Frequently Asked Questions for Drug & Product Development

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What is the 21st Century Cures Act?

Legislation designed to help accelerate medical product development and bring new innovations and advances to patients who need them faster and more efficiently.

A

What is an active pharmaceutical ingredient (API)?

Any substance or mixture of substances that is intended to be used in the manufacture of a drug product and that becomes an active ingredient of the drug product when used in the production of the drug.

What is adaptive design?

A clinical trial study design that may be more flexible by utilizing results accumulated during a trial to modify the trial’s course in accordance with pre-specified rules.

What is an adverse drug reaction (ADR)?

Any harmful or unintended response to a medicinal product related to any dose.

What is an adverse event (AE)?

Any untoward medical occurrence associated with the use of a medicinal product in humans, whether or not it is considered drug related.

What is the Association of American Cancer Institutes (AACI)?

An association of 100 of the top academic and independent cancer research centers in North America.

B

What are basket studies?

A more recent development for oncology clinical trials, also known as bucket studies, these studies include patients who have a certain genetic mutation in common regardless of the site or origin of cancer in the body.

C

What is a case report form (CRF)?

A paper or electronic questionnaire used in the context of a clinical trial to collect data from study participants.

What is the Center for Biologics Evaluation and Research (CBER)?

The branch of the U.S. Food and Drug Administration (FDA) that is responsible for regulating biological products for human use under applicable federal laws.

What is the Center for Cancer Research (CCR)?

The largest division of the intramural research program of the National Cancer Institute (NCI), this center comprises approximately 250 basic and clinical research groups.

What is the Center for Devices and Radiological Health (CDRH)?

The branch of the FDA that is responsible for premarket approval of all medical devices, as well as oversight of the manufacturing, performance, and safety of these devices.

What is the Center for Drug Evaluation and Research (CDER)?

The branch of the FDA that is responsible for monitoring the safety and efficacy of over-the-counter and prescription drugs, including biologics and generics.

What is the Centers for Medicare & Medicaid Services (CMS)?

A federal agency within the U.S. Department of Health and Human Services (DHHS) that administers Medicare and works with state governments to administer Medicaid and the Children’s Health Insurance Program (CHIP). CMS is also responsible for ensuring health insurance portability standards, long-term care facility quality standards, and clinical laboratory quality standards under the Clinical Laboratory Improvement Amendments (CLIA).

What is a centralized clinical trials office (CCTO) or clinical trials office (CTO)?

A central office that provides infrastructure and operational support for researchers, sponsors, and patients involved in the conduct of clinical trials.

What is the Clinical and Translational Science Award (CTSA) Program?

A program designed to develop innovative solutions that improve the efficiency, quality, and impact of the process for translating laboratory, clinic, and community observations into interventions that improve the health of individuals and the public.

What is the Clinical Data Acquisition Standards Harmonization (CDASH) guidance?

Part of the clinical data interchange standards consortium (CDISC) initiative, this is a set of best practices for developing case report forms.

What is clinical data management (CDM)?

A critical process in clinical research, which leads to generation of high-quality, reliable, and statistically sound data from clinical trials.

What is a clinical data management system (CDMS)?

A tool used in clinical research to manage the data generated in a clinical trial.

What is the Clinical Data Update System (CDUS)?

The main repository of clinical trial data for the NCI’s Division of Cancer Treatment and Diagnosis (DCTD) and Division of Cancer Prevention (DCP).

What is a clinical research associate (CRA)?

This role is responsible for organizing and administering clinical trials of new or current drugs in order to assess the benefits and risks.

What is a clinical research coordinator (CRC)?

This role is responsible for conducting clinical trials according to good clinical practice guidelines.

What is a clinical study report (CSR)?

A detailed document that integrates the clinical and statistical description, presentations, analyses, and all background information related to a clinical study.

What is a clinical trial management system (CTMS)?

A software system used by the biopharmaceutical industry to manage clinical trials in clinical research.

What is the Clinical Trials Reporting Program (CTRP)?

A comprehensive database that includes information on all NCI-supported clinical trials.

What is the Code of Federal Regulations (CFR)?

A system of general and permanent rules and regulations published in the Federal Register by the U.S. federal government.

What is a cohort study?

A longitudinal study that is designed to follow a group of subjects over time.

What are the Common Terminology Criteria for Adverse Events (CTCAE)?

Also called common toxicity criteria, this is a set of criteria developed by the NCI to standardize the classification of adverse effects of drugs used in cancer therapy.

What is a contract manufacturing organization (CMO)?

Also called a contract development and manufacturing organization (CDMO), this is a company that provides drug development and manufacturing services to other companies in the pharmaceutical industry on a contract basis.

What is a contract research organization (CRO)?

A company that provides clinical trial support to the pharmaceutical, biotechnology, and medical device industries in the form of research services outsourced on a contract basis.

What is a Corrective and Preventive Action (CAPA) system?

A system to collect information, analyze information, identify, and investigate product and quality problems, and take appropriate and effective corrective and/or preventive action to prevent their recurrence.

D

What is a data and safety monitoring board (DSMB)?

Also known as a data monitoring committee (DMC) or data and safety monitoring committee (DSMC), this is an independent group of experts who monitor study conduct and safety on an ongoing basis throughout a clinical trial.

What is a data manager (DM)?

In the context of a clinical trial, this role is responsible for ensuring that all data is collected, managed, and reported accurately.

What is a data monitoring committee (DMC)?

Also known as a data and safety monitoring board (DSMB) or data and safety monitoring committee (DSMC), this is an independent group of experts who monitor study conduct and safety on an ongoing basis throughout a clinical trial.

What is the Department of Health and Human Services (HHS)?

Also known as the Health Department, this department of the U.S. federal government is responsible for protecting the health of all Americans and providing essential human services.

E

What is an electronic case report form (eCRF)?

An electronic questionnaire used in clinical trial research to collect data from each study participant.

What is electronic data capture (EDC)?

The use of systems to collect clinical trial data in electronic form as opposed to paper form.

What is an electronic health record (EHR)?

A digital version of a patient’s health records, including information from all clinicians, laboratories, and health care institutions involved in the patients’ care.

What is an electronic medical record (EMR)?

A digital version of a patient’s medical and treatment history, usually from one health care practice.

What is an electronic patient-reported outcome (ePRO)?

Any patient-reported outcome that is collected by electronic methods.

What is an electronic trial master file (eTMF)?

A trial master file that is in digital format and contains the essential data from a clinical trial.

What is an ethics committee (EC)?

An independent body that is responsible for ensuring that medical experimentation and research on human subjects is conducted in an ethical manner in accordance with national and international law.

What is the European Medical Device Regulation (EU MDR)?

A European Union (EU) regulation which ensures high standards of quality and safety for medical devices being produced in or supplied into Europe.

F

What is Fast Track (FT) designation?

A process designed to facilitate the development and expedite the review of drugs to treat serious conditions and to address an unmet medical need.

What is Federalwide Assurance (FWA)?

Documentation of an institution’s assurance of compliance with U.S. federal regulations for the protection of human subjects in research.

What is Findable, Accessible, Interoperable, Reusable (FAIR)?

A set of guiding principles for scientific data management which were designed to enhance the ability of data or tools from non-cooperating resources to integrate or work together with minimal effort.

What is the Food and Drug Administration (FDA)?

A federal agency responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices, as well as by ensuring the safety of the food supply, cosmetics, and radiation-emitting products.

G

What is a general clinical research center (GCRC)?

Also known as a clinical research center, this is any designated medical facility used to conduct clinical research.

What is the General Data Protection Regulation (GDPR)?

A regulation in EU law on data protection and privacy for all individual citizens of the EU and the European Economic Area (EEA).

What is good clinical practice (GCP)?

An international ethical and scientific quality standard for designing, conducting, recording, and reporting trials that involve the participation of human subjects.

What is good documentation practice (GDP or GDocP)?

A term used in the pharmaceutical and medical device industries to describe standards and guidelines used to record raw data entries in a legible, traceable, and reproducible manner.

H

What is the Health Insurance Portability and Accountability Act (HIPAA)?

A law enacted in August 1996 which mandates industry-wide standards for improving the portability and continuity of health insurance coverage and ensuring the privacy, protection, and confidential handling of protected health information.

I

What is an in-house clinical research associate (IHCRA)?

This office-based role is responsible for supporting clinical research associates who are in the field.

What is an independent ethics committee (IEC)?

Also known as an institutional review board, this is a committee charged with protecting the rights and safety of clinical trial participants by reviewing all relevant study materials.

What is an informed consent form (ICF)?

A document containing information that allows individuals to make an informed decision about whether or not to participate in a clinical trial.

What is an institutional review board (IRB)?

Also known as an independent ethics committee, this is a committee charged with protecting the rights and safety of clinical trial participants by reviewing all relevant study materials.

What is intention-to-treat (ITT) analysis?

A technique used in randomized controlled trials where the results of the study are analyzed based on a participant’s initial treatment assignment and not the treatment eventually received.

What is an interactive response system (IRS)?

A standard industry term for interactive voice response (IVR) and interactive web response (IWR) systems that allow detection of voice, text, and web for gathering data related to clinical research.

What is the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)?

This initiative brings together the regulatory authorities and pharmaceutical industry to discuss scientific and technical aspects of drug registration.

What is the International Council for Harmonisation and Good Clinical Practice (ICH GCP E6 [R2])?

An amended guidance to encourage implementation of improved and more efficient approaches to clinical trial design, conduct, oversight, recording, and reporting, while continuing to ensure human subject protection and reliability of trial results.

What is an investigational device exemption (IDE)?

This approval allows an investigational device to be used in a clinical study for the purpose of collecting data on safety and efficacy.

What is an investigational new drug application (IND)?

An FDA program by which a biopharmaceutical company obtains permission to start human clinical trials and to ship an experimental drug across state lines (usually to clinical investigators) before a marketing application for the drug has been approved.

What is an investigator-initiated trial (IIT)?

A clinical study initiated, developed, designed, and managed by a qualified sponsor who assumes sole responsibility for conduct and management of the study.

What is an Investigator Site File (ISF)?

A file containing the essential clinical trial documents necessary for the principal investigators and the research team.

L

What is a local regulatory affairs associate (LRAA)?

A professional who is responsible for ensuring compliance with local government regulations.

What is long-term follow up (LTFU)?

In the context of a clinical trial, this refers to continuing monitoring to evaluate the long-term effects of a treatment or intervention.

N

What is the National Cancer Institute (NCI)?

Part of the National Institutes of Health (NIH), this agency leads, conducts, and supports cancer research across the U.S.

What is the National Institutes of Health (NIH)?

Part of the HHS, this is the largest biomedical research agency in the world.

What is the National Library of Medicine (NLM)?

Operated by the U.S. federal government, this is the world’s largest biomedical library.

What is a natural history study?

A study that follows a group of people over time who have, or are at risk of developing, a specific medical condition or disease.

What is a new drug application (NDA)?

A vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S.

What is a normal healthy volunteer (NHV)?

A person with no known significant health problems who participates in clinical research to test a new drug, device, or intervention.

O

What is an observational study?

A fundamental part of epidemiological research, this is a study in which researchers observe the effect of a diagnostic test, treatment, intervention, or risk factor without trying to change or control the variable under investigation.

What is the Office for Human Research Protections (OHRP)?

An office within the HHS that is responsible for protecting the rights, welfare, and well-being of human subjects involved in research conducted or supported by HHS.

What is an Office of Clinical Trials (OCT)?

An office within an institution that is responsible for supporting the execution and management of clinical research.

What is an orphan drug?

Defined by the Orphan Drug Act (ODA), this refers to a biological product granted special status to treat a rare disease or condition upon the request of a sponsor.

P

What is a pilot study?

A small feasibility study that is usually performed to test hypotheses in preparation for a larger interventional clinical trial.

What is a platform study?

A clinical trial with a single master protocol in which multiple treatments are evaluated simultaneously.

What is a principal investigator (PI)?

The primary individual responsible for the preparation, conduct, and administration of laboratory study or clinical trial.

What is protected health information (PHI)?

Under U.S. law, this refers to all individually identifiable health information, including demographic data, health status, test results, insurance information, and any other information used to identify a patient or provide healthcare services or coverage.

What is a protocol coordinator (PC)?

In the context of a clinical trial, this role is responsible for conducting the informed consent process and ensuring compliance with the protocol.

What is a protocol review and monitoring committee (PRMC)?

A committee responsible for assessing and overseeing the scientific merit and integrity of a clinical trial.

What is a protocol review and monitoring system (PRMS)?

Mandated by the NCI for every Cancer System, this is a system used to assess the scientific merit and feasibility of all clinical trial protocols studying subjects diagnosed with, or at risk for, cancer.

Q

What is a qualifying clinical trial (QCT)?

A trial that meets the requirements set forth by the CMS for coverage of routine costs.

What is quality control (QC)?

In the context of a clinical trial, this refers to the procedures utilized to ensure the protection of human subjects and the reliability of the data generated.

R

What is a randomized controlled trial (RCT)?

An intervention study in which a group of subjects with similar characteristics are randomized to receive one of several defined interventions.

What is real-world data (RWD)?

Data relating to patient health status and/or the delivery of health care collected from a variety of sources, such as electronic health records, claims and billing activities, product and disease registries, patient-generated data, data gathered from other sources (e.g., mobile devices).

What is real-world evidence (RWE)?

Clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data. RWE can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective).

What is a registry?

A data collection tool typically used to better understand long-term trends in a specific population, such as patients with a particular disease or exposure to a certain treatment.

S

What is a scientific review board (SRB)?

Also called a scientific review committee or scientific review panel, this is a group of clinicians, researchers, and other experts that evaluates the detailed plan of a clinical trial for scientific quality and appropriate study design.

What is a scientific review committee (SRC)?

Also called a scientific review board or scientific review panel, this is a group of clinicians, researchers, and other experts that evaluates the detailed plan of a clinical trial for scientific quality and appropriate study design.

What is a serious adverse event (SAE)?

An untoward medical occurrence associated with the use of a medicinal product in humans which is life-threatening or results in death, initial or prolonged hospitalization, disability or permanent damage, or a congenital anomaly or birth defect.

What is a site management organization (SMO)?

Also known as a trial management organization, this is an organization that provides clinical trial-related support services to a biopharmaceutical company, medical device company, contract research organization, or clinical site.

What is source document review or source data review (SDR)?

This refers to the review of source documentation to check quality, review compliance with the protocol, and ensure critical processes and source documentation are adequate.

What is source document verification (SDV)?

A verification of the conformity of the data presented in case report forms or other data collection systems with source data. This is conducted to ensure that the data collected are reliable and allow reconstruction and evaluation of the trial.

What are Specialized Programs of Research Excellence (SPOREs)?

Cancer research grants focused on a specific organ site or on a group of highly related cancers. SPOREs are designed to support rapid, efficient translation of basic scientific findings and to determine the biological basis for observations made in individuals with cancer or in populations at risk for cancer.

What is a standard operating procedure (SOP)?

Detailed, written instructions to record routine operations, processes, and practices followed within a business organization to ensure uniformity and compliance with institutional, federal, and state guidances.

What is a study coordinator (SC)?

This role is responsible for supporting the management and coordination of a clinical research study.

What is the Study Data Tabulation Model (SDTM)?

This defines a standard structure for study data tabulations that are to be submitted as part of an application to a regulatory authority such as the FDA.

What is a subject visit template (SVT)?

A template designed to ensure that adequate and complete documentation of a study visit is captured.

What is a suspected unexpected serious adverse reaction (SUSAR)?

A term that refers to an adverse event that occurs in a study participant and which is assessed by the sponsor or study investigator as being unexpected, serious, and likely to have a causal relationship with the study drug.

T

What is a trial management organization (TMO)?

Also known as a site management organization, this is an organization that provides clinical trial-related support services to a biopharmaceutical company, medical device company, contract research organization, or clinical site.

What is a trial master file (TMF)?

The collection of essential documents which allows the conduct of a clinical trial to be reconstructed and evaluated.

U

What are umbrella studies?

Studies designed to test the impact of different drugs on different mutations in a single type of cancer.

What is an unanticipated adverse device effect (UADE)?

Any serious adverse effect on health or safety or any life-threatening problem or death caused by, or associated with, a device, if that adverse effect was not previously identified in the investigational device exemption. This term may also be used for any other unanticipated serious problem associated with a device relative to the rights, safety, or welfare of study participants.

What is an unexpected adverse drug reaction (UADR)?

An adverse reaction for which the nature or severity is not consistent with information in the relevant source documents.