The probability of an event or outcome occurring in a defined population over a specified time period
Glossary
Absolute risk reduction
A measure of treatment effect that compares the decrease in risk in the control group with that of a treatment group i.e. Pc – Pt
Adverse event
An undesirable effect of a health technology
Base Case
Analysis conducted using the set of preferred methods most appropriate for the decision-maker
Bayesian Method
A branch of statistics that uses prior information based on subjective opinion and objective evidence, such as the results of previous research, for estimation and inference
Bias
Bias is any distorting influence that makes the results of a study an inaccurate reflection of the true situation
Birch bark extract (Filsuvez®). HTA ID: 23069
| Assessment Status | NCPE Assessment Process Complete |
| HTA ID | 23069 |
| Drug | Birch bark extract |
| Brand | Filsuvez® |
| Indication | Treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in patients 6 months and older. |
| Assessment Process | |
| Rapid review commissioned | 10/11/2023 |
| Rapid review completed | 08/12/2023 |
| Rapid review outcome | A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of birch bark extract compared with the current standard of care. |
| Full pharmacoeconomic assessment commissioned by HSE | 18/12/2023 |
| Pre-submission consultation with Applicant | 23/04/2024 |
| Full submission received from Applicant | 15/10/2024 |
| Preliminary review sent to Applicant | 27/02/2025 |
| NCPE assessment re-commenced | 26/03/2025 |
| Factual accuracy sent to Applicant | 31/03/2025 |
| NCPE assessment re-commenced | 07/04/2025 |
| NCPE assessment completed | 14/04/2025 |
| NCPE assessment outcome | The NCPE recommends that birch bark extract (Filsuvez®) be considered for reimbursement if cost-effectiveness is improved*. |
*This recommendation should be considered while also having regards to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.
Blinding
When patients, investigators, researchers and/or assessors are kept unaware of the allocation of treatments (investigational agent or control) to participants in a study
Budget Impact Analysis
Estimates the financial consequences of adoption and diffusion of a new health-care intervention within a specific health-care setting or system context given inevitable resource constraints.
Clinical outcome
An outcome of major clinical importance that is defined on the basis of the disease being studied (e.g. fracture in osteoporosis, relapse rates).
Clinical significance
A conclusion that an intervention has an effect that is of practical meaning to patients and health care providers. Effects identified as statistically significant are not always clinically significant, because the effect is small or the outcome is not important.
Clinical trial
A carefully controlled and monitored research study on human subjects or patients evaluating one or more health interventions. Each trial is designed to answer specific scientific questions.
Comparative effectiveness
The ability of one treatment, relative to another, to demonstrate clinical effectiveness
Comparator
the alternative to which the intervention is compared
Cost
the value of opportunity forgone as a result of engaging resources in an activity
Cost benefit analysis (CBA)
An economic evaluation comparing alternative interventions in which costs and outcomes are quantified using the same monetary units
Cost consequences analysis
An economic evaluation in which the components of costs and consequences (health outcomes, adverse effects etc.) are measured and presented in their natural units, without any attempt to aggregate the outputs
Cost minimisation analysis (CMA)
An economic evaluation comparing the costs of different interventions that are assumed to produce equivalent benefits
Cost of illness analysis
A determination of the economic impact of a disease or health condition, including treatment costs.
Cost per QALY
A measure used in cost-utility-analysis, expressed as monetary cost per unit of outcome
Cost utility analysis (CUA)
An economic evaluation in which costs are measured in monetary units and outcomes are measured in terms of their utility e.g. using the quality adjusted life year (QALY).
Cost-effective
A technology is considered cost effective for a specified indication if the incremental benefits of the technology versus its comparator justify its incremental costs and harms; value for money
Cost-effectiveness acceptability curve (CEAC)
Graphical representation of the probability that an intervention is cost-effective at various willingness-to-pay thresholds
Cost-effectiveness acceptability frontier (CEAF)
The CEAF plots the strategy with the highest expected cost-effectiveness (expected Net Monetary Benefit) as a function of the cost-effectiveness threshold
Cost-effectiveness analysis (CEA)
An economic evaluation in which costs are measured in monetary units and outcomes are measured in nonmonetary units related to health e.g. as life years gained or clinical events avoided
Cost-effectivenss plane
Graph plotting the difference in effect between the technology of interest and the comparator on the horizontal axis, and difference in costs on the vertical access.
Critical appraisal
The process of assessing and interpreting evidence by systematically considering its validity, results and relevance.
Decision analysis
A systematic approach to decision making under uncertainty. This involves the modelling of the sequences or pathways of multiple possible strategies (e.g. of diagnosis and treatment for a particular clinical problem) to determine which is optimal
Decision tree
A graphical framework for representing alternatives for use in decision analysis
Deterministic sensitivity analysis (DSA)
A method of capturing the level of uncertainty in the results of decision analysis which varies variables one at a time (one-way sensitivity analysis) or two or more at a time (multi-way sensitivity analysis).
Diagnosis Related Group (DRG)
A patient classification scheme that provides a clinically meaningful way of relating the types of patients treated in a hospital to the resources required by the hospital
Direct costs
The fixed and variable costs of all resources (goods, services, etc.) consumed in the provision of an intervention as well as any consequences of the intervention such as adverse effects or goods or services induced by the intervention – includes direct medical costs and direct nonmedical costs such as transportation or child care
Direct medical costs
A medical cost that varies directly with the provision of a health care intervention (e.g. physician salaries).
Direct non-medical costs
A non-medical cost associated with provision of medical services (e.g. transportation of a patient to a hospital).
Discount rate
The interest rate used to discount or calculate future costs and benefits so as to arrive at their present values, e.g. 4%
Discounting
the process used in economic analyses to convert future costs or benefits to present values using a discount rate. Discounting costs reflects societal preference for costs to be experienced in the future rather than the present, and for benefits to be realised in the present rather than at a later date
Distribution
A mathematical function describing the variability of a variable.
Dominance
a strategy is dominated if it is less effective and has higher costs than an alternative strategy
Economic evaluation
The comparison of alternative courses of action in terms of their costs and consequences, to inform and support the decision-making process
Economic model
a framework for bringing together different types of data from a range of sources to describe the patient pathway and the choices and consequences for the interventions under study. Modelling may be used to combine different data sets changing the information collected from a clinical trial into a form that can be used, to extrapolate short-term clinical data to longer term, to link intermediate with final endpoints, to generalise from clinical trial settings to routine practice and to estimate the relative effectiveness of technologies where these have not been directly compared in clinical trials
Effectiveness
The evaluation of benefit or risk, in a standard clinical setting, using outcomes measuring issues of importance to patients.
Efficacy
The evaluation of benefit or risk in a defined population in controlled or ideal circumstances e.g. in a laboratory setting, under research conditions
EVPI
Expected value of perfect information: describes the maximum amount a decision maker would be willing to pay in order to obtain perfect information, where perfect information would allow them to make a choice under certainty
Extended dominance
In the comparison of mutually exclusive programmes, the situation where one option has a higher incremental cost effectiveness ratio than a more effective alternative.
Extrapolation
Refers to the application of results to a wider population. Means to infer, predict, extend or project the results beyond what was recorded, observed or experienced
Health outomes
The results or impact of any type of intervention on health, (e.g. a clinical procedure, health policy or program, etc.).
Health technology assessment (HTA)
The systematic evaluation of properties, effects, and/or impacts of health care technology. It may address the direct, intended consequences of technologies as well as their indirect, unintended consequences. Its main purpose is to inform technology-related policymaking in healthcare. HTA is conducted by interdisciplinary groups using explicit analytical frameworks drawing from a variety of methods.
Health-related quality of life (HRQoL, QOL)
Patient outcome measures that include physical, social, and emotional aspects that are relevant and important to an individual’s wellbeing.
Heterogeneity
In meta-analysis heterogeneity refers to variability or differences between studies e.g. due to different study designs, different statistical methods, differences in key characteristics of the participants, interventions or outcome measures
Incremental cost effectiveness ratio (ICER)
The additional cost of an intervention as compared with the comparator, divided by the difference in effect or patient outcome between the interventions, e.g. additional cost per QALY.