Every industry has its own terminology; sometimes, it feels like its own language. The healthcare industry, specifically the life sciences and pharmaceutical industry, is riddled with phrases and acronyms that could make your head spin whether you’re just starting out in the field or are a tenured professional.
In three categories – terms related to drug development, risk assessment and AI – our ever-growing glossary briefly defines terms relevant to drug development, risk assessment and artificial intelligence, helping to put these common terms into plain English.
Glossary of Drug Development Terms
Abbreviated New Drug Application (ANDA): An application submitted to the FDA for the review and approval of a generic drug product. Unlike new drug applications (NDAs), ANDAs generally do not require preclinical or clinical data; they demonstrate bioequivalence to the innovator drug.
Active Ingredient: A component in a drug that provides pharmacological activity or other direct effects in treating or preventing disease. It’s the key therapeutic substance in a medication.
Adjuvant Treatment: Additional treatment given after the primary treatment (such as surgery or chemotherapy) for a disease, typically to prevent recurrence or to enhance the effectiveness of the primary treatment.
Adverse Event (AE): Any unfavorable medical occurrence in a patient or clinical investigation subject who received a pharmaceutical product. The adverse event does not necessarily have a causal relationship with the treatment. AE is a broad term and includes adverse drug reactions.
Adverse Event (AE) Reporting: AE reporting involves documenting and analyzing any undesirable or harmful effects experienced by patients during a clinical trial or after using a drug.
Adverse Drug Reaction (ADR): Any unintended or harmful reaction that occurs after administering a drug or medication under normal conditions of use. A causal relationship between the medical product and the occurrence is suspected.
ADR Database: Case management system that monitors ADR occurrence and trends.
Application: Refers to a New Drug Application (NDA), Abbreviated New Drug Application (ANDA), or Biologic License Application (BLA). These applications contain data submitted to the FDA for review and approval of a drug or biologic product.
Approval History: A chronological record of all FDA actions related to a specific drug product, including label changes, new routes of administration and expanded patient populations.
Approval Letter: An official communication from the FDA to a new drug application sponsor, allowing the commercial marketing of a new drug product.
Application Number: The unique identifier assigned by the FDA to each application for approval to market a generic drug or biologic in the United States.
Attribute: A feature or characteristic of a medical product (e.g., efficacy, safety, means of administration) that may affect benefit-risk considerations.
Attrition Rate: The rate at which drug candidates fail or are discontinued at various stages of development, reflecting the challenges and risks associated with advancing compounds through clinical testing.
Basket Trial: A clinical trial design in which a single drug or treatment is tested simultaneously across multiple diseases or conditions that share a common genetic or molecular alteration. This approach allows researchers to evaluate the drug’s effectiveness in treating various types of cancer or other diseases with specific genetic mutations or biomarkers rather than focusing on a single disease type.
Bayesian Analysis: Bayesian analysis is a statistical method that combines prior knowledge with new data to update and refine estimates of drug efficacy and safety. It is used to make informed decisions in drug development based on evolving evidence.
Benefit: The favorable effects of a medical product, including clinical benefit and other important characteristics (e.g., convenience) that impact patient compliance.
Benefit-Risk Assessment: An ongoing evaluation of a drug’s benefits (including efficacy) versus its risks (including safety) throughout its lifecycle. Regulatory agencies use this assessment to make informed judgments about drug approvals.
Blinded Trial: A study where participants, researchers, or both are unaware (blinded) of which treatment a participant is receiving. Blinding helps minimize bias in assessing the effects of a drug or treatment by preventing expectations or preferences from influencing outcomes.
Biologic License Application (BLA): A submission containing information on the manufacturing processes, pharmacology, and clinical effects of a biological product. If approved, a license is issued to market the biologic.
Biological Product: A medical product derived from living organisms used to diagnose, prevent, or treat diseases, including vaccines, blood components, gene therapies, and therapeutic proteins.
Biomarker: a biological molecule found in blood, other body fluids, or tissues that indicates a normal or abnormal process, or a condition or disease, and can be used to monitor and predict health states and responses to treatments
Brand Name: Name given for marketing purposes to any ready-prepared medicine placed on the market under a special name and in a special pack. A brand name may be a protected trademark.
Breakthrough Therapy: A designation by regulatory authorities (such as the FDA in the United States) for a drug that demonstrates substantial improvement over existing therapies for a serious or life-threatening condition. This designation expedites the development and review process, aiming to make promising treatments available to patients more quickly.
Clinical Benefit: Refers to a positive effect of a treatment on a patient’s health,
Clinical Outcome: Describes an individual’s well-being, functioning, or survival.
Clinical Trial: A research study involving human participants to evaluate the safety and efficacy of a medical intervention (e.g., a drug). Clinical trials follow specific protocols and phases (I-IV) to gather data for regulatory approval.
Cohort: A group of individuals who share specific characteristics or conditions and are studied together as part of a clinical trial or observational study. Cohorts are often used to assess the safety, efficacy, or outcomes associated with a particular drug or treatment strategy.
Combination Therapy: The use of two or more drugs or treatments together to enhance therapeutic efficacy, target multiple pathways or mechanisms of disease, or reduce the likelihood of drug resistance.
Conditional Marketing Authorization (CMA): A regulatory pathway by the EMA that allows early approval of a medicine that addresses unmet medical needs, especially in emergency situations. CMAs are granted based on less comprehensive data than normally required, with the understanding that additional data will be provided in the future. It allows patients earlier access to treatments while ensuring ongoing assessment of the benefits and risks as more information becomes available.
Cost-Effectiveness Analysis (CEA): CEA is a method used to evaluate the economic value of a drug by comparing its costs to its health outcomes. It helps stakeholders make informed decisions regarding resource allocation and pricing strategies.
Dose-Response Relationship: The dose-response relationship describes the correlation between the dose of a drug administered and its effect on the body. Understanding this relationship is crucial for determining the optimal dose that balances therapeutic benefit with safety.
Drug R&D: Expenses incurred by pharmaceutical companies and researchers while researching and developing new drugs. These costs typically include expenditures on clinical trials, preclinical studies, regulatory approval processes, and other associated research activities.
Dual Phase Trial: A clinical trial that includes two distinct phases of testing within the same study design. This could involve initial testing (Phase 1 or Phase 2a) to establish safety and preliminary efficacy, followed by a subsequent phase (Phase 2b or Phase 3) to further evaluate effectiveness and confirm safety before potential regulatory approval. This approach streamlines the development process and may expedite the transition from early-stage to later-stage clinical testing.
Efficacy Endpoint: An endpoint that assesses whether a drug or treatment is effective in achieving its intended therapeutic effect. Efficacy endpoints are crucial in clinical trials to demonstrate that a drug has the desired beneficial effect on the patient’s health condition.
Endpoint: A specific, measurable outcome used to determine a treatment’s effect. It can be a clinical event, a symptom, a biomarker, or a laboratory measurement. Endpoints can be primary (the main outcome of interest) or secondary (additional outcomes that provide supporting information).
European Medicines Agency (EMA): An agency of the European Union in charge of the evaluation and supervision of pharmaceutical products. Before 2004, it was known as the European Agency for the Evaluation of Medicinal Products or European Medicines Evaluation Agency.
Fast Healthcare Interoperability Resources (FHIR): A standard developed by Health Level Seven International (HL7) for exchanging healthcare information electronically. It is designed to enable health data, including clinical and administrative data, to be quickly and efficiently exchanged between different healthcare systems. The goal of FHIR is to create a more connected health ecosystem that enhances interoperability, supports innovative applications, and improves health outcomes.
Fast Track: A process designed to expedite the development and review of drugs intended to treat serious conditions and fill an unmet medical need. This designation allows for more frequent interactions with regulatory agencies, priority review of marketing applications, and potential eligibility for accelerated approval pathways based on early clinical data.
FDA-Approved Label: The official description of a drug product, including indications, side effects, and usage instructions.
First Line Treatment: The initial therapy or treatment regimen recommended for a disease or condition. It is typically the first choice of treatment considered by healthcare providers based on its proven efficacy, safety profile, and suitability for a particular patient population.
Food and Drug Administration (FDA): A U.S. federal agency of the Department of Health and Human Services. The FDA controls and supervises food safety, tobacco products, caffeine products, dietary supplements, prescription and over-the-counter pharmaceutical drugs, vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices, cosmetics, animal foods and feed and veterinary products.
Hazard Ratio: The measure of the relative risk of an event (e.g. disease progression or death) between two groups in a clinical trial. It quantifies the likelihood of an event occurring in one group compared to another over time, providing key information about the effectiveness of a drug or treatment intervention.
Health Economics: A branch of economics concerned with the efficient allocation and utilization of healthcare resources to maximize health outcomes. It analyzes the costs and benefits of healthcare interventions, policies, and systems to inform decision-making in healthcare.
Health Level Seven (HL7): A set of international standards for the exchange, integration, sharing, and retrieval of electronic health information. These standards aim to improve the interoperability between healthcare systems, enabling the seamless sharing of clinical and administrative data across different healthcare providers and organizations. HL7 covers a variety of aspects, including messaging formats, document standards, and data exchange protocols.
Health Technology Assessment (HTA): A multidisciplinary process that evaluates the medical, economic, social, and ethical implications of using a healthcare technology, including new drugs. It helps decision-makers understand the value and impact of adopting new therapies.
HIPAA (Health Insurance Portability and Accountability Act): a U.S. federal law enacted in 1996 that aims to protect patients’ sensitive health information from being disclosed without their consent or knowledge. HIPAA establishes standards for the privacy and security of health data, mandates secure handling of personal health information (PHI), and ensures that individuals can transfer and continue health insurance coverage when they change or lose jobs. It also sets guidelines for electronic health transactions and code sets, as well as national identifiers for providers, health insurance plans, and employers.
Indication: A specific medical condition or disease for which a drug is intended to be used based on its therapeutic properties and demonstrated efficacy and safety in clinical trials. Each indication typically requires separate regulatory approval.
Market Access: The processes and strategies pharmaceutical companies use to ensure their products are available and reimbursed by payers (e.g., insurance companies or government healthcare systems). It involves navigating regulatory requirements, demonstrating the value of the drug, negotiating pricing and reimbursement agreements, and overcoming barriers to ensure patients have access to the medication. Assessing market access risks and opportunities is essential for successfully commercializing a drug.
Mechanism of Action (MOA): The specific biochemical interactions through which a drug produces its pharmacological effect within the body. This includes how the drug binds to its target, modifies biological processes, and ultimately leads to therapeutic outcomes. Understanding the mechanism of action is crucial for designing and optimizing drugs for clinical use.
Modality: The specific type or category of therapeutic agent being developed. This can include small molecules, biologics (such as antibodies or proteins), gene therapies, cell therapies, or other types of therapeutic approaches aimed at treating diseases. Each modality may have distinct characteristics, mechanisms of action, and considerations in terms of development, manufacturing, and clinical application.
Monotherapy: Using a single drug or treatment regimen to treat a disease or condition. It contrasts with combination therapy, where multiple drugs are used simultaneously. Monotherapy is often employed when a single agent is sufficient to achieve the desired therapeutic effect or when testing the efficacy and safety of a new drug in clinical trials.
Neoadjuvant Treatment: Therapy given before the primary cancer treatment (such as surgery or radiation therapy) for a disease. Its purpose is to shrink the tumor or reduce the extent of the disease, making primary treatment more effective or feasible.
Orphan Drug: A pharmaceutical agent developed specifically to treat a rare medical condition known as an orphan disease or orphan indication. These diseases typically affect a small number of patients, and orphan drug designation by the FDA provides incentives to encourage their development, such as tax credits, market exclusivity, and exemption from certain fees.
Orphan Medicinal Product: The equivalent of an Orphan Drug in the EU. Like the FDA, the EMA provides incentives, such as market exclusivity, to encourage the development of orphan medicinal products.
Overall Response Rate (ORR): The proportion of patients in a clinical trial whose disease shows a predefined amount of partial response or complete response) following treatment with a drug. It is a key measure of the effectiveness of therapeutic intervention in treating a specific disease or condition.
Overall Survival: The length of time from starting treatment with a drug or therapy until the patient’s death from any cause. It is a critical endpoint in clinical trials and provides key information about the efficacy of a treatment in prolonging patients’ lives. Overall Survival is often considered the most reliable and direct measure of a treatment’s effectiveness in improving patient outcomes.
Pharmacodynamics (PD): The study of a drug’s effects on the body, including its mechanism of action, receptor binding, and physiological responses.
Pharmacokinetics (PK): The study of how the body absorbs, distributes, metabolizes, and excretes drugs.
Pharmacovigilance: The ongoing monitoring and assessment of drug safety once a product is on the market. It involves detecting, evaluating, and preventing adverse effects.
Phase I Clinical Trials: First trials of a new active ingredient or new formulations in humans, often carried out in healthy volunteers. They aim to make a preliminary evaluation of safety and an initial pharmacokinetic/ pharmacodynamic profile of the active ingredient.
Phase II Clinical Trials: The second phase of clinical trials is designed to determine activity and to assess the short-term safety of the active ingredient in patients suffering from a disease or condition for which it is intended. The trials are performed on a limited number of subjects and are often, at a later stage, of a comparative (e.g., placebo-controlled) design. This phase is also concerned with the determination of appropriate dose ranges/ regimens and (if possible) the clarification of dose-response relationships to provide an optimal background for the design of extensive therapeutic trials.
Phase III Clinical Trials: Trials in large (and possibly varied) patient groups to determine the short- and long-term safety-efficacy balance of formulation(s) of the active ingredient and assessing its overall and relative therapeutic value. These trials also establish any frequent adverse reactions and special features of the product e.g. clinically relevant drug interactions, and factors leading to differences in effect, such as age.
Phase IV Clinical Trials: Studies are performed after the pharmaceutical product has been marketed. They are based on the product characteristics on which the marketing authorization was granted. They normally take the form of post-marketing surveillance and assessment of therapeutic value or treatment strategies.
Placebo: An inactive substance (e.g., sugar pill) used in clinical trials as a control group to compare with the effects of an active drug.
Priority Review: An expedited review process by regulatory agencies, such as the FDA in the United States, for a drug application. This designation is granted to drugs that offer significant advancements in treatment or provide a therapy where no adequate alternative exists. Priority review aims to shorten the time from submission to approval, making important treatments available to patients sooner.
Progression Free Survival (PFS): The length of time during and after treatment with a drug or therapy that a patient lives with the disease without it progressing or worsening. It is typically measured from the start of treatment until the disease progresses or the patient experiences adverse effects that necessitate discontinuation of treatment. PFS is an important endpoint in clinical trials to evaluate the effectiveness of a treatment in controlling the progression of a disease.
Quality of Life (QoL) Assessment: Measures the impact of a drug on patients’ physical, emotional, and social well-being. Evaluating QoL outcomes in clinical trials provides insights into the holistic benefits and risks of a drug therapy.
Radiotherapy: The use of ionizing radiation, such as X-rays or gamma rays, to treat diseases, particularly cancer, by targeting and destroying cancer cells or tumors.
Randomized Controlled Trial (RCT): A type of clinical trial where participants are randomly assigned to different treatment groups (e.g., drug vs. placebo) to minimize bias and assess treatment efficacy.
Regulatory Hurdles: Regulatory requirements and standards that must be met for a drug candidate to progress through development stages and obtain marketing approval.
Risk-Benefit Analysis: Weighing the potential risks of a drug (e.g., side effects or safety concerns) against its potential benefits (such as efficacy and patient outcomes). It is crucial to determine whether the overall benefits of a drug outweigh its risks.
Risk Evaluation and Mitigation Strategies (REMS): These are comprehensive risk-management plans required by regulatory agencies for certain drugs. These are plans or actions aimed at reducing the potential risks associated with drug development and include implementing safety protocols, diversifying clinical trial populations, or exploring alternative drug formulations.
Risk Management Plan (RMP): A comprehensive strategy used by the EMA to identify, characterize, and minimize risks associated with a medicine. It includes safety monitoring, risk minimization measures, and communication plans.
Risk of Harm: The potential for adverse effects or negative consequences associated with using a pharmaceutical product, including known and potential risks to patients’ health and safety. This term encompasses risks identified during clinical trials, post-marketing surveillance, and regulatory assessments.
Safety Profile: An evaluation of the potential risks and adverse effects associated with a drug candidate, crucial for assessing its safety and tolerability in human subjects.
Safety Signal: An unexpected adverse event or pattern observed during clinical trials that warrants further investigation.
Second Line Treatment: The therapy given after initial treatment (first line treatment) has failed, either due to lack of effectiveness or because the disease has progressed. It is typically used when first line treatments are inadequate, and it may involve different drugs or therapeutic approaches.
Sponsor: An individual, organization, or company that takes responsibility for initiating, managing, and financing the clinical trial of a drug or medical device. The sponsor assumes legal and regulatory obligations to ensure the trial is conducted in accordance with applicable guidelines and standards.
Surrogate Endpoint: A biomarker that is intended to substitute for a clinical endpoint. A surrogate endpoint is expected to predict clinical benefit (or harm or lack of benefit or harm) based on epidemiological, therapeutic, pathophysiologic, or other scientific evidence.
Target: A specific molecule, protein, receptor, or pathway within the body that a drug aims to interact with or modify. Identifying and validating targets is a crucial step in developing new therapies, as it determines the focus of drug discovery efforts and potential treatment outcomes.
Therapeutic Area (TA): A specific medical field or category of diseases or conditions that a drug or therapy is intended to treat. Examples include oncology (cancer treatment), cardiology (heart diseases), neurology (brain and nervous system disorders), and infectious diseases. Drugs are often developed with a focus on a particular therapeutic area based on the disease mechanisms and patient needs within that area.
Third Line Treatment: A therapy administered after both first line treatment (initial therapy) and second line treatment have been unsuccessful or are no longer effective in managing a disease or condition. It represents a further attempt to control the disease using alternative medications or strategies.
Treatment-Related Discontinuations: The cessation of a drug or treatment regimen during a clinical trial or medical study due to adverse effects, lack of efficacy, or other treatment-related reasons.
Trial Arm: A specific group or segment within a clinical trial where participants receive a particular treatment or intervention. Different trial arms allow researchers to compare the effects and outcomes of various treatments or dosages, helping to evaluate the efficacy and safety of the drug being tested.
Toxicity: The harmful effects of a drug on living organisms.
Umbrella Trial: A clinical trial design where multiple sub-studies or cohorts are conducted under a single overarching protocol. Each sub-study typically evaluates a different drug or treatment regimen targeting a specific genetic mutation or biomarker across various cancer types or diseases. This approach allows for efficient testing of multiple therapies within a single trial framework, accelerating the evaluation and potential approval of targeted treatments.
Glossary of Risk Assessment Terms for the Pharmaceutical Industry
Competitive Landscape: The analysis and understanding of the market dynamics, including the presence, strategies, strengths, and weaknesses of current and potential competitors in the pharmaceutical industry.
Likelihood of Approval (LOA): Is closely related to POS and often used interchangeably. However, the focus is specifically on the final stages of development, i.e. Phase 3 trials and especially regulatory review. Some researchers use LOA for the overall probability of success, i.e. moving a drug from Phase 1 to approval, and therefore to express the same concept as POS.
Operational Risk: Operational risk involves logistical and management challenges in delivering robust clinical information about a candidate drug. It includes aspects like trial execution, data collection, and regulatory compliance.
Pharmaceutical Portfolio Optimization: The strategic process of evaluating and managing a company’s collection of drug candidates and products to maximize overall performance, profitability, and alignment with business objectives.
Portfolio Management: The strategic oversight and decision-making process aimed at effectively managing a company’s collection of drugs and therapies throughout its lifecycle. This includes prioritizing investments, allocating resources, and optimizing the balance of products to maximize overall performance, market competitiveness, and profitability.
Portfolio Strategy: The strategic process of selecting, prioritizing, and optimizing a company’s portfolio of drug candidates and products to maximize returns and minimize risks.
Probability of Launch: expresses the likelihood that a drug completes all clinical trial phases successfully, navigates regulatory approval and is launched.
Probability of Phase Transition: The probability that a drug candidate will successfully progress from one phase of clinical development to the next, indicating its potential for advancing toward eventual approval.
Probability of Pricing and Access Success: Measures an asset’s pricing and market access risk, considering the therapeutic area, region and expected launch timing.
Probability of Success (POS): navigate the entire development process and eventually be approved and marketed. It includes all stages from discovery through preclinical and clinical trials to regulatory approval and is a measure of the cumulative risks at each stage, including scientific, clinical, regulatory and commercial factors.
Probability of Regulatory Success: the probability that a drug successfully makes it through the regulatory review process and achieves market approval.
Probability of Technical and Regulatory Success (PTRS): A quantitative assessment of the likelihood that a drug candidate will meet both technical developmental milestones and regulatory requirements for approval.
Probability of Technical Success: The probability that a drug successfully completes the clinical trials. The focus is specifically on the technical challenges a drug candidate faces during clinical trials.
Return on Investment (ROI): In drug development, ROI refers to the financial gain or loss generated from investment in a drug development project. It is a critical metric for assessing the profitability and success of drug development efforts.
Risk Communication: The exchange or sharing of information about risk between the decision-maker and other stakeholders.
Risk Analysis: The process of understanding the nature of risk and determining the level of risk.
Risk Assessment: The process of identifying, evaluating, and estimating the levels of risk involved in a process, determining their significance, and determining an acceptable level of risk.
Risk Evaluation: The process of comparing the estimated risk against given risk criteria to determine the significance of the risk.
Risk Management: The systematic application of management policies, procedures, and practices to the tasks of identifying, analyzing, assessing, controlling, and monitoring risk.
Glossary of AI Terms
Artificial Intelligence (AI): The field of computer science focused on creating systems capable of performing tasks that typically require human intelligence. These tasks include learning, reasoning, problem-solving, perception, language understanding and decision-making. AI systems use algorithms and models to process data and make predictions or decisions.
Artificial Intelligence Explainability: Refers to the ability to understand and interpret the decision-making processes and outputs of AI systems. It involves making the operations of AI algorithms transparent and comprehensible to humans, enabling users to trust and effectively interact with AI by understanding how and why decisions or predictions are made.
Artificial Intelligence Model: a computational system designed to perform specific tasks or make predictions by learning patterns from data. It uses algorithms and statistical techniques to analyze and interpret information, mimicking aspects of human intelligence. AI models can range from simple decision trees to complex neural networks.
Data Mining: Also known as knowledge discovery in data, is the process of uncovering patterns and other valuable information from large data sets.
Data Transparency: Data transparency involves sharing clinical trial data and results openly with stakeholders, including researchers, regulators, and the public. Transparent data reporting is essential for assessing the reliability and credibility of findings in drug development.
Deep Learning: A subset of machine learning that uses artificial neural networks with multiple layers to learn and extract higher-level features from data.
FAIR Principles: A set of guidelines aimed at making AI models and datasets Findable, Accessible, Interoperable, and Reusable. These principles, originally developed for scientific data management, are being adapted to the field of artificial intelligence to promote better practices in AI research and development.
Generative AI: A subset of artificial intelligence that focuses on creating new content, such as images, text, music, or other data, based on learned patterns from existing data. It utilizes models like Generative Adversarial Networks (GANs) or transformer-based models to generate novel outputs that mimic the characteristics of the input data it was trained on.
Large Language Model (LLM): A large language model (LLM) is a type of artificial intelligence model that has been trained to recognize and generate vast quantities of written human language.
Machine Learning (ML): A branch of artificial intelligence that involves the development of algorithms and statistical models enabling computers to learn from and make predictions or decisions based on data. Machine learning systems are not explicitly programmed to perform a task, but improve their performance as they are exposed to more data over time. This process involves training models on large datasets to recognize patterns and make inferences.
Natural Language Processing: a subfield of computer science and artificial intelligence that uses machine learning to enable computers to understand and communicate with human language.
Neural Network: A neural network is an interconnected group of artificial neurons that processes information using a computational model inspired by the structure and function of biological neural networks in animal brains. Neural networks can learn from data, recognize patterns, and perform complex tasks such as image recognition, natural language processing, and predictive modeling.
Shapley Additive Explanations (SHAP) Analysis: A method in machine learning and data science used to explain the output of a machine learning model by attributing the contribution of each feature to the prediction. It provides insights into how each feature influences the model’s output and helps interpret its decision-making process.
Structured Data: Refers to data that is organized and formatted in a specific way to make it easily readable and understandable by both humans and machines. This is typically achieved through the use of a well-defined schema or data model, which provides a structure for the data.
Unstructured Data: Data which does not conform to a data model and has no easily identifiable structure such that it can not be used by a computer program easily. Unstructured data is not organized in a predefined manner or does not have a pre-defined data model, thus it is not a good fit for a mainstream relational database.