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The capability of a medicine to produce a desired or expected effect in the real world clinical setting. When talking in terms of efficacy vs. effectiveness, effectiveness relates to how well a treatment works in the practice of medicine, as opposed to efficacy, which measures how well a treatment works in clinical trials or laboratory studies.


Efficacy refers to the ability of a medicine to provide a beneficial effect (a positive benefit/risk ratio) when studied in a clinical trial. When talking in terms of efficacy vs. effectiveness, effectiveness relates to how well a treatment works in the real world practice of medicine, as opposed to efficacy, which measures how well a treatment works in clinical trials or laboratory studies.


In the context of health economics, efficiency of a medicine is a measure of its ability to provide a beneficial effect against its costs to individuals or society. The most efficacious treatment may not be the most efficient (cost-effective) option, for example making it unaffordable for the patients or the health system to implement.



Electroconvulsive Therapy

Electroconvulsive Therapy

Elements to Assure Safe Use

Elements to Assure Safe Use


Eligibility in medicines development usually refers to the requirements that participants must meet in order for them to have the possibility of being selected to participate in a clinical trial. The requirements (criteria) will typically contain not only elements which allow participation (inclusion criteria) but also details of what will prevent someone from participating (exclusion criteria).

Endocrine Therapy

Endocrine therapy, or hormone therapy, is a therapy that adds, blocks, or removes hormones in order to treat a disease. For certain conditions (such as diabetes or menopause), hormones are given to adjust low hormone levels. Hormones can also be given to block the production of natural hormones and to slow or stop the growth of certain cancers (such as prostate and breast cancer).


The endpoint of a clinical trial is a pre-defined event: for instance, the occurrence of a disease, the occurrence of a symptom, or a particular laboratory result. Once someone reaches the endpoint, they are generally excluded from further research in the trial.

Endpoints can be hard (objective) or soft (subjective). In some cases they can be replaced by surrogate endpoints. The endpoints used in a trial must be defined and documented as part of the trial protocol.

Epidermal Growth Factor Receptor

Epidermal Growth Factor Receptor


Epigenetics is the study of changes in gene activity (expression) that do not involve changes in the underlying DNA sequence (genotype). Epigenetic changes are a regular and natural occurrence, but they can also be influenced by several factors “ including age, environment/lifestyle, and disease state “ through mechanisms that switch genes on or off.


Epigenomics is the study of the complete set of epigenetic modifications on the genetic material of a cell, known as the epigenome. The epigenome marks the genome in two main ways, both of which play a role in turning genes on or off.

In epigenomics, researchers try to chart the locations and understand the functions of all the chemical tags that mark the genome. Epigenomic maps may someday help doctors make diagnoses and tailor a patient's response to therapies.

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?

Ethical Principles

Ethical principles exist both to protect the research participants and to ensure the integrity of the research. There are various codes and regulations which guide ethical research today, including the Declaration of Helsinki, although a number of common principles exist.

The World Medical Association (WMA) developed the Declaration of Helsinki as a statement of ethical principles specifically for medical research involving human subjects. It emphasises the procedures required to ensure subject safety in clinical trials including informed consent and review by research ethics committees.

Ethical Review Committee

Ethical Review Committee

Ethical, Legal, and Social Implications

Ethical, Legal, and Social Implications

Ethics Committee

An Ethics Committee is an independent body made up of a range of individuals including medical or scientific professionals and non-medical or non-scientific members (e.g. patients or lay members). An Ethics Committee may operate within an institution, or it may be national, or supranational or private.

Ethics committees have a responsibility to ensure the protection of the rights, safety and wellbeing of research participants, as well as assuring the public of that protection. It operates, among other things, expressing an opinion on the clinical trial protocol, the suitability of the investigators involved in the trial, the adequacy of facilities, and on the methods and documents to be used to inform trial participants and obtain their informed consent. A trial should only begin when a favourable opinion by an Ethics Committee has been given.

Ethics Committees may also monitor studies once they have begun and once they are complete.

EU Regulation

A regulation is a written rule or law. European regulations are binding legislative acts. They must be applied in their entirety across the EU simultaneously from the date agreed. Regulations can be distinguished from European directives which are legislative acts that set out a goal that all EU countries must achieve. However, it is up to the individual countries to devise their own laws on how to reach these goals. Directives need to be ˜converted™ into national law before they are enforceable.


The European network for Health Technology Assessment was established to create a network for HTA across Europe, developing a framework (HTA Core Model®) by which a technology (for example a new medicine) can be assessed. It facilitates efficient use of resources, creating a sustainable system of knowledge, and promoting good practice in HTA methods and processes. The network connects public national HTA bodies, research institutions and health ministries, to exchange information and to support policy decisions by member states.


The EuroBioBank network is the first operating network of biobanks in Europe providing human DNA, cell, and tissue samples as a service to the scientific community conducting research on rare diseases. It was established by patients and researchers.

A biobank is a large, organised collection of samples, usually human, used for research. Biobanks catalogue and store samples using genetic, clinical, and other characteristics such as age, gender, blood type, and ethnicity. Some samples are also categorised according to environmental factors, such as whether the donor had been exposed to some substance that can affect health. Biobanks play a crucial role in biomedical research, such as in genomics and personalised medicine. Researchers access biobanks when they need samples with similar characteristics for their research studies.

European Alliance for Personalised Medicine

European Alliance for Personalised Medicine (EAPM)"

European Association for Bioindustries

European Association for Bioindustries (EuropaBio)"

European Bioinformatics Institute

European Bioinformatics Institute (EBI)"

European Clinical Trials Database

European Clinical Trials Database (EudraCT)"

European Databank for Medical Devices

European Databank for Medical Devices

European Directorate for the Quality of Medicines and HealthCare

European Directorate for the Quality of Medicines and HealthCare (EDQM)"

European Economic Area

European Economic Area

European Federation of Pharmaceutical Industries and Associations

European Federation of Pharmaceutical Industries and Associations (EFPIA)"

European Free Trade Association

European Free Trade Association (EFTA)"

European Medicines Agency

European Medicines Agency (EMA) | European Competent Authority."

European Network of Paediatric Research at the European Medicines Agency

European Paediatric Formulation Initiative

European Paediatric Formulation Initiative (EuPFI)"

European Patent Office

European Patent Office

European Patients’ Academy on Therapeutic Innovation

European Patients’ Academy on Therapeutic Innovation (EUPATI)"

European Public Assessment Report

A European Public Assessment Report (EPAR) is an assessment produced for all medicines where marketing authorisation is sought through the centralised procedure at the European Medicines Agency (EMA). It is a series of documents, and includes:

EPARs are published on the EMA’s website once the European Commission has issued a decision regarding a marketing authorisation.

European Union

European Union (EU)"

European Union Clinical Trials Register

European Union Committee of Experts on Rare Diseases

European Union Committee of Experts on Rare Diseases

Evidence-based Medicine

Evidence-based medicine (EBM) applies the scientific method to medical practice, using techniques from science, engineering, and statistics - such as the meta-analysis of scientific literature, benefit-risk analysis, and randomised controlled trials. One of the goals of evidence-based medicine is that healthcare professionals should make 'conscientious, explicit, and judicious use of current best evidence' in their everyday practice.

Excerpta Medica Database

Excerpta Medica Database (EMBASE)"


Pharmaceutical excipients are natural or synthetic substances combined with the active substance in a dosage form. Excipients assist the drug or active substances to be better metabolised or administered. They protect, support or enhance stability or facilitate absorption of the medicine in the body. They may also assist in product identification (e.g. by colour of a tablet/capsule).

Exclusion Criteria

Exclusion criteria are characteristics that exclude people from taking part in a trial.

For example, depending on the requirements of the trial, exclusion criteria might include age, gender, type or stage of disease, and the presence or absence of other medical conditions. For a trial studying an anti-venom (snake bite) medicine, some of the criteria that would exclude an individual from taking part might be:
- Pregnancy
- Aged under 12 or over 70
- Previously received an anti-venom medicine
- Medical history includes wheezing, high blood pressure, heart disease
- Known adverse reaction to adrenaline.

Exclusion criteria (and inclusion criteria) are an important part of a trial protocol. If they are properly defined, exclusion and inclusion criteria will increase the chances of a trial producing reliable results. They also protect participants from harm and help avoid exploitation of vulnerable people (such as those unable to provide informed consent).

The reason for choosing the exclusion criteria should be documented with the trial protocol. Exclusion of certain groups can affect how realistic it is to generalise the trial results to the relevant patient population (external validity). This should be considered by researchers when they are designing a trial, and unnecessary exclusions should be avoided.

Exploratory trials

Exploratory trials are developed to select compounds or targeted medicines or approaches based on human data rather than animal data. They are conducted early in Phase I of the clinical development process, which involve limited human exposure, have no therapeutic intent, and are not intended to define a maximum tolerated dose.


Compounds that can be extracted from plastic or coatings of a container or closure system in direct contact with an active substance or medicinal product. Regulatory guidelines stipulate that an extractable profile should be determined for all materials in contact with the medicine or patient directly to ensure medicine safety.

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