ABLATION: A medical technique whereby a needle, containing a heating device or other element at its end, is placed through the skin into a tumour using imaging guidance. Typically the tumour is then burned off or frozen depending on the particular type of ablation being used. Different types of ablation include: CRYOBLATION, MICROWAVE ABLATION (MWA) or RADIOFREQUENCY ABLATION (RFA).

ADAPTIVE TRIAL: A type of CLINICAL TRIAL where learning takes place from previous results whilst the trial is ongoing. Where trial arms are seen to be effective or less effective the trial arms are tweaked to accelerate potential promising treatments and to drop or reduce those that seem less effective.

ADJUVANT: A therapy given after initial treatment for the primary cancer. The intention of an adjuvant therapy is to prevent future metastatic disease (spread of the disease).

ADVERSE REACTION or ADVERSE EVENT: Also known as side effects, adverse reactions include any undesired actions or effects of the experimental drug or treatment. Experimental treatments must be evaluated for both immediate and long-term side effects.

APPROVAL: A drug, device or biologic must be approved by a countryʼs regulatory agency before it can be marketed. The approval process involves several steps including preclinical (animal) studies, clinical trials for safety and efficacy, filing of a New Drug Application (NDA) in the United States or Marketing Authorization Application (MAA) in Europe by the manufacturer, regulatory agency review of the application, and agency approval/rejection of application.

ARM: Any of the treatment groups in a clinical trial. Most randomized trials have two “arms,” but some have three “arms,” or even more.

BASELINE: Baseline information is gathered at the beginning of a study from which variations found in the study are measured. Baseline can also be described as a known value or quantity with which an unknown is compared when measured or assessed. Safety and efficacy of a drug are often determined by monitoring changes from the baseline values.

BAYESIAN TRIAL: see ADAPTIVE TRIAL

BIAS: When a point of view prevents impartial judgment on issues relating to the subject of that point of view. In clinical studies, bias is controlled by blinding and randomization.

BLIND, BLINDED or BLINDING: A clinical trial is “blinded” if the participants are unaware on whether they are in the experimental or control arm of the study. Blinding may also be extended to the investigators so that their patient observations are less likely to be biased by their awareness of the treatment the patient is receiving.

BRACHYTHERAPY: A radiotherapy treatment for primary ocular (uveal) melanoma which involves attaching a round radioactive metal plaque to the outside of the eyeball. The plaque is left in place for a couple of days to burn off the tumour which is situated on the inside of the eye in the same location as the plaque has been attached. This treatment is usually sight-saving although some residual radiation damage will often occur. Its availability is dependent on the size and shape of the tumour as well as its location.  

CASE CONTROL STUDY: A scientific study that compares a group of people with a disease (such as leukemia) to a similar group of people without that disease. This type of study compares the levels of exposure (for example, radiation or chemical) each group had before appearance of the disease.

CHEMOEMBOLIZATION: A treatment where chemotherapy agents are supplied to the blood vessel supplying a tumour, usually in a form such as beads that then block the bloody supply so trapping the chemotherapy agent into the tumour site. When this process is performed in the liver it is called Transarterial Chemoembolization (TACE).

CHEMOSATURATION: See PERCUTANEOUS HEPATIC PERFUSION

CLINICAL: Pertaining to or founded on observation and treatment of human participants or patients, as distinguished from theoretical or basic science.

CLINICAL INVESTIGATOR: A medical researcher in charge of carrying out a clinical trial's protocol.

CLINICAL RESEARCH ASSOCIATE (CRA): Person employed by the study sponsor or clinical research coordinator to monitor a clinical trial at one or more participating sites. The CRA is responsible for ensuring all clinical studies are conducted according to study protocol, within regulations and ICH guidelines.

CLINICAL RESEARCH COORDINATOR (CRC): Site administrator for the clinical trial who is responsible for coordinate administrative activities between field and home offices staff, such as the collection of essential documents, distribution of supplies and site selection. Also called research, study or health care coordinator, data manager, research nurse or protocol nurse.

CLINICAL TRIAL (CT): Clinical trials, also known as clinical studies, test potential treatments in human volunteers or patients to see whether they should be further investigated or approved for wider use in the general population. A treatment could be a drug, medical device, or biologic, such as a vaccine, blood product, or gene therapy. Potential treatments, however, must first be studied in laboratory models or animals to determine its safety before they can be tried in people. Treatments having acceptable safety profiles for the disease or condition and showing the most promise are then moved into clinical trials. Clinical trials are an integral part of new product discovery and development, and are required by all regulatory agencies (e.g., the Food and Drug Administration (FDA) in the United States), before a new product can be brought to the market.

COMMUNITY-BASED CLINICAL TRIAL (CBCT): A clinical trial conducted primarily through primary care physicians rather than researchers at academic facilities.

COMPASSIONATE USE: A method of providing experimental therapeutics prior to final regulatory agency approval for use in humans. This procedure is used with very sick individuals who have no other treatment options. Often, case-by-case approval must be obtained by the patientʼs physician from the regulatory agency for “compassionate use” of an experimental drug or therapy.

COMPLETE RESPONSE (CR): The term used for the absence of all detectable cancer after treatment. Complete response doesn't necessarily mean that someone is cured, although it is the best result that can be reported. Any measurable cancer is gone and there is no evidence of disease.

COMPUTERISED TOMOGRAPHY (CT): A type of scan using computer software to resolve many X-Ray images taken from varying angles to provide a cross-sectional image of the body, or specific parts of the body.

CONFIDENTIALITY REGARDING TRIAL PARTICIPANTS: Refers to maintaining the confidentiality of trial participants including their personal identity and all personal medical information. The trial participants' consent to the use of records for data verification purposes must be obtained prior to the trial. The Informed Consent Document will explain how personal health information and study data will be used in accordance with pertinent data protection laws and regulations.

CONJUNCTIVAL MELANOMA: A melanoma found in the lining of the eye, conjunctival melanoma accounts for only 2% of all ocular malignancies, which themselves make up only 3-4% of all melanomas. It is a very rare condition which is often bracketed with other eye cancers although is genetically distinct. Conjunctival Melanoma has more in common with Mucosal Melanoma and often displays NRAS and BRAF mutations which are seen in about half of all conjunctival melanomas.   

CONTRACT RESEARCH ORGANIZATION (CRO): A commercial organization contracted by a research and development organization to perform one or more research-related functions.

CONTRAINDICATION: A specific circumstance in which the use of certain treatments are not allowed usually because they could be harmful or fatal.

CONTRAST AGENT: A substance given to a patient before a scan to help contrast lesions and their structure in the imaging process.

CONTROL GROUP: A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

CONTROLLED TRIALS: A control is a standard against which experimental observations may be evaluated. In a controlled clinical trial, one group of participants is given an experimental drug, while another group (i.e., the control group) is given either a standard treatment for the disease or a placebo.

CROSSOVER TRIAL: A clinical trial in which all participants receive both treatments, but at different times. At a predetermined point in the study, one group is switched from the experimental treatment to the control treatment (standard treatment), and the other group is switched from the control to the experimental treatment.

CRYOBLATION: Cryoablation is a type of ABLATION that uses extreme cold to destroy tumours. A hollow needle, through which cooled, thermally conductive, fluids are circulated, is placed through the skin and into the tumour using imaging guidance .

CYBERKNIFE: This is a method of delivering radiotherapy, with the intention of targeting treatment more accurately than standard radiotherapy. This is an example of STEREOTACTIC RADIOSURGERY.

DATA SAFETY MONITORING BOARD (DSMB): A committee, independent of the sponsor, composed of clinical research experts that reviews trial data while a clinical trial is in progress to ensure that participants are not exposed to undue risk. A DSMB may recommend that a trial be stopped if there are safety concerns or if the trial objectives have been achieved and further continuing the study would not benefit the patients.

DATA SAFETY MONITORING PLAN (DSMP): This plan is meant to assure that each clinical trial has a system for appropriate oversight and monitoring of the conduct of the trial. This oversight ensures the safety of the participants and the validity and integrity of the data.

DECLARATION OF HELSINKI: A series of guidelines adopted by the 18th World Medical Assembly in Helsinki, Finland in 1964. The Declaration addresses ethical issues for physicians conducting biomedical research involving humans. Recommendations include the procedures required to ensure subject safety in clinical trials, including informed consent and Ethics Committee reviews.

DELCATH: The company who provide the equipment and expertise for PERCUTANEOUS HEPATIC PERFUSION are Delcath Systems Ltd. Their name has become synonymous with the treatment itself, much like the terms ‘Xerox’ or ‘Hoover’ are used to mean ‘Photocopying’ or ‘Vacuum Cleaning’

DEMOGRAPHIC DATA: The characteristics of participant group or populations. This could include data on race, age, sex and medical history, all of which can be relevant to the clinical trial study findings.

DEVICE: An instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including any component, part or accessory, that is used to diagnose, cure, treat, or prevent disease. A device does not achieve its intended purpose through chemical action or metabolism in the body.

DIAGNOSTIC TRIALS: Refers to trials that are conducted to find better tests or procedures for diagnosing a particular disease or condition. Diagnostic trials usually include people who have signs or symptoms of the disease or condition being studied.

DOSE-RANGING STUDY: A clinical trial in which two or more doses of an agent (such as a drug) are tested against each other to determine which dose works best and is least harmful.

DOUBLE-BLIND STUDY: A clinical trial design in which neither the participating individuals nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo or another therapy). Double-blind trials are thought to produce objective results, since the knowledge, expectations and biases of the doctor and the participant about the experimental drug or treatment do not affect the outcome.

DRUG-DRUG INTERACTION: A modification of the effect of a drug when administered with another drug. The effect may be an increase or a decrease in the action of either substance, or it may be an adverse effect that is not normally associated with either drug.

EFFECTIVENESS: Whether a drug achieves its desired effect in the real world.

EFFICACY: (Of a drug or treatment) the ability of a drug or treatment to produce a beneficial result. A drug demonstrates efficacy if it is effective at the dose tested against the illness for which it is prescribed.

ELIGIBILITY CRITERIA: Summary criteria for participant selection; includes inclusion and exclusion criteria.

EMPIRICAL: Based on observation or experience, not experimental data.

ENDPOINT: Overall outcome that the protocol is designed to evaluate.

ENUCLEATION: A surgical procedure to completely remove the eye, keeping it and the surrounding muscles and orbital structures intact.

EPIDEMIOLOGY: The branch of medical science that deals with the study of incidence, distribution and control of a disease in a population.

ETHICS COMMITTEE: See institutional review board or "IRB" definition below

EUROPEAN MEDICINES AGENCY (EMA): The European regulatory agency responsible for the scientific evaluation of applications for European marketing authorization for medicinal products (centralized procedure). EMEAʼs main mission is to protect and promote public and animal health, through the evaluation and supervision of medicines for human and veterinary use.

EXCLUSION/INCLUSION CRITERIA: The medical or social standards determining whether a person may or may not be allowed to enter a clinical trial. These criteria are based on such factors as age, gender, pregnancy status, the type and stage of a disease, previous treatment history, and other medical conditions. It is important to note that inclusion and exclusion criteria are not used to reject people personally, but rather to identify appropriate participants to ensure the integrity of the study and to keep them safe.

EXENTERATION: The complete surgical removal of the eyeball and other contents of the eye socket.

EXPANDED ACCESS: Refers to any of the regulatory authority procedures, such as compassionate use, parallel track, and treatment Investigational New Drug Application.

EXPERIMENTAL/INVESTIGATIONAL DRUG: A drug that has been authorized for use in a clinical trial but has not been granted marketing approval as a treatment for a particular use.

EXTRA-HEPATIC: Outside the liver. Extra-hepatic disease in cancer refers to lesions that appear outside the liver.

EYE CANCER: Broadly used to describe cancers that arise in or around the eye. Sometimes used interchangeably with OCULAR MELANOMA or UVEAL MELANOMA. Eye cancers can also include diseases such as RETINOBLASTOMA, a childhood cancer of the eye.

FINE-NEEDLE ASPIRATION BIOPSY: Fine-needle aspiration is a diagnostic procedure used to investigate lumps or masses, in this case one on the inside of the eye. In this technique, a thin, hollow needle is inserted into the mass for sampling of cells that, after being stained, will be examined under a microscope and their genetic makeup determined. The genetic makeup of an eye tumour is a good indicator of possible future metastatic spread.

FIRST-IN-HUMAN/MAN STUDY: A clinical trial where a medical procedure or medicinal product, previously developed and assessed through laboratory model or animal testing, or through mathematical modeling is tested on human subjects for the first time.

FOOD AND DRUG ADMINISTRATION (FDA): The U.S. Department of Health and Human Services agency responsible for ensuring the safety and effectiveness of all drugs, biologics, vaccines, and medical devices. The FDA also works with the blood banking industry to safeguard the nation's blood supply.

GAMMA KNIFE: Not a knife at all, but a set of focused beams of gamma radiation used to treat lesions, usually those in the brain. The various beams of radiation enter the brain at several different locations so that only a relatively small dose is received in any given area, except the place where they are focused which receives a large dose of radiation in the expectation that the lesion is destroyed. This is an example of STEREOTACTIC RADIOSURGERY.

GENERIC DRUGS: A medicine with the same active ingredient, but not necessarily the same inactive ingredients, as a brand-name drug. A generic drug may be marketed only after the original drug's patent has expired.

GNAQ & GNA11:

GUT MICROBIOME: Gut microbiota (also called gut flora) is the name given today to the microbe population living in our intestine. Our gut microbiota contains tens of trillions of microorganisms, including at least 1000 different species of known bacteria and collectively the environment is known as the gut microbiome. The gut microbiome is known to be related to the effectiveness of our immune system, although exactly how this mechanism works is not well known and currently there are no proven ways to enhance immune response by altering it.

HEALTH TECHNOLOGY ASSESSMENT (HTA): The systematic evaluation of a health technology, such as a drug or device used for treatment, typically including assessment of safety, clinical relevance and cost-effectiveness. The body responsible for HTA in the UK is the National Institute for Health and Care Excellence (NICE). Other examples of HTA bodies include ZIN (Netherlands) and AIFA (Italy).  

HELSINKI AGREEMENT: See DECLARATION OFHEALTH TECHNOLOGY ASSESSMENT (HTA): The systematic evaluation of a health technology, such as a drug or device used for treatment, typically including assessment of safety, clinical relevance and cost-effectiveness. The body responsible for HTA in the UK is the National Institute for Health and Care Excellence (NICE). Other examples of HTA bodies include ZIN (Netherlands) and AIFA (Italy).  
HELSINKI AGREEMENT: See DECLARATION OF HELSINKI
HEPATIC: Relating to the liver. In cancer hepatic disease refers to lesions found in the liver.
HIPAA: The Health Insurance Portability and Accountability Act of 1996 (HIPAA) established national standards for electronic health care transactions and national identifiers for providers, health plans, and employers. It also addresses the security and privacy of health data. All clinical trial data and activities performed by covered entities must comply with HIPAA regulations.
HUMAN SUBJECT: A patient or healthy person participating in a research project.
HYPOTHESIS: A theory, supposition or assumption advanced as a basis for reasoning or argument, or as a guide to experimental investigation.
IMMUNOTHERAPY (IT): The set of treatments used to harness and enhance the body’s own immune system response to cancer. Usually provided intravenously over a series of infusions, different immunotherapy drugs have different effects to permit the immune system to better detect, infiltrate and ultimately destroy cancer cells. The set of “-mab” drugs, which stands for Monoclonal AntiBody, include such drugs as Ipilimumab, Pembrolizumab and Atezolizumab.  
INFORMED CONSENT: Informed consent is the verification of a personʼs willingness to participate in a research project. Prior to enrollment into a clinical trial, researchers inform participants about all relevant study details. Participants are provided an informed consent document that details all the important study information including its purpose, duration, risks, potential benefits, required procedures, and key contacts, and they will be asked to sign this document if they agree to participate in the trial. The informed consent document is not a contract. Participation in the clinical trial is voluntary and the participant may withdraw from the trial at any time without penalty or loss of benefits to which he/she is otherwise entitled. The research team actively maintains informed consent throughout the trial by providing the participant with any new or developing information.
INFORMED CONSENT DOCUMENT: A document that describes the rights of the study participants, and includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant will be asked to sign this document if they agree to.
INSTITUTIONAL REVIEW BOARD (IRB): A committee of physicians, statisticians, researchers, community advocates, and others that ensures that a clinical trial is ethical and that the rights of study participants are protected. Every institution that conducts or supports biomedical or behavioral research involving human participants must, by federal regulation, have an IRB that approves and periodically reviews the research in order to protect the rights of human participants.
INTENT TO TREAT: Analysis of clinical trial results that includes all data from participants in the groups to which they were randomized even if they never received the treatment.
INTERVENTION NAME: The generic name of the experimental treatment being studied.
INTERVENTIONS: Primary experimental treatments being studied. Types of treatments may include drug, gene transfer, vaccine, behavior, device, or procedure.
INVESTIGATIONAL NEW DRUG APPLICATION (IND): The petition through which a drug sponsor requests the FDA to allow human testing of a new drug, antibiotic drug, or biological drug in a clinical investigation. This includes an application for a biological product used in vitro for diagnostic purposes. INDs are made available to participants before general marketing begins, typically during Phase 3 studies. To be considered for a treatment IND a participant cannot be eligible to be in the definitive clinical trials.
IN VIVO: Testing or action inside an organism, such as a human subject or patient.
IN VITRO: Testing or action outside an organism (e.g. inside a test tube or culture dish.)
IPILIMUMAB: An IMMUNOTHERAPY drug and monoclonal antibody that works to activate the immune system by targeting CTLA-4, a protein receptor that dampens the effect of the immune system. 
IRIS MELANOMA: Accounting for about 5% of all Uveal Melanomas, this malignancy occurs in the coloured part of the eye, the iris.  
LIVER DIRECTED TREATMENT: Treatment options that pertain to the liver only and seek only to treat hepatic (liver) lesions. These include such treatments as PERCUTANEOUS HEPATIC PERFUSION, SIRT, LIVER RESECTION, TACE and CHEMOEMBOLIZATION. Other directed treatments exist to specifically treat other organs. A SYSTEMIC TREATMENT is used to treat the cancer across the entire body. 
MAGNETIC RESONANCE IMAGING (MRI): A type of scan using a strong magnetic field and radio waves to generate images of parts of the body that can't be seen as well with X-Rays, CT scans or ultrasound. MRI scans are often seen as the Gold Standard of imaging technology with little long term effect on the body. The very latest MRI scanners have such a high resolution that it may soon be possible to undergo scans without the need for contrast agents.
MARKETING AUTHORIZATION APPLICATION (MAA): An application submitted by a sponsor to the EMEA for approval to market a new drug (a new, non-biological molecular entity) for human use in Europe.
MEK INHIBITOR:
META-ANALYSIS: Systematic methods that use statistical techniques for combining results from similar studies to obtain a quantitative estimate of the overall effect of a particular intervention or variable on a defined outcome. This type of analysis is typically hypothesis generating.
MICROWAVE ABLATION (MWA): A type of ABLATION which uses microwaves to burn tumours. A needle, with a microwave source at its tip, enters via the skin to pierce the tumour before the microwave source is activated to burn off the tumour. This procedure is often used to destroy one or two metastatic liver tumours. 
MULTICENTER TRIAL: Clinical trial conducted according to a single protocol but at more than one site, and, therefore, carried out by more than one investigator.
MULTIPLE-RISING DOSE STUDY: see dose ranging study above. HELSINKI

HEPATIC: Relating to the liver. In cancer hepatic disease refers to lesions found in the liver.

HIPAA: The Health Insurance Portability and Accountability Act of 1996 (HIPAA) established national standards for electronic health care transactions and national identifiers for providers, health plans, and employers. It also addresses the security and privacy of health data. All clinical trial data and activities performed by covered entities must comply with HIPAA regulations.

HUMAN SUBJECT: A patient or healthy person participating in a research project.

HYPOTHESIS: A theory, supposition or assumption advanced as a basis for reasoning or argument, or as a guide to experimental investigation.

IMMUNOTHERAPY (IT): The set of treatments used to harness and enhance the body’s own immune system response to cancer. Usually provided intravenously over a series of infusions, different immunotherapy drugs have different effects to permit the immune system to better detect, infiltrate and ultimately destroy cancer cells. The set of “-mab” drugs, which stands for Monoclonal AntiBody, include such drugs as Ipilimumab, Pembrolizumab and Atezolizumab.  

INFORMED CONSENT: Informed consent is the verification of a personʼs willingness to participate in a research project. Prior to enrollment into a clinical trial, researchers inform participants about all relevant study details. Participants are provided an informed consent document that details all the important study information including its purpose, duration, risks, potential benefits, required procedures, and key contacts, and they will be asked to sign this document if they agree to participate in the trial. The informed consent document is not a contract. Participation in the clinical trial is voluntary and the participant may withdraw from the trial at any time without penalty or loss of benefits to which he/she is otherwise entitled. The research team actively maintains informed consent throughout the trial by providing the participant with any new or developing information.

INFORMED CONSENT DOCUMENT: A document that describes the rights of the study participants, and includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant will be asked to sign this document if they agree to.

INSTITUTIONAL REVIEW BOARD (IRB): A committee of physicians, statisticians, researchers, community advocates, and others that ensures that a clinical trial is ethical and that the rights of study participants are protected. Every institution that conducts or supports biomedical or behavioral research involving human participants must, by federal regulation, have an IRB that approves and periodically reviews the research in order to protect the rights of human participants.

INTENT TO TREAT: Analysis of clinical trial results that includes all data from participants in the groups to which they were randomized even if they never received the treatment.

INTERVENTION NAME: The generic name of the experimental treatment being studied.

INTERVENTIONS: Primary experimental treatments being studied. Types of treatments may include drug, gene transfer, vaccine, behavior, device, or procedure.

INVESTIGATIONAL NEW DRUG APPLICATION (IND): The petition through which a drug sponsor requests the FDA to allow human testing of a new drug, antibiotic drug, or biological drug in a clinical investigation. This includes an application for a biological product used in vitro for diagnostic purposes. INDs are made available to participants before general marketing begins, typically during Phase 3 studies. To be considered for a treatment IND a participant cannot be eligible to be in the definitive clinical trials.

IN VIVO: Testing or action inside an organism, such as a human subject or patient.

IN VITRO: Testing or action outside an organism (e.g. inside a test tube or culture dish.)

IPILIMUMAB: An IMMUNOTHERAPY drug and monoclonal antibody that works to activate the immune system by targeting CTLA-4, a protein receptor that dampens the effect of the immune system.

IRIS MELANOMA: Accounting for about 5% of all Uveal Melanomas, this malignancy occurs in the coloured part of the eye, the iris.  

LIVER DIRECTED TREATMENT: Treatment options that pertain to the liver only and seek only to treat hepatic (liver) lesions. These include such treatments as PERCUTANEOUS HEPATIC PERFUSION, SIRT, LIVER RESECTION, TACE and CHEMOEMBOLIZATION. Other directed treatments exist to specifically treat other organs. A SYSTEMIC TREATMENT is used to treat the cancer across the entire body.

MAGNETIC RESONANCE IMAGING (MRI): A type of scan using a strong magnetic field and radio waves to generate images of parts of the body that can't be seen as well with X-Rays, CT scans or ultrasound. MRI scans are often seen as the Gold Standard of imaging technology with little long term effect on the body. The very latest MRI scanners have such a high resolution that it may soon be possible to undergo scans without the need for contrast agents.

MARKETING AUTHORIZATION APPLICATION (MAA): An application submitted by a sponsor to the EMEA for approval to market a new drug (a new, non-biological molecular entity) for human use in Europe.

MEK INHIBITOR:

META-ANALYSIS: Systematic methods that use statistical techniques for combining results from similar studies to obtain a quantitative estimate of the overall effect of a particular intervention or variable on a defined outcome. This type of analysis is typically hypothesis generating.

MICROWAVE ABLATION (MWA): A type of ABLATION which uses microwaves to burn tumours. A needle, with a microwave source at its tip, enters via the skin to pierce the tumour before the microwave source is activated to burn off the tumour. This procedure is often used to destroy one or two metastatic liver tumours.

MULTICENTER TRIAL: Clinical trial conducted according to a single protocol but at more than one site, and, therefore, carried out by more than one investigator.

MULTIPLE-RISING DOSE STUDY: see dose ranging study above.

NATIONAL INSTITUTES OF HEALTH (NIH): Agency within the Department of Health and Human Services that funds research, conducts studies, and funds multicenter national studies.

NEW DRUG APPLICATION (NDA): An application submitted by a sponsor to the FDA for approval to market a new drug (a new, non-biological molecular entity) for human use in interstate commerce in the United States.

NO EVIDENCE of DISEASE (NED): Every cancer patient’s favourite words. This means that there is no detectable disease on scans and is the best result that can be reported.

OBSERVATIONAL STUDY: An epidemiologic study that does not involve any intervention, experimental or otherwise. Such a study may be one in which nature is allowed to take its course, with changes in one characteristic being studied in relation to changes in other characteristics. Analytical epidemiologic methods, such as case-control and cohort study designs, are properly called observational epidemiology because the investigator is observing without intervention other than to record, classify, count, and statistically analyze results.

OCULAR MELANOMA (OM): Typically used to describe any melanoma found in or around the eye. This broadly includes two types of cancers: Uveal Melanoma, a cancer that develops in the pigmented uveal tract within the eye, and Conjunctival Melanoma found on the surface of the eye and more similar to mucosal melanoma. Note that in medical circles uveal and conjunctival melanomas are usually treated separately. As such medical literature often refers to uveal melanoma whilst we talk about ocular melanoma — this is especially useful to know if you’re searching for treatments or information online when the term Ocular Melanoma might not be used.

OFF-LABEL USE: A drug prescribed for conditions other than those approved by a countryʼs regulatory agency.

OFFICE FOR HUMAN RESEARCH PROTECTIONS (OHRP): The Office for Human Research Protections (OHRP) supports, strengthens and provides leadership to the nationʼs system for protecting volunteers in research that is conducted or supported by the U.S. Department of Health and Human Services (HHS). OHRP provides clarification and guidance to research institutions, develops educational programs and materials, and promotes innovative approaches to enhancing human subject protections.

OPEN-LABEL TRIAL: A clinical trial in which doctors and participants know which treatment is being administered.

ORPHAN DRUGS: A drug category that refers to medications used to treat rare diseases and conditions. Orphan drug status provides manufacturer specific incentives to develop and bring such treatments to market.

OUTCOMES TRIAL/STUDY: An outcomes trial evaluates the effect of a treatment on patients. Treatments may include medications or other therapies and outcomes may include changes in disease status, morbidity or mortality.

OVERALL RESPONSE RATE (ORR): Measured as a percentage, this figure gives the proportion of patients with a tumour size reduction (of a predefined amount) for a specified minimum time period in a study cohort or other patient group. Often defined as the total proportion of patients who attained either a PARTIAL RESPONSE (PR) or COMPLETE RESPONSE (CR).

OVERALL SURVIVAL (OS): The length of time from either the date of diagnosis or the start of treatment that patients diagnosed with cancer are still alive. In a clinical trial, measuring the overall survival is one way to see how well a new treatment works.

OVERALL SURVIVAL RATE (OSR): The percentage of people in a study or treatment group who are still alive for a certain period of time after they were diagnosed or started treatment. The overall survival rate is often stated as a five-year survival rate, which is the percentage of people in a study or treatment group who are alive five years after their diagnosis or the start of treatment. Also called survival rate.

P-VALUE: A p-value demonstrates the likelihood that sample data do not adequately represent the population from which they were drawn. The accepted standard for a statistically significant p-value is <0.05, meaning that the likelihood that the result could occur by random chance is less than 5 in a hundred.

PARALLEL STUDY: A parallel designed clinical trial compares the results of a treatment on two separate groups of patients. The sample size calculated for a parallel design can be used for any study where two groups are being compared.

PARTIAL RESPONSE (PR): Describes when a cancer partially responds to treatment, yet has still not gone away. This may mean that some tumours reduced by a certain amount where others appear unchanged.

PATHWAY: An established and accepted set of clinical guidance for treatment of a particular disease. Having such a mechanism in place helps standardise care and reduces variability in treatment across different centres. Also called Clinical Pathway or Care Pathway.

PEER REVIEW: Review of a clinical trial by independent experts. These experts review the trials for scientific merit, validity, participant safety, and ethical considerations.

PEMBROLIZUMAB: An IMMUNOTHERAPY drug and monoclonal antibody that works by blocking one of the protective mechanisms of cancer cells. The drugs targets the Programmed Cell Death receptor (PD-1) on cancer cells, whose mechanism is to cause the body’s immune cells to self destruct.

PERCUTANEOUS HEPATIC PERFUSION (PHP): A treatment specific to the liver whereby the organ is isolated and a high dose of chemotherapy (usually Melphalan) is administered before being filtered out again. This treatment allows a far higher dose of chemotherapy to be given than could safely be tolerated were it given systemically across the entire body. This treatment has shown very encouraging results for those with liver metastases and is now a safe and well tolerated treatment option.  

PHARMACOLOGY: The study of how drugs interact with living organisms to produce a change in function. Pharmacology deals with how drugs interact within biological systems to affect function.

PHARMACOKINETICS: The processes (in a living organism) of absorption, distribution, metabolism, and excretion of a drug or vaccine.

PHARMACOVIGILANCE: The science of collecting, monitoring, researching, assessing and evaluating information from healthcare providers and patients on the adverse effects of medications, biological products, herbalism and traditional medicines with a view to identify new information about hazards associated with medicines and preventing harm to patients.

PHASE 1 CLINICAL TRIAL: Sometimes called dosing studies, these trials may test methods of administering the treatment (e.g. by mouth, injection, etc.) and how often, as well as the safety of the treatment. Depending in the disease and drug being tested these trials can sometimes involve a small number of healthy participants.

PHASE 2 CLINICAL TRIAL: These trials continue to test the safety of the treatment and evaluate how well the treatment works. Phase 2 studies usually evaluate the treatment in a specific condition. These trials usually involve 100-300 patients.

PHASE 3 CLINICAL TRIAL: These trials compare the new treatment to the current standard of treatment for a specific condition, establishing both efficacy and adverse events. Participants are usually assigned to either receive the new treatment or the current standard. Phase 3 trials typically enroll large numbers of patients (1,000-3,000 or many more) and may be carried out at hospitals and doctorsʼ offices nationwide.

PHASE 4 CLINICAL TRIAL: Post-marketing studies to gain a greater understanding of the treatment, including its risks, benefits, and optimal use.

PIVOTAL STUDY: A study, usually Phase 3, which presents the data used by regulatory agencies to decide whether to approve a drug. A pivotal study will generally be well- controlled, randomized, and whenever possible, double-blind.

PLACEBO: A placebo is an inactive pill, liquid or powder that has no treatment value. In clinical trials, a placebo is administered instead of an active drug or experimental treatment to assess the experimental treatmentʼs effectiveness.

PLACEBO CONTROLLED STUDY: A method of drug investigation in which an inactive substance (a placebo) is given to one group of participants, while the drug being tested is given to another group. The results obtained in the two groups are then compared to see if the investigational treatment is more effective than the placebo in treating the condition.

PLACEBO EFFECT: A physical or emotional change, occurring after an inactive substance is taken or administered, that is not the result of any special property of the substance. The change may be beneficial, reflecting the expectations of the participant and, often, the expectations of the person giving the substance.

PLAQUE BRACHYTHERAPY: see BRACHYTHERAPY

POSITRON EMISSION TOMOGRAPHY (PET): A type of scan, usually a series of X-Rays, that is used to provide 3D images of structures within the body based on metabolism. Before a PET scan patients are given a radioactive glucose solution which causes areas of the body with high metabolic rate, such as metastatic cancer lesions, to be highlighted on the scan.   

PRECLINICAL STUDIES: Experiments performed in the laboratory and in animals to study a drug before it is tested in humans.

PREVENTION TRIALS: Conducted to find better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.

PRINCIPAL INVESTIGATOR: A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator who is the responsible leader of the team may be called the principal investigator.

PROGRESSIVE DISEASE (PD): The term used to describe when tumours have grown, new tumours have appeared or that the cancer has spread to other parts of the body.

PROGRESSION FREE SURVIVAL (PFS): The length of time during and after treatment that a patient lives with the disease but it does not progress. In a clinical trial, measuring the progression-free survival is one way to see how well a new treatment works.

PROSPECTIVE STUDY: A prospective study identifies subjects, applies a treatment and follows them over time to measure their progress/outcomes relative to a predetermined set of criteria or endpoints.

PROTOCOL: A protocol is the study plan on which a clinical trial is based. Each trial is carefully designed to safeguard the health of participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial, the schedule of tests, procedures, medications, dosages, and length of the study.

PROTON BEAM THERAPY: A form of radiotherapy used to kill cancerous tissue. A beam of protons is targeted at the cancer and unlike conventional radiotherapy is less damaging to surrounding tissue. Commonly used as a treatment for primary ocular (uveal) melanoma.

QUALITY ADJUSTED LIFE YEAR (QALY): A combined measure of both quality and quantity of life with a disease burden. It is used as a metric in the economic evaluation of treatments to assess the value for money of medical interventions.

QUALITY OF LIFE TRIALS: (or Supportive Care trials): Refers to trials that explore ways to improve comfort and quality of life for individuals with a chronic illness.

RADIOFREQUENCY ABLATION (RFA): A type of ABLATION which uses heat from an electric current to burn tumours. A needle enters via the skin to pierce the tumour before the device is activated to burn off the tumour. This procedure is often used to destroy one or two metastatic liver tumours.

RANDOMIZATION: A method based on chance by which study participants are assigned to different treatment groups. This minimizes the differences among groups by equally distributing people with particular characteristics among all the trial arms, thereby avoiding “selection bias.” Randomization allows for researchers to comparably test different treatments in similar groups.

RANDOMIZED CONTROL TRIAL or RANDOMIZED TRIAL (RCT): A study in which participants are randomly (i.e. by chance) assigned to one of two or more treatment arms of a clinical trial.

RESECTION: A surgical procedure to remove all or part of an organ, tissue or other structure in the body. A liver resection entails removing those parts of the liver affected by cancerous lesions.

RESCUE MEDICATION: A quick-relief or fast-acting medication patients in clinical trials may be given besides the investigational drug or control that can alleviate symptoms due to disease or lack of efficacy of the study treatment. It acts quickly to stop symptoms, but the effects are not long lasting.

RETINOBLASTOMA: A rare cancer that rapidly develops from the immature cells of a retina, the light-detecting tissue of the eye. It is the most common primary malignant intraocular cancer in children, and it is almost exclusively found in young children. An initial step in testing for the condition, called the ‘Red Reflex Test’, involves shining a light into the eye in a darkened room and with an ophthalmoscope seeing if there is a red reflection (normal) or white (sometimes indicative of an eye condition).  

RETROSPECTIVE STUDY: A study in which investigators select groups of patients that have already been treated and analyze data from the events experienced by these patients. These studies are subject to bias because investigators can select patient groups with known outcomes.

RISK-BENEFIT RATIO: The risk a treatment places on individual participants versus the potential benefits of the treatment. The risk/benefit ratio may differ depending on the condition being treated and the stage of the disease.

SCREENING TRIALS: Refers to trials which test the best way to detect certain diseases or health conditions.

SELECTIVE INTERNAL RADIATION THERAPY (SIRT): A LIVER DIRECTED TREATMENT where tiny radioactive beads are administered to the liver via the hepatic artery, also known as Radioembolization. The beads get stuck in the small blood vessels supplying the tumours and remain there emitting radiation which damages and kills the nearby tumours.

SIDE EFFECTS: Any undesired actions or effects of a drug or treatment. Experimental drugs must be evaluated for both immediate and long-term side effects.

SINGLE-BLIND STUDY: A study in which subject or patient is unaware of what medication they are taking, while the physician does know.

SIRTEX: A company providing the tiny radioactive microsphere beads for SIRT treatment, sometimes called SIR-Spheres or Theraspheres.

SMALL MOLECULE INHIBITOR DRUGS:

SPONSOR: A person or an organization that manages and finances a clinical trial.

STABLE DISEASE (SD): Used to describe a tumour that is neither growing nor shrinking. Also to describe how no new tumours have appeared and that cancer has not spread to another part of the body.

STANDARD TREATMENT: A treatment currently in wide use by the medical community and approved by a countryʼs regulatory agency considered to be effective in the treatment of a specific disease or condition.

STANDARD OF CARE (SOC): Treatment regimen or management based on state of the art medical care.

STATISTICAL SIGNIFICANCE: The probability that an event or difference occurred by chance alone. In clinical trials, the level of statistical significance depends on the number of participants studied and the observations made, as well as the magnitude of differences observed.

STATISTICIAN: One versed in or engaged in compiling statistics.

STEREOTACTIC RADIOSURGERY (SRS): A method of killing cancer cells by focusing several separate beams of radiation onto a single small area. Often used to destroy brain lesions, although also used elsewhere e.g. the lungs, several beams of radiation are aimed at a single spot, the benefit being that other areas of the brain (or other organ) are not seriously damaged because they only receive the lower dosage from any one single beam. Examples of this treatment include CYBERKNIFE and GAMMA KNIFE.

STEREOTACTIC RADIOTHERAPY (SRT): Used to describe a course of STEREOTACTIC RADIOSURGERY, which itself refers only to a single treatment.

STUDY ENDPOINT: An outcome used to judge the safety or effectiveness of a treatment.

SURROGATE ENDPOINT: A biomarker or endpoint that is intended to substitute for a clinical endpoint. A surrogate endpoint is expected to predict a clinical endpoint or lack thereof.

SYSTEMIC TREATMENT: Systemic treatments are drugs that work across the entire body to treat cancer cells wherever they are situated. Examples are systemic chemotherapy, targeted drugs, and immunotherapy.

TARGETED DRUGS:

TRANS-ARTERIAL CHEMOEMBOLIZATION (TACE): See CHEMOEMBOLIZATION

THERASPHERES: Another name for the tiny irradiating beads used to treat the liver using SELECTIVE INTERNAL RADIATION THERAPY (SIRT).

TOXICITY: A treatment-related adverse effect that may be detrimental to the recipient's health. The level of toxicity associated with a treatment will vary depending on the attributes of the treatment itself and the condition the drug is being used to treat.

TREATMENT TRIALS: Refers to trials which test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.

ULTRASOUND (US) SCAN: This method of scanning uses sound waves with frequencies above the audible range for humans. Pulses of ultrasound are sent to build images, also called sonograms, of internal structures, including tumours, by recording and interpreting the echoes. The reporting of ultrasound scans is dependent upon the skill of the operator in detecting lesions particular to the disease in question.  

UVEAL MELANOMA (UM): Refers to melanomas that occur within the eye’s pigmented uveal tract. The three main types within this are Choroidal Melanoma, Ciliary Body Melanoma and Iris Melanoma. The majority of uveal melanomas are choroidal melanomas. This is the term used in medical circles, because OCULAR MELANOMA brackets both uveal and conjunctival melanomas which are essentially different diseases.

CBCT: COMMUNITY-BASED CLINICAL TRIAL

CR: COMPLETE RESPONSE

CRA: CLINICAL RESEARCH ASSOCIATE

CRC: CLINICAL RESEARCH COORDINATOR

CRO: CONTRACT RESEARCH ORGANIZATION

CT: CLINICAL TRIAL

CT: COMPUTERISED TOMOGRAPHY

DSMB: DATA SAFETY MONITORING BOARD

DSMP: DATA SAFETY MONITORING PLAN

EMA: EUROPEAN MEDICINES AGENCY

FDA: FOOD & DRUG ADMINISTRATION

FNAB: FINE-NEEDLE ASPIRATION BIOPSY

HTA: HEALTH TECHNOLOGY ASSESSMENT

IND: INVESTIGATIONAL NEW DRUG APPLICATION

IRB: INSTITUTIONAL REVIEW BOARD

IT: IMMUNOTHERAPY

MAA: MARKETING AUTHORIZATION APPLICATION

MRI: MAGNETIC RESONANCE IMAGING

MWA: MICROWAVE ABLATION

NDA: NEW DRUG APPLICATION

NED: NO EVIDENCE of DISEASE

NIH: NATIONAL INSTITUTES OF HEALTH

OHRP: OFFICE FOR HUMAN RESEARCH PROTECTIONS

OM: OCULAR MELANOMA

ORR: OVERALL RESPONSE RATE

OS: OVERALL SURVIVAL

OSR: OVERALL SURVIVAL RATE

PD: PROGRESSIVE DISEASE