatment group study participants are assigned. This study technique is known as a double-blind study and is common among respected research studies.

Eligibility

To be eligible to participate in a clinical trial, there are specific criteria, or guidelines, that must be met. These guidelines are developed by the researchers to define the target population. Eligibility guidelines that allow par­ticipation in a clinical trial are called inclusion criteria, and those that prevent participation are called exclusion criteria.

Eligibility criteria can include the following:

  • Type of illness or diagnosis
  • Stage (extent) of the cancer, if cancer diagnosis is required
  • Previous treatments that you must, or could not, have had
  • Length of time since you last received treatment
  • Results of certain laboratory tests
  • Medicines that you are taking
  • Other medical conditions
  • Previous history of any other cancer or diagnosis of interest
  • Other conditions specific to each clinical trial4
  • Age and gender

The more representative the final clinical trial group is of the national population, the better researchers are able to adequately measure the outcomes and final­ize the protocols for distribution among patients in all areas. So, no matter what race or ethnic background you have, what your gender and age are, or where you live, we encourage you to review the eligibility criteria for the clinical trials in your area.


RESOURCES

Lazarex Cancer Foundation

www.lazarex.org or (877) 866-9523

Provides financial assistance to defray costs associated with clinical trial participation; also assists patients with navigation through clinical trial options and education.

My Resource Search Mobile App

www.patientadvocate.org/myresources

This mobile search tool allows patients to search any of 35 available categories of assistive programs, including transportation, utilities, clinical trial assistance, pharmaceutical support, and more. It is available for free from both Apple and Android app stores.


 

How to Find A Clinical Trail

Talk to your doctor if you are interested in a clinical trial for your medical diagnosis, as he or she may be able to connect you to relevant and current studies. You may also search through clinical trial databases at any point through a registry website:


 

Your Rights Within A Clinical Trial

Once you are evaluated and deemed eligible for the trial, the trial research team must clearly and completely explain the trial’s purpose, procedures, risks, and benefits and obtain informed consent before you can participate. You will receive documentation regarding the details of the study; purpose of the trial; duration, or length of time the trial will run; required testing such as lab work and scans; key contacts; and what costs, if any, you are responsible to cover. You may discuss these documents with your doctors or other trusted medical professionals to help you understand any terminology as well as seek their guidance if desired.

Even if you decide to move forward under the clinical trial, by signing the informed consent you are not obligated to participate for the full duration of the trial—you have the right to leave the study at any time. If you choose to leave the study, your doctor will discuss other treatment options.

You have the following rights as a clinical trial participant:

  • To understand all of your treatment options
  • To know what is involved in the trial, such as tests, risks, and benefits
  • To have the opportunity to discuss the trial with the investigator and research team before and during the trial
  • To hear and read information in a language you can understand
  • To end care through the clinical trial and leave the clinical trial at any point

References

  1. NCI Dictionary of Cancer Terms: standard of care. National Cancer Institute website. Available at: http://www.cancer.gov/dictionary. Accessed October 5, 2014.
  2. NIH Clinical Research Trials and You. National Institutes of Health website. Available at: http://www.nih.gov/health/clinicaltrials/basics.htm. Accessed Octo­ber 5, 2014.
  3. Learn about Clinical Studies. ClinicalTrials.gov website. Available at: http://clini­caltrials.gov/ct2/info/understand. Accessed October 5, 2014.
  4. Learn about Clinical Trials. National Cancer Institute website. Available at: http://www.cancer.gov/clinicaltrials/learningabout/deciding-to-take-part. Accessed October 5, 2014.