Glossary

Adverse Event (AE)

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

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Blinding

A process in which one or more parties within the clinical trial are unaware of the treatment the patients are assigned. Blinding is performed to avoid bias. For example, if a patient knows they are receiving the active treatment, this could skew the results, making the treatment seem more effective than it actually is.

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Clinical Study Design

There are several types of clinical studies; however, the two main types are:

• Interventional study: This study compares the new medical approach to the standard approach.

• Observational study: Participants may receive an intervention or procedure but are not specifically chosen to receive a particular intervention.

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Clinical Trial

A clinical trial is a research project that assigns human participants to one or more interventions to evaluate the effects of those interventions on health outcomes.

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Compliance

Adherence to all trial-related requirements, including ensuring that the clinic follows Good Clinical Practice (GCP) requirements and applicable regulatory standards. Additionally, compliance refers to patients adhering to the study schedule.

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Concomitant Treatment or Medication

A treatment or condition a patient is undergoing that is not being investigated in the research project in which the patient is participating.

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Control Group

A control group is one that receives either standard treatment, a placebo, or no treatment.

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Clinical Research Associate (CRA)

A clinical research associate is responsible for collecting and organizing data obtained during the clinical study. They ensure that the study stays on track with the clinical objectives and complies with relevant regulations. The CRA also analyzes data and is appointed by either the sponsor or the contract research organization (CRO).

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Clinical Research Coordinator (CRC)

The clinical research coordinator assists the investigator with clinical study-related activities and is based at the hospital or site where the clinical study is conducted.

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Case Report Form (CRF)

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

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Clinical Research Organization (CRO)

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

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Dosage

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

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Double-Blinded

In a double-blinded clinical trial, two parties are blinded. Often, this includes the patient and the trial monitors, and sometimes even the individuals analyzing the trial data.

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Efficacy

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

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Eligibility Criteria/Inclusion Criteria

Eligibility/inclusion criteria must be met by all patients enrolled in a clinical trial. These criteria may include factors such as age, gender, and medical history.

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Enrollment

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

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Exclusion Criteria

Exclusion criteria must not be met by any patient enrolled in a clinical trial. These criteria may include medical history, for example.

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Food and Drug Administration (FDA)

Relevant only in the USA, the FDA decides whether new medical products can enter the market.

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Good Clinical Practice (GCP)

A standard for the design, conduct, performance, monitoring, auditing, recording, analysis, and reporting of clinical trials that ensures data and reported results are credible and accurate while protecting the rights, integrity, and confidentiality of trial subjects.

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Good Documentation Practice (GDP)

An international ethical and scientific quality standard for designing, conducting, monitoring, recording, auditing, analyzing, and reporting studies. GDP ensures that data from clinical trials are credible and accurate and that the subjects' rights and confidentiality are protected.

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Generics

Commonly known as "copy drugs," generics contain the same active ingredient as the original drug, but the excipients may vary. This is why some patients cannot use generic drugs.

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Good Laboratory Practice (GLP)

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

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Good Manufacturing Practice (GMP)

The GMP guidelines provide the minimum requirements that manufacturers must meet to ensure their products are consistently high in quality, from batch to batch, for their intended use. GMP requirements may vary depending on the industry (e.g., food, cosmetics, pharmaceutical products, and medical devices). The main purpose of GMP is to prevent harm to the end user.

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Health Insurance Portability and Accountability Act (HIPAA)

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

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Investigator's Brochure (IB)

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

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International Conference on Harmonisation (ICH)

A regulatory and industry project aimed at improving the efficiency of the process for developing and registering new products in Europe, Japan, and the USA.

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Independent Ethics Committee (IEC)

An independent body (a review board or a committee that may be institutional, regional, national, or supranational), consisting of medical professionals and non-medical members. Its responsibility 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 reviewing and approving or providing a favorable opinion on the trial protocol, the suitability of the investigator(s), facilities, and the methods and materials used to obtain and document informed consent from 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 IEC to act in accordance with GCP guidelines.

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In Vitro

Tests conducted on cultures in an artificial environment, such as in petri dishes in a laboratory, rather than on living organisms.

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In Vivo

Tests conducted on living animals and/or human systems.

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Informed Consent

A process in which a healthcare provider gives information (educates) about the risks, benefits, and alternatives of a given procedure or intervention to a potential patient wishing to participate in a study. Informed consent must be signed before enrollment in the study. However, the decision to participate is completely voluntary.

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Investigator

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

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Investigational Product (IP)

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) differently from the approved form, when used for an unapproved indication, or when used to gain further information about an approved use.

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Institutional Review Board (IRB)

An independent body comprising medical, scientific, and non-scientific members, responsible for ensuring the protection of the rights, safety, and well-being of human subjects involved in a trial. This is achieved through reviewing, approving, and providing ongoing review of trial protocols and amendments, as well as the methods and materials used to obtain and document informed consent from trial subjects.

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Multicentre Trial

A clinical trial conducted according to a single protocol at more than one site, involving multiple investigators.

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Open-Label Study

A type of study in which no information is withheld from participants. Both the participants and the researchers know which treatment the patient is receiving.

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Orphan Diseases

An orphan disease is one that has not been "adopted" by the pharmaceutical industry, meaning no treatment has been developed, often because there is little financial incentive for the private sector to create and market new medications to prevent or treat it.

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Over-The-Counter Drugs (OTC)

Drugs that can be purchased without a prescription, such as painkillers.

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Paediatrics (Pediatrics)

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

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Participant

A patient who matches the study criteria, volunteers, and is enrolled in the study.

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Potential Candidate

A potential candidate is someone who has expressed interest in a specific study and is considered a possible participant. After signing up, they undergo a pre-screening process to determine whether they meet the study's inclusion and exclusion criteria and whether they are qualified to participate.

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Phases of a Trial

Clinical studies are divided into four phases. Each phase has a specific purpose to help researchers answer different questions and ensure the procedure is as safe as possible for participants.

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

• Phase 2: The study drug is given to a larger group of participants with a specific disease (100-300) to assess its effect and further evaluate its safety.

• Phase 3: The study drug is given to a large group of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it with existing treatments, and collect information to ensure it is used safely.

• Phase 4: Also called post-marketing studies, this phase examines the safety of medications after they have entered the market. These studies provide additional information about the drug's risks, benefits, and very rare side effects.

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Patient Information Forum (PIF)

A UK-based organization focused on promoting high-quality health and care information. It supports healthcare professionals and organizations in creating and disseminating reliable, accessible, and easy-to-understand information for patients, ensuring they can make informed decisions about their health and treatment. PIF provides resources, training, and advocacy to improve the standards of patient information.

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Principal Investigator (PI)

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

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Placebo

A placebo is an inert study drug that has no medical effect. In clinical studies, the investigational medication is often compared to a placebo to determine if the study drug has any effect on patients. In some clinical studies, one group of participants receives a placebo instead of the study drug. The participants will not know whether they receive the placebo or the investigational medication until the project is completed.

Example: Two groups diagnosed with the same disease participate in a clinical study. One group receives a pill containing the medication, while the other group receives a pill containing only sugar. The sugar pill has no medical effect. Researchers compare the groups to determine if the new study drug is effective.

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Patient-Reported Outcome (PRO)

Any health-related outcome reported by the patient who experienced it. In clinical trials, patient-reported outcomes can be documented through patient diaries or questionnaires.

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Patient Recruitment

The process of identifying, enrolling, and retaining eligible participants for clinical trials.

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Prescreener

A prescreener refers to a person or process used to assess the suitability of potential candidates for a specific research study. The process filters candidates based on specific criteria, identifying those who meet the requirements to proceed to further screening or enrollment.

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Protocol

A detailed written description of how to perform a clinical study. The protocol includes the aim, design, and methods used in the study. It also contains background information, highlights of scientific research, questions to address, and the expected duration of the trial. The protocol's goal is to ensure patient safety and secure the integrity of the data collected.

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Quality Assurance (QA)

Planned and systematic actions established to ensure that a trial is performed, and data is generated, documented, and reported in compliance with Good Clinical Practice (GCP) and applicable regulatory requirements.

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Quality Control (QC)

Operational techniques and activities conducted within the quality assurance system to verify that trial-related activities meet the required quality standards.

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Randomization

A process in which patients are assigned to different treatment groups by chance. This ensures that participants have an equal chance of receiving the treatment or being allocated to the control group.

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Rare Diseases

A rare disease affects a small percentage of the population. However, there is no universally accepted definition of rare diseases, so specific criteria vary. Rare diseases are often genetic, and many manifest early in life. Approximately 30% of children with rare diseases die before reaching the age of 5.

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Risk

Patients participating in a clinical study may experience potential risks, such as uncomfortable or serious side effects.

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Serious Adverse Event (SAE)

In human drug trials, an SAE is defined as any untoward medical occurrence at any dose that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage.

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Screening Period

Before participating in a clinical trial, potential patients are screened to determine whether they qualify for the study. Once the screening period is over, the patient may be randomized and enrolled in the study.

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Screening Visit

When a patient matches the study criteria, they visit the clinic for a screening visit. Depending on the study, the visit can last from 30 minutes to several hours. During this time, the patient meets the trial coordinator and the principal investigator and receives a copy of the informed consent form for review.

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Side Effects

Unwanted actions or impacts related to the experimental treatment.

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Single-Blinding

A procedure in which one or more parties to the trial are kept unaware of the treatment assignment(s). In a single-blinded trial, it is usually the patient who does not know which medication they are receiving.

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Site / Clinic

The site is where the patient participates in the clinical trial, and all data, such as blood samples, CT scans, and X-rays, are collected.

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Site Management Organization (SMO)

An organization that finds and manages the performance of clinical sites.

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Standard Operating Procedure (SOP)

Detailed, written instructions designed to ensure uniformity in the performance of specific functions.

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Sponsor

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

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Sponsor-Investigator

An individual who both initiates and conducts a clinical trial and under whose direction the investigational product is administered, dispensed, or used by a subject. The term does not refer to entities other than individuals (e.g., corporations or agencies). A sponsor-investigator holds the responsibilities of both a sponsor and an investigator.

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Standard of Care

Also referred to as standard treatment, it is the most widely accepted and used approach for a specific medical condition. In some studies, patients receive the standard-of-care treatment alongside the investigational treatment.

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Study Withdrawal

Patients participating in a clinical trial are allowed to withdraw from the study at any time without providing a reason and without facing any penalties.

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Subinvestigator

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

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Therapeutic Area (TA)

A field of knowledge that focuses on the research and development of treatments for diseases and pathological conditions, as well as the prevention of conditions that negatively impact health. Patiro’s therapeutic areas include cardiovascular diseases, endocrine/hormonal disorders, and more.

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Treatment Group or Experimental Group

The group of participants in a clinical study exposed to an experimental intervention. The opposite of the experimental group is the control group, which is not exposed to the experiment.

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Unexpected Adverse Drug Reaction

An adverse reaction whose nature or severity is not consistent with the applicable product information.

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Vulnerable Subjects

Individuals whose willingness to volunteer in a clinical trial may be unduly influenced by expectations of benefits from participation or fear of repercussions for refusing to participate. Examples include members of hierarchical structures (e.g., students, employees, military personnel), patients with incurable diseases, persons in nursing homes, ethnic minorities, the homeless, refugees, minors, and those unable to provide informed consent.

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Wash-Out Period

A pre-defined time period in a clinical trial during which the patient does not receive any active medication, allowing all traces of drugs to clear from the patient’s system.

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