You are here Biopharmaceuticals Glossary Homepage/Search > Business > Drug discovery & development > Regulatory Affairs

Regulatory Affairs: drugs & diagnostics Glossary & taxonomy
Evolving Terminologies for Emerging Technologies
Comments? Questions? Revisions? Mary Chitty 
mchitty@healthtech.com
Last revised May 07, 2014

 



Drug discovery term index Finding guide to terms in these glossaries  Site Map   Ethics  This is a sub-category of Drug discovery & development  Related glossaries include Biologics  Biomaterials & medical devices  Clinical trials   Drug safety & pharmacovigilance   Molecular Medicine   Pharmacogenomics   Informatics  Clinical informatics   Research Technologies: Bioprocessing  

21 CFR Part 11, Electronic records, Electronic signatures, Office of Regulatory Affairs ORA, FDA  http://www.fda.gov/ora/compliance_ref/part11/
21CFRPart11.com, Waters Corp. http://www.21cfrpart11.com/index.html   

21 CFR Part 50 Human Subject Protection Informed Consent :

21 CFR Part 56 Institutional Review Boards IRBs   

accelerated approval: in 1992 FDA instituted the Accelerated Approval regulation, allowing earlier approval of drugs to treat serious diseases, and that fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker - a laboratory measurement, or physical sign - that is used in clinical trials as an indirect or substitute measurement that represents a clinically meaningful outcome, such as survival or symptom improvement. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. Approval of a drug based on such endpoints is given on the condition that post marketing clinical trials verify the anticipated clinical benefit. http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm 

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.  Title 21  Food and Drugs  Chapter I FDA Dept HHS Subchapter H Medical Devices Part 809 IN VITRO DIAGNOSTIC PRODUCTS FOR HUMAN USE Subpart C--Requirements for Manufacturers and Producers http://en.wikipedia.org/wiki/Analyte-specific_reagent   FDA    See also Laboratory Developed Tests

bioequivalence:  Scientific basis on which generic and brand- name drugs are compared. To be considered bioequivalent, the bioavailability of two products must not differ significantly when the two products are given in studies at the same dosage under similar conditions. Some drugs, however, are intended to have a different absorption rate. FDA may consider a product bioequivalent to a second product with a different rate of absorption if the difference is noted in the labeling and doesn't affect the drug's safety or effectiveness or change the drug's effects in any medically significant way. Drug Review Glossary, FDA Consumer Magazine, 25 definitions http://www.fda.gov/fdac/special/newdrug/bengloss.html  See also therapeutic equivalency

bioinequivalence: www.fda.gov/ohrms/DOCKETS/ac/04/slides/2004-4078S2_07_YU.ppt 

biogenerics: So far, drugs based on large biological molecules have been immune from copycat competition since most are still patent- protected and, critically, regulators in major markets have yet to set clear rules for approving generic versions. But so- called "biogenerics" are gaining a foothold in Asia, where patents on original versions have expired or patent protection does not exist, and generics firms are looking hungrily at Europe as their next major outlet. Ben Hirschler, Biotech drug copycats get ready to pounce, San Diego Union Tribune, Sept, 18, 2002  http://www.signonsandiego.com/news/business/biotech/ 20020918-0547-health-biotech-generics.html    Broader terms: follow ons, generic drugs, me toos.   Related terms: biosimilars,  analogue based drug discovery,

biosimilars:
Biosimilars promise to lower costs through increased competition, expand access to medicines, and promote further biomedical innovation. However, biosimilars are subject to an approval process, which requires substantial additional data compiled through clinical, animal and analytical studies. It is thus difficult and costly to recreate biologics.   Biosimilars and Biobetters March 19-20, 2013 • Baltimore, MD Program | 

biological products, which are biological prescription drugs that are demonstrated to be “highly similar” (biosimilar) to or “interchangeable” with an FDA-approved biological product. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/Biosimilars/default.htm Related terms: analogues, biogenerics, follow ons, generics, me-toos   
Wikipedia 
http://en.wikipedia.org/wiki/Biosimilar 

biotechnology: Biologics

borderline products:  European Union http://ec.europa.eu/consumers/sectors/cosmetics/cosmetic-products/borderline-products/

brand name drug: A drug marketed under a proprietary, trademark- protected name. Glossary, Drugs@FDA, CDER, 2004 http://www.fda.gov/cder/drugsatfda/glossary.htm 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. http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/default.htm

Categories of Therapeutic Biological Products Remaining in CBER Cellular products, including products composed of human, bacterial or animal cells (such as pancreatic islet cells for transplantation), or from physical parts of those cells (such as whole cells, cell fragments, or other components intended for use as preventative or therapeutic vaccines). Gene therapy products. Human gene therapy/gene transfer is the administration of nucleic acids, viruses, or genetically engineered microorganisms that mediate their effect by transcription and/or translation of the transferred genetic material, and/or by integrating into the host genome. Cells may be modified in these ways ex vivo for subsequent administration to the recipient, or altered in vivo by gene therapy products administered directly to the recipient. Vaccines (products intended to induce or increase an antigen specific immune response for prophylactic or therapeutic immunization, regardless of the composition or method of manufacture).  Allergenic extracts used for the diagnosis and treatment of allergic diseases and allergen patch tests. Antitoxins, antivenins, and venoms Blood, blood components, plasma derived products (for example, albumin, immunoglobulins, clotting factors, fibrin sealants, proteinase inhibitors), including recombinant and transgenic versions of plasma derivatives, (for example clotting factors), blood substitutes, plasma volume expanders, human or animal polyclonal antibody preparations including radiolabeled or conjugated forms, and certain fibrinolytics such as plasma-derived plasmin, and red cell reagents. http://www.fda.gov/AboutFDA/CentersOffices/CBER/ucm133463.htm

CDER Center for Drug Evaluation and Research: 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.  http://www.fda.gov/cder/  Categories of Therapeutic Biological Products Transferred to CDER from CBER, June 30, 2003: Monoclonal antibodies for in-vivo use, Proteins intended for therapeutic use, including cytokines (e.g. interferons), enzymes (e.g. thrombolytics), and other novel proteins, except for those that are specifically assigned to CBER (e.g., vaccines and blood products). This category includes therapeutic proteins derived from plants, animals, or microorganisms, and recombinant versions of these products. Immunomodulators (non-vaccine and non-allergenic products intended to treat disease by inhibiting or modifying a pre-existing immune response). Growth factors, cytokines, and monoclonal antibodies intended to mobilize, stimulate, decrease or otherwise alter the production of hematopoietic cells in vivo   http://www.fda.gov/AboutFDA/CentersOffices/CBER/ucm133463.htm 

cGMP: current Good Manufacturing Practice: Bioprocessing

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  http://grants1.nih.gov/grants/guide/notice-files/not98-024.html

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). . http://www.cms.hhs.gov/clia/  

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 http://commonfund.nih.gov/clinicalresearch/overview-policy.aspx

combination products: 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; (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; (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; 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. Definition of a Combination Product, FDA, Office of Combination Products, As defined in 21 CFR § 3.2(e), http://www.fda.gov/oc/combination/definition.html  See also Biomaterials & Medical Devices glossary & taxonomy

Combination Drug Diagnostics: Fueling Growth of Personalized Medicine  Insight Pharma Reports  2010

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. http://www.mhra.gov.uk/home/idcplg?IdcService=SS_GET_PAGE&nodeId=301  

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 http://www.ich.org/products/ctd.html 
Wikipedia http://en.wikipedia.org/wiki/Common_Technical_Document 

compassionate use: See expanded access

cosmeceuticals: While the Food, Drug, and Cosmetic 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.  The Food, Drug, and Cosmetic Act defines drugs as those products that cure, treat, mitigate or prevent disease or that affect the structure or function of the human body. While drugs are subject to a review and approval process by FDA, cosmetics are not approved by FDA prior to sale. If a product has drug properties, it must be approved as a drug.  FDA, Cosmetics 2009  http://www.fda.gov/Cosmetics/ProductandIngredientSafety/ProductInformation/ucm127064.htm 

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 http://www.chem.qmul.ac.uk/iupac/medchem/ah.html

Should be considered synonymous with investigational (medicinal) product, medicinal product and pharmaceutical (including vaccines and other biological products). E15 terminology in Pharmacogenomics, ICH Draft 2 (Revision 2), 2008  http://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm129296.pdf Narrower terms: specialty pharmaceuticals. Compare biologics. Related terms: CDER, FDA. In the US biologics and drugs are regulated by different Centers at the FDA.

efficacy: Pharmaceutical biology

EMEA: European Agency for the Evaluation of Medicinal Products now EMA: European Medicines Agency  http://www.ema.europa.eu/

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 http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm 

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 http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm078933.pdf  Related term: Phase zero, Phase 0

FDA: Food and Drug Administration, US regulatory agency http://www.fda.gov/   Narrower terms: CBER, CDER, CDRH

FDA compliance:  FDA compliance references http://www.fda.gov/ora/compliance_ref/default.htm 

FDA Drug Development and Review definitions: 2010 http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm176522.htm 

FDA Regulatory Procedures Manual: Glossary http://www.fda.gov/ora/compliance_ref/rpm/pdf/ch11.pdf 2004, 25 pages

FDAMA: The Food and Drug Modernization Act of 1997 reauthorized the collection of user fees by the FDA and amended the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act to improve the regulation of food, drugs, devices, and biological products, and facilitate the development and evaluation of new drugs and biologics designed to treat serious and life - threatening illnesses. Tufts Center for the Study of  Drug Development, Glossary, 2007 http://csdd.tufts.edu/InfoServices/Glossary.asp 

FDA drug approvals: http://www.centerwatch.com/patient/drugs/drugls01.html 1995 - present

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. http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm 

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 http://www.who.int/intellectualproperty/submissions/Submission_DiMasi.pdf

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, 2012 http://www.barnettinternational.com/EducationalServices_Publication.aspx?p=10067&id=115409 

Good Clinical Practice in FDA Regulated Clinical Trials http://www.fda.gov/oc/gcp/default.htm  Broader term: GxP

generic drugs: Drugs whose drug name is not protected by a trademark. They may be manufactured by several companies. MeSH, 1992 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. barnettinternational.com/ EducationalServices_ Publication.aspx?p=7802&id= 97212 
Wikipedia http://en.wikipedia.org/wiki/Good_Laboratory_Practice  Non-human studies Broader term: GxP

GMP Good Manufacturing Practice: Bioprocessing

guidance documents:   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. http://www.fda.gov/regulatoryinformation/guidances/

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.   http://en.wikipedia.org/wiki/GxP 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 http://www.hhs.gov/ocr/hipaa/ 

home brew:  Reagents or the combination of reagents made in a laboratory, or purchased reagents used by that laboratory for clinical tests and not for sale to other laboratories. Neal Holtzman, Michael Watson "Promoting Safe and Effective Genetic Testing in the United States: Final Report" glossary, 1997, last updated 2004 http://www.genome.gov/10002399  Related terms: Genetic & genomic testing

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  http://www.ich.org/home.html 

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. http://www.fda.gov/%20Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/default.htm 

Narrower terms: exploratory IND, screening IND  
IND
reports   http://www.fda.gov/cder/rdmt/default.htm  
IND reports for biologicals
http://www.fda.gov/cber/ind/ind.htm

informed consent: Guide to Informed Consent, Guidance for Institutional Review Boards and Clinical Investigators
FDA, 1998 Update
http://www.fda.gov/oc/ohrt/irbs/informedconsent.html   
Guide to understanding informed consent, cancer.gov, NCI, US http://www.nci.nih.gov/clinicaltrials/conducting/informed-consent-guide
Presidential Commission for the Study of Bioethical Issues http://www.bioethics.gov/ 
International Standard Randomised Controlled Trial Number Register:
http://www.controlled-trials.com/isrctn/
   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 http://www.hhs.gov/ohrp/archive/irb/irb_glossary.htm 
Institutional Review Boards http://www.hhs.gov/ohrp/assurances/irb/index.html

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. http://www.cms.gov/medicare-coverage-database/details/technology-assessments-details.aspx?TAId=72&bc=BAAgAAAAAAAA&   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: The executive agency of the Department of Health safeguarding public health by ensuring that all medicines on the UK market meet appropriate standards of safety, quality and efficacy.  http://www.mca.gov.uk/

MedWatch: FDA Safety Information and Adverse Event Reporting Program http://www.fda.gov/medwatch/index.html  Related terms: drug safety, pharmacovigilance 

minorities- research: Women and minorities as participants in  research involving human subjects - Policy implementation stage, Office of Extramural Research, NIH, 2003   http://grants1.nih.gov/grants/funding/women_min/women_min.htm

NCE New Chemical Entity: A compound not previously described in the literature. IUPAC Medicinal Chemistry

Any new molecular compound not previously approved for human use, excluding diagnostic agents, vaccines and other biologic compounds not approved by the FDA's Centers for Drug Evaluation and Research (CDER). Also excluded are new salts, esters and dosage forms of previously approved compounds. Tufts Center for the Study of  Drug Development, Glossary, 2007  http://csdd.tufts.edu/InfoServices/Glossary.asp  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. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/

Wikipedia http://en.wikipedia.org/wiki/New_Drug_Application

NDA New Drug Approvals, New Drug Applications reports http://www.fda.gov/cder/rdmt/default.htm

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 http://www.fda.gov/Drugs/informationondrugs/ucm079436.htm 
NME reports  http://www.fda.gov/cder/rdmt/default.htm

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 http://www.nhgri.nih.gov/ELSI/TFGT_final/glossary.html  

openFDA: will offer easy access to FDA public data and highlight projects using these data in both the public and private sector to further regulatory or scientific missions, educate the public, and save lives. Company types and subtypes will provide API and raw download access to a number of high-value structured datasets. Additionally, openFDA will provide a platform for public challenges issued by the FDA and a place for the community to interact with each other and FDA domain experts with the goal of spurring innovation around FDA data. We're currently focused on working on datasets in the following areas:
Adverse Events: FDA’s Adverse Event Reporting System, a database that contains millions of adverse event and medication error reports submitted to FDA covering all regulated drugs.
Recalls: Enforcement Report and Product Recalls Data, containing information gathered from public notices about certain recalls of FDA-regulated products
Documentation: Structured Product Labeling Data, containing detailed product label information on many FDA-regulated products. http://open.fda.gov/

orphan drug: Drugs developed for rare diseases and conditions which, in the U.S., affect fewer than 200,000 people or, in the European Union, affect 5 or fewer per 10,000 people. Because sales of orphan drugs are likely to be small compared to their development costs, pharmaceutical companies are awarded exclusive rights to market these medicines for a period of time as an incentive to develop them. Tufts Center for the Study of  Drug Development, Glossary, 2007  http://csdd.tufts.edu/InfoServices/Glossary.asp 

orphan designation:  Committee for Orphan Medicinal Products COMP, EMA, European Union http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp 

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  http://www.fda.gov/orphan/designat/index.htm increasing frequency for post-approval 

OTC drugs: Over the counter drugs. See also non-prescription drugs  

PDUFA Prescription Drug User Fee Act (1992): The Prescription Drug User Fee Act of 1992. Legislation passed by Congress authorizing the FDA to collect "user fees" for regulatory review of human drug applications. The FDA agreed to use the income from the fees to hire more reviewers to speed up drug review without compromising review quality. Tufts Center for the Study of  Drug Development, Glossary of terms, 2002  http://csdd.tufts.edu/InfoServices/Glossary.asp

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 http://www.fda.gov/oc/pdufa/reports.html   Ss) are bein

pharmacovigilance: Drug safety  Related terms: Phase IV/postmarketing surveillance

Phase IV Planned post-marketing studies of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques that have been approved for general sale. These studies are often conducted to obtain additional data about the safety and efficacy of a product. This concept includes phase IV studies conducted in both the U.S. and in other countries. MeSH  Clinical trials, Phase IV as topic 
2008 (1993) 

post approval drug safety: Drug safety & pharmacovigilance  
postmarketing surveillance: See under Phase IV/postmarketing surveillance

preclinical drug development:
Drug Discovery & development

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 22013 http://en.wikipedia.org/wiki/Title_21_CFR_Part_11

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. http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm  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 and toxicology

regulated information systems: Regulatory demands are changing the way information systems will be designed and implemented in the life sciences industry. With the introduction of 21 CFR Part 11 and HIPAA in the U.S., and new requirements from EMEA, CEOs and CIOs are paying much more attention to security, access and control issues. These requirements are causing the development of a new information systems market - the regulated systems market. The regulated systems market requires the use of information systems that can protect personal health information and ensure that every document and data set has been secured, audited for changes, and authenticated with respect to signature authorities. New Regulatory Requirements Are Changing the Way Pharmaceutical Companies Do Business and Creating a New Market for Informatics Systems: An Interview with Bernard P. Wess, Jr., of PERSEID, CHI's GenomeLInk 24.2 http://www.chidb.com/newsarticles/issue24_2.asp

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

regulatory compliance drug diagnostic co-development: Molecular Diagnostics

regulatory science:  The NIH and the U. S. Food and Drug Administration (FDA) have formed an interagency partnership to foster regulatory science, a specialized and inter-disciplinary area of biomedical research that serves to generate new knowledge and tools for assessing experimental therapies, preventives, and diagnostics. A key goal of this new Regulatory Science program is to accelerate the development and use of new tools, standards and approaches to efficiently develop products and to more effectively evaluate product safety, efficacy and quality. Regulatory Science, NIH Common Fund http://commonfund.nih.gov/regulatoryscience/overview.aspx

REMS Risk Evaluation and Mitigation Strategy : Drug Safety

safe harbor: Implementation of new microbiological methods poses significant problems and risks. These include validation of methods that will not yield results equal to traditional methods. Additionally, comparative parallel testing is not always informative. Experimental designs for validating the methods can be developed, but may not be a perfect solution. Therefore, some risk is involved and there is a need to create a "safe harbor" for firms willing to undertake new microbiological tests. The safe harbor concept for sterility testing using new/rapid microbiological test methods (a specification change using the ICH definition of specification) may be limited because the test is qualitative and the established release test requirement is a critical parameter. For a critical parameter, the batch cannot be released if the parameter is not met. The safe harbor concept for sterility testing may not afford much latitude because a failed criterion prohibits retesting by the same method or even a compendial method. The batch must be rejected. Otherwise, the batch is "tested into compliance." Process Analytical Technology Initiative, Microbiology Methods, FDA Dockets, 2002 http://www.fda.gov/ohrms/dockets/ac/02/briefing/3901B1_02_Rapid%20Michtm 

screening IND: In general, CDER policy has been to encourage separate INDs for different molecules and dosage forms. However, in the early phase of drug development, before the developmental path is clear, exploratory studies may be conducted on a number of closely related drugs to choose the preferred compound or formulation. These studies may be best and most efficiently conducted under a single IND, referred to as a screening IND. CDER, FDA, Manual of policies and procedures  MAPP 6030.4, 2002 http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/ucm082026.pdf 

specified biotechnology products: Was well characterized biotechnology product. 
FDA Guidance for Industry .... well characterized therapeutic, biotechnology derived products 1995
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071597.pdf

third party review: Another example of the FDA making use of external resources to ensure quality submission and flexibility in its approval process. In the pilot program for third party review, we obtained the assistance of the State of California Department of Health. The FDA provided them with a guidance document, clearly outlining the objectives, the type of information we are interested in, the process of review and the key points to look for when considering an application. Based on those guidelines, the Department of Health reviewed one 501(k) case and concluded that it was a substantial equivalent to a marketed product. The FDA then had 30 days to review it. Actual review time was only 15 days. The role of the FDA was that of quality control, essentially to oversee the procedure and make sure the third party did everything right. The procedure has worked out fairly well, but the actual number of submissions to this program was low. In 1999, there were only two. To date, the total is only eleven. Joseph Hackett FDA

unapproved drugs, access to: See expanded access   

well characterized biotechnology products See specified biotechnology products

women and minorities- research: Women and minorities as participants in research involving human subjects - Policy implementation stage, Office of Extramural Research, NIH, 2003   http://grants1.nih.gov/grants/funding/women_min/women_min.htm

Bibliography
Bioprocessing International, International Pharmaceutical Regulation, 2009 http://www.bioprocessintl.com/default.asp?page=glossary&TopicID=3 
FDA, CDER Glossary,
Drugs@FDA,  2004, 30+ definitions. http://www.fda.gov/cder/drugsatfda/glossary.htm
FDA Drug Development and Review definitions: 2010 http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm176522.htm
FDA, Drug advertising a glossary of terms, 2012, 19 terms  http://www.fda.gov/Drugs/ResourcesForYou/Consumers/PrescriptionDrugAdvertising/ucm072025.htm
FDA Glossary of Computerized System and Software Development Terminology 2009 http://www.fda.gov/iceci/inspections/inspectionguides/ucm074875.htm 
FDA, Innovation or stagnation: Challenge and opportunity on the critical path to new medical products, 2004
http://www.fda.gov/oc/initiatives/criticalpath/whitepaper.html
FDA Review.org Glossary, Independent Institute, 2003, about 60 terms http://www.fdareview.org/glossary.shtml 
Findlaw, Health Hippo
http://hippo.findlaw.com/hippohome.html Policy and regulatory material related to healthcare
ICH International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use Glossary http://www.ich.org/glossary.html 
Institutional Review Board Glossary, Office Human Research Protection, HHS, US, 1993 150+ terms
http://www.hhs.gov/ohrp/archive/irb/irb_glossary.htm  Some portions valid, others obsolete
ISPE International Society Pharmaceutical Engineering ISPE Glossary of Pharmaceutical and Biotechnology Terminology http://www.ispe.org/glossary  Manufacturing and regulatory
Mayo Clinic, IRB definitions, 2008
http://mayoresearch.mayo.edu/mayo/research/irb/definitions.cfm 
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.
http://www.meddramsso.com/   
Medicines & Device Regulation: What you need to know, MHRA Medicine & Healthcare products Regulatory Agency, UK http://www.mhra.gov.uk/home/groups/comms-ic/documents/websiteresources/con2031677.pdf 
National Research Ethics Services NRES Glossary, UK 2013  http://www.nres.nhs.uk/applications/glossary/ 
NIH, Glossary of terms for human subjects
http://grants1.nih.gov/grants/peer/tree_glossary.pdf 
Roche, Key Clinical Trial Terms: Glossary of Terms and Abbreviations, 2000 http://www.rochetrials.com/glossary.action 
Tufts Center for the Study of Drug Development, Glossary of Terms, 20+ terms.
http://csdd.tufts.edu/InfoServices/Glossary.asp  
WHO glossary, 2007, 28 definitions
http://www.who.int/ictrp/glossary/en/index.htm
http://www.genomicglossaries.com/content/drug_a3.jpg
Live seminars, Web seminars, Publications on clinical trials and Regulatory Affairs

Barnett books http://www.barnettinternational.com/EducationalServices/Publications.aspx
Barnett Live Seminars http://www.barnettinternational.com/EducationalServices/Seminars.aspx
Barnett Web Seminars http://www.barnettinternational.com/EducationalServices/Webinars.aspx


Alpha glossary index

How to look for other unfamiliar  terms

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

Contact | Privacy Statement | Alphabetical Glossary List | Tips & glossary FAQs | Site Map