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Regulatory Glossary & taxonomy
Evolving Terminologies for Emerging Technologies
Comments? Questions? Revisions?
Mary Chitty MSLS
Last revised January 09, 2020

Drug discovery term index   Ethics  Regulatory Affairs  is a sub-category of Drug discovery & development 
Related glossaries include
Biologics    Biomaterials & medical devices     
Clinical trials  
Drug safety & pharmacovigilance       Molecular Medicine    Pharmacogenomics   
Clinical informatics    
Research Technologies:    Bioprocessing  

21 CFR Part 11, Electronic records, Electronic signatures: This guidance is intended to describe the Food and Drug Administration's (FDA's) current thinking regarding the scope and application of part 11 of Title 21 of the Code of Federal Regulations; Electronic Records; Electronic Signatures (21 CFR Part 11).2

510K: Biomaterials & Medical Devices

accelerated approval: The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint.  A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. FDA Accelerated Approval Program

Advanced therapy medicinal products (ATMPs): medicines for human use that are based on genes, tissues or cells. They offer groundbreaking new opportunities for the treatment of disease and injury.  ATMPs can be classified into three main types:  gene therapy medicines: these contain genes that lead to a therapeutic, prophylactic or diagnostic effect. They work by inserting 'recombinant' genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources;  somatic-cell therapy medicines: these contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases; tissue-engineered medicines: these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue; In addition, some ATMPs may contain one or more medical devices as an integral part of the medicine, which are referred to as combined ATMPs. An example of this is cells embedded in a biodegradable matrix or scaffold. European Medicines Agency

analyte specific reagents: antibodies, both polyclonal and monoclonal, specific receptor proteins, ligands, nucleic acid sequences, and similar reagents which, through specific binding or chemical reaction with substances in a specimen, are intended for use in a diagnostic application for identification and quantification of an individual chemical substance or ligand in biological specimens. CFR Title 21

Wikipedia     See also Laboratory Developed Tests

bioavailability, bioequivalence: Drug delivery   See also therapeutic equivalency

biogenerics: A biopharmaceutical or other biological product ... that has emerged from patent protection and can be manufactured by a party other than the original developer using either identical or different manufacturing processes, for FDA approval the product must be bioequivalent or comparable to the original innovative product.
Broader terms: follow ons, generic drugs, me toos.   Related terms: biosimilars, 
analogue based drug discovery,

biological products, which are biological prescription drugs that are demonstrated to be “highly similar” (biosimilar) to or “interchangeable” with an FDA-approved biological product. 
Related terms: analogues, biogenerics, follow ons, generics, me-toos 

borderline products: 
European Union

brand name drug: A drug marketed under a proprietary, trademark- protected name. Glossary, Drugs@FDA, CDER, 2004 See also proprietary drug, proprietary name

CBER Center for Biologics Evaluation and Research: CBER is the Center within FDA that regulates biological products for human use under applicable federal laws, including the Public Health Service Act and the Federal Food, Drug and Cosmetic Act. CBER protects and advances the public health by ensuring that biological products are safe and effective and available to those who need them.

CBER has undergone an internal restructuring.  The new CBER structure includes the Office of Blood Research and Review (OBRR), the Office of Vaccines Research and Review (OVRR), and the Office of Tissues and Advanced Therapies (OTAT, formerly known as the Office of Cellular, Tissue and Gene Therapies, or OCTGT).  The formation of OTAT involves the transfer of OBRR’s Division of Hematology Clinical Review and Division of Hematology Research and Review, along with appropriate support staff, to OCTGT to constitute the new office.  The products now regulated by OTAT include all purified and recombinant versions of therapeutic proteins for hematology.  Antivenins have also been transferred to OTAT.  This new organizational structure became effective on October 16, 2016.   FDA, CBER

CDER Center for Drug Evaluation and Research:  The Center for Drug Evaluation and Research (CDER) performs an essential public health task by making sure that safe and effective drugs are available to improve the health of people in the United States.  As part of the U.S. Food and Drug Administration (FDA), CDER regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs. This work covers more than just medicines. For example, fluoride toothpaste, antiperspirants, dandruff shampoos and sunscreens are all considered "drugs."

children in research: Guidelines on involving human subjects, including, but not limited to, clinical trials, supported or conducted by the NIH. NIH Policy and Guidelines on the inclusion of children as participants in research involving human subjects, NIH, US Mar. 8, 1998

CLIA Clinical Laboratory Improvement Amendments: Regulates all laboratory testing (except research) performed on humans in the U.S. Part of the Centers for Medicare and Medicaid Services (CMS). .  

Clinical Research Policy Analysis and Coordination: CRPAC: The Re-engineering the Clinical Research Enterprise Roadmap set of initiatives is also addressing the difficulties clinical researchers confront in satisfying the multiple requirements of diverse regulatory and policy agencies. Clinical researchers must understand and fulfill these varying requirements that often overlap and may even contradict one another. NIH aims to take a leadership role in working with other agencies, institutional review boards, and other organizations to develop better processes and to standardize requirements for reporting adverse events, human subjects protections, privacy and conflict-of-interest policies, and standards for electronic data submission. Harmonizing policies and reporting requirements will help minimize unnecessary burdens that slow research, while at the same time enhancing patient protections. NIH Common Fund

combination products: A combination product is a product composed of any combination of a drug and a device; a biological product and a device; a drug and a biological product; or a drug, device, and a biological product. Under 21 CFR 3.2 (e), a combination product is defined to include: 1. A product comprised of two or more regulated components (i.e., drug/device, biologic/device, drug/biologic, or drug/device/biologic) that are physically, chemically, or otherwise combined or mixed and produced as a single entity [often referred to as a “single-entity” combination product]; 2. Two or more separate products packaged together in a single package or as a unit and comprised of drug and device products, device and biological products, or biological and drug products [often referred to as a “co-packaged” combination product]; 3. A drug, device, or biological product packaged separately that according to its investigational plan or proposed labeling is intended for use only with an approved individually specified drug, device, or biological product where both are required to achieve the intended use, indication, or effect and where, upon approval of the proposed product, the labeling of the approved product would need to be changed (e.g., to reflect a change in intended use, dosage form, strength, route of administration, or significant change in dose) [often referred to as a “cross-labeled” combination product]; or 4. Any investigational drug, device, or biological product packaged separately that according to its proposed labeling is for use only with another individually specified investigational drug, device, or biological product where both are required to achieve the intended use, indication, or effect [another type of “cross-labeled” combination product]. FDA, Frequently asked questions aboout Combination Products

Committee on Human Medicines:  The MHRA was set up in April 2003 from a merger of the Medicines Control Agency and the Medical Devices Agency. The MHRA is the government agency which is responsible for ensuring that medicines and medical devices work, and are acceptably safe. The MHRA is an executive agency of the Department of Health.  

common technical document: The agreement to assemble all the Quality, Safety and Efficacy information in a common format (called CTD - Common Technical Document ) has revolutionised the regulatory review processes, led to harmonised electronic submission that, in turn, enabled implementation of good review practices. For industries, it has eliminated the need to reformat the information for submission to the different ICH regulatory authorities.  International Conference on Harmonisation 

compassionate use: See expanded access

cosmeceuticals: The term "cosmeceutical" has no meaning under the law. While the Federal Food, Drug, and Cosmetic Act (FD&C Act) does not recognize the term "cosmeceutical," the cosmetic industry uses this word to refer to cosmetic products that have medicinal or drug-like benefits. A product can be a drug, a cosmetic or both. The FD&C Act defines drugs as those products that cure, treat, mitigate or prevent disease or that affect the structure or function of the human body, if a product makes such claims it will be regulated as a drug. Cosmetics are intended to beautify, promote attractiveness, alter appearance or cleanse; they are not approved by FDA for sale nor are they intended to effect structure or function of the body. 2017

drug: Any substance which when absorbed into a living organism may modify one or more of its functions. The term is generally accepted for a substance taken for a therapeutic purpose, but is also commonly used for abused substances. Synonymous with medicine, pharmaceutical.  IUPAC Compendium

A drug is any substance presented for treating, curing or preventing disease in human beings or in animals. A drug may also be used for making a medical diagnosis or for restoring, correcting, or modifying physiological functions (e.g., the contraceptive pill). IUPAC Medicinal Chemistry

Should be considered synonymous with investigational (medicinal) product, medicinal product and pharmaceutical (including vaccines and other biological products). E15 terminology in Pharmacogenomics
Narrower terms: specialty pharmaceuticals. Compare biologics. Related terms: CDER, FDA.
In the US biologics and drugs are regulated by different Centers at the FDA.

effective: a drug product must be found to be effective and safe before it may be approved for general marketing. The Act explicitly gives the legal definition of the evidence necessary for the Agency to determine that a drug product has been found to be effective; that standard is “substantial evidence of effectiveness,” and is defined as “evidence consisting of adequate and well-controlled investigations, including clinical investigations, by experts qualified by scientific training and experience to evaluate the effectiveness of the drug involved, on the basis of which it could fairly and responsibly be concluded by such experts that the drug will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in the labeling or proposed labeling thereof.”  FDA: Evidentiary Standards for Drug Development and Approval Russell Katz  NeuroRx. 2004 Jul; 1(3): 307–316.  doi:  10.1602/neurorx.1.3.307 PMCID: PMC534930 htttps:// 

electronic signatures: This guidance is intended to describe the Food and Drug Administration's (FDA's) current thinking regarding the scope and application of part 11 of Title 21 of the Code of Federal Regulations; Electronic Records; Electronic Signatures (21 CFR Part 11).2  FDA, CBER, CDER< Part 11, Electronic Records; Electronic Signatures — Scope and Application

EMEA: European Agency for the Evaluation of Medicinal Products now EMA: European Medicines Agency

expanded access: Expanded access, sometimes called "compassionate use," is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options. FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials. FDA Access to Investigational drugs outside a clinical trials   

exploratory IND: Exploratory IND studies usually involve very limited human exposure and have no therapeutic or diagnostic intent. Such studies can serve a number of useful goals. For example, an exploratory IND study can help sponsors • Determine whether a mechanism of action defined in experimental systems can also be observed in humans (e.g., a binding property or inhibition of an enzyme)  • Provide important information on pharmacokinetics (PK) • Select the most promising lead product from a group of candidates designed to interact with a particular therapeutic target in humans, based on PK or pharmacodynamic (PD) properties.  FDA, CDER, Draft Guidance for Industry, Investigators and Reviewers, Exploratory IND Studies, 2006  Related term: Phase zero, Phase 0

fast- track: Speeding the development and availability of drugs that treat serious diseases are in everyone's interest, especially when the drugs are the first available treatment or have advantages over existing treatments.  The Food and Drug Administration (FDA) has developed three distinct and successful approaches to making such drugs available as rapidly as possible: Priority Review, Accelerated Approval, and Fast Track.  Because each of these approaches implies speed, there can be confusion about the specific meaning of each and the distinctions among them. ... Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious diseases and fill an unmet medical need.  The purpose is to get important new drugs to the patient earlier.

FDA: Food and Drug Administration, US regulatory agency   Narrower terms: CBER, CDER, CDRH

FDA Compliance Manuals

FDA Drug Development and Review definitions: 2010 

FDA Regulatory Procedures Manual: a reference manual for FDA personnel. It provides FDA personnel with information on internal procedures to be used in processing domestic and import regulatory and enforcement matters.  Regulatory Procedures Manual Glossary

FDAMA: The Food and Drug Administration Modernization Act (FDAMA), enacted Nov. 21, 1997, amended the Federal Food, Drug, and Cosmetic Act relating to the regulation of food, drugs, devices, and biological products. With the passage of FDAMA, Congress enhanced FDA's mission in ways that recognized the Agency would be operating in a 21st century characterized by increasing technological, trade and public health complexities.

FDA drug approvals: 1995 - present

follow ons: The development of so-called ‘me-too’, or ‘follow-on’, drugs by the pharmaceutical industry has been viewed by some as duplicative and wasteful, while others have argued that these drugs often provide needed therapeutic options and inject some price competition into the marketplace. This study examines data on the trends in the speed with which competitive entry has occurred in the pharmaceutical marketplace and the competitive nature of the industry’s development of these drugs. The Economics of Follow-on Drug Research and Development  Trends in Entry Rates and the Timing of Development Joseph A. DiMasi and Cherie Paquette Pharmacoeconomics 2004; 22 Suppl. 2: 1-14 1170-7690/04/0002-0001

GCP Good Clinical Practice:  At its core, Good Clinical Practice (GCP) is a set of broad regulatory requirements, standards, and recommendations that apply to thousands of highly specific tasks, processes, and roles in the conduct of clinical research. It is important to remember that much of the practical standards used in the conduct of clinical trials are "best practices" derived from regulations, guidances, and industry standards and practices and not all found in black and white in the regulations. Given the disparity between the detailed nature of clinical trial processes and tasks and the general GCP requirements and standards under which they occur, it is not surprising that interpreting and implementing GCP standards continue to represent challenges for the pharmaceutical, biotechnology, and medical device industries. Barnett International, Good Clinical Practice: A Question & Answer Reference Guide, 

Good Clinical Practice FDA The Office of Good Clinical Practice is the focal point within FDA for Good Clinical Practice (GCP) and Human Subject Protection (HSP) issues arising in human research trials regulated by FDA.

GCP FDA Adherence to the principles of good clinical practices (GCPs), including adequate human subject protection (HSP) is universally recognized as a critical requirement to the conduct of research involving human subjects.  Many countries have adopted GCP principles as laws and/or regulations.  The Food and Drug Administration’s (FDA’s) regulations for the conduct of clinical trials, which have been in effect since the 1970s, address both GCP and HSP.  These FDA regulations and guidance documents are accessible from this site.  International GCP guidance documents on which FDA has collaborated and that have been adopted as official FDA guidance are also be found here.  Finally, this site includes links to other sites relevant to the conduct of clinical trials, both nationally and internationally.  Broader term: GxP

generic drugs: Drugs whose drug name is not protected by a trademark. They may be manufactured by several companies. MeSH, 1992

Generic drug products are defined as drug products that are identical to an innovative (brand-name) drug which is the subject of an approved NDA with regard to active ingredient(s), route of administration, dosage form, strength, and conditions of use. Since ANDA submissions for generic applications do not require lengthy clinical evaluation of the generic drugs under investigation (see Table 1), the price of generics are usually much lower than that of the originals. On average, it is about 20% of the price of the brand-name originals. In 1984, the United States Food and Drug Administration (FDA) was authorized to approve generic drug products under the Drug Price Competition and Patent Term Restoration Act. The purpose is to make less expensive, safe, and equally efficacious generics available to general public after the expiration of patent protection of expensive brand-name drugs. For approval of generic drug products, the FDA requires that evidence of average bioequivalence in drug absorption be provided through the conduct of bioavailability and bioequivalence studies. Bioequivalence assessment is considered as a surrogate for clinical evaluation of the therapeutic equivalence of drug products. Bioavailability and Bioequivalence in Drug Development. Wiley Interdiscip Rev Comput Stat. 2014;6(4):304-312.   Narrower terms: authorized generics, biogenerics

GLP Good Laboratory Practice:   Good Laboratory Practices: Guide to Compliance, provides clear recommendations for performing preclinical laboratory studies according to 21CFR58 and the OECD Principles of Good Laboratory Practice. The Guide includes templates for SOPs and other forms that can be copied and used directly in the laboratory, including a full set of GLP inspection sheets.  http://www. EducationalServices_ Publication.aspx?p=7802&id= 97212 
Wikipedia  Non-human studies Broader term: GxP

guidance documents FDA:   Guidance documents represent FDA's current thinking on a topic.  They do not create or confer any rights for or on any person and do not operate to bind FDA or the public.  You can use an alternative approach if the approach satisfies the requirements of the applicable statutes and regulations.

GxP: GxP:  A general term for Good Practice quality guidelines and regulations. These guidelines are used in many fields, including the pharmaceutical and food industries. accessed  Jan 11, 2011 
Narrower terms: GCP, GLP, GMP

harmonization of pharmaceuticals regulations: See ICH International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use 

HIPAA Health Insurance Portability and Accounting Act: Health & Human Services, US 

HIPAA for individuals   Most of us believe that our medical and other health information is private and should be protected, and we want to know who has this information. The Privacy Rule, a Federal law, gives you rights over your health information and sets rules and limits on who can look at and receive your health information. The Privacy Rule applies to all forms of individuals' protected health information, whether electronic, written, or oral. The Security Rule is a Federal law that requires security for health information in electronic form. 

HIPAA for professionals  To improve the efficiency and effectiveness of the health care system, the Health Insurance Portability and Accountability Act of 1996 (HIPAA), Public Law 104-191, included Administrative Simplification provisions that required HHS to adopt national standards for electronic health care transactions and code sets, unique health identifiers, and security. At the same time, Congress recognized that advances in electronic technology could erode the privacy of health information. Consequently, Congress incorporated into HIPAA provisions that mandated the adoption of Federal privacy protections for individually identifiable health information. HIPAA for professionals

home brewn the past, LDTs were referred to as “home brew” or “in-house” devices. The term “laboratory developed test” and its acronym “LDT” replaced “home brew” over time, but the regulatory considerations are not affected by the change in terminology.  FDA, CBER  Draft Guidance for Industry, Food and 2 Drug Administration Staff, and Clinical  Laboratories  Framework for Regulatory Oversight of 7 Laboratory Developed Tests (LDTs 2014 Oct   [PDF]Draft Guidance for Industry, Food and Drug Administration Staff ... - FDA Related terms: diagnostics

ICH International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use:  The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) is unique in bringing together the regulatory authorities and pharmaceutical industry of Europe, Japan and the US to discuss scientific and technical aspects of drug registration. Since its inception in 1990, ICH has evolved, through its ICH Global Cooperation Group, to respond to the increasingly global face of drug development, so that the benefits of international harmonisation for better global health can be realised worldwide. ICH's mission is to achieve greater harmonisation to ensure that safe, effective, and high quality medicines are developed and registered in the most resource-efficient manner. ICH Homepage 

IND Investigational New Drug Application: Current Federal law requires that a drug be the subject of an approved marketing application before it is transported or distributed across state lines. Because a sponsor will probably want to ship the investigational drug to clinical investigators in many states, it must seek an exemption from that legal requirement. The IND is the means through which the sponsor technically obtains this exemption from the FDA.  Narrower terms: exploratory IND, screening IND  

informed consent: Guide to Informed Consent, Guidance for Institutional Review Boards and Clinical Investigators FDA,   
National Cancer Institute, Informed Consent 2016 

Presidential Commission for the Study of Bioethical Issues   See also ethics

IRB Institutional Review Board: A specially constituted review body established or designated by an entity to protect the welfare of human subjects recruited to participate in biomedical or behavioral research [Federal Policy §§___.102(g), ___.108, ___.109]. Institutional Review Board glossary  

Institutional Review Boards

Laboratory Developed Tests: The Coverage and Analysis Group at the Centers for Medicare & Medicaid Services (CMS) requested from The Technology Assessment Program (TAP) at the Agency for Healthcare Research and Quality (AHRQ) a horizon scan to summarize the available scientific evidence on the quality of laboratory-developed ("home brew" or "in-house") molecular tests, which are currently not actively regulated by the U.S. Food and Drug Administration (FDA). CMS has concerns about the quality of laboratory-developed tests and the validation currently being performed on these tests . AHRQ assigned this report to the following Evidence-based Practice Center (EPC): ECRI EPC (Contract Number: 290 2007 10063 I). To help CMS to address its concerns, this horizon scan is intended to: 1) identify types of laboratory-developed molecular tests (LDMTs) currently available for conditions relevant to the Medicare over-65-year-old population, 2) identify the methodologies and the processes that have been developed for the assessment of analytical and clinical performance of molecular tests, 3) summarize the role of Federal agencies in regulating LDMTs, and 4) identify the quality standards that have been developed for molecular tests by regulatory bodies, the industry, and the medical community.   See also Analyte Specific Reagents

me too drug: A compound that is structurally very similar to already known drugs, with only minor pharmacological differences. IUPAC Medicinal Chemistry  Related terms: follow-ons  Drug discovery& development analogue based drug  discovery

Medicines Control Agency: Now Medicine and Healthcare Regulatory Authority MHRA

MedWatch: FDA Safety Information and Adverse Event Reporting Program  Related terms: Drug safety, pharmacovigilance, post marketing surveillance

Medicines and Healthcare Regulatory Authority

minorities- research: Women and minorities as participants in  research involving human subjects - Policy implementation stage, Office of Extramural Research, NIH, 2003

NCE New Chemical Entity: A compound not previously described in the literature. IUPAC Medicinal Chemistry
NCE Wikipedia definition   Compare: me too drug  

NDA New Drug Application: For decades, the regulation and control of new drugs in the United States has been based on the New Drug Application (NDA). Since 1938, every new drug has been the subject of an approved NDA before U.S. commercialization.  The NDA application is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S.  The data gathered during the animal studies and human clinical trials of an Investigational New Drug (IND) become part of the NDA.


NDA New Drug Approvals, New Drug Applications reports

NME New Molecular Entity: An active ingredient that has never before been marketed in the United States in any form. Drugs@FDA Glossary of terms 

Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product; these products frequently provide important new therapies for patients. Some drugs are characterized as NMEs for administrative purposes, but nonetheless contain active moieties that are closely related to active moieties in products that have previously been approved by FDA. For example, CDER classifies biological products submitted in an application under section 351(a) of the Public Health Service Act as NMEs for purposes of FDA review, regardless of whether the Agency previously has approved a related active moiety in a different product. FDA’s classification of a drug as an “NME” for review purposes is distinct from FDA’s determination of whether a drug product is a “new chemical entity” or “NCE” within the meaning of the Federal Food, Drug, and Cosmetic Act. FDA  New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products

non-prescription drugs: Drugs that can be sold legally without a prescription. MeSH, 1974

off label: The use of an FDA- approved drug or device for a purpose other than that intended by the manufacturer and described on the label. FDA only approves drugs or devices for their intended use as described on the label. Neal Holtzman, Michael Watson "Promoting Safe and Effective Genetic Testing in the United States: Final Report" glossary, 1997  

openFDA: The openFDA project was created to provide easy access to public data, to create a new level of openness and accountability, to ensure the privacy and security of public FDA data, and ultimately to educate the public and save lives. The concept was to index high-value, high priority and scalable public-access data, format and document that data in developer and consumer-friendly standards, and make that data available via a public-access portal that enables developers to quickly and easily use it in applications. .

orphan designation:  Committee for Orphan Medicinal Products COMP, EMA, European Union 

orphan drugs: See orphan products

orphan products: The Orphan Drug Act (ODA) provides for granting special status to a product /indication combination upon request of a sponsor, and if the product/indication combination meets certain criteria. This status is referred to as orphan designation. Orphan designation qualifies the sponsor of the product for the tax credit and marketing exclusivity incentives of the ODA. FDA, US Orphan Product Designation incrasin frequency for post-approval 
OTC drugs: Over the counter drugs. See also non-prescription drugs  

PDUFA Prescription Drug User Fee Act (1992): Another effort to shorten the FDA review process. Under this act, fees the FDA collected from drug developers between 1993 and 1997 were to be used to shorten the timelines needed for evaluating certain drug applications, in part through the hiring of more reviewers. At the same time, the FDA committed to a set of goals for streamlining the review process. The original PDUFA act expires on September 30, 1997, but the FDA Modernization Act of 1997 extended the act through September 30, 2002.  See also   Ss)

pharmacovigilance: Drug safety, pharmacovigilance, post marketing surveillance

predicate rules: A predicate rule is any requirement set forth in the Federal Food, Drug and Cosmetic Act, the Public Health Service Act, or any FDA regulation other than Part 11. Wikipedia accessed Nov 5 2013

prescription drugs: Drugs whose use must be authorized by a professional health provider.  

priority review: A Priority Review designation is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.  A Priority Review means that the time it takes FDA to review a new drug application is reduced.  The goal for completing a Priority Review is six months.  Related terms: accelerated approval,  fast track

proprietary drug, proprietary name:  Trademarked, brand name.  Compare generic.

protocol: The formal design or plan of an experiment or research activity; specifically, the plan submitted to an IRB for review and to an agency for research support. The protocol includes a description of the research design or methodology to be employed, the eligibility requirements for prospective subjects and controls, the treatment regimen(s), and the proposed methods of analysis that will be performed on the collected data. IRB

PSUR Periodic Safety Update Report: Drug safety, pharmacovigilance, post marketing surveillance

regulatory agencies: See EMA, FDA, ICH, MHRA. Related term:  harmonization  

regulatory compliance drug diagnostic co-development: Molecular Diagnostics

Regulatory Intelligence (RI):  the act of gathering and analyzing regulatory information for impact or changes in laws, regulations, directives, guidance documents, etc. There is more to RI than the black and white of regulations or guidance documents; there are many colors of possible interpretations found through researching regulatory precedents, industry practices, Health Authority (HA) opinions, competitor information, etc RAPS Learning Portal

REMS Risk Evaluation and Mitigation Strategy: Drug safety, pharmacovigilance, post marketing surveillance

safe harbor:  In patent law, the research exemption or safe harbor exemption is an exemption to the rights conferred by patents, which is especially relevant to drugs. According to this exemption, despite the patent rights, performing research and tests for preparing regulatory approval, for instance by the FDA in the United States, does not constitute infringement for a limited term before the end of patent term.[1] This exemption allows generic manufacturers to prepare generic drugs in advance of the patent expiration. In the United States, this exemption is also technically called § 271(e)(1) exemption or Hatch-Waxman exemption. In 2005, the U.S. Supreme Court considered the scope of the Hatch-Waxman exemption in Merck v. Integra. The Supreme Court held that the statute exempts from infringement all uses of compounds that are reasonably related to submission of information to the government under any law regulating the manufacture, use or distribution of drugs. In Canada, this exemption is known as the Bolar provision or Roche-Bolar provision, named after the case Roche Products v. Bolar Pharmaceutical. In the European Union, equivalent exemptions are allowed under the terms of EC Directives 2001/82/EC (as amended by Directive 2004/28/EC) and 2001/83/EC (as amended by Directives 2002/98/EC2003/63/EC2004/24/EC and 2004/27/EC).  Wikipedia accessed 2018 Aug 29

specified biotechnology products: Was well characterized biotechnology product. 
FDA Guidance for Industry .... well characterized therapeutic, biotechnology derived products 1995

third party review: The Accredited Persons Program was created by the FDA Modernization Act of 1997 (FDAMA), based on an FDA pilot. The purpose of the program is to improve the efficiency and timeliness of FDA's 510(k) process, the process by which most medical devices receive marketing clearance in the United States. Under the program, FDA has accredited third parties (Accredited Persons) that are authorized to conduct the primary review of 510(k)s for eligible devices. Persons who are required to submit 510(k)s for these devices may elect to contract with an Accredited Person and submit a 510(k) directly to the Accredited Person. The Accredited Person conducts the primary review of the 510(k), then forwards its review, recommendation, and the 510(k) to FDA. By law, FDA must issue a final determination within 30 days after receiving the recommendation of an Accredited Person. 510(k) submitters who do not wish to use an Accredited Person may submit their 510(k)s directly to FDA.

unapproved drugs, access to: See expanded access   

well characterized biotechnology products See specified biotechnology products

women and minorities- human subjects research: NIH  

Regulatory Resources
FDA, CDER Glossary, Drugs@FDA,
FDA Drug Development and Review definitions: 2010
FDA Drug advertising a glossary of terms, 2012, 19 terms
FDA Glossary of Computerized System and Software Development Terminology 2009 
FDA Glossary Strategic Plan for Regulatory Science
FDA Glossary, Independent Institute, 2003, about 60 terms
ICH International Council on Harmonisation Efficacy guidances
Institutional Review Board Glossary, Office Human Research Protection, HHS, US
Mayo Clinic IRB Definition of Terms 

MedDRA Medical Dictionary for Regulatory Activities, Maintenance and Support Services Organization. An international medical terminology designed to support the classification, retrieval, presentation, and communication of medical information throughout the medical product regulatory cycle.   
Medicines & Device Regulation: What you need to know, MHRA Medicine & Healthcare products Regulatory Agency, UK 
WHO glossary,

Barnett Publications
Barnett Core Curriculum
Barnett Web Seminars

How to look for other unfamiliar  terms

IUPAC definitions are reprinted with the permission of the International Union of Pure and Applied Chemistry.

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