Volunteers frequently asked questions.

What is clinical research?

Clinical Research is a section of healthcare science which determines the effectiveness and safety of devices, medications, diagnostic products and treatment for human use.  These may be used to treat and/or diagnosis symptoms of a disease or medical issue. In clinical research evidence is collected to establish a treatment. The term clinical research refers to the entire bibliography of a drug, device, or biologic from its inception in the lab through to consumer use. If initial laboratory research is successful, the data is sent to the Food and Drug Administration (FDA) for approval to continue research. Once approved by the FDA for human testing, a group of promising candidates are identified to perform a clinical trial.

What are clinical trials?

Clinical trials are only a small part of the overall research that goes into the development of a new treatment. Clinical Trials are intended to gather data about the safety and effectiveness of new drugs and medications, medical devices, and other diagnostic products. Clinical trials take place with healthy subjects with no pre-existing medical conditions as well as with patients with specific health conditions who seek otherwise unavailable treatments. In preliminary phases, participating patients are healthy volunteers who are typically financially compensated. The clinical sponsor designs particular trials with physicians, called clinical investigators. Sponsors typically enroll patients from a number of different facilities for trials for any given trial and once a clinical trial is ready, Investigators will enroll patients that fit within the scope of the trial parameters, treat the patients in accordance of the trial, and collect data based on the treatment. Data can include but not be limited to measurements of vital signs, blood and/or tissues concentration of drugs, and symptom changes. The data is later sent to the trial sponsor, to be analyzed. There are typically four phases of approval in the clinical trial process in order for the drug or device to become safe and useful in the consumer market, if ever. Each phase of the clinical trial is considered a separate trial. At the end of each phase, clinical trial investigators are required to submit their findings to the FDA for approval in order to move onto the next phase of the trial.

What are clinical trial phases?

Phase I

Phase I studies are the first phase of human testing that determine the pharmacovigilance (drug safety), tolerability (safe dosage range), pharmacokinetics (the movement of drug into, through, out, and the absorption rate in the body), and pharmacodynamics (biochemical and physiological effects of a specific drug on the body).  This initial phase of testing normally includes a small group of patients, typically 20-100.  Patients are typically compensated for the time they spend in the trial, which can last several months.  Phase I clinical trials are typically performed in a clinical trial clinic where patients are observed full time.  This phase of trials are run by contract research organizations (CRO’s) on behalf of pharmaceutical companies or research investigators.  Patients that participate in the study are more often healthy volunteers, but occasionally there are test performed with terminal patients.  Approximately 70% of experimental drugs pass this phase.

Phase II

Phase II studies test the the ability to produce a desired or intended result of a drug or device. After a range of doses is determined, it’s determined if the drug has any biological activity or effect.  Phase II of testing can last from several months to two years, and typically involves a larger group, from 100-300 patients. Most phase II studies are random trials where group “A” receives the experimental drug, while group “B” receives the “control” or placebo. These are what you call “blind test” which means that neither group “A”, group “B” or the researchers know who has received the drug or the placebo.  This allows investigators to provide the pharmaceutical company, sponsors, and the FDA with comparative data concerning safety and effectiveness of the drug.  About 20% of experimental drugs successfully make it to phase III.  If the clinical research of  a new drug fails, it typically occurs during Phase II trials because the drug is discovered to not perform as planned, or become toxic.

Phase III

Phase III studies are designed to determine the effectiveness, monitor side effects, and compare commonly used treatments to the new drug or treatment.  Phase III trials are randomized and “blind tested” in very large groups, typically 100’s to 1,000’s of patients which can last several years.  These trials provide pharmaceutical companies sponsors, and the FDA with a more thorough understanding of the effectiveness of the drug or device, the benefits, and the range of possible adverse reactions.  Phase III trials are typically the most costly, time-consuming, and difficult to run.  It is common that certain Phase III trials continue while the regulatory submission is pending at the FDA or other regulatory agency, allowing patients to continue to receive what may be considered life saving treatment. It is also common for each new drug or treatment to be required to perform a phase III trial more than once demonstrating safety and efficacy. This is one of the reasons that it’s common to see phase III drugs on the market. Only between 10% and 30% of drugs that are in phase III fail.

Phase IV

Phase IV studies are often called “Post Marketing Surveillance Trials” and are conducted after a drug or device has been approved for the consumer market.  The goals in phase IV is to compare new drugs with existing drugs, monitor long-term effectiveness on quality of life, and to determine the cost-effectiveness.  During a phase IV study, a new drug can become restricted within a certain patient demographic or even removed from the market if determined to be harmful.

Who can participate?

All clinical trials have specific guidelines about who can and cannot participate. Anyone can apply for any study, but before actually joining a clinical trial, each volunteer must qualify. “Inclusion Criteria” are the factors that allow volunteers to participate.  Similarly, “Exclusion Criteria” are factors that disallow volunteers from participating.   These criteria often differ from study to study but they typically 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, while others require healthy participants. It is important to note that inclusion and exclusion criteria are used to identify appropriate participants, ensure participants’ safety, and make certain that researchers learn the information they need.

What should I expect?

Before Starting

Before participating in a clinical trials, a member of the research staff will sit down with you to determine your eligibility and document your medical history. It’s during this time, the research staff will go through the inclusion and exclusion criteria to determine if you are able to participate in the study. If you qualify for the study, and wish to move onto the next step, the research staff will give you an informed connect form which will explain:

  • The purpose of the clinical trial
  • The risks, side effects, or discomforts that might be reasonably expected
  • Any benefits that can be reasonably expected
  • What will happen in the study and whether any procedures, drugs, or devices are different than those that are used in standard medical treatment
  • Available options to you and how they are better or worse than being in a clinical trial
  • That you may ask any questions about the trial prior to consenting and/or at any time during the course of the trial
  • That you may refuse participate for any reason at any time
  • What to do if complication should occur

During The Trial

Research study staff such as clinical research coordinators and physicians (Investigators) closely monitor the participants throughout the study through physical exams, laboratory tests and other medical procedures specified in each trial. Often, these procedures are described as an extension of what is traditionally done to manage the patient’s health and medical condition. Patients in research studies receive study-medication and study related care at no cost and are often compensated for time and travel. Studies can last for weeks, months or even years, during which time patients can save the enormous cost associated with paying for medications and office visits.

After The Trial

At the end of each trial, you will have a final visit with the research staff and the study will close out. The collected data is used to determine the drug’s effectiveness, if it is safe and if there are any side effects. Depending on the results, researchers then determine whether to stop testing or move to the next phase of study.

Why should I participate?
There are a number of reasons a patient would want to participate in a clinical trial.  Participating patients will:
  • Have access to the newest treatments and medications, which they otherwise would not have access to at this time or maybe ever
  • Receive close medical oversight throughout the study
  • Gain a deeper knowledge of their conditions and newest treatments
  • Receive study related medical care at no cost
  • Receive compensation for time and travel
  • Be directly aiding in the advancement of medicine, helping millions of people in the future

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