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Clinical Research

Table of Contents:

Sr Topics Page No.1. Definition of Clinical Research2. Goals of Clinical Research3. Types of Clinical Research4. Structure & Types of Research Investigations5. Steps in Planning6. Phases of Clinical Research7. Clinical Trials & its Phases8. Criteria of Participation9. Randomization10. Informed Consent11. Risks & Benefits of Clinical Research12. Ethical Issues13. Glossary14. References

Clinical Research

Definition:

Clinical research is a branch of healthcare science that determines the safety and effectiveness of medications, devices, diagnostic products and treatment regimens intended for human use. These may be used for prevention, treatment, diagnosis or for relieving symptoms of a disease.

Clinical research directly involves a particular person or group of people, or that uses materials from humans, such as their behavior or samples of their tissue.

The National Institutes of Health defines “clinical research” as research conducted with human subjects (or on material of human origin such as tissues, specimens and cognitive phenomena) for which an investigator directly interacts with human subjects.

In clinical research people participate as patients or volunteers. Different terms are used to describe clinical research, including clinical studies, clinical trials, studies, research, trials, and protocols.

Goals of Clinical Research:

Clinical research may have a number of goals, such as:

To develop new treatments or medications. To identify causes of illness. Studying trends. To evaluate ways in which genetics may be related to an illness.

Clinical research can be based on any of the following four concepts:

1. Treatment of a disease2. Diagnosis of a disease or disorder or dysfunction3. Systematic review of several clinical studies4. Prognosis of a particular disease.

In general, clinical research or studies are designed to add to medical knowledge related to the treatment, diagnosis, and prevention of diseases or conditions. Some common reasons for conducting clinical studies include:

• Evaluating one or more interventions (for example, drugs, medical devices, approaches to surgery or radiation therapy) for treating a disease, syndrome, or condition

• Finding ways to prevent the initial development or recurrence of a disease or condition. These can include medicines, vaccines, or lifestyle changes, among other approaches.

• Evaluating one or more interventions aimed at identifying or diagnosing a particular disease or condition

• Examining methods for identifying a condition or risk factors for that condition

• Exploring and measuring ways to improve the comfort and quality of life of people with a chronic illness through supportive care

Types of Clinical Research:

Clinical research includes:

Patient-oriented research:

This type of research involves a particular person or group of people, or uses materials from humans. This research can include:

Mechanisms of human disease. Therapeutic interventions. Clinical trials. Development of new technologies.

Epidemiological and behavioral studies:

These types of studies examine the distribution of disease, the factors that affect health, and how people make health-related decisions.

Outcomes and health services research:

These studies seek to identify the most effective and most efficient interventions, treatments, and services.

http://keck.usc.edu/Research/Clinical_Research/Definition.aspx

There are Different types and levels of clinical research, which are used accordingly. As a Research Subject, it is important to understand the type of research study. Below are descriptions of some different kinds of clinical research.

Treatment

Treatment research (also called “clinical trials”) generally involves an intervention such as medication, psychotherapy, new devices, or new approaches to surgery or radiation therapy.

Prevention

Prevention research looks for better ways to prevent disorders from developing or returning. Different kinds of prevention research may study medicines, vitamins, vaccines, minerals, or lifestyle changes.

Diagnostic

This refers to the practice of looking for better ways to identify a particular disorder or condition.

Screening

Screening research aims to find the best ways to detect certain disorders or health conditions.

Quality of life

Also known as “supportive care,” this research explores ways to improve comfort and the quality of life for individuals with a chronic illness.

Genetic studies

Genetic studies aim to improve the prediction of disorders by identifying and understanding how genes and illnesses may be related. Research in this area may explore ways in which a person’s genes make him or her more or less likely to develop a disorder. This may lead to development of tailor-made treatments based on a patient’s genetic make-up.

Epidemiological studies

Epidemiological studies seek to identify the patterns, causes, and control of disorders in groups of people.

Other kinds of clinical research

Many people believe that all clinical research involves testing of new medications or devices. This is not true, however. Some studies do not involve testing medications and a person’s regular medications may not need to be changed. Healthy volunteers are also needed so that researchers can compare their results to results of people with the illness being studied. Some examples of other kinds of research include the following:

• A long-term study that involves psychological tests or brain scans

• A genetic study that involves blood tests but no changes in medication

• A study of family history that involves talking to family members to learn about people’s medical needs and history.

The Structure & Types of Clinical Research investigation:

It can be can be broadly classified into three phases:

Planning and design of research project. Execution of the investigation. Analysis and meaningful interpretation of the results.

Research investigations are classified into two types:

Observational Studies: Experimental Studies:

Observational studies:

Observational studies include prevalence, incidence, case-control and cohort studies. Prevalence and incidence studies are designed to obtain estimates of certain simple characteristics of the population. These investigations focus on describing the distribution of diseases, which populations do or do not develop a disease, its geographical distribution, and how the disease frequency varies over time (trend). Prevalence studies are also known as cross-sectional studies in which individuals are observed only once at a given time. Incidence studies, on the other hand, are longitudinal in nature; investigate changes over time, and record observations on more than one occasion in the same population.

Case-control and cohort studies are conducted to ascertain the associations between certain variables with outcome of interest or to identify the causal factors. These studies focus on the determinants of a disease by testing the hypothesis formulated from earlier similar descriptive

studies. In these studies, the researcher collects information on the attributes or measurements of interest but, as in prevalence and incidence studies, does not influence events. In a case-control study, the numbers of subjects with the disease in question (the cases) are identified along with some unaffected subjects (controls). The past history of these groups in relation to exposure(s) of interest is then compared. In contrast, in a cohort study, a group of subjects of interest is identified and then followed prospectively till the outcome of interest occurs or for a specified time period.

Experimental studies:

Experimental studies include clinical trials and animal and laboratory studies. These studies are classified into prophylactic and therapeutic trials and interventional studies. These are usually carried out to assess the effects of various treatments on different experimental groups. The distinguishing feature of experimental studies is that there is planned interference by the investigator.

Experimental studies often provide the most reliable evidence regarding the hypothesis being tested. The crucial question in experimental studies is how to allocate the intervention to the available units or subjects. It is necessary to arrange the groups being compared. The groups are made similar and as alike as possible except for the features of direct interest (matching). Ethical considerations form an important issue in the finalization of all studies.

Steps in Planning Clinical Research:

Selecting a problem for research

The most important step in the formulation of a research protocol is to define the problem to be studied. For this purpose, one has to study the patterns and changes which have already taken place, understand what has already been done, what has yet to be done, what are the priorities and prior knowledge regarding the problem.

This can be achieved by a detailed study of the available literature and discussions with peer groups.

Formulation of objectives and hypothesis

After selecting a problem, the aims and objectives of the study need to be defined through statements in a precise and clear manner. Such statements are known as hypotheses. Ultimately, these hypotheses will be proved or disapproved on the basis of the results of the proposed research.

Area of study

After selecting a problem, the study area has to be selected. In a community-based study, the geographical area for the study could be a district, a state or a smaller unit. In certain circumstances, a special group can be further selected, e.g. a religious group, or people belonging to one profession, etc. In a hospital-based study, on the other hand, the contributing departments or hospitals have to be identified prior to commencing the investigation.

Duration of the study

Temporal trends over years or seasonal trends over months have to be kept in mind while designing a study. The duration for a particular type of study has to be decided, depending upon the problem. Questions such as duration of recruitments of subjects into the study and the follow up time, the schedule of follow up, the rationale for a particular schedule, etc. need to be defined. For studies involving infective agents, a shorter period of observation usually suffices while investigations into causal factors may require years of observation.

Design of the study

The study design varies according to the type of research. In most cases, it is tailored to suit a particular study. In clinical and Para clinical fields studies are usually experimental, while observational studies are more frequently done in community- or hospital-based settings. A therapeutic study can be a two- or three arm randomized or non-randomized, blind or double-blind trial.

Similarly, observational studies must also be designed carefully. The help of a statistician or a research methodology expert is particularly valuable at the designing stage. Fallacies in study design can render extremely good data worthless.

Investigators of the study

As medical research usually involves "a group of people, it is beneficial to assign specific tasks to each person at the beginning of the study. Identifying key personnel in certain areas helps in the smooth execution of the research project.

Subjects to be studied

The subjects for the study may suffer from a disease or different grades of the disease such as mild, moderate or severe. Alternatively, they may just be residing in an area chosen for the study.

The inclusion and exclusion criteria of subjects for the study need to be defined and adhered to rigidly. Any violation of this ultimately leads to exclusion of that subject or set of observations from the analysis. This can lead to biased and invalid results.

Selection of variables

On the basis of the defined objectives and hypothesis to be tested, a list of relevant variables, including the co-variants pertinent to the problem, should be defined. While choosing the variables it is necessary to assess the feasibility of collecting the data in a reliable and accurate manner. The tools and equipment required for collecting the information are dependent on the variables selected for the study.

Designing tools for data collection

Experimental study designs are based on definite methodologies while observational studies are carried out using questionnaires or peroformas for eliciting data. All the subjective questions used in the peroforma such as satisfactory or unsatisfactory, exposed or unexposed need to be standardized to obtain uniform answers from all the respondents. The conditions being studied must be defined explicitly. It would be ideal to adopt standardized definitions for various terms such as severity of a disease, stage, distribution, etc. It is also best to test the tool developed in a pilot study.

Sample design

In medical research, the aim is to make general statement based on observations about a wider set of subjects. The set of all subjects (human or animal under investigation) is called the population of interest. Some examples include institutional populations, armed personnel, factory workers, school children, and a population suffering from a disease.

The various methods of selecting the samples are:

1. Simple random sampling:

In this technique, each unit has the same probability of being selected.

2. Stratified sampling:

In this method of sampling, the population is first divided into subgroups or strata, according to some characteristics, and a random sample is then taken from each subgroup.

3. Systematic sampling:

In this method of sampling, the first unit of the sample is selected at random and the subsequent units are selected in a systemic manner.

4. Multistage sampling:

In this type of sampling, sampling units are selected at various stages. For example, if the prevalence of a disease is to be estimated, a sample of village may be selected at random in the first stage, out of these selected villages, a random sample of houses can be selected in the second stage and out of these sampled houses, a random sample of individuals may be selected in the third stage.

5. Cluster sampling:

A cluster of individuals (villages, localities, etc.) are treated as units and a sample of these units are drawn and observations for each member of every sample cluster are recorded.

Clinical Research Methods A step towards quality medical research

By A. MATHEW, N. S. MURTHY

Phases of clinical research

Clinical studies/ research are classified under four phases:

Phase i: Researchers test an investigational drug in a small number of people by giving it to either healthy volunteers or patients to evaluate its safety, determine a safe dosage range and identify side effects.

Phase ii: The investigational drug is given to a larger number of people who have a particular disease or condition to further evaluate its safety and often to assess its effectiveness.

Phase iii: Several hundred to several thousand people with the appropriate disease or condition participate in these studies. The investigational drug undergoes additional testing to further determine its safety and effectiveness, monitor side effects, and compare it to commonly used treatments or placebo.

Phase iv: After a drug is available by prescription, additional information about a drug's risks, benefits and use may be gathered.

http://www.bms.com/StudyConnect/Pages/clinical_study_phases.a

Clinical Trials

The development of a new treatment generally progresses through several steps that have become known as phases. There are three major phases (I, II, III) in the development of a new drug or procedure.

Volunteer Research Subjects participate in all three of these phases.

As an example the research testing of a new drug can be describe through these phases.

Phase I Trials

One of the first things we want to know about a new drug is how well humans tolerate it. Phase I trials focus on the safety of a new drug or device. The trial may involve various doses and/or methods of administration to study the symptoms, side effects or laboratory changes that may occur.

Research Subjects should be aware of certain special features about Phase I trials.

• First, in order to increase the safety of these trials, it is required that a new drug has been studied extensively in laboratory animals. Therefore, in the informed consent process with the PI, the Research Subject will have an opportunity to find out the results of those animal experiments.

• Second, the Research Subject must have no expectation of personal benefit because the value of the new drug is unknown at that time.

• Third, the Research Subject must be aware that the risks of unexpected complications will be higher in this phase of research than in the next two phases.

Phase II Trials

Assuming the Phase I trial of this new drug has shown it to be well tolerated without serious side effects, the next question to answer is "Does it work?"

For example, a sponsor has a new drug it thinks will treat headaches better than available medications. A Phase I trial showed it was well tolerated with few side effects. In the Phase II trial, it will be given to persons with headaches, in various doses, to see if the drug helps relieve headaches. If the persons do experience relief, it may be worth studying further. If they report no relief, it probably isn't worth further study.

Importantly, Phase I and Phase II trials include small groups of Research Subjects. Once a drug or device is determined to have a desirable effect on a specific disease or symptom, a larger study is still necessary to confirm both the safety and effectiveness of the treatment.

Phase III Trials

Again, assuming that both the Phase I and Phase II trials were successful, now the questions are, "How well does this drug work?" and "How good is it when compared with the drugs we already have?" Now the number of Research Subjects must become very large. In order to have enough subjects, these trials often are carried out by many PIs in different institutions throughout the country. The results of the Phase III trial will determine whether or not the new drug or device will get Federal Food and Drug Administration (FDA) approval. FDA approval is required before it can be used in medical practice and sold in pharmacies and/or used in hospitals.

Phase III trials also have special requirements that the Research Subject must understand. These rules include techniques to avoid the effects of bias in the collection of information. The scientific use of the word bias refers to anything that could falsely affect the results of the study. Avoiding bias in a Phase III study obviously is of critical importance. Therefore, it is common that these studies are blinded so that no one, including the PI, knows what drug(s) or treatment(s) any Research Subject is getting.

Another comparison technique that prevents bias is to give some of the Research Subjects as placebo as a control. A placebo is a pill that looks like all the others but has nothing effective in it. It is often referred to as a "sugar pill." Sometimes the control is not a placebo but is the currently accepted treatment for the disease. In contrast to the other Phases, the Research Subjects in Phase III trials will receive information on all the drugs to be used, including the placebo, but will not know which one they are taking!

Phase IV Trials (After Approval and Sale)

Following approval and sale of new drugs on the market, the FDA continues to gather data on these medications. Sometimes complications that did not show up when hundreds of people used a new drug in the clinical trials will appear when thousands of people use them. If serious complications do appear, the FDA or the manufacturer may decide to withdraw the drug from the market, even after all that careful study.

http://clinicaltrials.gov/ct2/about-studies/learn

ctsi.med.nyu.edu/patient.../clinical-research/clinical-trials

Criteria of participation in clinical research

Each clinical research study has different requirements that determine whether a person can participate. These requirements are called “inclusion” and “exclusion” criteria. The criteria are used to help researchers answer the study questions and to ensure the safety of all volunteers.

Depending on what the study is testing, inclusion and exclusion criteria may include a person’s:

• Illness

• Health history

• Past or current treatment

• Age or sex

• Address (A person living too far from where the study takes place may not be eligible to participate).

Randomization in Clinical research:

In clinical research studies that seek to test the effectiveness of a new intervention (for example: a new medication or a new psychotherapy technique), participants will usually be evenly placed in different study groups. Participants do not know to which group they have been assigned.

One group receives the experimental intervention; the other receives standard or no treatment. This process is called randomization. It allows researchers to compare results between the two groups. It is important to note that many studies (such as those involving brain imaging or genetics) may not require randomization.

People who take part in studies requiring randomization are generally placed in a group based on chance, not choice. Some participants are placed in the “experimental” group, and the others are placed in the “control” group. The control group will receive standard treatment, a placebo, or other intervention based on the design of the study.

For example 100 people decide to participate in a study that compares a new treatment to a standard treatment. Each participant is randomly assigned to either the treatment group or the control group. To do this, researchers use a “coin toss” or similar method. Half of the people are assigned to the treatment group and half are assigned to the control group. The researchers then compare the results of the two groups to see which treatment works better. They also try to figure out why it works better.

Informed consent:

Before starting any research or study, it is important to fully understand the nature of participation. Researchers will provide an “informed consent” statement to the participants.

This is a document that has detailed information about the study, including its length, the number of visits required, and the medical procedures and medications in which one will take part. The document also provides expected outcomes, potential benefits, possible risks, any available treatment alternatives, expenses, terms of confidentiality, and contact information for people to call if there are any questions or concerns. When needed, a translator may be provided.

Researchers will review the informed consent statement with the participant and answer the questions. After reviewing the statement, getting all the information If participant is agreed to participate in research, he/she will need to sign the informed consent statement. The signature of participant indicates that he/she understands the study and agrees to participate voluntarily. The participant may leave a study at any time and for any reason even after signing the informed consent document.

Sometimes, a potential participant may not be able to give informed consent because of memory problems or mental confusion. Someone else, usually a family member with a durable power of attorney, can give consent for that participant. That caregiver must be confident there is small risk to the participant, and that he or she would have agreed to consent if able to do so.

Risks and benefits of participating in clinical Trials/ research:

Risks

The nature of the risks depends on the kind of study. Often, clinical studies pose the risk of only minor discomfort that lasts for a short time.

Risk can occur in many different ways, and it is important to speak with the research team to understand the risks in a particular study.

All research sites are required to review their studies for any possible harm, and to share any potential risks with study volunteers. Possible risks include the following:

• The treatment involved may have health risks, such as unwanted side effects.

• The study may require more time and attention than standard treatment. May be there are more visit of study site, additional blood tests, stay in the hospital, or manage complex dosage requirements for medication.

• The treatment may not make participants better.

• Participant may hope to receive a new treatment, but may receive a standard treatment or placebo (inactive pill).

• Whether a new treatment will work cannot be known ahead of time. There is always a chance that a new treatment may not work better than a standard treatment, may not work at all, or may be harmful.

• The treatment may cause side effects that are serious enough to require medical attention.

Benefits

Benefits to participating in a study include:

• Treatment with experimental or study medications not widely available elsewhere.

• Care from a research team that includes doctors and other health care professionals who are familiar with the most advanced treatments available.

• Treatment that has been reviewed by many people, including other doctors and researchers.

• Research-related care or medicine at no cost.

• The opportunity to learn more about an illness and how to take care of it.

• The satisfaction of helping others by contributing to medical knowledge, or helping to identify possible new treatments.

Ethics in Clinical Research:

The purpose of ethical guidelines is both to protect patient volunteers and to preserve the integrity of the science.

The ethical guidelines in place today were primarily a response to past abuses, the most notorious of which in America was an experiment in Tuskegee, Alabama, in which treatment was withheld from 400 African American men with syphilis so that scientists could study the course of the disease. Various ethical guidelines were developed in the 20th century in response to such studies.

Some of the influential codes of ethics and regulations that guide ethical clinical research include:

Nuremberg Code (1947) Declaration of Helsinki (2000) Belmont Report (1979) CIOMS (2002) U.S. Common Rule (1991)

While conducting any clinical studies, the following points should be strongly considered:

Social and clinical value

Every research study is designed to answer a specific question. Answering certain questions will have significant value for society or for present or future patients with a particular illness. An answer to the research question should be important or valuable enough to justify asking people to accept some risk or inconvenience for others. In other words, answers to the research question should contribute to scientific understanding of health or improve our ways of preventing, treating, or caring for people with a given disease. Only if society will gain useful knowledge — which requires sharing results, both negative and positive — can exposing human subjects to the risk and burden of research be justified.

Scientific validity

A study should be designed in a way that will get an understandable answer to the valuable research question. This includes considering whether the question researchers are asking is answerable, whether the research methods are valid and feasible, and whether the study is designed with a clear scientific objective and using accepted principles, methods, and reliable practices. It is also important that statistical plans be of sufficient power to definitively test the objective, for example, and for data analysis. Invalid research is unethical because it is a waste of resources and exposes people to risk for no purpose.

Fair subject selection

The primary basis for recruiting and enrolling groups and individuals should be the scientific goals of the study — not vulnerability, privilege, or other factors unrelated to the purposes of the study. Consistent with the scientific purpose, people should be chosen in a way that minimizes risks and enhances benefits to individuals and society. Groups and individuals who accept the risks and burdens of research should be in a position to enjoy its benefits, and those who may benefit should share some of the risks and burdens. Specific groups or individuals (for example, women or children) should not be excluded from the opportunity to participate in research without a good scientific reason or a particular susceptibility to risk.

Favorable risk-benefit ratio

Uncertainty about the degree of risks and benefits associated with a drug, device, or procedure being tested is inherent in clinical research — otherwise there would be little point to doing the research. And by definition, there is more uncertainty about risks and benefits in early-phase research than in later research. Depending on the particulars of a study, research risks might be trivial or serious, might cause transient discomfort or long-term changes. Risks can be physical (death, disability, infection), psychological (depression, anxiety), economic (job loss), or social (for example, discrimination or stigma from participating in a certain trial). Has everything been done to minimize the risks and inconvenience to research subjects, to maximize the potential benefits, and to determine that the potential benefits to individuals and society are proportionate to, or outweigh the risks? Research volunteers often receive some health services and benefits in the course of participating, yet the purpose of clinical research is not to provide health services.

Independent review

To minimize potential conflicts of interest and make sure a study is ethically acceptable before it even starts, an independent review panel with no vested interest in the particular study should review the proposal and ask important questions, including: Are those conducting the trial sufficiently free of bias? Is the study doing all it can to protect research volunteers? Has the trial been ethically designed and is the risk–benefit ratio favorable? In the United States, independent evaluation of research projects is done through granting agencies, local institutional review boards (IRBs), and data and safety monitoring boards. These groups also monitor a study while it is ongoing.

Informed consent

For research to be ethical, most agree that individuals should make their own decision about whether they want to participate or continue participating in research. This is done through a process of informed consent in which individuals (1) are accurately informed of the purpose, methods, risks, benefits, and alternatives to the research, (2) understand this information and how it relates to their own clinical situation or interests, and (3) make a voluntary decision about whether to participate.

There are exceptions to the need for informed consent from the individual — for example, in the case of a child, of an adult with severe Alzheimer’s, of an adult unconscious by head trauma, or of someone with limited mental capacity. Ensuring that the individual’s research participation is consistent with his or her values and interests usually entails empowering a proxy decision maker to decide about participation, usually based on what research decision the subject would have made, if doing so were possible.

Respect for potential and enrolled subjects

Individuals should be treated with respect from the time they are approached for possible participation—even if they refuse enrollment in a study—throughout their participation and after their participation ends. This includes:

1. Respecting their privacy and keeping their private information confidential.

2. Respecting their right to change their mind, to decide that the research does not match their interests, and to withdraw without penalty.

3. Informing them of new information that might emerge in the course of research, which might change their assessment of the risks and benefits of participating.

4. Monitoring their welfare and, if they experience adverse reactions, untoward events, or changes in clinical status, ensuring appropriate treatment and, when necessary, removal from the study.

5. Informing them about what was learned from the research. Most researchers do a good job of monitoring the volunteers’ welfare and making sure they are okay. They are not always so good about distributing the study results. If they don’t tell you, ask

Sources:

http://clinicalcenter.nih.gov/recruit/ethics.html

Glossary

Essential terms for understanding mental health clinical research

Comparison–To learn more, researchers compare results from patients in the experimental groups with results from patients in the control groups.

Confidentiality regarding participants–This refers to the practice of maintaining as private all information related to clinical trial participants, including their personal identity and all personal medical information. Results from the study will usually be presented in terms of trends or overall findings and will not mention any specific participants.

Control group–The group of participants that receives standard treatment or a placebo. The control group may also be made up of healthy volunteers. Researchers compare results from the control group with results from the experimental group to find and learn from any differences.

Experiment– A study done to answer a question. Other words to describe an experiment are “research,” “study,” and “protocol.”

Experimental group– The group of participants in a study that receive the experimental or study intervention (such as medication or psychotherapy).

Food and Drug Administration (FDA)–The FDA is the Federal agency responsible for ensuring that foods are safe, wholesome and sanitary; human and veterinary drugs, biological products, and medical devices are safe and effective; cosmetics are safe; and electronic products that emit radiation are safe. Some of the agency’s specific responsibilities include regulating medications and devices.

Healthy volunteer–In a clinical study, a person who does not have the disorder or disease being studied. Results from healthy controls are compared to results from the group being studied.

Inclusion/exclusion criteria–Inclusion criteria are the factors that allow someone to participate in a clinical trial. Exclusion criteria are the factors that prevent someone from participating in the trial. These factors may include a person’s illness, health history, past treatment, age, sex, or where he or she lives.

Informed consent–When a participant provides informed consent, it means that he or she has learned the key facts about a research study and agrees to take part in it. For more detailed information, see the “What is informed consent?” section in this guide.

National Institutes of Health (NIH)–Part of the U.S. Department of Health and Human Services, NIH is the primary Federal agency for conducting and supporting medical research.

Placebo– An inactive pill. This is sometimes called a “sugar pill.” In some studies, participants may be assigned to take a placebo rather than the study medication.

Placebo effect–Sometimes people taking a study medication receive benefits that are not from the chemicals in the medicine. This is called a “placebo effect

Post-marketing studies–Studies done after a treatment, medication or device is approved for use by the FDA. These studies gather additional information about a product’s safety, effectiveness, or best use.

Protocol– A study done to answer a question. Other words to describe a protocol are “research,” “study,” and “experiment.” “Protocol” also refers to the plan that details what researchers will do during the study.

Randomization/random assignment–This is the process in which researchers evenly assign study participants into a group receiving the experimental treatment being studied, and others into a group receiving standard or no treatment. Participants are assigned to a group based on chance, not choice.

Research–A study done to answer a question. Scientists do research when they’re not sure what will work best to help people with an illness. Other words to describe clinical research are “clinical trial,” “protocol,” “study,” and “experiment.”

Sponsors–Clinical trials are sponsored or funded by various organizations or individuals, including physicians, foundations, medical institutions, voluntary groups, and pharmaceutical companies, as well as Federal agencies such as NIH, FDA, the Department of Defense, and the Department of Veterans Affairs.

Standard treatment–The treatment that medical professionals consider at the time of the study to be the most prevalent and best available treatment.

Standardized procedures–These are study rules that researchers must follow exactly for every participant, regardless of what each participant is used to. For example, if you normally take a medicine by injection but the experiment is testing the same medicine in pill form, the researcher must prescribe pills to you. The researcher cannot use a different method for you.

Study– Conducted by a principal investigator who is often a doctor. Members of the research team regularly monitor the participant’s health to determine the study’s safety and effectiveness. Other words to describe a study are “clinical trial,” “protocol,” “experiment,” and “research.”

Single-blind research design–A study in which one party, either the investigator or participant, is unaware of what medication or intervention the participant is taking; also called single-masked study.

References

1. NIH Clinical Center

2. ClinicalTrials.gov: Glossary of Clinical Trials Terms

3. National Cancer Institute

http://www.nimh.nih.gov/health/publications/a-participants-guide-to-mental-health-clinical-research/index.shtml