HEOR & HTA Glossary

A category of medicines based on genes, tissues, or cells, including gene therapies, somatic cell therapies, and tissue-engineered products. ATMPs face distinct HTA challenges due to small patient populations, limited long-term evidence, and one-time administration with uncertain durability of effect.

A financial analysis estimating the total cost to a payer or health system of adopting a new treatment over a defined time horizon, typically three to five years. BIMs complement cost-effectiveness analyses by translating per-patient cost-effectiveness into aggregate budget consequences.

A biological medicine that is highly similar to an already approved reference biologic, with no clinically meaningful differences in safety, purity, or potency. Biosimilars enter the market after the reference product's patent expires and typically at lower prices, creating significant budget impact considerations for payers.

An economic evaluation comparing the costs and health outcomes of two or more interventions, typically expressed as an incremental cost-effectiveness ratio (ICER). CEA is the dominant framework for HTA submissions to bodies such as NICE, where results are assessed against a cost-effectiveness threshold.

Any measure used in clinical trials to assess how a patient feels, functions, or survives. COAs include patient-reported outcomes (PROs), clinician-reported outcomes, observer-reported outcomes, and performance outcomes. Regulatory and HTA bodies increasingly require robust COA evidence to support labelling and reimbursement claims.

A form of cost-effectiveness analysis where health outcomes are measured in quality-adjusted life years (QALYs). CUA is the standard approach required by most European HTA bodies, enabling comparison across disease areas using a common unit of health benefit.

The formal evidence package submitted to an HTA body by a manufacturer seeking reimbursement or market access. A dossier typically includes clinical evidence, economic analyses, budget impact models, and patient-reported outcome data, structured according to the receiving body's template and requirements.

The most widely used preference-based health-related quality of life instrument in HTA, measuring five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Utility values derived from EQ-5D responses are used to calculate QALYs in cost-utility analyses. NICE updated its value set from EQ-5D-3L to EQ-5D-5L in 2026.

The EU agency responsible for the scientific evaluation, supervision, and safety monitoring of medicines in Europe. EMA's Committee for Medicinal Products for Human Use (CHMP) issues opinions on marketing authorisations for centrally approved medicines, which are then adopted by the European Commission.

A multidisciplinary field combining health economics, epidemiology, and outcomes research to generate evidence supporting healthcare decision-making. HEOR encompasses cost-effectiveness modelling, real-world evidence generation, patient-reported outcomes, and budget impact analysis — all central to HTA submissions and market access strategies.

A numerical value between 0 (death) and 1 (perfect health) representing the preference for a particular health state. Utility values are used to weight life years in QALY calculations. They are typically derived from preference-based instruments such as EQ-5D or from direct elicitation methods such as time trade-off or standard gamble.

A systematic evaluation of the clinical, economic, social, and ethical implications of a health technology — typically a medicine, device, or diagnostic. HTA informs coverage and reimbursement decisions by payers and health systems. Major HTA bodies include NICE (UK), HAS (France), G-BA (Germany), and the EU HTA Coordination Group (HTACG).

The EU body responsible for coordinating joint clinical assessments (JCAs) under the EU HTA Regulation (2021/2282). The HTACG comprises representatives from member state HTA bodies and is supported by the European Commission. It oversees the scientific work of joint assessments and determines the scope of JCA reports.

The ratio of the difference in costs between two interventions to the difference in their health outcomes, typically expressed as cost per QALY gained. An ICER is compared against a cost-effectiveness threshold to determine whether a technology represents value for money. NICE's threshold range was raised from £20,000–£30,000 to £25,000–£35,000 per QALY in 2025.

US legislation passed in 2022 that allows Medicare to negotiate drug prices directly with manufacturers for the first time. The IRA has significant implications for global pricing strategies, as US list prices are now subject to mandatory negotiation, with spillover effects on reference pricing in European markets.

A statistical method for comparing treatments that have not been directly compared in head-to-head trials, using a common comparator as a bridge. ITCs form the basis of network meta-analyses and are frequently required in HTA submissions where direct comparative evidence is unavailable.

The EU-level clinical assessment conducted under the EU HTA Regulation (2021/2282), producing a joint report on the relative clinical effectiveness and safety of new medicines across member states. JCAs are mandatory for oncology and ATMP products from 2025, with expansion to other therapeutic areas by 2030. Member states must consider the JCA report in national pricing and reimbursement decisions.

A prospective clinical study jointly commissioned by the EU HTACG to address evidence gaps identified during the JCA process. JCS represents an emerging mechanism for post-authorisation evidence generation at EU level, particularly relevant for ATMPs and rare disease products where trial evidence is limited at launch.

The cross-functional process of securing reimbursement, coverage, and appropriate pricing for a medicine across target markets. Market access strategy integrates regulatory planning, HTA requirements, payer engagement, health economics, and evidence generation to ensure patients can access a product once it receives marketing authorisation.

The smallest change in a patient-reported or clinical outcome measure that patients and clinicians consider meaningful in the context of a given disease. MCID thresholds are critical for interpreting whether statistically significant trial results translate into clinically relevant benefits, and are increasingly scrutinised by HTA bodies assessing PRO data.

A pricing policy requiring that a manufacturer charge no more than the lowest price offered to any other country or payer. MFN clauses create global pricing tensions: a low price negotiated in one market can trigger automatic price reductions in others through international reference pricing linkages.

The UK HTA body responsible for producing guidance on the use of medicines, diagnostics, and medical technologies in the NHS. NICE technology appraisals assess clinical and cost-effectiveness using a QALY-based framework, with outcomes that directly determine NHS commissioning. NICE is widely regarded as one of the most rigorous and influential HTA bodies globally.

A statistical technique that simultaneously compares multiple treatments using both direct (head-to-head) and indirect evidence from a network of trials. NMA is a standard component of HTA submissions, enabling relative effectiveness estimates for treatments that have never been directly compared. The credibility of NMA results depends heavily on the assumption of consistency across the network.

A medicine developed for the treatment of rare diseases affecting fewer than 5 in 10,000 people in the EU. Orphan designation confers regulatory incentives including market exclusivity and fee reductions. HTA assessment of orphan drugs presents particular challenges due to small trial populations, surrogate endpoints, and limited comparator data.

The length of time from treatment initiation or study entry until death from any cause. OS is considered the gold standard endpoint in oncology trials, as it directly measures the most meaningful outcome for patients. HTA bodies increasingly require OS data to support reimbursement, particularly where surrogate endpoints have been used for regulatory approval.

The framework used by HTA bodies to define the scope of a clinical assessment. PICO specifies the patient population of interest, the intervention under review, the relevant comparators, and the outcomes to be assessed. In EU JCA, the PICO is determined collaboratively by member state HTA bodies and significantly shapes the evidence requirements for submissions.

The length of time during and after treatment that a patient lives with the disease without it worsening. PFS is widely used as a surrogate endpoint in oncology trials, enabling earlier regulatory approval. However, HTA bodies often require demonstration of OS benefit or strong evidence of PFS-to-OS correlation before granting full reimbursement.

Any report of a patient's health status coming directly from the patient, without interpretation by a clinician or anyone else. PROs capture symptoms, functioning, and health-related quality of life that may not be observable in clinical assessments. Regulatory agencies and HTA bodies increasingly require robust PRO data to support labelling claims and reimbursement decisions.

A measure of health outcome that combines quantity and quality of life, calculated by multiplying the number of life years gained by a utility value representing health-related quality of life (0 = death, 1 = perfect health). QALYs are the standard outcome metric in cost-utility analyses and are used by HTA bodies such as NICE to assess value for money relative to a cost-effectiveness threshold.

A pricing policy where a country sets or benchmarks the price of a medicine based on prices in a basket of reference countries. IRP creates global pricing interdependencies: a low launch price in one market can automatically trigger price reductions in countries that reference it, incentivising manufacturers to sequence launches strategically.

A decision by a payer or health system to fund the cost of a medicine for eligible patients. Reimbursement decisions are typically made following HTA and price negotiation, and may include conditions such as patient population restrictions, managed access arrangements, or outcome-based agreements.

Clinical evidence derived from real-world data sources outside randomised controlled trials, including electronic health records, claims databases, registries, and patient surveys. RWE is increasingly used to supplement trial data in HTA submissions, particularly for long-term outcomes, safety data, and evidence in subpopulations underrepresented in trials.

A biomarker or intermediate outcome measure used as a substitute for a direct clinical endpoint such as overall survival. Surrogate endpoints enable earlier regulatory approval but are viewed critically by HTA bodies unless their relationship to clinical benefit is well established. The disconnect between regulatory approval on surrogates and HTA requirements for clinical outcomes is a central tension in oncology market access.

A strategic document defining the desired characteristics of a medicine at various stages of development, including target indication, patient population, clinical endpoints, safety profile, and comparator. The TPP guides development decisions and should be informed by HTA requirements to ensure the evidence package generated will support reimbursement in target markets.

A pricing approach where the price of a medicine is linked to the value it delivers to patients and the health system, rather than solely to production costs or market dynamics. In practice, value-based pricing is operationalised through HTA-informed price negotiations and outcomes-based agreements, where reimbursement or rebates are tied to real-world performance.

The maximum amount a payer or health system is willing to pay for a unit of health benefit, typically one QALY. WTP thresholds vary by country and inform cost-effectiveness decisions: a technology with an ICER below the threshold is generally considered cost-effective. NICE's current threshold range is £25,000–£35,000 per QALY.