The process of looking for patterns in information to identify cause and effect and/or answer specific questions, such as whether it works and what the risks are. The information is usually collected in research. HTAI
A multi-criteria decision analytic method used to elicit patients’ preferences for specific treatment characteristics or outcomes being assessed in, for example, an HTA. Danner & Gerber-Grote
An alliance of more than 50 clinical trials networks, coordinating centres and clinical quality registries that works together to promote effective and cost‐effective healthcare in Australia through investigator‐initiated clinical trials and clinical quality registries that generate evidence to support decisions made by health practitioners, policy‐makers, and consumers.
A publicly available report about the evaluation of a prescription medicine and the considerations that led the TGA to approve or not approve an application.
Occurs when a factor influences a research study’s outcomes or interpretation of outcomes. There are many types of bias. Some are by accident, some are on purpose, some are caused by the research and some are caused by other factors. Sampling bias occurs when the people or entities chosen to represent the entire population are not representative. For example, a study that enrols patients on a first‐come, first‐served basis may be biased towards those who are already receiving better health care. Publication bias occurs because journals are more likely to publish only the results of studies that show a benefit. Studies that show no effect are often not published, even though their findings are just as important. HTAI
A study to determine whether a treatment is safe and effective. It is carried out with a sample of intended patients, usually after laboratory studies and studies with healthy volunteers have been conducted. The trial is set up to answer one or more questions. For example, does the treatment cause adverse effects and, if so, how serious are these? Does the treatment result in desired outcomes for patients, and if so, how much improvement takes place? What is the safest dose to avoid serious adverse effects while still achieving the desired outcomes? HTAI
Groups of practicing clinician researchers (often several 100 per network) that come together to identify important clinical questions and design large multi-centre clinical trials to answer them. Some also conduct trials with industry but the majority have a strong focus on investigator-initiated trials that can provide unbiased, high-quality scientific evidence of the effectiveness or cost effectiveness of interventions. ACTA
A benefit from treatment that relates to an important outcome, such as length of life, and is large enough to have practical importance to patients and health professionals. Effects that are identified as statistically significant are not always clinically significant, because the effect is small or on an outcome that is unimportant.
For example, a treatment may improve blood flow but for that condition, there is no evidence that this leads to an important clinical outcome, such as lower risk of blood clots or heart attack. HTAI
The medicine or treatment currently being used which the new medicine or treatment is being compared to in an assessment. If the new medicine or treatment is recommended for the PBS the comparator will usually be replaced by the new treatment.
There is always some uncertainty in research. This is because a small group of patients (called the sample) is studied to predict the effects in the wider population who may eventually use the treatment. The confidence interval (CI) shows the amount of uncertainty. It gives a range of results from the study that is likely to include the ‘true’ value for the population. The CI is usually stated as ‘95% CI’, which means that the range of values have a 95 in 100 chance of including the ‘true’ value. For example, a study may state that ‘based on our sample findings, we are 95% certain that the ‘true’ population blood pressure is not higher than 150 and not lower than 110; thus 95% CI is 110 to 150’. HTAI
The unit established by the Department of Health in 2019 to develop structured projects to engage consumer and patient groups in HTA bodies like PBAC, MSAC and PLAC.
The national peak body representing the interests of Australian healthcare consumers. CHF works to achieve safe, quality, timely healthcare for all Australians, supported by accessible health information and systems.
This analysis is a method of considering the advantages and disadvantages of alternative health care technologies. The scope of the advantages and disadvantages considered in an analysis depends on the perspective taken. Cost‐benefit analysis differs from other forms of economic analysis, like cost‐effectiveness analysis, mainly in putting monetary values on outcomes.
For example, the costs of an insulin injection may include the costs of the drug, the needle, the nursing time, the monitoring tests and the patient’s time. The outcomes (both positive and negative) are also given in terms of money. For example, one outcome may be the savings in potential costs to manage severe diabetes, including kidney failure, circulation and cardiovascular complications, foot problems, and time in hospital. The outcomes might also include the patient’s contribution at work, lack of social welfare costs, and increased cost of healthy foods.
The more challenging part of cost‐benefit analysis is that it assigns money values to outcomes such as better health or improved access. For example, the outcomes might include a monetary value of the expected health gain measured though willingness to pay. The costs and benefits of a comparator treatment are also worked out. For example, the cost of insulin taken by mouth includes the higher cost of the drug, but no cost for needles, and increased cost for more monitoring tests, but no cost for nursing time to make sure patients take it as prescribed. The outcomes of the treatment being compared are expressed as money. For example, insulin taken by mouth saves the cost of being in hospital and long‐term organ failure because more patients take it as prescribed. Also included in the outcomes may be the cost of drugs to treat adverse effects such as stomach problems.
The difference in costs and the difference in benefits of the two treatments can be directly compared. For example, the total cost of insulin taken by mouth may be more than the total cost of insulin given by needle, but the total savings due to increased benefits may result in total lower costs to the system. HTAI
This analysis compares two or more drugs, devices, tests, or procedures to find out which provides more outcomes for the cost of treatment or which has the lowest cost for a given outcome. This means that the outcomes of all treatments being compared must be measured in the same units.
For example, Drug A for epilepsy results in 90 days without seizure. Drug B costs twice as much, but increases the number of days without seizure to 240. So Drug B gives better outcomes for the money spent as 240 (the outcome) divided by two (the cost) equals 120, which is more than Drug A’s 90 days. Examples of other uniform outcome measures are reduced blood sugar levels, days without cancer symptoms progressing, and years of survival. The more specific an outcome measure the less useful it is in making comparisons between technologies. For example, a measure in terms of cancer symptoms will be less useful in comparing cancer drugs with drugs for multiple sclerosis, so there is a preference for common measures such as life years gained (or quality‐adjusted life‐years gained). HTAI
A study similar to a cost‐effectiveness analysis. The costs are measured in units of money and the benefits are stated in a value that reflects patient preferences (known as utilities), such as a quality‐adjusted life year. HTAI
A process to find valid and relevant evidence or methods in a systematic review or HTA. Evidence is considered using a system of agreed rules to check its quality and decide if it should be included in the HTA or not.
For example, evidence from a particular study may not be included because it is an uncontrolled study or uses a different form of treatment from that studied in the HTA. HTAI
The government department responsible for delivering health care. In Australia there are departments of health at the state level and one Department of Health at the federal level, called the Australian Government Department of Health. The Australian Government Department of Health is responsible for the PBS and Medicare.
A physical item or artificial body part (called a prosthesis) used to treat a disease or condition or diagnose it. For example, a device might be a pacemaker, knee replacement, xray or blood pressure kit (but not a drug). HTAI
In HTA, an economic analysis is an assessment that compares the costs and benefits of using different tests or treatments for the same condition. Sometimes called an economic evaluation. HTAI
Responsible for protecting public health in the United States of America by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices; and by ensuring the safety of our nation's food supply, cosmetics, and products that emit radiation. FDA
In HTA, a gold standard is a method, procedure or measure that is widely agreed among the medical profession to be the best available to test for or to treat a disease. New tests or treatments are often compared against the gold standard. HTAI
The level of health of a person or group of people that is measured either by the person or people themselves and/or by scientific means. The level is usually based on the patients’ ability to carry out everyday activities such as dress and feed themselves or freedom from pain. For example, the status may be measured according to whether a person can walk by themselves, walk with a stick, needs a wheelchair or is bedridden. HTAI
Any form of intervention to improve health, such as drugs, devices, medical equipment and procedures relating to health care and its services, including prevention, diagnosis and treatment of a condition. HTAI
The systematic evaluation of the clinical effectiveness and/or cost effectiveness and/or the social and ethical impact of a health technology on the lives of patients and the health care system. Its main purpose is to inform health care policy makers. The process advises whether a health technology should be used, and if so, how it is best used and which patients will benefit most from it. Assessments vary, but most look at the health benefits and risks of using the technology. They also look at costs and any wider impacts that the technology might have on a population or on society. HTAI
In Australia, PBAC, MSAC, PLAC and hospitals do health technology assessments.
The number of years spent in good health that a patient would see as equal to the actual number of years they spend in ill health. For example, if someone spent 10 years ill, they may see it as equal to five years spent healthy. HTAI
A medicine that is listed in the PBS to treat chronic conditions but to which only public and private hospitals with appropriate specialist facilities are allowed access because of its clinical use or other specialised features. Funding is provided under the HSD Program within s. 100 of the National Health Act 1953. CoA
The committee – made up of the consumer representatives from PBAC, MSAC, PLAC and their subcommittees – helps the Department of Health work more closely with consumers and communities in HTA decision making; brings consumer and community evidence and views into HTA processes; informs policy on consumer and patient matters in HTA of significance to Australian consumers and community; creates opportunities to promote greater public understanding of HTA processes and enhances methods for formal patient inputs.
A set of conditions that must be met for a person to take part in a clinical trial, such as gender, age and type or stage of disease, as well as medical history. It may also be a set of rules to decide if evidence is included in a systematic review or HTA. HTAI
A ratio that shows the extra cost of a more expensive test or treatment, compared with a cheaper treatment or no treatment, divided by the difference in health outcome. HTAI
A principle of analysis that includes data from all participants allocated to a specified clinical management group as representing that group irrespective of whether they received or completed the prescribed regimen, or whether they were followed for the full duration of the trial or study. This involves following up participants to contribute data and/or prespecifying procedures to deal with missing data. CoA
A procedure, such as treatment with medicine drug, surgery, behaviour change (such as diet or exercise), psychotherapy (such as counselling), early detection (such as screening) or use of patient educational materials. HTAI
The program through which the Australian Government provides fully subsidised access for eligible patients with rare and life-threatening diseases to essential medicines (currently 16 medicines eligible patients with one of 10 conditions).
A summary of the information published in books, journals, etc (the literature) on a topic. A literature review may be a general overview and interpretation of the research, or a more formal review (such as a systematic review) of all published studies on a specific topic. HTAI
An independent HTA advisory committee of the Australian Government that primarily advises the health minister on whether it supports the public funding of proposed health technologies and other medical services. CoA
Under the authority of the Health Insurance Act 1973, a listing and description of the professional services for which a Medicare benefit is payable by the Australian Government, the amount of a patient’s cost that is met through a government rebate, and any conditions applying to the use of that service. CoA
The representative body for the pharmaceutical industry in Australia, responsible for the Code of Conduct which sets the standard for the ethical marketing and promotion of prescription medicines.
Cooperative endeavour to bring about better health outcomes for all Australians, focusing especially on people’s access to, and wise use of, medicines. The term “medicine” includes prescription and non-prescription medicines, including complementary healthcare products. DoH
Information provided by patients, their representative groups or caregivers. It can be written or verbal and is based on knowledge gained from living with a condition. It is not mediated by researchers and aims to aid value judgements and add value to decision-making in an assessment like PBAC
In HTA the term is often used for two different activities which complement each other: (1) patient participation (two way communication with patients including patient input such as taking Consumer Comments and enabling patients to take part in HTAs, to enable committees and patients to learn from each other and solve problems before, during and after an HTA); (2) research into patient aspects (patient-based evidence from primary or secondary research into patients’ experience, preferences and perspectives using robust, scientific methodology).
A measurement based on a report that comes directly from the patient (i.e., study subject) about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else. PROM is the instrument or tool, typically a questionnaire or diary, used to gather the health status of the patient. FDA
An incorporated association which brings together patients, patient groups, researchers and industry to promote and improve the use of the patient voice in healthcare decision making in Australia.
Evidence from research into patients’ experiences, preferences and perspectives, conducted using robust scientific methodology and able to be critically assessed like other scientific evidence.
An independent HTA advisory committee of the Australian Government that primarily makes recommendations to the health minister on the listing of medicines in the PBS. PBS
Under the authority of the National Health Act 1953, a listing and description of the medicines that are subsidised by the Australian Government, the amount of that subsidy and any conditions applying to the use of that medicine. PBS
Different phases of clinical trials that are run to develop a new test or treatment, such as a drug. Phase I (one) involves using healthy human volunteers to check the safety of the test or treatment. In phases II–IV (two to four), patients with the disease that the researchers are interested in are given the treatment and the optimal dose is worked out. Researchers study these patients to see whether it works, how long the effects last and whether there are any adverse effects. HTAI
A group of people with a common link, such as the same medical condition or living in the same area or sharing the same characteristics. The population for a clinical trial is all the people whom the test or treatment is designed to help (such as adults with diabetes, women at high risk of breast cancer). The group taking part in a clinical trial need to be typical of the whole population of interest. HTAI
How common a disease or condition is within a population either at a point in time or over a given period of time (it includes new and existing cases). For example, in 2007, the prevalence of diabetes in Scottish heath boards varied from 3.7% to 4.6%. HTAI
The result(s) of most interest to the researchers. A test or treatment can give results for several outcomes, but primary outcomes are of greatest importance when assessing the outcome. HTAI
The likelihood that an event will occur. In statistics, the probability (or P‐value) shows the likelihood that a research result could have occurred by chance alone.
For example, a P‐value of 0.05 means there is a five in 100 chance that the effect observed in the trial could have been due to chance. Results with P‐values of 0.05 or less are usually considered to be a reliable indication of an effect in the wider population and are called statistically significant. HTAI
An independent HTA advisory committee of the Australian Government that primarily makes recommendations to the health minister on appropriate listing of, and benefits for, prostheses in the Prostheses List. CoA
A method used to bring together multiple studies into patients attitudes, belief and feelings about a treatment or condition to provide decision-makers with a diverse range of experiences found in robust research. Also known as qualitative systematic review.
The act of exploring and understanding people’s beliefs, experiences, attitudes or behaviours. It asks questions about how and why. Qualitative researchers use methods like focus groups and interviews.8
For example, in a qualitative research study, a researcher might ask people why they want to stop smoking, rather than asking how many people have tried to stop. HTAI
A measure of the state of health of a person or group of people in which the benefits, in terms of length of life, are adjusted to reflect the quality of life. That is, a year of active normal life gained as the result of a treatment is rated higher than a year of living with reduced quality (such as being in extreme pain or being in hospital).
It is often measured in terms of the level of a person’s ability to perform activities of daily living, their freedom from pain and mental disturbance. The patients, or observers with knowledge in the area, rate these various states by giving them scores. HTAI
Researchers collect data in the form of numbers, that is, they measure things or count things. Quantitative research might ask a question like how many people visit their GP each year, or what proportion of children have had a particular vaccine, or whether a new drug lowers blood pressure more than the drugs that are usually used. Quantitative researchers use methods like surveys and clinical trials.
A study in which the people taking part are assigned by chance (randomisation) into groups (such as the control group or the study group). The groups are managed in exactly the same way except they are given different treatments, or exposed to a risk factor of interest. Outcomes are measured at specific time points and any difference in response between the groups is assessed statistically. This method is used to reduce bias. HTAI
The data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. RWD can come from a number of sources, for example: electronic health records, claims and billing activities, product and disease registries, patient-generated data including in home-use settings, and data gathered from other sources that can inform on health status, such as mobile devices. FDA
The clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real world data. Real world evidence can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective). FDA
A social enterprise to to speed up access to better treatments by making sure people are empowered with a knowledge of clinical trials and how to get involved, and are not alone in their journey to learn about, take part in, and partner with researchers to make trials a better experience, accessible, faster and more relevant.
The probability of an event is the risk of it occurring. Another meaning of risk is the chance that a test or treatment will cause injury or harm. Risks are a product of the effect of a hazard and the level of exposure. HTAI
An arrangement agreed between the supplier of a PBS-listed medicine and the Australian Government that adequately monitors identified risks (or undesired events such as cost-ineffective use or greater-than-expected use) and manages them by appropriate mechanisms for sharing the impact of these risks between the supplier and the government should they arise. CoA
The organisation that makes a submission to PBAC or MSAC or PLAC to have a health technology (such as a medicine or treatment) assessed. It is usually the manufacturer, but patient groups can be sponsors.
The probability of observing a treatment effect as large as that seen in the sample (such as in randomised controlled trial), when there is no treatment effect in the wider population, is less than 0.05. HTAI
Work that aims to bring together the results of all studies that address a particular research question. They provide a comprehensive and unbiased summary of the research.
For example, one clinical trial may not give a clear answer about the effectiveness of a treatment. This may be because the difference between the treatments being tested was very small, or because only a small number of people took part in the trial. So systematic reviews are used to bring the results of a number of similar trials together, to piece together and assess the quality of all the evidence. Combining the results from a number of trials (using meta‐analysis) may give a clearer picture. HTAI
Part of the Australian Government Department of Health responsible for regulating therapeutic goods including prescription medicines, vaccines, sunscreens, vitamins and minerals, medical devices, blood and blood products.
A measure of how desirable an outcome is, generally expressed as a number between zero and one. For example, a full healthy life would be given a value of one, whereas death is given a value of zero. Utility can also mean a patient’s preferred outcome. HTAI
In a study, validity is the degree to which the conclusions that the researchers make can be considered to be ‘true’, based on how well the study was designed and how well the study matched ‘real life’ situations.
External validity is the extent to which the cause‐and‐effect relationships in a study are true for a wider population beyond the study. For example, the external validity of the study may be questioned if the population is people in Australia and the study was in Spain, or for old people if the study was in young people.
Internal validity is the extent to which the cause‐and‐effect relationships in a study are true for the people and conditions of the study. HTAI
The maximum amount of money that an individual is prepared to give up to ensure that a proposed beneficial change occurs. A beneficial change could include an improved health outcome or ensuring that the proposed health technology is substituted for its main comparator based on valuing the resulting difference(s) in outcomes. CoA
Definitions used with the thanks to the following sources
ACTA – Australian Clinical Trials Alliance. About ACTA: What is a Clinical Trials Network?
CoA – Commonwealth of Australia (2013) Glossary: Key terms for preparing submissions to a health technology assessment (HTA) advisory committee for funding of a medicine, medical service or prosthesis (Version 1).
FDA – US Food and Drug Administration (2019) Real World Evidence.
Danner & Gerber-Grote – Danner, A & Gerber-Grote A (2017). Analytic Hierarchy Process. In Patient involvement in health technology assessment. (Eds Facey KM, Hansen HP, Single ANV). Springer Nature, Singapore.
HTAi – Health Technology Assessment international (2009) HTAi consumer and patient glossary: A beginner’s guide to words used in health technology assessment.