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You are here Biopharmaceuticals Glossary Homepage/Search > Business > Drug discovery & development > Regulatory Affairs Regulatory Affairs:
drugs, devices, diagnostics Glossary & taxonomy
Applications Map: Finding guide to terms in these glossaries Site Map Ethics 21 CFR Part 11, Electronic
records, Electronic signatures, Office
of Regulatory Affairs ORA, FDA http://www.fda.gov/ora/compliance_ref/part11/ 21 CFR Part 50 Human
Subject Protection Informed Consent :
http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=50 21 CFR Part 56
Institutional Review Boards IRBs http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=56 510(K) A premarketing submission made to FDA to
demonstrate that the device to be marketed is as safe and effective, that is,
substantially equivalent (SE), to a legally marketed device that is not subject
to premarket approval (PMA). Applicants must compare their 510(k) device to one
or more similar devices currently on the U.S. market and make and support their
substantial equivalency claims. A legally marketed device is a device that was
legally marketed prior to May 28, 1976 (preamendments device), or a device which
has been reclassified from Class III to Class II or I, a device which has been
found to be substantially equivalent to such a device through the 510(k)
process, or one established through Evaluation of Automatic Class III
Definition. The legally marketed device(s) to which equivalence is drawn is
known as the "predicate" device(s). FDA, Center for Devices and
Radiologic Health, 2004 http://www.fda.gov/cdrh/devadvice/314.html A 510(k) application
involves demonstrating that the new product is substantially equivalent
to an existing product on the market. It is limited to devices and diagnostics,
and by definition, applies only to "me- too" type devices. That is, it
represents an incremental improvement over something that is already on the
market ... Because of its similarity to a product that has already had a
thorough regulatory review, it does not bring up any new issues. .. For 510(k)s,
we [the FDA] have been averaging about 1,000 a year. [Joseph Hackett, in
CHI Summit Pharmacogenomics Report] accelerated approval: Intended to speed the clinical development and
regulatory review of important new medicines through the use of surrogate
endpoints to predict the clinical benefit of the treatment. Tufts Center
for the Study of Drug Development, Glossary of terms, 2007 http://csdd.tufts.edu/InfoServices/Glossary.asp ADE
Adverse Drug Effect: Drug Safety &
Pharmacovigilance analyte
specific reagents: In the past, the FDA's
major regulatory efforts with regard to laboratory testing have involved
"analyte specific reagents" (ASRs) which are the active components in
many laboratory-developed tests, especially genetic tests. In the ASR
regulation, issued in November 1997, the FDA stated that most ASRs would be
considered Class I and therefore exempt from pre-market approval or clearance by
the FDA. The ASR rule did indicate that some tests for contagious diseases, such
as tuberculosis or HIV, would be considered Class III, and thus subject to FDA
pre-market clearance or approval. In more recent statements, FDA officials
have indicated that they are concerned about the lack of oversight of in-house
developed laboratory tests and are considering a revision of the ASR rule.
Issues Oversight of Clinical Laboratory Tests, American Clinical
Laboratory Association, 2004-2007 http://www.clinical-labs.org/issues/fda/index.shtm A new class of regulated
product: analyte specific reagents. These products are usually a
singular reagent, such as an antibody, which can be used toward
developing a test by third parties, such as another company or a hospital. The
manufacturer of this product is not planning to sell it as part of a kit, but
only as an independent reagent. Therefore, if is of low risk to the user, it can
be exempt from 501(k) [device] requirements, and by definition must
be used "for identification and quantification of an individual chemical
substance or ligand in biological substances." [Joseph Hackett, in
CHI Summit Pharmacogenomics Report] Related term: FDA draft
guidelines 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 Exploring the Pathway to
Generic Biologics, National Organization for
Rare Diseases, 2004 http://www.rarediseases.org/pdf/GB_White_Paper_0114_2.pdf Google =
about 107,000 Nov 10, 2006, about 68,900 Oct 23, 2008 Broader terms: follow ons,
generic drugs, me toos. Related terms: analogue based drug
discovery, biosimilars
biologics: Biologics biopharmaceutical:
Biologics biosimilars:
At this time, the U.S. FDA acknowledges that
biosimilars have not been demonstrated to be interchangeable through any
scientific process. The world community may ultimately decide that INN policy
for this class of products should be treated differently than that for small
molecule drugs. A different naming scheme for these products might involve
utilizing a different level of granularity, which may be more detailed or less
detailed depending upon the utility in the INN system. Considering the inherent
difficulties in additional INN product distinctions (e.g. retroactive and
lifecycle changes in naming, additional INN responsibility and liability), if
the world community decides to proceed with a change in the policies regarding
the assigning of INNs, it should be preceded by (a) appropriate exploration of
alternatives (e.g. improvements in education and/or labeling), (b) assuring the
such changes fall within the scope, competence, and expertise of the INN
program, and (c) the performance and independent validation of a formal risk
assessment and/or documentation of events with appropriate statistical
treatment. Discussion by National Regulatory Authorities with World Health
Organization (WHO) On Possible International Non-proprietary Name (INN) Policies
for Biosimilars, 2006 http://www.fda.gov/cder/news/biosimilars.htm Related terms: analogues,
biogenerics, follow ons, generics, me-toos biotechnology: Genetic manipulation &
disruption glossary borderline products: Products which are close to
the boundary between medicines, which need a licence, and others, such as
nutritional supplements, cosmetics etc., which do not. Classification depends
either on the ingredient or the claim or both. Medicines Control Agency, UK,
Pilot publication scheme, Glossary of terms, 2003 http://www.mca.gov.uk/pilot/app1.htm#A 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 bundling: Refers to the inclusion of
multiple devices or multiple indications for use for a device in a single
premarket submission, including products subject to the device and biologics
license application (BLA) authorities, for purposes of review and user fee
payment. In CBER, the term may also include the designation of separate
submissions as one premarket submission for review and user fee payment.
Multiple devices may include different models within a generic type of device2
or devices that are of differing generic types. Center for Devices &
Radiological Health, FDA, Guidance for Industry and FDA Staff: Bundling Multiple
Devices or Multiple Indications in a Single Submission, 2003. http://www.fda.gov/cdrh/mdufma/guidance/1215.html#2a CBER Center for Biologics
Evaluation and Research: Part of the US FDA.
CBER is responsible for ensuring the safety of this nation's entire blood
supply and the products derived from it; the production and approval of safe and
effective childhood vaccines, including any future AIDS vaccines; the
proper oversight of human tissue for transplantation; an adequate and safe
supply of allergenic materials and anti- toxins; the safety and efficacy of
biological therapeutics, including an exciting new array of biotechnology-
derived products used to treat diseases such as cancer and AIDS. http://www.fda.gov/cber/index.html Categories
of Therapeutic Biological Products Remaining in CBER
include 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). 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. Transfer of Therapeutic Products to the Center for Drug Evaluation and
Research, CBER, FDA, US, 2004 http://www.fda.gov/cber/transfer/transfer.htm Related terms: biologics; Molecular Medicine glossary blood and blood safety Pharmaceutical biology
glossary: vaccines CDER Center for Drug
Evaluation and Research: http://www.fda.gov/cder/ Part of the US FDA. CDRH Center for Devices and
Radiologic Health: http://www.fda.gov/cdrh/index.html Part of the US FDA. cGMP current Good
Manufacturing Practice: Bioprocessing glossary to
ddv Often
refers to cancer treatments, but is also used more generally for drug therapy,
particularly antimicrobial drugs.. Google = about 5,060,000
Dec. 3, 2004 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). Was Health Care Financing
Administration. http://www.cms.hhs.gov/clia/ 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 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: A common formatting for marketing applications
developed in the venue of the International Conference on Harmonisation (ICH).
As parties to the ICH, CBER and CDER worked with their ICH partners to achieve
consensus on this common format to be accepted in all the ICH regions. The ICH
regions include the United States, the European Union and Japan, as well as the
Observer countries of Canada and Switzerland (as representative for the European
Free Trade Area). CBER, FDA, Common Technical Document, Implementation
Plan, 2002 http://www.fda.gov/cber/ctd/ctdplan.htm 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, Center for Food Safety and
Applied Nutrition, Office of Cosmetics and Colors Fact Sheet, 1995, revised 2000
http://www.cfsan.fda.gov/~dms/cos-217.html 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), 2006 http://www.fda.gov/cder/guidance/7619dft.pdf Narrower terms: specialty
pharmaceuticals. Compare biologics. drug safety: Drug safety &
pharmacovigilance CDER [FDA] evaluates the
safety profiles of drugs available to American consumers using a variety of
tools and disciplines throughout the life cycle of the drugs. We maintain a
system of postmarketing surveillance and risk assessment programs to identify
adverse events that did not appear during the drug development process. We learn
about adverse events through required reporting by companies and through
voluntary reports submitted to FDA’s MedWatch program, which together total
more than 250,000 reports per year. Staff in the Office of Drug Safety use this
information to identify drug safety concerns and recommend actions to improve
product safety and protect the public health. Activities include updating
drug labeling, providing more information to the community, implementing or
revising a risk management program, and, on rare occasions, reevaluating
approval or marketing decisions. Office of Drug Safety, CDER, FDA http://www.fda.gov/cder/Offices/ODS/default.htm Drug Safety Initiative,
FDA http://www.fda.gov/cder/drugSafety.htm EMEA: European Agency for the
Evaluation of Medicinal Products http://www.emea.eu.int/ efficacy: Pharmaceutical biology
glossary electronic data: New technology is available and being created
everyday to make the collection, correction, and assessment of data from
clinical trials more efficient. The goal is to better integrate systems and data
across departments and regions in order to optimize the speed and cost of trials
and drug development. Electronic Data in Clinical
Trials: Collecting and Leveraging Data to Optimize Clinical Trials, March 2010
Philadelphia PA
electronic records -- FDA:
http://www.fda.gov/ora/compliance_ref/part11/ eCTD: Filing new submissions is time consuming and costly
to both regulators and drug development organizations. Global regulatory
agencies are committed to improving the approval process and the electronic
Common Technical Document (eCTD) is seen as a practical solution. It has been
mandated in some countries and, to date, over 30,000 eCTD sequences have been
submitted to the FDA alone. Despite the promise of eCTD to move your data
through the approval process more efficiently, there are challenges to adoption
and implementation. eCTD 2010: Achieving Efficiency and Compliance in
Electronic Submissions March 2010 Philadelphia PA
Electronic standards for
the transfer of regulatory information,
Glossary of Abbreviations and Terms http://www.fda.gov/cder/m2/pdf/glossary.pdf 21CFRPart11.com, Waters Corp. http://www.21cfrpart11.com/index.html EMEA: European Agency for the Evaluation of Medicinal
Products http://www.emea.eu.int/ efficacy: Pharmaceutical biology ethics: Ethics
exploratory
IND: Exploratory IND studies, which usually involve very
limited human exposure and have no therapeutic intent, can serve a number of
useful goals. For example, an exploratory IND study can help sponsors:
Gain an understanding of the relationship between a specific mechanism of action
and the treatment of a disease, Provide important information on
pharmacokinetics, including, for example, biodistribution of a candidate drug,
Select the most promising lead product from a group of candidates[5] designed to interact with a particular therapeutic
target in humans, Explore a product’s biodistribution characteristics using
various imaging technologies. FDA, CDER, Draft Guidance for Industry,
Investigators and Reviewers, Exploratory IND Studies, 2005 http://www.fda.gov/cder/guidance/6384dft.htm#_Toc100638010 FDA: Food and Drug Administration, US regulatory agency http://www.fda.gov/ FDA compliance: FDA compliance references http://www.fda.gov/ora/compliance_ref/default.htm FDA Drug Development and
Review definitions: 28 terms, 2001 http://www.fda.gov/cder/about/smallbiz/definitions.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 FDAMA
was the most sweeping piece of legislation in FDA's modern history. Not only did
it renew the Prescription Drug User Fee Act because of the agency's successful
performance, but it codified many activities the agency had undertaken through a
variety of reinvention initiatives. Jane E. Henney, FDA, Life After PDUFA, FDAMA
and ICH, Millennial World Congress, San Francisco CA, Apr. 16, 2000 http://www.fda.gov/oc/speeches/2000/pharmsciences41600.html See also http://www.fda.gov/ora/fdama/default.htm FDA draft guidelines -
multiplex tests: Guidance on preparing and
reviewing premarket approval (PMA) submissions for multiplex tests, or tests
that assay multiple analytes simultaneously. Array- based tests, such as
oligonucleotide, cDNA, protein and tissue arrays, are a subset of multiplex
tests. The following recommendations for elements of a multiplex test submission
apply to array- based tests as well as other types of multiplex tests. This
guidance primarily considers nucleic acid based analysis, but many of the
principles apply to protein and tissue arrays as well. Multiplex Tests for
Heritable DNA Markers, Mutations and Expression Patterns; Draft Guidance for
Industry and FDA Reviewers, Office of In Vitro Diagnostic Device Evaluation and
Safety, Division of Immunology and Hematology Devices, Center for Devices and
Radiological Health, FDA, Apr. 2003 http://www.fda.gov/cdrh/oivd/guidance/1210.html Related
term: analyte specific reagent; Microarrays & protein
chips glossary FDA drug approvals: http://www.centerwatch.com/patient/drugs/drugls01.html 1995 - present fast- track: The Food and Drug Administration Modernization Act
of 1997 (FDAMA) includes Section 112, “Expediting study and approval of
fast track drugs.” This section mandates the Agency to facilitate the
development and expedite review of drugs and biologics intended to treat serious
or life- threatening conditions and that demonstrate the potential to address
unmet medical needs. Fast track adds to existing programs, such as accelerated
approval, the possibility of a “rolling submission” for a marketing
application. An important feature of fast
track is that it emphasizes the
critical nature of close early communication between the FDA and sponsor to
improve the efficiency of product development. CBER, FDA, US "CBER
Fast Track Designation Request Performance" 2001 http://www.fda.gov/cber/inside/fastrk.htm The fast track process was
established in the FDA Modernization Act of 1997. Under this act, NDAs are
deemed either "standard" or "priority" (fast track). With
the "standard" designation, the FDA’s goal is to complete the review
and make a decision on the NDA within ten months after it has been filed. With
the "priority" designation, used for drugs that address unmet medical
needs, the target date is six months after the filing. However, actual approval
times are typically longer. In certain cases, the FDA also offers
"accelerated approval" to allow the marketing of drugs for
life-threatening diseases, before the benefits to patients are formally
demonstrated. This approval is made on the basis of a surrogate marker (e.g., a
drug’s effect on survival).
follow ons: http://www.biotechnologyhealthcare.com/journal/fulltext/1/2/BH0102020.pdf http://www.boinetpopulation.bio.org/ip/newsletter/RIPLNewsletter.Issue5.pdf GCP Good
Clinical Practice: 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: Bioprocessing glossary GMP Good Manufacturing
Practice: Bioprocessing glossary guidance documents:
Documents prepared for FDA staff, applicants/
sponsors, and the public that describe the agency`s interpretation of or policy
on a regulatory issue. Guidance documents include, but are not limited to,
documents that relate to: The design, production, labeling, promotion,
manufacturing, and testing of regulated products; the processing, content, and
evaluation or approval of submissions; and inspection and enforcement policies.
Guidance documents do not include: Documents relating to internal FDA
procedures, agency reports, general information documents provided to consumers
or health professionals, speeches, journal articles and editorials, media
interviews, press materials, warning letters, memoranda of understanding, or
other communications directed to individual persons or firms. ...Guidance
documents do not establish legally enforceable rights or responsibilities. They
do not legally bind the public or FDA. FDA, CDRH, Administrative Practices
and Procedures, 21CFR10.115, April 2004 http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=10.115 GxP: A collective term used to refer to the regulations
and guidances governing the research, development, testing, and manufacturing of
drugs, medical devices, and biologics. John Stromp, The Basics of GxPs, Journal
of GXP Compliance http://www.ivthome.com/free/gxp/gxpbasics.htm 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 human factors: The study of how people use technology. It
involves the interaction of human abilities, expectations, and limitations, with
work environments and system design. The term “human factors engineering”
(HFE) refers to the application of human factors principles to the design of
devices and systems. It is often interchanged with the terms "human
engineering," "usability engineering," or "ergonomics."
The goal of HFE is to design devices that users accept willingly and operate
safely in realistic conditions. In medical applications, HFE helps improve human
performance and reduce the risks associated with use error. FDA's Human
Factors Program, 2003 http://www.fda.gov/cdrh/humanfactors/whatis.html ICH International
Conference on Harmonisation of Technical Requirements for Registration of
Pharmaceuticals for Human Use: a unique project that
brings together the regulatory authorities of Europe, Japan and the United
States and experts from the pharmaceutical industry in the three regions to
discuss scientific and technical aspects of product registration. The purpose is
to make recommendations on ways to achieve greater harmonisation in the
interpretation and application of technical guidelines and requirements for
product registration in order to reduce or obviate the need to duplicate the
testing carried out during the research and development of new medicines. ICH
Homepage, http://www.ich.org/UrlGrpServer.jser?@_ID=276&@_TEMPLATE=254 IND
Investigational New Drug Application: A request for Food and Drug
Administration (FDA) authorization to administer an investigational drug to
humans. Such authorization must be secured prior to interstate shipment and
administration of any new drug that is not the subject of an approved new drug
application. CDER, FDA, US "Information for Sponsor- Investigators
Submitting Investigational New Drug Applications (INDs) 2001 http://www.fda.gov/cder/forms/1571-1572-help.html 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 See also ethics Guide to understanding
informed consent, cancer.gov, NCI, US http://www.nci.nih.gov/clinicaltrials/conducting/informed-consent-guide International
Standard Randomised Controlled Trial Number Register: http://www.controlled-trials.com/isrctn/ 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/irb/irb_glossary.htm IRB -- National Human
Genome Research Institute
http://www.genome.gov/10004794 in
silico clinical trials: See computer trials simulations MDUFA Medical Device User
Fee and Modernization Act: FDA, 2002 http://www.fda.gov/oc/mdufma/ Summary http://www.fda.gov/cdrh/mdufma/mdufmasummary.html 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 glossary analogue based drug
discovery medical device: Medical devices range from simple tongue depressors
and bedpans to complex programmable pacemakers with micro- chip technology and
laser surgical devices. In addition, medical devices include in vitro
diagnostic products, such as general purpose lab equipment, reagents, and test
kits, which may include monoclonal antibody technology. Certain
electronic radiation emitting products with medical application and claims meet the
definition of medical device. Examples include diagnostic ultrasound products,
x-ray machines and medical lasers. If a product is labeled, promoted or used in
a manner that meets the following definition in section 201(h) of the Federal
Food Drug & Cosmetic (FD&C) Act it will be regulated by the Food and Drug Administration (FDA) as a medical device and is subject to premarketing
and postmarketing regulatory controls. A device is: "an instrument,
apparatus, implement, machine, contrivance, implant, in vitro reagent, or
other similar or related article, including a component part, or accessory which
is: recognized in the official National Formulary, or the United States
Pharmacopoeia, or any supplement to them, intended for use in the diagnosis of
disease or other conditions, or in the cure, mitigation, treatment, or
prevention of disease, in man or other animals, or intended to affect the
structure or any function of the body of man or other animals, and which does
not achieve any of it's primary intended purposes through chemical action within
or on the body of man or other animals and which is not dependent upon being
metabolized for the achievement of any of its primary intended purposes."
FDA, Center for Devices and Radiological Health, US "Is the product a
medical device?" 1998 http://www.fda.gov/cdrh/devadvice/312.html#contents Device Advice, FDA, CDRH http://www.fda.gov/cdrh/devadvice/ 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 meta-analyses,
meta-analysis: Research glossary microarrays
regulating: See Microarrays glossary FDA -- microarrays - regulating microdosing: See Pharmacogenomics glossary 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 Minorities, race and
Genetics: Human Genome Project Information, DOE, US http://www.ornl.gov/TechResources/Human_Genome/elsi/minorities.html 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: CDER (FDA) New and generic drug approvals interim
index http://www.fda.gov/cder/approval/index.htm NDA New Drug Approvals, New
Drug Applications reports http://www.fda.gov/cder/rdmt/default.htm NME New Molecular Entity: A medication containing an active substance
that has never before been approved for marketing in any form in the United
States. [Center for Drug Evaluation and Research, FDA, US "FDA's Drug
review and approval times" 2001] http://www.fda.gov/cder/reports/reviewtimes/ NME reports http://www.fda.gov/cder/rdmt/default.htm non-prescription drugs: Drugs that can be sold legally without a
prescription. MeSH, 1974 OTC drugs: Over the counter
drugs. See also non-prescription drugs 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 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 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, 2001 http://www.fda.gov/orphan/designat/index.htm increasing
frequency for post-approval 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 pharmacoepidemiology: The study of the utilization and effects of drugs
in large numbers of people. To accomplish this study, pharmacoepidemiology
borrows from both pharmacology and epidemiology. About Pharmacoepidemiology,
International Society Pharmacoepidemiology, 2004 http://www.pharmacoepi.org/aboutpe.cfm pharmacovigilance: The science and activities relating to the
detection, assessment, understanding and prevention of adverse effects or any
other drug-related problems. The Importance of Pharmacovigilance, WHO 2002
http://www.who-umc.org/defs.html#pvr The process of (a)
monitoring medicines as used in everyday practice to identify previously
unrecognised or changes in the patterns of their adverse effects; (b) assessing
the risks and benefits of medicines in order to determine what action, if any,
is necessary to improve their safe use; (c) providing information to users to
optimise safe and effective use of medicines; (d) monitoring the impact of any
action taken. Medicines Control Agency, UK, Pilot publication scheme,
Glossary of terms, 2003 http://www.mca.gov.uk/pilot/app1.htm#A Related terms: Phase
IV/postmarketing surveillance Broader term: drug safety
Phase zero, Phase O: Phase Zero is a novel
pre-clinical testing service that combines a range of integrated technologies
and involves the introduction of human tissue at the earliest stages of drug
development. It allows target identification and validation as well as testing
the viability of drug leads and candidates in human tissue before entering the
clinic. This enables rationalization of the drug development process and
improves the outcome at several points along the developmental path.
Pharmagene signs new Phase Zero agreement with Taisho, Friday, March 09, 2001
http://www.pharmabiz.com/article/detnews.asp?articleid=6449§ionid=14 Related terms: microdosing
Biomarkers glossary type 0 biomarker post approval drug safety: Drug safety &
pharmacovigilance postmarketing surveillance:
See under Phase IV/postmarketing surveillance preclinical drug
development: Drug Discovery &
development predicate rules: This document provides guidance to persons who, in
fulfillment of a requirement in a statute or another part of FDA's regulations
to maintain records or submit information to FDA,3 have chosen to maintain the records or submit
designated information electronically and, as a result, have become subject to
part 11. Part 11 applies to records in electronic form that are created,
modified, maintained, archived, retrieved, or transmitted under any records
requirements set forth in Agency regulations. Part 11 also applies to electronic
records submitted to the Agency under the Federal Food, Drug, and Cosmetic Act
(the Act) and the Public Health Service Act (the PHS Act), even if such records
are not specifically identified in Agency regulations (§ 11.1). The underlying
requirements set forth in the Act, PHS Act, and FDA regulations (other than part
11) are referred to in this guidance document as predicate rules. CDER, FDA,
Guidance for Industry, Electronic Records, Electronic Signatures, 2003 http://www.fda.gov/cder/guidance/5667fnl.htm premarket approval PMA: The Medical Device Amendments of 1976 to the
Federal Food, Drug, and Cosmetic Act (the act) established three regulatory
classes for medical devices. The three classes are based on the degree of
control necessary to assure that the various types of devices are safe and
effective. The most regulated devices are in Class III. The amendments define a
Class III device as one that supports or sustains human life or is of
substantial importance in preventing impairment of human health or presents a
potential, unreasonable risk of illness or injury. [Information on
Premarket Approval Applications, FDA, CDRH, 2001] http://www.fda.gov/cdrh/pmapage.html Related term medical device For the Pre-Market Approval
(PMA) application, the issue is not substantial equivalence. It involves instead
an entirely new product, something that has not been previously introduced into
the market. For these products, the FDA has no knowledge or precedent for its
safety and efficacy, and therefore it has to be shown to be safe and effective
on its own merit. For these applications, we invariably make use of an external
panel review. ... For PMA’s, we [the FDA] will receive 13-15 submissions a
year. [Joseph Hackett prescription drugs: Drugs whose use must be authorized by a
professional health provider. 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 public policy and
biotechnology: US President's Council on Bioethics http://www.bioethics.gov/topics/biotech_index.html 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.healthtech.com/newsarticles/issue24_1.asp regulatory agencies: See
FDA, EMEA. Related term: harmonization
REMS
Risk Evaluation and Mitigation Strategy :
Drug
Safety 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 6040.4 INDs: Screening INDS http://www.fda.gov/cder/mapp/6030-4.pdf side effect: Molecular medicine glossary 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 unapproved
drugs, access to: Access to unapproved drugs
can occur through many legal mechanisms, including enrolling in clinical trials,
a special exception or compassionate exemption, an emergency Investigational New
Drug (IND), and a Treatment IND. FDA Oncology Tools, Access to Unapproved Drugs,
CDER, FDA, 2004 http://www.fda.gov/cder/cancer/access.htm 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 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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