Access to Promising New Treatments.
Your doctor may tell you about an opportunity to consider participating in a clinical trial, which could give you access to a new treatment in development. Here are a few frequently asked questions about clinical trials and what is involved in participation.
What Is a Clinical Trial or Research Study and Why Is It Important?
The only way to improve care is to test new treatments, devices or processes. Current treatments had to be proven safe and efficacious prior to approval through this process.
Clinical trials or research studies may be interventional (in which a new treatment, procedure or device is studied) or observational (looking at aspects of your medical condition or care such as symptoms, surgery outcomes, length of hospital stay, pain, body image, etc.). The goal is to identify new medications, procedures and processes that improve medical care.
Who Monitors Research?
Research studies are monitored by the Food and Drug Administration (FDA), Institutional Review Boards (IRBs), and Data Safety Monitoring Boards (DSMBs). The study physician treating you, medical staff and research personnel assisting with your care report any safety concerns, as well as study progress to these groups.
What Does “Phase Numbers” Mean?
Phase 1: Testing of a new drug or device occurs in a very small group of people (<50), following initial tests in a laboratory setting. The goal is to determine safety and treatment dose, as well as how the drug is processed, or metabolized, by the body.
Phase 2: Testing of a new drug or device occurs in a larger group of people (<100) to further evaluate safety, dose and drug metabolism.
Phase 3: Testing of a new drug or device occurs in a very large group of people (hundreds) in which patients are randomized to different treatment groups. For example, a study will have one group of patients that receive a standard of care drug in addition to the new drug being studied, while the other group of patients will receive the standard of care drug and a placebo. In this case, patients are randomized 1:1. The number of patients and randomization process is different in each study. If the new treatment is found to be safe and efficacious, the study sponsor may apply for approval from the FDA.
Phase 4: These studies have already obtained FDA approval and look at long term outcomes (safety, efficacy, side effects).
Why Should I Participate in an Early Phase Clinical Trial That Hasn’t Been Tested in Many People?
Patients that have already exhausted all standard of care treatments, decline or cannot tolerate standard of care treatments, or for which no treatment exists, may consider an early phase clinical trial.
Why Would I Participate in a Clinical Trial If I Could Just Be Getting a Placebo?
In the study, you receive the standard of care treatment together with the study treatment or placebo. Even if you do not receive the study treatment in addition to the standard of care, you will benefit from the medical attention involved in the study.
If I Participate in a Clinical Trial With More Than One Treatment Group, Will I Know What Treatment I Am Receiving?
If the trial involves at least a standard of care treatment, you will know that you are receiving the standard of care treatment. If the trial is “double blind,” neither the doctor nor study participant will know what study medication you are receiving. If the trial is “single blind,” the doctor will know, but the participant will not know. If the trial is “open label,” the treating doctor and the study participant will know.
What Is the Benefit of a “Blinded” Study?
Blinded studies help eliminate bias that the treating doctor or patient may have towards the study treatment.
How Do I Enroll in a Research Study?
If you decide to participate in research, you will likely be asked to sign an informed consent. This form is regularly reviewed and approved by IRBs and provide detailed information on the study. It will tell you what the research is, what the purpose is, what will be required of you (study visits over a specified time period), risks, benefits, costs, other treatment options, your rights to privacy and to withdraw at any time, and who to contact. When you sign this form, it is an agreement to participate and a statement that you understand all the information presented to you. You should always ask questions if you do not understand something in the informed consent. A copy of the informed consent will be given to you for your records.
How Do I Find a Clinical Trial?
Clinical trials are regularly registered and updated on www.clinicaltrials.gov. You can search for specific key words and by location. There are many disease specific organizations that offer assistance in finding clinical trials. Talk to your doctor to see if he is aware of any clinical trials or resources for your condition.
If I Decide I Want to Participate in a Clinical Trial, Am I Guaranteed a Spot?
Clinical trials have a set number of people that will be enrolled. If the trial is enrolling, or still has spots to fill, you may be considered. Your desire to participate does not guarantee a spot. Every study has eligibility requirements that vary among studies. The requirements often center on prior treatment, medical history, age and current health status.
What Are the Benefits of Participating in Research?
- Since research may involve unanticipated risks, you will be monitored more closely.
- You increase the number of treatment options available.
- You contribute to advancing medicine and helping future patients.
- You may receive a treatment that will help you more than what is currently available.
What Are the Risks of Participating in Research?
- You may experience side effects, expected or unexpected, minor or severe.
- You may not receive the investigational treatment.
- The investigational treatment may not be effective.
- You will be followed closely, which means you may have to spend more time communicating with your doctor and medical staff. You may also be required to complete additional tests, which may involve longer than normal or more frequent appointments.
Will I Be Paid to Participate in Research?
There are some research studies that pay participants. Some will provide a small monetary payment or gift card for completing questionnaires or study visits while others do not. Some will cover the cost of your transportation to your appointments, or your hotel/food if you are required to be at your appointment early. If the research study requires you to have certain medical procedures (labs, EKG, etc.) beyond what you would have as part of standard of care for your condition, those costs may be covered by the sponsor of the study. Care that you would normally receive for your condition is considered standard of care and will be billed to you/your insurance, regardless of your participation in research. The informed consent will provide details on what costs are considered research versus standard of care, and questions about your medical costs may be answered by the research team and your insurance company.
How Long Do Research Studies Last?
Every study is different. Some require as little as a one-time questionnaire, while others that involve new drugs may last for years. You may be contacted periodically over a period of weeks, months, or years to monitor your condition. The informed consent will provide information on what is considered the end of treatment as well as define the long term follow up period.
What Research Studies Do You Currently Offer?
The number and type of studies we offer are constantly changing. Some studies involve testing new drugs and devices while others are observational. Some have a sponsor while others are investigator initiated. Contact the Digestive Health Institute Research Office at Call813-615-7068 to learn more.