FAQs
| Printer Friendly Format |
| 1. IFPMA CLINICAL TRIALS PORTAL |
Q: Why is the pharmaceutical industry developing a portal for searching information on clinical trials?
Q: What information can I find by use of the portal?
Q: How does the portal work?
Q: How can I print out my search results?
2. CLINICAL TRIALS |
Q: Why participate in a clinical trial?
Q: Where do the ideas for trials come from?
Q: Who sponsors clinical trials and where are they conducted?
Q: What is a protocol?
Q: What is a placebo?
Q: What is a control or control group?
Q: What are the different types of clinical trials?
Q: What are the phases of clinical trials?
| 3. PARTICIPATION IN A CLINICAL TRIAL |
Q: What happens during a clinical trial?
Q: What is informed consent?
Q: What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
Q: What should people consider before participating in a trial?
Q: Does a participant stay in contact with a primary health care provider while in a trial?
Q: What are side effects and adverse reactions?
Q: What are the potential benefits and risks of participating in a clinical trial?
Q: How is the safety of the participant protected?
Q: Can a participant leave a clinical trial after it has begun?
4. ABOUT INFORMED CONSENT |
Safeguards
History
What to Expect
What Will I Find in the Informed Consent Document?
Other Useful Tips
More Information
1. IFPMA CLINICAL TRIALS PORTAL | Top |
Q: What information can I find by use of the portal?
Q: How does the portal work?
Q: How can I print out my search results?
Q: Why is the pharmaceutical industry developing a portal for searching information on clinical trials? | Top |
Q: What information can I find by use of the portal? | Top |
Secondly, the portal enables the user to find results of clinical trials conducted on medicinal products that have been approved for marketing. Industry committed to disclose these results in a non-promotional summary, regardless of the trial outcome.
Q: How does the portal work? | Top |
Q: How can I print out my search results? | Top |
2. CLINICAL TRIALS | Top |
Q: What is a clinical trial?
Q: Why participate in a clinical trial?
Q: Where do the ideas for trials come from?
Q: Who sponsors clinical trials and where are they conducted?
Q: What is a protocol?
Q: What is a placebo?
Q: What is a control or control group?
Q: What are the different types of clinical trials?
Q: What are the phases of clinical trials?
Q: What is a clinical trial? | Top |
Q: Why participate in a clinical trial? | Top |
Q: Where do the ideas for trials come from? | Top |
Q: Who sponsors clinical trials and where are they conducted? | Top |
Q: What is a protocol? | Top |
Q: What is a placebo? | Top |
Q: What is a control or control group? | Top |
Q: What are the different types of clinical trials? | Top |
Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
Observational trials address health issues in large groups of people. Trial participants may be asked to answer questions about their family histories or give blood samples, but they do not receive treatment for their diseases.
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 the quality of life for individuals with a chronic illness.
Q: What are the phases of clinical trials? | Top |
Phase I starts with the initial administration of an investigational new drug into humans.
Although human pharmacology studies are typically identified with Phase I, they may also be indicated at other points in the development sequence. Studies in this phase of development usually have non-therapeutic objectives and may be conducted in healthy volunteer subjects or certain types of patients, e.g. patients with mild hypertension. Drugs with significant potential toxicity, e.g. cytotoxic drugs, are usually studied in patients. Studies in this phase can be open, baseline controlled or may use randomisation and blinding, to improve the validity of observations.
Studies conducted in Phase I typically involve one or a combination of the following aspects:
a) Estimation of Initial Safety and Tolerability
b) Pharmacokinetics
c) Assessment of Pharmacodynamics
d) Early Measurement of Drug Activity
Phase II (Most typical kind of study: Therapeutic Exploratory):
Phase II is usually considered to start with the initiation of studies in which the primary objective is to explore therapeutic efficacy in patients.Initial therapeutic exploratory studies may use a variety of study designs, including concurrent controls and comparisons with baseline status. Subsequent trials are usually randomised and concurrently controlled to evaluate the efficacy of the drug and its safety for a particular therapeutic indication. Studies in Phase II are typically conducted in a group of patients who are selected by relatively narrow criteria, leading to a relatively homogeneous population and are closely monitored.
An important goal for this phase is to determine the dose(s) and regimen for Phase III trials. Early studies in this phase often utilise dose escalation designs to give an early estimate of dose response and later studies may confirm the dose response relationship for the indication in question by using recognised parallel dose-response designs (could also be deferred to phase III).
Confirmatory dose response studies may be conducted in Phase II or left for Phase III. Doses used in Phase II are usually but not always less than the highest doses used in Phase I.Additional objectives of clinical trials conducted in Phase II may include evaluation of potential study endpoints, therapeutic regimens (including concomitant medications) and target populations (e.g., mild versus severe disease) for further study in Phase II or III. These objectives may be served by exploratory analyses, examining subsets of data and by including multiple endpoints in trials.
Phase III (Most typical kind of study: Therapeutic Confirmatory / Hypothesis-testing):
Phase III usually is considered to begin with the initiation of studies in which the primary objective is to demonstrate, or confirm therapeutic benefit.
Studies in Phase III are designed to confirm the preliminary evidence accumulated in Phase II that a drug is safe and effective for use in the intended indication and recipient population. These studies are intended to provide an adequate basis for marketing approval. Studies in Phase III may also further explore the dose-response relationship, or explore the drug's use in wider populations, in different stages of disease, or in combination with another drug. For drugs intended to be administered for long periods, trials involving extended exposure to the drug are ordinarily conducted in Phase III, although they may be started in Phase II. These studies carried out in Phase III complete the information needed to support adequate instructions for use of the drug (official product information).
Phase IV (Variety of Studies: - Therapeutic Use):
Phase IV begins after drug approval. Therapeutic use studies go beyond the prior demonstration of the drug's safety, efficacy and dose definition.
Studies in Phase IV are all studies (other than routine surveillance) performed after drug approval and related to the approved indication. They are studies that were not considered necessary for approval but are often important for optimising the drug's use. They may be of any type but should have valid scientific objectives. Commonly conducted studies include additional drug-drug interaction, dose-response or safety studies and studies designed to support use under the approved indication, e.g. mortality/morbidity studies, epidemiological studies.
3. PARTICIPATION IN A CLINICAL TRIAL | Top |
Q: Who can participate in a clinical trial?
Q: What happens during a clinical trial?
Q: What is informed consent?
Q: What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
Q: What should people consider before participating in a trial?
Q: Does a participant stay in contact with a primary health care provider while in a trial?
Q: What are side effects and adverse reactions?
Q: What are the potential benefits and risks of participating in a clinical trial?
Q: How is the safety of the participant protected?
Q: Can a participant leave a clinical trial after it has begun?
Q: Who can participate in a clinical trial? | Top |
Q: What happens during a clinical trial? | Top |
Q: What is informed consent? | Top |
Q: What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor? | Top |
- Plan ahead and write down possible questions to ask.
- Ask a friend or relative to come along for support and to hear the responses to the questions.
- Bring a tape recorder to record the discussion to replay later.
Q: What should people consider before participating in a trial? | Top |
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the new treatment being tested may be effective? Has it been tested before?
- What kinds of tests and treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Who will pay for the treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the treatment is working? Will results of the trials be provided to me?
- Who will be in charge of my care?
| Top |
Q: What are side effects and adverse reactions? | Top |
Q: What are the potential benefits and risks of participating in a clinical trial? | Top |
Clinical trials that are well-designed and well-executed are the best treatment approach for eligible participants to:
- Play an active role in their own health care.
- Gain access to new research treatments before they are widely available.
- Obtain expert medical care at leading health care facilities during the trial.
- Help others by contributing to medical research.
Risks
There are potential risks to participating in a clinical trial.
- There may be unpleasant, serious or even life-threatening side effects to treatment.
- The treatment may not be effective for the participant.
- The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.
Q: How is the safety of the participant protected? | Top |
Every clinical trial must be approved and monitored by an Ethics Committee (called Institutional Review Board (IRB) in the U.S. or Independent Ethics Committee (IEC) in the EU) to make sure the risks are as low as possible and are worth any potential benefits. An Ethics Committee is an independent committee of physicians, statisticians, community advocates (lay persons), and other suitably qualified experts that ensures that a clinical trial is ethical and the rights of study participants are protected.
Q: Can a participant leave a clinical trial after it has begun? | Top |
4. ABOUT INFORMED CONSENT | Top |
Introduction
Safeguards
History
What to Expect
What Will I Find in the Informed Consent Document?
Other Useful Tips
More Information
INTRODUCTION: Definition of Informed Consent | Top |
The informed consent document provides a summary of the clinical trial (including its purpose, the treatment procedures and schedule, potential risks and benefits, alternatives to participation, etc.) and explains your rights as a participant. It is designed to begin the informed consent process, which consists of conversations between you and the research team. If you then decide to enter the trial, you give your official consent by signing the document. You will receive a copy and should use it as an information resource throughout the course of the trial.
The informed consent process provides you with ongoing explanations that will help you make educated decisions about whether to begin or continue participating in a trial. Researchers and health professionals know that a written document alone may not ensure that you fully understand what participation means. Therefore, before you make your decision, the research team will discuss with you the trial's purpose, procedures, risks and potential benefits, and your rights as a participant. If you decide to participate, the team will continue to update you on any new information that may affect your situation. Before, during, and even after the trial, you will have the opportunity to ask questions and raise concerns. Thus, informed consent is an ongoing, interactive process, rather than a one-time information session.
SAFEGUARDS: Informed Consent and the Larger System of Protections | Top |
HISTORY | Top |
Over the past half-century, the international medical communities have taken numerous steps to protect people who take part in clinical research. The following timeline provides an overview of some of the key events that have contributed to the development of the current system.
1947 - The Nuremberg Code
Developed in response to the Nuremberg Trials of Nazi doctors who performed unethical experimentation during World War II, the Code was the first major international document to provide guidelines on research ethics. It made voluntary consent a requirement in clinical research studies, emphasizing that consent can be voluntary only if:
- participants are able to consent;
- they are free from coercion (i.e., outside pressure); and
- they comprehend the risks and benefits involved.
The Code also states that researchers should minimize risk and harm, make sure that risks do not significantly outweigh potential benefits, use appropriate study designs, and guarantee participants' freedom to withdraw at any time. The Nuremberg Code was adopted by the United Nations General Assembly in 1948.
1964 - Declaration of Helsinki
At the 18th World Medical Assembly in Helsinki, Finland, the World Medical Association adopted 12 principles to guide physicians on ethical considerations related to biomedical research. It emphasizes the distinction between medical care that directly benefits the patient and research that may or may not provide direct benefit. These guidelines were revised at subsequent meetings in 1975 (Tokyo, Japan), 1983 (Venice, Italy), 1989 (Hong Kong), 1996 (Somerset West, Republic of South Africa), 2000 (Edinburgh, Scotland) and 2008 (Seoul, South Korea), and notes for clarification were added in 2002 (Washington) and 2004 (Tokyo).
1996 - ICH Guideline on Good Clinical Practice
The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) is a unique project that brings together the regulatory authorities of Europe, Japan and the United States and experts from the pharmaceutical industry in the three regions to discuss scientific and technical aspects of product registration. ICH guidelines aim to harmonize differences in the drug development processes in these three major pharmaceutical regions. Many other countries like Australia, Canada and the EFTA states have subsequently adopted these recommendations.
In this guideline ICH states the principles and practices concerning protection of clinical trial subjects.
| Top |
- An initial meeting during which a member (or members) of the research team provides you with the informed consent document and explains its content to you. This discussion may also include your specialist and primary care doctor, as well as a nurse, social worker, patient representative, and/or staff psychologist. You may wish to bring along a family member or friend for support, and to help you keep track of the information presented. This information should be given logically and at a comfortable pace, with plenty of time allowed for you to consider it and ask questions. You may be given a video, audiotape, or even an interactive computer program to help you better understand the information in the consent document.
If your child (e.g., under age 18) is the one who will participate in the clinical trial, you will go through this process on his or her behalf. More and more frequently, children over the age of 6 are being asked to give their "assent" to participation as well. If this is the case, you will be asked to give permission for your child to take part in the assent process. During this process, a child or teenager is provided with a form that explains, in concrete and age-appropriate terms, the purpose of the research, what they will be asked to do, and what procedures they will undergo. Usually, a nurse or other health professional also explains the information and gives the child a chance to ask questions. Then they are asked to indicate their assent either by signing the form or making a mark in a specific place. - Time to digest the information. It can be very difficult to absorb this information in one sitting, especially at a time of emotional distress. You should be given a copy of the document so that you can take it home, review it as many times as you need, and discuss it with family, friends, social workers, clergy, a patient representative, or other trusted advisors.
- Assessment of your understanding. The research team should take some steps to ensure that you comprehend the information, either by having you fill out a questionnaire, asking you questions orally, or having you explain certain aspects of the trial in your own words. You also should alert team members to anything you do not understand. If you find that the document is written in words that are too difficult for you, don't hesitate to let them know that.
- Opportunities to ask questions. Both during the initial meeting and in follow-up discussion(s), you should be given the chance to ask questions and raise concerns. Keep asking questions until you have all the information you need to make your decision.
- Continuing updates on new information. As the trial proceeds, the research team may discover new information that could affect your health, welfare, or willingness to remain in the study. They will share this with you and may ask you to sign a new informed consent document. Of course, you are free to leave the study if this information leads you to have doubts about continuing to participate.
WHAT WILL I FIND IN THE INFORMED CONSENT DOCUMENT? | Top |
Purpose [Why is this clinical trial being done?]
In this section, researchers explain why they are conducting the trial. Their reasons will depend on the type of disease and the trial type (i.e., whether they are investigating new prevention, screening, supportive care, or treatment methods). Researchers conduct treatment trials either because they have not found an effective treatment for a certain type of disease, or they are not sure which treatment method works best.
Description of Procedures [What is involved in the trial?]
This section describes the procedures that you will undergo, how frequently you will have them, and where they will take place (at home, in the hospital or clinical center, or in an outpatient setting). For treatment trials, this section should include:
- procedures that are part of regular disease care and may be done even if you do not join the trial;
- standard procedures being done because you are in the trial; and
- procedures that are being tested or evaluated by the trial.
Duration [How long will I be in the trial?]
This section indicates how long the trial will last and whether it involves follow-up, and if so, for how long. It also includes information about any circumstances under which the researcher might remove you from the trial (for example, if your condition worsens or new information indicates you shouldn't continue). The document should make clear that you have the right to stop participating at any time, and it should describe any possible medical consequences of sudden withdrawal.
Risks [What are the potential risks of the trial?]
This section includes the foreseeable physical and nonphysical risks of participating in the trial. A nonphysical risk might be time away from work, while physical risks might include side effects such as nausea, vomiting, pain, or susceptibility to infection, among others. The document should indicate the likelihood of these risks occurring, how serious they may be, and whether they are more likely to be short-term (last only during the trial or shortly afterward) or long-term (last weeks, months, or even years after the trial is over). The document should make clear which risks are related to the investigational aspects of the trial. It also should include specific information about reproductive risks (Could participating make you infertile? Should you not get pregnant or father a child while on the trial? Can you nurse a child during the trial?).
Benefits [Are there benefits to taking part?]
The document describes any benefits to you or to others which may reasonably be expected. A trial may or may not involve direct medical benefits to you, but it might lead to new knowledge that can help others in the future.
Alternatives to Participation [What are my options if I don't participate?]
For treatment trials, this section describes what care options you have besides participating in the trial, such as other commonly-used therapies or no treatment at all.
Confidentiality
This statement tells you the extent to which your information will be kept confidential. It should inform you about any groups or organizations that may have access to your records for quality assurance and data analysis (e.g. the trial sponsor).
Costs / Additional Expenses [What are the costs?]
This section indicates whether participating in the trial will result in added costs to you or your insurance company. It also covers other cost issues, such as who will pay for emergency medical treatment in case of injury or illness, whether you will have to pay for drugs that become commercially available during the trial (if this is a drug trial), and whether or not you will receive payment for participating.
Participants' Rights [What are my rights as a participant?]
The document should specify that:
- your participation is voluntary;
- you can choose not to take part or leave at any time without penalty or loss of benefits; and
- any new information that might affect your participation will be shared with you.
Contact Information [Whom do I call if I have questions or problems?]
You should have a contact name and phone number (usually of a member of the research team) for getting answers to questions about the study or a research-related injury. You also should be given a phone number for the Ethics Committee or a patient representative, in case you have questions about your rights as a research participant.
Supplemental Information [Where can I get more information?]
This section lists additional resources that may prove useful as you make your decision, such as informational booklets, community organizations, and Web resources.
The Signature
Your dated signature represents your legal consent to participate in the trial. In case of illiteracy of the participant, researchers need to pay special attention that the trial information they give is understood by the participant and a literate and disinterested person must witness the oral consent of the participant and sign the document on behalf of the participant.If any of these sections appears to be incomplete or missing from the informed consent document, don't hesitate to ask for the information.
OTHER USEFUL TIPS | Top |
- Keep a copy of the informed consent document as a helpful resource for the duration of the trial. Ask for a copy if one isn't offered to you. You may also request a copy of the protocol (full study plan).
- According to Good Clinical Practice principles, no informed consent document may include any language that asks or appears to ask you to waive your legal rights, or that releases or appears to release the investigator, the sponsor, or the institution from liability for negligence.
- If you cannot understand the forms you are signing, don't be afraid to let someone know that you are having trouble. If you have difficulties reading the document at first, try not to get upset. Many people feel anxious about reading and signing documents and communicating with physicians. Just take your time and ask for help when you need it.
MORE INFORMATION: Resources for Understanding Informed Consent | Top |
- local hospitals
- Ethics Committees
- patient advocacy and support groups
- library and Internet research.
Source: based on clinicaltrials.gov, cancer.gov and ich.org FAQ dated 20 July 2005
myPortal
You are a patient?
Gain a better understanding of clinical trials through personalized information.
You are a physician?
Advise and guide your patients with updated data on clinical trials.
Save Time
Save time and resources with free myPortal email alerts, specifically defined to meet your needs.
- Save your search
- Repeat your search
- Receive an email when a new matching trial is posted
