Glossary

A

Administration mode: The way a questionnaire is administered to the patient (Caregiver-administered; Clinician-rated; Interviewer-administered; Proxy-administered; Proxy-rated; Self-administered).

ATC code: In the Anatomical Therapeutic Chemical (ATC) classification system, the drugs are divided into different groups according to the organ or system on which they act and their chemical, pharmacological and therapeutic properties. Drugs are classified in groups at five different levels. The drugs are divided into fourteen main groups (1st level), with one pharmacological/therapeutic subgroup (2nd level). The 3rd and 4th levels are chemical/pharmacological/therapeutic subgroups and the 5th level is the chemical substance. The 2nd, 3rd and 4th levels are often used to identify pharmacological subgroups when that is considered more appropriate than therapeutic or chemical subgroups. The ATC Index 2005 is available on the WHO Collaborating Centre for Drugs Statistics Methodology.

B

Biomarker: A physiologic, pathologic, or anatomic patient characteristic that is measured by an automated process or algorithm as an indicator of normal biologic processes, pathologic processes, or biological responses to a therapeutic intervention (source FDA).

C

Caregiver-administered: A caregiver asks the patient questions and completes the instrument from the patient’s perspective.

Centralized procedure: Applications are made directly to the European Medicines Agency (EMA) and lead to the grant of a European marketing authorization by the Commission. This procedure was established in 1995.

CHMP: Committee of Human Medicine Products.

Clinician-reported outcome (ClinRO): A ClinRO is based on a report that comes from a trained health-care professional after observation of a patient's health condition. A ClinRO measure involves a clinical judgment or interpretation of the observable signs, behaviors, or other physical manifestations thought to be related to a disease or condition. ClinRO measures cannot directly assess symptoms that are known only to the patient (e.g., pain intensity) (source FDA).

Clinician-rated: The instrument is completed by a clinician from his/her observation of the patient.

COA: Clinical outcome assessment. A COA is any assessment that may be influenced by human choices, judgment, or motivation and may support either direct or indirect evidence of treatment benefit. Unlike biomarkers that rely completely on an automated process or algorithm, COAs depend on the implementation, interpretation, and reporting from a patient, a clinician, or an observer. The four types of COAs are patient-reported outcome (PRO) measures, clinician-reported outcome (ClinRO) measures, observer-reported outcome (ObsRO) measures, and performance outcome (PerfO) measures (source FDA).

Composite measures: A composite measure includes at least one COA.

Computer administered: The instrument is completed on a computer.

Concept of interest: The thing measured by an assessment (e.g., pain intensity) (source FDA)
Health-Related Quality of life (HRQL) (incl. health status); Health-Related Quality of end of life; Patient satisfaction; Physical functioning; Psychological functioning (incl. coping); Signs and symptoms; Social functioning (incl. work); Utility.
The way an assessment will be used as a study result and statistically compared among treatment groups to assess the effect of treatment. Endpoints are often named by the assessment measured, but a complete statement of the endpoint should include a full description of what data are collected, and how they are analyzed to support a specific study objective (source FDA).

E

EPAR: European Public Assessment Report.

e-version: The instrument is completed on an electronic device. The data is compiled automatically.

F

FDA: Food and Drug Administration.

H

Health-related quality of life (HRQL): HRQL is a multi-domain concept that represents the patient’s general perception of the effect of illness and treatment on physical, psychological, and social aspects of life. Claiming a statistical and meaningful improvement in HRQL implies: (1) that all HRQL domains that are important to interpreting change in how the clinical trial’s population feels or functions as a result of the targeted disease and its treatment were measured; (2) that a general improvement was demonstrated; and (3) that no decrement was demonstrated in any domain (source FDA).

Health-Related Quality of end of life: Same as HRQL at the end of life.

I

INN: An International Nonproprietary Name (INN) identifies a pharmaceutical substance or active pharmaceutical ingredient by a unique name that is globally recognized and is public property. A nonproprietary name is also known as a generic name (Source World Health Organization).

Instrument: A means to capture data (e.g., questionnaire, diary) plus all the information and documentation that supports its use. Generally, that includes clearly defined methods and instructions for administration or responding, a standard format for data collection, and well-documented methods for scoring, analysis, and interpretation of results (source FDA).

Interviewer administered: An interviewer asks the patient questions and completes the instrument from the patient’s perspective.

Item: An individual question, statement, or task that is evaluated by the patient, clinician or caregiver to address a particular concept.

IVR version (IVR: Interactive Voice Response System): The subject completes the instrument using a software application which enables users to enter data on a telephone keypad or to input information by voice.

M

MAH: the Marketing Authorization Holder.

MeSH classification: the Medical Subject Headings (MeSH®) thesaurus is a controlled vocabulary produced by the U.S. National Library of Medicine and used for indexing, cataloging, and searching for biomedical and health-related information and documents.
Mutual recognition means that EU countries may approve the decision made about a medicinal product by another EU country. The pharmaceutical company submits its application to the country chosen to carry out the investigative works, which then approves or rejects the application. The other countries have to decide within 90 days whether they approve or reject the decision made by the original country. If the countries cannot agree, the EMA and the CHMP are responsible for making the decision. The formal decision is taken by the Commission.

O

Observer-reported outcome (ObsRO): An ObsRO is a measurement based on an observation by someone other than the patient or a health professional. This may be a parent, spouse, or other non-clinical caregiver who is in a position to regularly observe and report on a specific aspect of the patient’s health. An ObsRO measure does not include medical judgment or interpretation. Generally, ObsROs are reported by a parent, caregiver, or someone who observes the patient in daily life. For patients who cannot respond for themselves (e.g., infants or cognitively impaired), we encourage observer reports that include only those events or behaviors that can be observed. As an example, observers cannot validly report an infant’s pain intensity (a symptom) but can report infant behavior thought to be caused by pain (e.g., crying). For example, in the assessment of a child’s functioning in the classroom, the teacher is the most appropriate observer. Examples of ObsROs include a parent report of a child’s vomiting episodes or a report of wincing thought to be the result of pain in patients who are unable to report for themselves (source FDA).

Outcomes: The benefits or harms to a patient who receives an intervention.

P

Paper and pen: Data collection method is paper based.

Patient satisfaction: Evaluation of treatments, patients preference, health care delivery systems and professionals, patient education programs and medical devices.

Patient-reported outcome (PRO): A PRO is a measurement based on a report that comes from the patient (i.e., study subject) about the status of a patient’s health condition without amendment or interpretation of the patient’s report by a clinician or anyone else. A PRO can be measured by self-report or by interview, provided that the interviewer records only the patient’s response. Symptoms or other unobservable concepts known only to the patient (e.g., pain severity or nausea) can only be measured by PRO measures. PROs can also assess the patient perspective on functioning or activities that may also be observable by others (source FDA).

Performance outcome (PerfO): A PerfO is a measurement based on a task(s) performed by a patient according to instructions that is administered by a health care professional. Performance outcomes require patient cooperation and motivation. These include measures of gait speed (e.g., timed 25 foot walk test), memory recall, or other cognitive testing (e.g., digit symbol substitution test) (source FDA).

Physical functioning: Physical limitations and activity restrictions, e.g., self-care, walking, mobility, sleep, sexual, disability.

Proxy administered: A proxy (closed person of the patient) asks the patient questions and completes the instrument from the patient’s perspective.

Proxy rated: The instrument is completed by a proxy (closed person of the patient) from his/her observation of the patient.

Psychological functioning (incl. coping): Positive or negative affect and cognitive, e.g. anger, alertness, self-esteem, sense of wellbeing, distress, coping.

Q

Questionnaire: A set of items shown to a respondent in order to get answers for research purposes (source FDA).

R

Response: Prespecified change in the measure corresponding to a treatment benefit (source FDA).

S

Scale: The system of numbers or verbal anchors by which a value or score is derived. Examples include visual analogue scales, Likert scales, and rating scales (source FDA).

Self-administered: The instrument is completed by the patient himself.

Sign: Any evidence of a disease, health condition, or treatment-related effect that can be observed. Signs can be assessed using PRO, clinician-reported (ClinRO), observer-reported (ObsRO), or performance outcome (PerfO) assessments, as appropriate (source FDA).

Symptom: Any subjective evidence of a disease, health condition, or treatment-related effect that can be noticed and known only by the patient (source FDA).

Signs and symptoms: Any evidence of a disease, health condition, or treatment related effect that can be observed (signs) or that can be noticed and known only by the patient (symptoms).

Social functioning (incl. work): Limitations in work or school, participation in community.

T

Telephone administered: During a telephone interview an interviewer asks the patient questions and completes the instrument from the patient’s perspective.

Therapeutic area: The PROQOLID list of therapeutic areas is in accordance with an established classification system well known to scientists (MeSH: Medical Subject Headings of the National Library of Medicine). However some conditions such as “menopause” have been added in relation to a therapeutic area so that they can be searched through the advanced search engine.

  • Bacterial infections and mycoses: Infections caused by bacteria and fungi, general, specified, or unspecified.
  • Cardiovascular diseases: Pathological conditions involving the cardiovascular system including the heart, blood vessels, or the pericardium.
  • Congenital, hereditary and neonatal diseases and abnormalities: Diseases existing at birth and often before birth, or that develop during the first month of life (infant or newborn diseases), regardless of causation. Of these diseases, those characterized by structural deformities are termed congenital abnormalities.
  • Digestive system diseases: Diseases in any part of the gastrointestinal tract or the accessory organs (e.g., liver, biliary tract, pancreas).
  • Disorders of environmental origin: Disorders representing collectively the results of assault by external forces, rather than by organic or physiologic dysfunction or by pathogens (e.g., substance related disorders, wounds, and injuries).
  • Endocrine system diseases: Pathological processes of the endocrine glands and diseases resulting from abnormal level of available hormones.
  • Eye diseases: Diseases pertaining to the eye.
  • Female genital diseases and pregnancy complications: Pathological processes of the female reproductive system (genitalia, female), and disorders related to pregnancy. Female sexuality and menopause are included.
  • Generic: Therapeutic are linked to COAs that do not involve an actual pathology of the patient.
  • Hemic and Lymphatic diseases: Hematologic diseases and diseases of the lymphatic system collectively. Hematic diseases include disorders involving the formed elements (e.g., erythrocyte aggregation, intravascular) and chemical components (e.g., blood protein disorders) as well as lymphatic diseases that include disorders relating to lymph, lymph nodes, and lymphocytes.
  • Immune system diseases: Disorders caused by abnormal or absent immunologic mechanisms, whether humoral, cellmediated, or both Male Genital diseases Pathological processes involving the male reproductive tract (genitalia, male). Male sexuality is included.
  • Musculoskeletal diseases: Diseases of the muscles and their associated ligaments and other connective tissue and of the bones and cartilage viewed collectively.
  • Neoplasms: New abnormal growth of tissue. Malignant neoplasms show a greater degree of anaplasia and have the properties of invasion and metastasis, compared to benign neoplasms.
  • Nervous System diseases: Diseases of the central and peripheral nervous system. This includes disorders of the brain, spinal cord, cranial nerves, peripheral nerves, nerve roots, autonomic nervous system, neuromuscular junction, and muscles.
  • Nutritional and metabolic diseases: A collective term for nutritional disorders resulting from poor absorption or nutritional imbalance, and metabolic disorders resulting from defects in biosynthesis (anabolism) or breakdown (catabolism) of endogenous substances.
  • Otorhinolaryngologic diseases: Pathological processes of the ear, the nose, and the throat, also known as the ENT diseases.
  • Parasitic diseases: Infections or infestations with parasitic organisms. They are often contracted through contact with an intermediate vector, but may occur as the result of direct exposure.
  • Pathological conditions signs and symptoms: Abnormal anatomical or physiological conditions and objective or subjective manifestations of disease, not classified as disease or syndrome (e.g., inflammation, alopecia, fatigue, pain).
  • Psychiatry/Psychology:
    • Psychiatry: the medical science that deals with the origin, diagnosis, prevention, and treatment of mental disorders;
    • Psychology: the science dealing with the study of mental processes and behavior.
  • Respiratory tract diseases: Diseases pertaining to the respiratory system.
  • Skin and connective tissue diseases: A collective term for diseases of the skin and its appendages and of connective tissue.
  • Stomatognathic diseases: General or unspecified diseases of the stomatognathic system, comprising the mouth, teeth, jaws, and pharynx Surgical procedures, operative. Operations carried out for the correction of deformities and defects, repair of injuries, and the diagnosis and cure of certain diseases.
  • Urologic diseases: Pathological processes of the urinary tract in both males and females.
  • Virus diseases: A general term for diseases produced by viruses.

V

VAS: Visual Analog Scale.