3. Processes and methods

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1. Processes and methods

1.1. Assessment

HTA processes pertaining to medicines or devices typically begin with a company submitting a dossier of relevant information to an HTA body.

By default, the dossier includes detailed evidence relating to the safety and efficacy of the new technology as well as ‘added therapeutic benefit’, in other words a comparison of the clinical effectiveness of the new product with the existing standard practice (the comparator).

Some HTA systems in Europe also estimate the impact the new product may have on the health system’s budget (a budget impact evaluation) or the effectiveness of the medicine in comparison to its costs to the system (i.e. a cost-effectiveness analysis or economic evaluation). Not all HTA systems in Europe place the same emphasis on comparative cost-effectiveness analysis, but all focus on the added therapeutic benefit.

The most common components of an application dossier or ‘submission’ are listed below: note that some of these components are more quantitative than others. Equity, legal, and public health aspects may be more qualitative and therefore may be included in the appraisal part of HTA rather than the evaluation part.


  • Disease burden: Also known as ‘unmet need’ or ‘therapeutic need’ (while these terms may be used interchangeably, they are often context-related and may carry a different emphasis). It may be a measure of the number of people affected by a particular disease for whom current treatments are inadequate. It may include the number of new diagnoses of a disease, or the costs to society or a government representing those affected. It may also include more qualitative aspects about the burden of disease and current treatments available to patients.

  • Medicine description: A description of the medicine, how it works, method of delivery (e.g. injection, tablet), where it is administered to patients, (e.g. in hospital, community, primary care, at home, self-administered), how often, and it's appropriate use in therapy along with other interventions and medicines.

  • Clinical efficacy: In medicine, clinical efficacy indicates a positive therapeutic effect. If efficacy is established, an intervention is likely to be at least as good as other available interventions to which it will have been compared. When talking in terms of efficacy versus effectiveness, efficacy measures how well a treatment works in clinical trials or laboratory studies. Effectiveness, on the other hand, relates to how well a treatment works in real-world settings.

  • Relative efficacy: The extent to which an intervention does more good than harm under ideal circumstances compared to one or more alternative interventions.

  • Clinical effectiveness: Clinical effectiveness is a measure of how well a particular treatment works in real-world settings. It depends on the application of the best knowledge derived from research, clinical knowledge and experience, and patient preferences.

  • Relative clinical effectiveness: Can be defined as the extent to which an intervention does more good than harm compared to one or more intervention alternatives for achieving the desired results when provided under the usual circumstances of health care practice.

  • Economic evaluation and cost-effectiveness: In the context of pharmacoeconomics, cost effectiveness is studied by looking at the results of different interventions by measuring a single outcome, usually in 'natural' units (for example, life-years gained, deaths avoided, heart attacks avoided, or cases detected). Alternative interventions are then compared in terms of cost per (natural) unit of effectiveness in order to assess how it provides value for money. This helps decision-makers to determine where to allocate limited healthcare resources. Cost effectiveness, however, is only one of a number of criteria that should be used to determine whether or not interventions are made available. Other issues, such as equity, needs, impact on working life, and patient priorities should also be part of the economic evaluation.

  • Budget impact: Costs within a particular timeframe and related to a particular healthcare budget rather than a country’s overall budget. This assumes robust data on epidemiology and treatment patterns, along with assumptions of uptake and displacement of current treatments.

  • Innovative characteristics: An assessment of whether there are advantages to using the medicine beyond the added clinical benefit (such as convenience to patients of, for example, a different mode of delivery, or other characteristics that may improve adherence to therapy, with resulting improvements in clinical outcomes and / or quality of life).

  • Availability of therapeutic alternatives: A description of what else is available to treat the disease. This may or may not be another medicine.

  • Equity considerations: An assessment of how adoption of the new therapy might impact measures of fairness within the health system. For example, will the therapy lead to more benefits for people who are socially or economically disadvantaged?

  • Public health impact: An examination of how the new therapy might have a broader impact on public health. For example, a new therapy to treat HIV/AIDS may reduce the rate of HIV transmission within a community.

Most HTA bodies have developed guidelines for companies in order to make this process consistent and create fair comparisons. They may be available on the websites of most HTA bodies and can help explain how recommendations/decisions about new medicines are made. However, applicants have to be aware that guidelines vary from country to country and take this into consideration.

Dossiers are assessed by HTA bodies either directly or outsourced. Some HTA bodies conduct independent reviews of the clinical and the economic evidence in order to reduce conflicts of interest. Depending on the HTA organisation, a detailed evaluation of the company’s submission will be carried out rather than conducting an independent review.