Glossary
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Adverse event
See adverse drug reaction.
Adverse Drug Reaction (ADR)
A reaction to a medicine which is harmful, or potentially harmful, to a patient. If a reaction happens while a patient is taking a medicine, it may have been caused by the drug, but might have another cause such as the disease being treated. For this reason, they are sometimes called adverse events, with the term ADR being reserved for those which are likely to have been caused by the medicine.
Area Drug and Therapeutics Committees (ADTCs)
Committee responsible for providing advice to NHS Boards on all matters affecting efficacy, safety and economy in the use of medicinal products to help meet the health needs of each local Health Board population.
Association of British Pharmaceutical Industries (ABPI)
The trade association that represents the pharmaceutical industry operating in the UK in a way that assures patients access to the best available medicines by creating a favourable political and economic environment that encourages innovative research and development, and affords fair commercial returns.
Biotechnology Company
A biotechnology company is a company whose products or services primarily use biotechnology methods for their production, design or delivery.
Budget impact
A key factor for NHS boards to be able to plan the introduction of a new medicine is to know its budget impact so that finance officers can make sure the money is available in the budget. This will refer to the financial cost of making the new medicine available, after allowing for financial savings. It might be the impact on the medicines budget only, or the overall NHS budget impact.
Clinical effectiveness
The evaluation of benefit or risk, in a standard clinical setting, using outcomes of importance to the patient.
Clinical judgement
A decision made by a clinician based on their medical experience.
Clinical need
A person’s ability to benefit from a medicine (or other health intervention)
Comparative effectiveness
The ability of one treatment, relative to another, to demonstrate clinical effectiveness
Comparative efficacy
The ability of one treatment, relative to another, to demonstrate efficacy.
Comparative safety
The ability of one treatment, relative to another, to demonstrate safety.
Cost benefit
This is a tricky one, as it is a phrase in common use amongst NHS people but not amongst economists. It seems to have two meanings. First, it can be used interchangeably with cost-effectiveness e.g. “I’ve looked at the cost-benefits of this medicine and they seem favourable.” Secondly, it can mean savings, for example, “There would certainly be cost-benefits in terms of hospital beds freed.” Generally, this phrase should be avoided, however.
Cost effectiveness
The balance of costs and health benefits, a measure of value for money. For example, “The medicine is likely to be cost-effective in patients at high risk of getting the disease but not in the general population.” A statement like this will often be based on cost per QALY data.
Cost-minimisation
When a new medicine is compared to an existing treatment and their benefits are identical then we can compare them on the basis of their cost alone and choose the cheapest (cost-minimisation): easy!
Cost per QALY
A new medicine is compared to the existing treatment. The difference in costs is estimated, including differences in the cost of the medicine, side-effects, savings on future treatment, etc. The benefit can be in terms of living longer or having reduced levels of symptoms and illness (or both). Each year of life when a patient is alive is called a life-year. A weighting factor is applied to them to reflect the patient’s quality-of-life: ideally this estimate would come from questionnaires to patients in the clinical study. So if a patient lives longer, we can measure it in life-years, and if they live a better quality-of-life we measure it by changes in the weighting factor. Bringing it all together we divide the additional costs by the difference in quality-adjusted life-years gained, and we get “the cost per QALY” (which is the less cumbersome name for it).
Cost utility analysis
When a new medicine is compared to an existing treatment this type of economic evaluation compares the additional costs to the health benefits, where the latter is measured in terms of quality-adjusted life-years (QALYs).
Declaration of conflicting interests
A statement of personal and/or professional interests which have the potential to bias an individual when considering a product from a particular company. Examples are share-holding in a pharmaceutical company or working in a department which receives fees from a company.
Double blind study
A study in which neither the patients nor the investigators know which of the treatments being compared are being taken by an individual patient. Studies which are not blinded may bias the results if there is an expectation that one treatment will be better than another.
Economic model/modelling
When a new medicine is compared to an existing treatment the costs and benefits are often estimated with the help of a model. We could just work out the costs and benefits seen in the clinical study but that would not be very helpful, because we might want to know the costs and benefits after the clinical study has ended or we might want to estimate the costs and benefits for a medicine that wasn’t included in a study. A model is a way of structuring all of these pieces of data in a way that helps economists to bring all of these data together, change some the key values to test the effect on the results, to use as a basis for predicting the future, and so on.
Efficacy
The effect that a medicine has on the patient e.g. lowering blood pressure. While it is assumed that the effect will be beneficial to the patient, it cannot automatically be assumed that a medicine which has shown efficacy has shown clinical effectiveness.
End point
A measurement of the effect of a given treatment on a given patient in a study. It should be defined before the start of the study and be designed to provide clinically useful information in order to answer a relevant clinical question about the treatment.
Evidence
Information indicating whether a belief or proposition is true or valid.
Formulations
Material or mixture prepared according to a formula.
Horizon Scanning
The process of identifying new medicines or new uses of existing medicines that are expected to receive marketing authorisation from the Medicines and Healthcare products Regulatory Authority (MHRA) or the European Medicines Agency (EMA) in the near future and estimating their potential impact on patient care.
Incidence
The number of patients who have been newly identified with a disease or condition within a given time period e.g. 100 per 100 000 population per annum is the number presenting with a particular condition within a year.
Information
Facts or knowledge provided or learned as a result of research or study.
Licensed indication
The medical conditions or diseases for which a medicine is licensed (i.e. has a marketing authorisation). Pharmaceutical companies may not promote a medicinal product for conditions for which it is not licensed. Clinicians can use medicines outside the licensed indication if this is clinically justified, but the company is not liable for such use, and the prescriber takes full responsibility.
Marketing
The action or business of promoting and selling products.
Marketing authorisation
Before a medicine can be prescribed or sold in the UK, it must have a marketing authorisation (previously called a product licence) from the Medicines and Healthcare products Regulatory Agency or the European Medicines Agency (EMA) to show that it meets the standards of safety, quality and efficacy in clinical trials.
Medicines Healthcare and products Regulatory Agency/European Medicines Agency
The MHRA and EMA evaluate requests for licences (see licensed indication) for new medicines. Once a licence is granted, companies are free to promote their products to healthcare professionals in the area covered by the licence (United Kingdom or European Union respectively). Licences are based on demonstration by the company of safety, efficacy and quality of the product; efficacy normally being demonstrated in rigorously conducted randomised controlled clinical studies. In approving a medicine, no regard can be paid to its possible cost, nor to whether there is a need for the product at all.
New Drugs Committee (NDC)
A sub-working group which will advise and make recommendations to the SMC on the issues surrounding newly licensed products to the SMC.
NHS Boards
The role of the NHS Boards is to ensure the efficient, effective and accountable governance of the local NHS system. There are 14 NHS Boards in Scotland.
NHSScotland
National Health Service in Scotland.
New indication
When a medicine’s license is updated to cover additional medical conditions, this is called a new indication.
NICE
National Institute for Health and Clinical Excellence. Provides advice to the National Health Service in England on a wide range of topics relevant to health care. Compared with SMC, when NICE reviews medicines, the review is more in-depth, but usually occurs at a considerably later stage.
Pharmacodynamics
The mechanisms by which a medicine exerts its activity on the body.
Pharmacokinetics
The way the body handles a drug as it is taken into the body, circulated in the bloodstream, delivered to the target site and eliminated e.g. in urine. These will influence the strength of the effect of the medicine and how long the effects will last.
Pharmacodynamics and Pharmacokinetics in a nutshell
Pharmacodynamics is what the medicine does to the body.
Pharmacokinetics is what the body does to the medicine.
Pharmacovigilance
Systems and arrangements designed to detect and quantify adverse drug reactions after a medicine has been marketed, to inform prescribers and patients of possible risks and, in some cases, to raise and answer questions about whether a product should stay on the market. For example, the UK system for reporting of adverse drug reactions - the 'Yellow Card' scheme.
Prevalence
The number of patients can be identified with a disease or condition at any given time in a given population. e.g. UK prevalence 2004 = 1000 per 100 000 population is the proportion of people who suffered from a given disease when surveyed in the UK in 2004.
Product License see Marketing Authorisation
Phase I, II, II, IV studies
These are stages through which a medicine must pass before and after it is licensed. They progress from safety testing in animals and healthy volunteers to clinical trials involving patients (Phase III). Phase III trials are the most important in reviewing new medicines, and Phase IV studies gather information after marketing.
Placebo
A substance which has no known medicinal properties. It is usually made up to be identical in appearance to an active medicine for use in double- or single-blinding and/or in placebo-controlled trials.
Placebo-controlled study
A randomised controlled clinical study in which one group of patients receives placebo. The difference in effect between the active treatment and placebo is taken to show the 'true' effect of the treatment after allowing for factors such as chance and natural changes in the patient's condition. However placebo-controlled studies do not provide direct evidence as to how the treatment compares to other treatments in clinical use.
Pharmaceutical companies
Organisations that develop and make medicines.
Postcode prescribing
The availability of certain treatments is dependent on where in Scotland a patient lives.
Primary end-point
An end-point which provides information to help answer the main clinical question which the study was designed to answer.
Randomised controlled clinical studies
Studies which demonstrate the efficacy of a treatment by comparing it with another treatment or the standard treatment in a process in which the clinician cannot choose whether a patient gets the new treatment or its comparator.
Safety
An absence, or relative absence of side-effects which may bring harm to a patient (adverse effects).
Secondary end-point
An end-point which may provide useful information but is less essential to the main purpose of the study than the primary end-point. Secondary end-points are more likely to point the way to further research than to answer a research question on their own.
SIGN
Scottish Intercollegiate Guidelines Network. Provides evidence-based guidance on the diagnosis and management of specific conditions for the NHS in Scotland.
Single-blind study
As double-blind study, but where it is not possible to conceal the treatment from either the patient or the investigator. For example if there is no practical way to use a placebo to conceal treatment from the patient, it can be administered by someone other than the investigator, and the study can still be investigator-blind.
Tolerability
The extent to which adverse effects bring distress to a patient and/or limit daily activity. For a given set of adverse effects, tolerability may be influenced by the characteristics of the patient, such as their ability to tolerate discomfort, and of the disease where the benefits of treatment may offset any discomfort which it causes.
Willingness to pay
Once a cost per QALY gained has been estimated, the question is whether this represents good value. In an ideal world the question would not arise as cost would not be a factor but unfortunately we can’t afford to do everything so we have to make judgments like this. So the way we might use this is to say, “This is good value so long as the NHS is willing-to-pay £30,000 for a QALY.” Imagine you were going to an auction and there was something there you wanted to buy but you set your self a limit in advance of £100; then to an economist £100 is your maximum willingness-to-pay. The challenge for the NHS is to identify its willingness-to-pay for good health.