Dictionary
The Dictionary provides concise definitions of health economics terms, arranged alphabetically for quick reference. Use it to understand unfamiliar terminology or confirm the meaning of a specific term.
B
- BLA SubmissionBiologics License Application, BLA
- The formal Biologics License Application submitted to regulators seeking approval to market a new biological product, detailing safety, efficacy, and manufacturing quality.
C
- Clinical Benefit AssessmentClinical Efficacy Assessment, Therapeutic Benefit Assessment
- A benefit assessment focused on clinical endpoints, such as survival or symptom relief, rather than economic or quality-of-life measures.
- Clinical Effectiveness ReviewClinical Evidence Review, Efficacy Review
- A systematic evaluation of available clinical evidence regarding a technology's efficacy and safety, foundational to a broader assessment.
- Clinical EvidenceClinical Research Evidence, Trial Evidence
- Data derived from clinical research, including trials and observational studies, used to establish an intervention's safety and efficacy or effectiveness.
- Convenience OutcomeAdministration Burden Outcome, Treatment Convenience Measure
- A clinical outcome measure capturing the ease or burden of a treatment's administration, such as dosing frequency, distinct from its clinical efficacy.
D
- Data Safety Monitoring BoardDSMB, Data Monitoring Committee
- An independent group reviewing accumulating safety and efficacy data from an ongoing trial, with authority to recommend stopping, modifying, or continuing it.
- Dose FindingDose-Finding Study, Dose Optimisation
- The overall process of identifying an appropriate dose or regimen for a new treatment, balancing efficacy against tolerability during early development.
- Drug ApprovalRegulatory Approval, Marketing Approval
- The formal regulatory decision authorising a pharmaceutical product for marketing and clinical use, based on review of safety and efficacy evidence.
E
- Economic EvidenceCost-Effectiveness Evidence, Economic Analysis Evidence
- Data and analysis addressing the costs, resource use, and cost-effectiveness of health interventions, distinct from clinical evidence on safety and efficacy alone.
- Effectiveness EvidencePragmatic Evidence, Practice-Based Evidence
- Data demonstrating how well an intervention performs under real-world practice conditions, unlike efficacy evidence from controlled trial conditions.
- Effectiveness ResearchEffectiveness Studies, Real-World Effectiveness Studies
- Research evaluating how treatments perform in routine practice, distinguishing effectiveness observed under real-world conditions from efficacy demonstrated in controlled trials.
- EfficacyIdeal-Condition Benefit, Explanatory-Trial Benefit
- The extent to which an intervention produces a beneficial result under the ideal, controlled conditions of a clinical trial.
- Efficacy EvaluationExplanatory Evaluation, Controlled-Condition Evaluation
- An assessment of a treatment's effect under the controlled, idealised conditions of a clinical trial, estimating its maximum potential benefit.
- Efficacy EvidenceControlled-Trial Evidence, Explanatory Evidence
- Data demonstrating an intervention's effect under the controlled, often idealised conditions of a clinical trial.
- Efficacy OutcomeControlled-Trial Outcome, Explanatory Outcome
- A clinical outcome measured under the controlled, often idealised conditions of a trial, representing the maximum potential benefit achievable.
- Efficacy-Effectiveness GapEfficacy-to-Effectiveness Gap, Translation Gap
- The observed difference between a treatment's trial-demonstrated benefit and its actual benefit once used in routine clinical practice.
F
- FDAUS Food and Drug Administration, Food and Drug Administration
- The US Food and Drug Administration, the federal agency responsible for regulating the safety and efficacy of drugs, biologics, and devices.
- First-Line TreatmentInitial Therapy, Primary Treatment Choice
- The treatment recommended as the initial therapy for a condition, typically selected on the strongest available evidence of efficacy and safety.
I
- Interim AnalysisInterim Look, Mid-Trial Analysis
- A planned analysis of trial data conducted before final completion, typically to assess safety, efficacy, or futility and inform continuation decisions.
M
- Marketing AuthorizationProduct Licence, Market Authorisation Grant
- The formal regulatory approval required before a pharmaceutical product can be legally marketed within a given jurisdiction, based on safety and efficacy review.
- Medical Products BlockMedical Products Pillar, Medicines and Technologies Block
- A core health system building block encompassing access to essential medicines, vaccines, and technologies of assured quality, safety, and efficacy.
- MHRAMedicines and Healthcare products Regulatory Agency, UK Medicines Regulator
- The Medicines and Healthcare products Regulatory Agency, the UK body responsible for the safety, quality, and efficacy of medicines and devices.
- Multiple Sclerosis EconomicsMS Economics, Multiple Sclerosis Cost of Illness
- The application of health economic evaluation to multiple sclerosis, comparing an expanding range of disease-modifying therapies with varying costs and efficacy.
N
- NDA SubmissionNew Drug Application, NDA
- A New Drug Application submitted to the US FDA seeking approval to market a new small-molecule drug, detailing safety, efficacy, and manufacturing.
P
- Paediatric ExclusivityPediatric Exclusivity
- An additional market exclusivity period granted for conducting requested paediatric studies of an already-approved product, incentivising paediatric-specific safety and efficacy data.
- Phase I Design
- The methodological structure of an early-phase trial, typically small and focused on safety, tolerability, and pharmacokinetics rather than efficacy.
- Phase I Trial
- The first stage of human clinical testing for a new treatment, typically small and assessing safety, tolerability, and pharmacokinetics rather than efficacy.
- Phase II Design
- The methodological structure of a mid-stage trial, typically moderate in size, providing preliminary efficacy evidence alongside continued safety assessment.
- Phase II Trial
- A trial after phase I, typically enrolling a moderate number of participants to gather preliminary efficacy evidence while continuing safety monitoring.
- Phase III Design
- The methodological structure of a large-scale, definitive trial confirming efficacy and further characterising safety in a broader population, typically supporting approval.
- Phase III Trial
- A large-scale trial confirming a treatment's efficacy and further characterising its safety in a broad population, typically the primary basis for approval.
- Pivotal Trial
- A trial, typically phase III, whose results form the primary basis for a regulatory approval decision, showing significant and meaningful efficacy.
R
- Real-World Value
- An assessment of a technology's actual benefit and cost-effectiveness observed under routine clinical practice, unlike trial-based efficacy value estimates.
- Recommended Phase 2 Dose
- The dose identified during phase I dose-finding studies as offering the best balance of efficacy and tolerability, carried forward into phase II trials.
- Relative Effectiveness
- A measure comparing how well a treatment performs against an alternative under real-world practice conditions, unlike relative efficacy measured under trial conditions.
S
- Self-Efficacy
- An individual's belief in their own capacity to successfully perform actions needed to manage a specific situation, such as a chronic condition.
- Stopping Rule
- A predefined statistical criterion set before a trial begins specifying the conditions under which it should stop early, for efficacy, futility, or safety.
T
- Targeted Therapy Economics
- The application of health economic evaluation to cancer treatments acting on a specific molecular target, jointly considering efficacy and the companion diagnostic test.
- Treatment Discontinuation
- The cessation of a prescribed treatment before its originally planned completion, whether from adverse effects, lack of efficacy, or patient preference.
V
- Vaccine Effectiveness
- The degree to which a vaccine reduces disease risk under real-world conditions in a general population, unlike efficacy measured under trial conditions.
- Vaccine Efficacy
- The degree to which a vaccine reduces disease risk among vaccinated individuals under the controlled conditions of a clinical trial.