Glossary

See compliance.

Any unexpected and unfavorable and unintended sign (including abnormal laboratory finding), symptom or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

A process, where one or more parties within the clinical trial are not aware of the treatment the patients are assigned. Blinding is performed to avoid bias. E.g. if a patient know that he/she is getting the active treatment, this could skew the results, so that it might seem better to get the active drug, than it actually is.

There are several types of clinical studies, however, the two main types are; Inverventional study; this study compares the new medical approach to the normal/standard approach. Observational study; the participants may receive an intervention or procedure, but are not chosen to receive a specific intervention.

A clinical trial is a research project which assigns human participants to one or more intervention(s) in order to evaluate what effect the intervention has on health outcome.

Adherence to all the trial-related requirements. Both considering that the clinic follows the GCP requirements, and the applicable regulatory requirements. Furthermore, compliance can be used in relation to patients adhering to the study schedule.

Treatment or condition that a patient is taking, which is not investigated in the research project, which the patient is participating in.

A control group is either; receiving standard treatment, placebo or no treatment.

A clinical research associate is responsible for collecting and organizing data which is obtained during the clinical study. Furthermore they ensure that the clinical study stays on track in relation to the clinical objective of the study and also makes sure that the clinical study complies with the respective regulations and analyze data. The CRA is appointed by either the Sponsor or by the CRO.

The clinical research coordinator assist the investigator with clinical study related activities, and is based at the hospital or site where the clinical study is being performed.

A printed, optical, or electronic document designed to record all of the protocol required information to be reported to the sponsor on each trial subject.

A person or an organization (commercial, academic, or other) contracted by the sponsor to perform one or more of a sponsor's trial-related duties and functions.

Dosage is a defined (prescribed) administration of a specific amount, number and frequency of doses over a pre-specified period of time.

Two parties are blinded in the clinical trial. Often this is the the patient and the persons performing the trial (monitors) and sometimes even the persons analysing the data from the trial.

The ability of the drug or medical intervention to produce the desired outcome under what would be ideal and controlled circumstances.

Eligibility/inculsion criteria must be met by all patients who are enrolled in a clinical trial. These criteria can e.g. be: age, gender and medical history.

When patients meet all eligibility/inclusion criteria the patient can participate in the study, and thereby is enrolled in the study.

Exclusion criteria must not be met by any patients who are enrolled in a clinical trial. These criteria can e.g. be medical history.

Only relevant in USA. They decide if new medical products can enter the market.

A standard for the design, conduct, performance, monitoring, auditing, recording, analyses, and reporting of clinical trials that provides assurance that the data and reported results are credible and accurate, and that the rights, integrity, and confidentiality of trial subjects are protected.

International ethical and scientific quality standard for designing, conducting, monitoring, recording, auditing, analyzing and reporting studies. GCP ensures that the data from clinical trials are credible and accurate, making sure that the subjects rights and confidentiality is protected.

In general terms also known as "copy drugs", meaning that the drug contains the same active ingredient as the original drug, but the excipients can vary from the original drug. This is the reason for why some patients cannot use generic drugs.

A quality system of management controls for research laboratories and organizations to ensure the uniformity, consistency, reliability, reproducibility, quality and integrity of chemical (including pharmaceuticals) non clinical safety tests; from physio-chemical properties through acute chronic toxicity tests.

The GMP guidelines provide the minimum requirements which a manufacturer must meet to assure that their products are consistently high in quality, from batch to batch for their intended user. The requirements for the GMP might vary in relation to the different industry (e.g. food, cosmetics, pharmaceutical products and medical devices). The main purpose of GMP is to avoid that harm is done to the end user.

A law in the United State designed to provide privacy standards to protect patients' medical records and other health information which is provided to health plans, doctors, hospitals and other health care providers.

A compilation of the clinical and nonclinical data on the investigational product(s) which is relevant to the study of the investigational product(s) in human subjects.

A regulatory/industry project to improve the efficiency of the process for developing and registering new products in Europe, Japan and USA.

An independent body (a review board or a committee institutional, regional, national, or supranational), constituted of medical professionals and non-medical members, whose responsibility it is to ensure the protection of the rights, safety and well-being of human subjects involved in a trial and to provide public assurance of that protection, by, among other things, reviewing and approving/providing favourable opinion on, the trial protocol, the suitability of the investigator(s), facilities, and the methods and material to be used in obtaining and documenting informed consent of the trial subjects. The legal status, composition, function, operations and regulatory requirements pertaining to Independent Ethics Committees may differ among countries, but should allow the Independent Ethics Committee to act in agreement with GCP as described in this guideline.

Test made on cultures and in an artificial enviroment - not tested in humans, more like tested in petri dishes at the lab.

Test made in living animals and/or human systems.

A process where a health care provider gives information (educates) about the risk, benefits and alternatives of a given procedure or intervention to a potential patient wishing to participate in a study. The informed consent must be signed before being enrolled in the study. However, the decision is totally voluntary.

A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called the principal investigator.

A pharmaceutical form of an active ingredient or placebo being tested or used as a reference in a clinical trial, including a product with a marketing authorization when used or assembled (formulated or packaged) in a way different from the approved form, or when used for an unapproved indication, or when used to gain further information about an approved use.

An independent body constituted of medical, scientific, and non-scientific members, whose responsibility is to ensure the protection of the rights, safety and well-being of human subjects involved in a trial by, among other things, reviewing, approving, and providing continuing review of trial protocol and amendments and of the methods and material to be used in obtaining and documenting informed consent of the trial subject.

A clinical trial conducted according to a single protocol but at more than one site, and therefore, carried out by more than one investigator.

A type of study, where no information is withheld from participants. This means that both the participant and the researchers knows which treatment that the patient is receiving.

An orphan disease is a disease which has not been "adopted" by the pharmaceutical industry. Meaning that no treatment has been developed, often due to the fact that it provides little financial incentive for the privat sector to make and market new medications to prevent it.

Drugs that you can buy without having a prescription e.g. painkillers etc.

A branch of medicine involving medical care of infants, children and adolescents. Pediatrics means "healer of children".

A patient matching the study who volunteers and is enrolled in the study.

Clinical studies are divided into four phases. Each phase has its unique purpose to help researchers answer various questions and make the procedure as safe as possible for the participant.

Phase 1: The study drug is tested for the first time in small groups with only healthy participants (20-80 participants) to evaluate safety, determine a safe dose of therapy and identify possible side effects.

Phase 2: The study drug is given to a larger group of participants with a specific disease (100-300 participants) to see if the study drug has an effect and to evaluate the investigational medication’s safety.

Phase 3: The study drug is given to a large group of people (1000-3000) to confirm the investigational medication’s effect, to monitor side effects, to compare the study drug with older, existing medications, and to collect information so the study drug is used safely.

Phase 4: This stage is also called post-marketing studies that examined how safe medications are after the products have entered the market. These examinations will take place after the study drug has been approved. This stage provides additional information about the drug’s risks, benefits and very rare side effects.

The person(s) in charge of a clinical trial or a scientific research grant. The principal investigator prepares and carries out the clinical trial protocol (plan for the study) or research paid for by the grant. The principal investigator also analyzes the data and reports the results of the trial or grant research.

Placebo is an inert study drug that does not have any medical effect. In clinical studies, the investigational medication is often compared to placebo to be able to see if the study drug shows any effect on patients. In some clinical studies, one group of participants receive placebo instead of the study drug. The groups of participants will not know if they receive placebo or the investigational medication until the project is completed.

An example of placebo being used would be the following scenario: Two groups diagnosed with the same disease receive a study drug, since they are participating in a clinical study. One of the two groups will receive a pill containing the medication, while the other group will receive a pill only containing sugar. Since the pill containing sugar does not contain medicine, this pill has no medical effect. When using placebo, the scientists looks for indifferences between the groups to see if the new study drug worked or not.

Any health-related outcome that is reported by the patient who experienced this outcome. When participating in a clinical trial a patient reported outcome can e.g be reported as patient diaries or questionnaires.

The protocol is a detailed written description of how to perform the clinical study. The protocol contains the aim, design and methods used in relation to the study. Furthermore, the protocol contains background information for the trial, highlight of scientific research and questions to address as well as noting how long the trial is expected to last. The aim of the protocol is to ensure safety of the patients in the trial and secure the integrity of the data collected.

All those planned and systematic actions that are established to ensure that the trial is performed and the data are generated, documented (recorded), and reported in compliance with Good clinical Practice (GCP) and the applicable regulatory requirement(s).

The operational techniques and activities undertaken within the quality assurance system to verify that the requirements for quality of the trial-related activities have been fulfilled.

Patients are allocated to different types of treatment by the use of randomization. Patients are assigned to different treatment groups by chance. Since the allocation is done by chance, there is an equal chance of receiving treatment or being allocated to the control group.

A rare disease affects a small percentage of the population. However, there is not single widely accepted definition for rare diseases, so a specific cutoff has not been established. Often, the rare disease is genetic and many rare diseases appear early in life, and about 30 % of children with rare diseases will die before reaching 5 years of age.

Patients participating in a clinical study will experience potential risk, which e.g. could be potentially experiencing uncomfortable or serious side effects.

A SAE in human drug trials is defined as any untoward medial occurence that any dose; results in death, is life-threatening, requres inpatient hospitalization or causes prolongation of existing hospitalization , results in persistent or significant disability/incapacity, is a congenital anomality/birth defact, or requires intervention to prevent permanent impairment or damage.

Before participating in a clinical trial, a potential patient will be screened in order to determine whether he or she qualifies for the study. Only when the screening period is over, the patient can be randomized and enrolled in the study.

When a patient match the study, the patient will visit the clinic in order to have a screening visit performed. Depending on the study, the visit can last 30 minutes to several hours, where the patient meet the trial coordinater and the principal investigator. Furthermore, the patient receives a copy of the informed consent form to review.

Side effects are often defined as unwanted actions or impacts related to the experimental treatment.

A procedure in which one or more parties to the trial are kept unaware of the treatment assignment(s). Single-blinding usually refers to the subject(s), investigator(s), monitor, and, in some cases, data analyst(s) being unaware of the treatment assignment(s). Often, at a single blinded trial, it is the patient who does not know which medication they recieve.

The site is were the patient participate in the clinical trial. This is were all data is collected e.g. Blood samples, CT scan, X-ray etc.

Organisation thats finds and manage performing of clinical sites.

Detailed, written intructions to achieve uniformity of the performance of a specific function.

An individual, company, institution, or organization which takes responsibility for the initiation, management, and/or financing of a clinical trial.

An individual who both initiates and conducts, alone or with others, a clinical trial, and under whose immediate direction the investigational product is administered to, dispensed to, or used by a subject. The term does not include any person other than an individual (e.g. it does not include a coroperation or an agency). The obligations of a sponsor-investigator include both those of a sponsor and those of an investigator.

Standard of care is also described sometimes as standard treatment. It refers to the most widely used and accepted in relation to a specific medical condition. In some studies the patient will receive the existing standard-of-care treatment in addition to the treatment which is being investigated.

When participating patients are at any time allowed to withdraw themselves fom a clinical trial without giving any reason and without receiving any form of penalty.

Any individual member of the clinical trial team designated and supervised by the investigator at a trial site to perform critical trial-related procedures and/or make important trial-related decisions (e.g. associates, residents, research fellows).

A knowledge field that focuses on research and development of treatments for diseases and pathologic findings, as well as prevention of conditions that negatively impact the health of an individual. The different TA's Patiro has are e.g. cardiovascular disease, endocrine/hormonal diseases etc.

An experimental group is a group of participants who participate in the clinical study and are exposed to an experiment/intervention. The opposite of the experimental group is the control group, which is not exposed to an experiment.

An adverse reaction, the nature or severity of which is not consistent with the applicable product information.

Individuals whose willingness to volunteer in a clinical trial may be unduly influenced by the expectation, whether justified or not, of benefits associated with participation, or a retaliatory response from senior members of a hierachy in case of refusal to participate. Examples are members of a group with a hierarchial structure, such as medical, pharmacy, dental and nursing students, subordinate hospital and laboratory personnel, employees of the pharmaceutical industry, members of the armed forces and persons kept in detention. Other vulnerable subjects include patients with incurable diseases, persons in nursing homes, unemployed or impoverished persons, patients in emergency situations, ethnic minority groups, homeless persons, nomads, refugees, minors and those incapable of giving consent.

A pre-defined time period in a clinical trial where the patient does not receive active medication so that all traces of drugs are not within the patients system.