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.