Clinical Trial Information for Patients

The purpose of clinical trials is to collect data about the safety and efficacy of new drug and device development. There are several steps and stages of approval in the clinical trials process a drug or device must go through before it can be sold in the consumer market. If it fails any of the stages, it may not be approved. Since there are no devices used for P/P, this page will focus on treatments.

Drug testing begins with extensive laboratory research that can last years. During this time, experiments in animals and human cells will occur. If this initial laboratory research is successful, researchers send the data to the United States Food and Drug Administration (FDA) for approval to continue research and testing in humans. If approved, human testing of experimental drugs begins. This testing is usually done in five phases (Phase 0-IV) with each considered a separate trial. After each phase is completed, investigators must submit their data back to the FDA for approval before continuing to the next phase.

Human Clinical Trial Phases

Phase 0 exploratory studies involve very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies).

Phase I studies assess the safety of a drug and is the initial phase of testing. This phase can take several months to complete, usually including 20-100 healthy volunteers who are typically paid for their participation. This phase determines the effects of the drug on humans including how it is absorbed, metabolized, and excreted. This phase also investigates any side effects that occur as dosage levels are increased.

Phase II studies test the efficacy of a drug. This second phase of testing can last from several months to two years. It can involve several hundred patients. Most phase II studies are randomized trials where one group of patients receives the experimental drug, while a second “control” group receives a standard treatment, or placebo. These studies are “blinded” meaning neither the patients nor the researchers know who has received the experimental drug. This allows investigators to provide the pharmaceutical company and the FDA with comparative information about the relative safety and effectiveness of the new drug.

Phase III studies involve randomized and blind testing in several hundred to several thousand patients. This large-scale testing can last several years and provides the pharmaceutical company and the FDA with a thorough understanding of the effectiveness of the drug, its benefits, and the range of possible adverse reactions. Once Phase III is complete, a pharmaceutical company can request FDA approval for marketing the drug.

Phase IV studies (Post Marketing Surveillance Trials) are conducted after a drug has been approved for consumer sale. Pharmaceutical companies have several objectives at this stage: (1) to compare a drug with other drugs already in the market; (2) to monitor a drug’s long-term effectiveness and impact on a patient’s quality of life; and (3) to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies. Phase IV studies can result in a drug being taken off the market or restrictions of use could be placed on the product depending on the findings in the study.

Participating in a Clinical Trial

For detailed information and an FAQ about participating in clinical trials, please visit Should I Volunteer? And how?

Funding Clinical Trials

Funding for clinical research comes from the federal government (e.g., National Institutes of Health, Department of Defense, Department of Veteran’s Affairs), private industry (e.g., pharmaceutical and biotech companies), medical institutions, and foundations.

Participating in a clinical trial can be a difficult decision to make. Is it right for me? Would I qualify? What if I get worse? Can I quit? These are very real concerns and you should have those answers before deciding.

What is a clinical trial?

A clinical trial is a research study where volunteers receive investigational treatments under the supervision of a physician and research professionals. These treatments are developed by pharmaceutical and biotechnology companies who select qualified physicians, also known as investigators, to conduct clinical trials to determine the benefits of investigational drugs.

Who can participate in a clinical trial?

All clinical trials have guidelines about who can participate. Before joining a clinical trial, volunteers must qualify for the study. These factors are called “inclusion criteria” and the factors that disallow volunteers from participating are called “exclusion criteria.” These criteria can include age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.

Some research studies seek participants with specific illnesses or conditions to be studied in a clinical trial (like PV or MMP opposed to anyone with pemphigus or pemphigoid). It is important to note that inclusion and exclusion criteria are used to identify appropriate participants, promote participants’ safety, and ensure that researchers learn the information they need.

How does a clinical trial work?

In a clinical trial, a volunteer is generally assigned a specific study group. Volunteers in one study group may receive an investigational treatment (the study drug) while other volunteers may receive a placebo (a treatment already available).

A placebo is an inactive product used to assess the experimental treatment’s effectiveness. The participant, physician, and research staff may not know which volunteer receives a placebo and which receives the active treatment. This is called a “blind study.” Not knowing which participants receive the active treatment lets physician and research staff objectively observe the volunteers during the study. Regardless of which treatment volunteers receive, the level of medical attention and care that each receives is the same.

What questions should be asked before choosing to participate?

Patients thinking about participating in a clinical trial should talk about it with their physicians and medical caregivers. Potential volunteers should know and understand the credentials and experience of the staff and the facility involved in conducting the study.

Questions to ask a physician or medical caregiver:

• How long will the trial last?
• Where is the trial being conducted?
• What treatments will be used and how?
• What is the main purpose of the trial?
• How will patient safety be monitored?
• Are there any risks involved?
• What are the possible benefits?
• What are the alternative treatments besides the one being tested in the trial?
• Who is sponsoring the trial?
• Do I have to pay for any part of the trial?
• What happens if I am harmed by the trial?
• Can I opt to remain on this treatment, even after termination of the trial?

What can volunteers expect if they participate?

Sometimes, participants receive a physical examination and their medical histories are reviewed once they are enrolled in the study. The volunteers’ health continues to be monitored during and after the trial. A detailed description of what is expected of volunteers will be outlined in consent forms and specific clinical trial information.

What are the benefits and risks of joining a trial?

Volunteers in a clinical trial participate in the development of medical therapies that may offer better treatments (and even cures) for P/P and other chronic diseases. However, there are risks involved.

Possible benefits:

• Play an active role in their own health care
• Gain access to research treatments before they are widely available
• Obtain medical care at health care facilities during the trial
• Help other P/P patients by contributing to medical research

Possible risks:

• There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.
• The experimental treatment may not be effective.
• The protocol may require more time and attention than a non-protocol treatment. This may include trips to the study site, more treatments, hospital stays, or complex dosage requirements.

Volunteers may withdraw from a study at any time for any reason.

Is my personal and medical information confidential and private?

Access to personal information is usually available to the investigator and research team conducting the clinical trial. In some circumstances, the Institutional Review Board (IRB) overseeing the research and the sponsor or contract research organization coordinating the trial may have access to personal information. This is different in each clinical trial, however, it will be specifically stated in the consent form volunteers are asked to sign. As a clinical trial progresses, researchers report the results of the trial (not personal information) at scientific meetings, to medical journals, and to various government agencies.

What happens after the trial?

After a study phase is complete, the data is collected to determine the drug’s effectiveness, if it is safe, and if there are any side effects. Depending on the results, researchers determine whether to stop testing or move to the next phase of study. Researchers decide if the results are medically important and may submit them to journals for peer-review. Data then may be submitted to the Food and Drug Administration (FDA) for approval.

If a drug is approved, pharmaceutical companies may continue to conduct studies that compare the safety, effectiveness, and cost of the new drug to other drugs already on the market. They may also choose to assess a drug’s long-term effectiveness and its impact on the quality of a person’s life.

What is informed consent?

When you give written consent to participate in a clinical trial, you are acknowledging that you understand and accept all aspects of the research study including any risks or benefits involved. However, informed consent is more than signing a document — it is ongoing conversations between the research staff and you before, during, and after a study.

Millions of volunteers participate in government- and industry-sponsored clinical trials in the United States each year. Prior to agreeing to participate, every volunteer has the right to know and understand what will happen during a clinical trial. This is called informed consent and it is a process that can help you decide whether or not participating in a trial is right for you.

If you have given consent to participate in a clinical trial, or if you have given consent on behalf of another person, you both are entitled to the following rights:

• To be told the purpose of the clinical trial
• To be told about all the risks, side effects, or discomforts that might be reasonably expected
• To be told of any benefits that can be reasonably expected
• To be told what will happen in the study and whether any procedures, drugs, or devices are different than those that are used in standard medical treatment
• To be told about all options available to you and how they are better or worse than being in a clinical trial
• To be allowed to ask any questions about the trial prior to consenting and/or at any time during the course of the trial
• To be allowed ample time, without pressure, to decide whether or not to consent to participate
• To refuse to participate, for any reason, before and after the trial has started
• To receive a signed and dated copy of the informed consent form
• To be told of any medical treatments available if complications occur during the trial

What you should know

When you give written consent to participate in a clinical trial, you are acknowledging that you understand and accept all aspects of the research study including any risks or benefits involved. However, informed consent is not strictly about signing a document. It is a process that involves ongoing conversations between the research staff and you before and after a study.

To begin, the research staff is obligated to discuss all the pertinent information about the trial—its purpose, the procedures involved, the potential risks and benefits—with you. It is your responsibility to ask questions if there is something you do not understand.

If you do not understand any part of the process, ask the researcher to repeat the information or to explain it in another way, using everyday words. If English is not your first language, inform the researcher that you are not comfortable speaking about a clinical trial in English. Upon request, research centers can and should produce documents that explain every aspect of the study and study personnel should be able to explain the information to you in your preferred language. If this is not possible, you should not participate in the study.

It is the responsibility of the research staff to help you understand the information they provide you and to give you enough time to ask any additional questions you may have. In some instances, this may not be possible to accomplish in a single visit, therefore it is essential to take the time you need to make an informed decision. You may discover important concerns that you did not think about during the first visit.

What questions you should ask

The following represents a sample list of questions that you should ask during the informed consent process:

About the clinical trial

• What is the main purpose of the study?
• Why is this study important to me?
• What are the chances that this drug will work?
• What kinds of risks are involved?
• How much of my time will this take?
• Does the study involve a placebo or a treatment already on the market?

About your care

• What kinds of tests will be done? Will they hurt? If so, for how long?
• How will the tests in the study compare to tests I would have outside the study?
• Will I be able to continue to see my own doctor during the study?
• Will I be able to continue to take my regular medications during the study?
• If I have side effects, can they be treated during the study?

About personal matters

• Who will review information collected about me during the trial?
• What happens if I decide to quit the study?
• Can the investigator take me out of the study even if I want to continue?

About compensation and costs

• Do I have to pay for any part of the study? If so, will insurance cover these costs?

Take time to understand

A recent CenterWatch survey of 672 study volunteers offers insight into the informed consent process and whether or not it is working to help volunteers understand their roles and responsibilities. Some key takeaways from the study are:

• Nearly 98% of volunteers said they received the informed consent form and 90% said they read the form completely.
• The vast majority of volunteers said they understood the expectations of the study, including additional risks and their ability to withdraw at any time.
• More than three-quarters understood that they could contact someone outside the study if they had additional questions about their rights during the trial.
• More than half the volunteers were unaware that neither their doctor nor they would know what medication they would receive during the study; more than two-thirds understood that they could receive a placebo in the trial.
• Nine out of 10 volunteers said that the information received prior to the trial matched their actual experience during the trial.
• Approximately 75% of the volunteers indicated that the main reason for participating in a study was to help themselves or others and to advance science.

Research studies are very involved so it is important to learn as much as possible about the study you may participate in before you consent. The U.S. Food and Drug Administration (FDA) guidelines state that study volunteers should understand the risks they’re taking, which may mean spending additional time with the research staff to make sure you get the information you need. If you want to know details, such as any documented side effects of a particular drug observed in earlier clinical trials, you must ask for that information as well as any other information you may want to know.

The decision to participate in a clinical trial is a personal one and one that you are entitled to make freely, without influence or coercion. Being properly informed so that you fully understand the responsibilities of becoming a study volunteer is the best way to ensure that you are making the right decision for you.

Sometimes it sounds like doctors and researchers speak a foreign language. “Phase II is a longitudinal, double-blind placebo…” From A to Z find we can help you find out what the Belmont Report is, what double-bind means, or what a pilot study is.

Get our Clinical Research Terms and Definitions Glossary here.