How do Clinical Trials Provide Access to Investigational Therapies?
Patients and their physicians can pursue access to investigational therapies and treatments through clinical trials, established expanded access programs (EAPs), and single-patient expanded access (EA). The most common way to access an investigational therapy is through a clinical trial, and, in fact, patients and their physicians must first confirm that clinical trials are not an option before pursuing expanded access programs or single-patient expanded access.
Clinical trials determine how well an investigational therapy works as compared to the existing standard of care or a placebo, and whether the potential therapy is safe and effective. Clinical trials also explore whether the benefits of the investigational therapy outweigh the risks.
All therapies have risks, and it is important to know that the investigational therapies in clinical trials are not yet approved by the FDA. In fact, many therapies tested in clinical trials never get approved. The testing process in a clinical trial follows a clear protocol, or study plan, which is overseen by regulatory bodies such as the FDA, and ethical bodies known as Institutional Review Boards (IRBs).
The biggest obstacle to completing a clinical trial is recruiting enough patients. Often, patients or their physicians are not aware of clinical trials that are open for enrollment. In other cases, many patients do not meet the study requirements and cannot be admitted to the trial. Eligibility requirements ensure that all trial participants share certain characteristics, such as age, gender, and medical condition, so that the results of the study are not affected by complicating factors. Clinical trial requirements may also help prevent patients from enrolling in studies that are not appropriate for them.
As a patient who is interested in participating in a clinical trial, you will first work with your physician to determine whether you are eligible and able to join a study. In addition to discussing the investigational therapy in question, you should also consider the clinical trial’s location, cost of transportation, childcare, meals, places to stay, and other costs that are often associated with trial participation.
Clinical trials are the first option to consider before exploring expanded access through a program or single-patient expanded access. Most pharmaceutical companies do not allow access to an investigational therapy via EA while a clinical trial is underway for the same therapy, as this could delay or jeopardize the trial’s completion for a variety of reasons. Delaying a clinical trial affects not only those in the trial, but the broader population, by slowing the therapy’s path to market. The successful completion of a clinical trial is an important step toward marketing approval, which assures the broadest availability of promising effective treatments to the patients who are most likely to benefit from them.
To determine the existence, location, and details of specific clinical trials, including eligibility criteria, you and your physician can search ClinicalTrials.gov. Contact information is provided for each trial recruiting patients and can be used to obtain more information. For a practical guide to navigating the ClinicalTrials.gov website, refer to our ClinicalTrials.gov User Guide. Other sites helpful in researching clinical trials for rare diseases include Trials Today at ResearchMatch.org and the National Organization for Rare Disorders. If you are researching cancer-related investigational therapies you can also search for clinical trials through the National Cancer Institute.
If your physician has told you that you are ineligible for or unable to participate in a clinical trial, keep reading for more information on established expanded access programs (EAPs) and single-patient EA.