In a project like PREFER, a glossary helps researchers by providing a common taxonomy for our day-to-day interactions. We have agreed on definitions for a number of terms that we use in reports and publications. During the course of the project, the glossary is a living document. At the end of PREFER, some of the terms will be used in the recommendations we will publish.
The definitions in the PREFER glossary are written for expert stakeholders (people in academia, industry, regulators and HTA bodies). We have also worked to explain some of these terms in plain language using video. Below you can find some of the terms in both formats.
About the glossary
The glossary is a living document, and these terms should be considered working defintions for PREFER.The terms have been reviewed by one or more of our internal expert review panels for 1) methods/methodologies, 2) regulatory/safety, 3) HTA/payer, and 4) medical (with at least one member representing each core case study disease area: lung cancer, neuromuscular disease and rheumatoid arthritis).
The political process of making a decision about a medicinal product and/or medical device, taking account of assessment information as well as values and other factors. (Source: Stevens A, Milne R (2004). Health technology assessment in England and Wales. Int J Technol Assess Health Care, 20(1), 11—24).
The scientific/technical analytical process of gathering and summarizing information on the relevant aspects of a medicinal product and /or medical device.
A judgment, conclusion or determination reached after consideration. A decision is a response in a situation that is composed of three parts: 1) There is more than one possible course of action in the choice set. 2) The decision maker can form expectations about the outcomes that follow from each course of action. 3) The consequences of the outcomes can be assessed relative to current goals and personal values. (Source: Adapted from definition of decision in The American Heritage Dictionary of the English Language, Collins English Dictionary, Compact Oxford English Dictionary, Merriam-Webster's Online Dictionary, New Oxford American Dictionary. Expanded on by Hastie, R. & Dawes, R.M. Rational Choice in an Uncertain World: The Psychology of Judgment and Decision Making. Second Edition. SAGE Publications, Inc.: 2010./ WP2.3).
An instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including a component part, or accessory which is: 1) recognized in the official National Formulary, or the United States Pharmacopoeia, or any supplement to them, 2) intended for use in the diagnosis of disease or other conditions, or in the cure, mitigation, treatment, or prevention of disease, in man or other animals, or 3) intended to affect the structure or any function of the body of man or other animals, and which does not achieve its primary intended purposes through chemical action within or on the body of man or other animals and which is not dependent upon being metabolized for the achievement of any of its primary intended purposes. (Source: Section 201(h) of the Federal Food Drug & Cosmetic (FD&C) Act).
1) Any substance or combination of substances presented as having properties for treating or preventing disease in human beings; or 2) Any substance or combination of substances which may be used in or administered to human beings either with a view to restoring, correcting or modifying physiological functions by exerting a pharmacological, immunological or metabolic action, or to making a medical diagnosis. (Source: Article 1(2) of Directive 2001/83/EC3 of the European Parliament and of the Council of 6 NOVEMBER 2001 on the Community Code Relating to Medicinal Products for Human Use).
Qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions. (Source: FDA Guidance Patient Preference Information – Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling, 2016).
Patient Relevant Outcome
An outcome that is meaningful to the patient, which can be health or non-health related. Examples may include outcomes related to how a patient feels or functions in daily life, quality of life or utility measures, or economic outcomes. (Adapted from definition of patient relevant outcomes in the Pharmaceutical Benefits Advisory Committee (PBAC) glossary).
Patient Reported Outcome
Any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else. (Source: Guidance for Industry Patient-Reported Outcome Measures - U.S. Department of Health and Human Services, Food and Drug Administration; 2009).
Qualitative Preference (Exploration) Method
Methods that collect descriptive data through participant or phenomenon observation, and examining the subjective experiences and decisions made by participants. (Source: PREFER WP2.4).
Quantitative Preference (Elicitation) Method
Methods collecting quantifiable data that can be reported through statistical inferences or analysis. (Source: PREFER WP2.4).
Unmet Healthcare Need
A condition for which there exists no satisfactory method of diagnosis, prevention, or intervention to improve patient care available in the Community or, even if such methods exists, in relation to which the alternative method concerned will be of major therapeutic advantage to those affected. (Adapted from definition of unmet medical need from the IMI Adapt Smart project).