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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] adaptive clinical trials: A process for improving the efficiency of clinical trials based on interim analyses of clinical data, potentially leading to reductions in overall sample size, shorter project duration, improved quality of results, and reduced costs. Tufts Center for the Study of Drug Development, Glossary of terms, 2007 http://csdd.tufts.edu/InfoServices/Glossary.asp Tools such as toxicogenomic assays can provide important information about a drug's effectiveness and safety. An adaptive trial would generate and incorporate such information to help guide the more effective use of medicines. In an adaptive trial, for example, patient outcomes could be used as they became available to adjust the allocation of future patients or some other aspect of the study design. Adaptive Clinical Trials Are Steppingstone toward Personalized Medicine, PharmaWeek, July 10, 2006 http://www.healthtech.com/news/strategic_briefings/2007/When%20Smaller%20is%20Better.asphttp://www.pharmaweek.com/Regulatory_And_Legal/Adaptive%20Clinical.asp Within the past few years many new approaches have been developed to address this goal, e.g., adaptive trial design, Bayesian analysis, and the use of biomarkers. Adaptive Clinical Trial Designs, Oct. 2007, Toronto 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 Bayesian clinical trials: In recent years, there has been an explosion in predictive technologies to help researchers select only the most promising candidates for clinical development. The need for such tools is driven by the disastrous economic consequences of late-stage failures, which account for over 60% of all drug terminations. Insight Pharma Reports, Bayesian Forecasting of Phase III Outcomes: The Next Wave in Predictive Tools, 2007 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 Broader term: generic drugs Related term: biosimilars biologics: Include blood, vaccines, tissue, allergenics and biological therapeutics. CBER, FDA, US website http://www.fda.gov/cber/about.htm Compare drug. In the US biologics and drugs have been regulated by different Centers at the FDA (CBER, CDER) , but this is changing. Biologics, in contrast to drugs that are chemically synthesized, are derived from living sources (such as humans, animals, and microorganisms). Most biologics are complex mixtures that are not easily identified or characterized, and many biologics are manufactured using biotechnology. Biological products often represent the cutting- edge of biomedical research and, in time, may offer the most effective means to treat a variety of medical illnesses and conditions that presently have no other treatments available. http://www.fda.gov/cber/about.htm Veterinary biologics (vaccines, bacterins, diagnostics, etc, which are used to prevent, treat, or diagnose animal diseases) are regulated by the U.S. Department of Agriculture. http://www.aphis.usda.gov/vs/cvb/ biopharmaceutical: Any therapeutic biological compound, including recombinant proteins, monoclonal and polyclonal antibodies, antisense oligonucleotides, therapeutic genes, and recombinant and DNA vaccines. Tufts Center for the Study of Drug Development, Glossary, 2007 http://csdd.tufts.edu/InfoServices/Glossary.asp biopharmaceuticals: Biopharmaceuticals are generally complex macromolecules derived from recombinant DNA technology, cell fusion, or processes involving genetic manipulation. They include recombinant proteins, genetically engineered vaccines; therapeutic monoclonal antibodies; and nucleic acid based therapeutics, including gene therapy vectors. Industry Canada, Biopharma Companies and Products in the Pipeline, 2004 http://strategis.ic.gc.ca/epic/internet/inbio-pha.nsf/en/Home 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 biotechnology drugs: Defined as products based on recombinant DNA, monoclonal antibodies, continuous cell lines, gene therapy, and cellular therapy. Product Definition, The Biopharmaceutical Sector, Industry Canada, 2003 http://strategis.ic.gc.ca/epic/internet/inbio-pha.nsf/en/df00020e.html#2.1 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. CDISC Clinical Data Interchange Standards Consortium: An open, multidisciplinary, non- profit organization committed to the development of industry standards to support the electronic acquisition, exchange, submission and archiving of clinical trials data and metadata for medical and biopharmaceutical product development. http://www.cdisc.org/ 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 chemotherapy:
Treatment with anticancer drugs 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/ Clinical Data Interchange Standards Consortium: http://www.cdisc.org/ clinical development: The phase of drug development in which investigational new drugs (INDs) are tested on humans under the control of clinicians. Clinical development is necessary to obtain approval to market new drugs. http://www.biotechshares.com/glossary.htm clinical forecasting: It is clear that late-stage clinical failures account for a large proportion of the expenses. This can be as a result of both the large out-of-pocket investments in Phase III clinical trials and because unsuccessful trials tie up capital resources during their conduct, and potentially also for the time spent during any attempted recovery following regulatory rejection. So, there is an interest in strategies that could halt, as early as possible, the development of drugs that eventually fail. Clinical forecasting in drug development, Asher D. Schachter and Marco F. Ramoni, Nature Reviews Drug Discovery 6, 107-108 (February 2007) | doi:10.1038/nrd2246 http://www.nature.com/nrd/journal/v6/n2/full/nrd2246.html Narrower term: Bayesian clinical forecasting, Bayesian clinical trials clinical genomics - clinical trials impact: Clinical genomics is the application of large-scale, high-throughput genomics technologies in clinical settings, such as clinical trials or primary care of patients. Clinical genomics promises to allow a molecular understanding of disease and drug response, with benefits in all areas of medicine. Contributing to the growth of genomics, in 2005 the FDA issued guidelines for applications of genomics in drug development, with the stated hope that genomics will improve the safety and effectiveness of medicines. Given this mandate, clinical genomics applications appear to have crossed a threshold with the recent approval of several clinical genomics products. Insight Pharma Reports, Impact of Genomics on Clinical Trials, 2006 http://www.insightpharmareports.com/reports/2006/61_Clinical_Genomics/overview.asp clinical informatics: Integration of clinical workflow and business strategies of any healthcare organization will spell success for the providers of the future. Efficient exchange of data and information is essential for this merger, and information technology is the tool with which to accomplish the consolidation. Clinical Informatics is the practice evolving from this need in healthcare. HIMSS Clinical informatics http://www.himss.org/ASP/topics_clinicalInformatics.asp The American Medical Informatics Association (AMIA) will attempt to elevate applied clinical informatics to the level of medical subspecialty with the help of a $300,000 grant from the Robert Wood Johnson Foundation (RWJF). .... AMIA sees applied clinical informatics as not being essentially different for doctors as it is for pharmacy or nursing or any other health profession,” Detmer says. AMIA to Develop Clinical Informatics as Certifiable Specialty, Neil Versel, Digital Healthcare and Productivity, May 2007 http://www.health-itworld.com/newsitems/hitw/amia-clinical-informatics The application of informatics approaches to the clinical- evaluation phase of drug development. These approaches can include clinical- trial simulations to improve trial design and patient selection, as well as electronic capturing and storing of clinical data and protocols. The goal is to reduce expenses and time to market. clinical trial data model: Phase Forward Submits XML-based Clinical Trial Data Model to Worldwide Standards Organizations, 1999 http://www.oasis-open.org/cover/phaseCDISC19990621.html clinical trial simulation: A relatively new effort to devise in silico simulations of human physiology and genetic variation to help identify which compounds will eventually fail in the drug development process. Related term: Genetic manipulation & disruption systems biology clinical trials: The current clinical evaluation process is fraught with inefficiencies, resulting in numerous compound failures and exploding development costs. Until recently, the industry has reacted to the clinical evaluation problem essentially by "streamlining" the existing processes and by introducing information technology in a cautious and evolutionary fashion. While the FDA’s Critical Path Initiative of 2004 showed that the agency is willing to take the lead in working with representatives from industry and academia towards a remedy, this report suggests that a more radical solution is needed. Insight Pharma Reports, Clinical Trial in 2015: A new paradigm for clinical development, 2006 http://www.insightpharmareports.com/reports/2005/59_Clinical_Trials/overview.asp The NIH defines a clinical trial as a prospective biomedical or behavioral research study of human subjects that is designed to answer specific questions about biomedical or behavioral interventions (drugs, treatments, devices, or new ways of using known drugs, treatments, or devices). Clinical trials are used to determine whether new biomedical or behavioral interventions are safe, efficacious, and effective. Behavioral human subjects research involving an intervention to modify behavior (diet, physical activity, cognitive therapy, etc.) fits this definition of a clinical trial. Human subjects research to develop or evaluate clinical laboratory tests (e.g. imaging or molecular diagnostic tests) might be considered to be a clinical trial if the test will be used for medical decision making for the subject or the test itself imposes more than minimal risk for subjects. Biomedical clinical trials of experimental drug, treatment, device or behavioral intervention may proceed through four phases: Phase I clinical trials test a new biomedical intervention in a small group of people (e.g., 20-80) for the first time to evaluate safety (e.g., to determine a safe dosage range and to identify side effects). Phase II clinical trials study the biomedical or behavioral intervention in a larger group of people (several hundred) to determine efficacy and to further evaluate its safety. Phase III studies investigate the efficacy of the biomedical or behavioral intervention in large groups of human subjects (from several hundred to several thousand) by comparing the intervention to other standard or experimental interventions as well as to monitor adverse effects, and to collect information that will allow the intervention to be used safely. Phase IV studies are conducted after the intervention has been marketed. These studies are designed to monitor effectiveness of the approved intervention in the general population and to collect information about any adverse effects associated with widespread use. NIH Human Subjects Research Definitions, 2004 http://grants.nih.gov/grants/funding/PHS398/instructions2/p2_human_subjects_definitions.htm See also Phase zero - Phase IV below CenterWatch
http://www.centerwatch.com/
A listing of more than 41,000 industry- and government- sponsored clinical
trials as well as new drug therapies recently approved by the FDA. Related terms: CRO Clinical Research Organization,
clinical informatics, comparative data mining, FDA drug approvals, Phase I, Phase II, Phase III,
Phase IV/ postmarketing surveillance, preclinical, predictive data mining clinical trials Asia: The globalization of R&D into emerging regions, especially India, China and Southeast Asia continues. Growing R&D spending in these regions has been driven by many factors including the attractiveness of commercial markets there, more favorable economics including lower relative costs for greater speed, access to highly skilled professional labor and a large pool of treatment-naive patients. Pharmaceutical and biotechnology companies are seeing rapid growth in the volume of their clinical trial activity in India, China and Southeast Asia. At the same time these regions present unique challenges that must be anticipated and managed. Clinical Trials Asia Strategies for Planning, Placing and Implementing Clinical Trials in India, China and Southeast Asia, March 2008, San Francisco CA h Google = about 1,840,000 July 31, 2007 See also global drug development: Business glossary clinical trials- ethics: Ethics glossary clinical trials - imaging: Molecular imaging glossary 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 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 comparative data mining: Algorithms & data management glossary Useful for clinical trial meta-analyses 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 Critical path: On March 16 [2004], FDA released a report addressing the recent slowdown in innovative medical therapies submitted to the FDA for approval, "Innovation/ Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products." That report describes the urgent need to modernize the medical product development process -- the Critical Path -- to make product development more predictable and less costly. FDA, The critical path to new medical products, http://www.fda.gov/oc/initiatives/criticalpath/ CRO: Contract Research Organization or Clinical Research Organization. Clinical trials are increasingly being run by outsourcing to these groups. developmental site agreements: Companies work with hospitals to develop instrumentation and clinical research applications. 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 discovery pipeline: Drug discovery & development glossary 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 Related terms: MedWatch, pharmacovigilance EMEA: European Agency for the Evaluation of Medicinal Products http://www.emea.eu.int/ efficacy: Pharmaceutical biology glossary electronic records -- FDA: http://www.fda.gov/ora/compliance_ref/part11/ Electronic Common Technical Document,
FDA http://www.fda.gov/cber/ctd/ctd.htm 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 ethical drugs: The old term ethical drugs signified drugs advertised only to doctors. The expression refers to the original 1847 code of ethics of the AMA, which deemed advertising directly to the public to be unethical. Over time, the term came to mean legal drugs. FDAReview.org, Independent Institute, 2003 http://www.fdareview.org/glossary.shtml#ethical Related term: prescription drugs 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 Related term: Phase zero, Phase 0 explanatory trials: Explanatory trials generally measure efficacy—the benefit a treatment produces under ideal conditions, often using carefully defined subjects in a research clinic. Martin Roland, David J Torgerson, Understanding controlled trials: What are pragmatic trials? BMJ 316: 285 24 January, 1998 http://www.bmj.com/cgi/content/full/316/7127/285 Compare pragmatic trials 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 global drug development: Business glossary 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 HIPAA Health Insurance Portability and Accounting Act: Health & Human Services, US http://www.hhs.gov/ocr/hipaa/ New Regulatory Requirements Are Changing the Way Pharmaceutical Companies Do Business http://www.healthtech.com/newsarticles/issue24_2.asp harmonization of pharmaceuticals regulations: See ICH International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use 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 Large Streamlined Studies LSS: lure of initial value: What we observe [in clinical trials] is not so much a placebo response, but a so- called lure of initial value, where extreme symptoms tend to get spontaneously better. To get a handle on this problem, it would be useful to identify genes that are predictive of rapid disease remission, because many of our problems in terms of testing drugs are driven by the placebo group. ... One needs very large groups to detect that small difference. Obtaining genes predictive of response might also lead to defining the group of people who, because they are likely to get better spontaneously, are going to get minimal benefit from the drug. management trials: Management questions are less focused on a treatment's biologic activity; instead, they concern a treatment's effect on patient outcomes in common practice. Thus, a management trial compares treatment policies or strategies that can be widely applied, enrolling patients as diverse as those likely to use the strategy. Intrusion on the standard of care is small, and clinicians have as much discretion over patient management and study drug discontinuation or re-initiation as they would in common practice. Endpoints are clinical and easily ascertained, e.g., mortality or serious disease, and data collection is minimal. Primary analyses are intent- to- treat, with other analyses performed sparingly. MAPS: A Proposal for More Clinically Relevant Research in AIDS, Carlton Hogan et. al, Univ. of Minnesota http://www.biostat.umn.edu/~carlton/MAPS.html 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 medicinal products: Committee for Orphan Medicinal Products COMP, European Union http://www.emea.europa.eu/htms/human/comp/orphapp.htm 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 patient: People with a specific disease or condition, particularly those being treated by a health professional. Compare subject patient recruitment: Patient recruitment if not adequately planned for can extend your development timeline by a number of years. Retention of patients throughout the life of a clinical trial is essential in order have complete data sets for your analysis and subsequent filings. In order to optimize both you have to have a plan. Patient Recruitment in Clinical Trials Drafting a Successful Recruitment and Retention Plan May 7-8, 2008 • Cambridge, MA The same trial treatment options available at the University of Maryland are available at four affiliated community hospitals where university-employed clinical research associates (CRAs) have been placed, says [Mohan] Suntha, [MD].. Over the past several years, 25 percent of participants in radiation oncology trials – or roughly 50 patients a year – have enrolled in their own communities rather than at the university’s main campus in downtown Baltimore. “We’ve integrated telemedicine technology with our commitment to clinical trial access,” says Suntha. Telemedicine links provide a means for university-based investigators to communicate with patients while they’re in a study. They also allow CRAs to seamlessly transmit data mined at community- based sites. Patient Recruitment: A Growing Role for Telemedicine? Deborah Borfitz, eCliniqua, Oct 1, 2007 http://www.bio-itworld.com/archive/eclinica/index_10012007.htm 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 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 Phase I: The main aim of this phase is to determine drug safety. At this stage, drugs are tested in a small group of healthy volunteers to determine the drug’s activity. Phase II: These trials are aimed at identifying the optimal dose to be used in Phase III trials and, ideally, they identify drugs that will not make it through the next phase of testing. Typically, Phase II trials are double-blinded and have placebo controls. phase II risk reduction: See Chemistry glossary backup compounds Phase III: These studies, which take several years, can involve thousands of patients at multiple trial centers. They are aimed at definitively determining the drug’s effectiveness and its side- effect profiles. These studies are also typically double- blinded and placebo- controlled. Phase IIIb: Subjects entered on a phase III trial may want to stay on the drug after the study is completed and closed and while the data is being readied for presentation. A Phase IIIB study allows "compassionate" use of the drug during this interim time. [Human Subjects Manual, Medical Issues, 10.2 Drugs and Biologics, Stanford Univ., 2001 http://humansubjects.stanford.edu/manual/chapters/ch10_2b_med.html Phase IV/ postmarketing surveillance: At this stage, after a drug has been launched, pharmaceutical companies may conduct further studies of its performance, often examining long- term safety. See also clinical trials, phase I, II, III, IV placebo non- responders: Non- responders on placebo] define a group that would never improve their condition unless given the drug. They may be a group that, if we could identify them, could be used to reduce clinical trial size. Using this group in a proof- of -concept, it may be possible to test a drug even without a comparative placebo and determine whether it is likely to be active. Related terms: disease resistant individuals, lure of initial value, placebo responders placebo responders: Most people think of the placebo response as a true response. But much of it is actually regression to the mean. Clinical trial subjects with more extreme symptoms are often selected because it is desirable to see a dramatic effect upon treatment with the drug. Related terms: disease resistant individuals, lure of initial value, placebo non- responders post approval drug safety: Drug safety & pharmacovigilance postmarketing surveillance: See under Phase IV/postmarketing surveillance pragmatic trials: Pragmatic trials measure effectiveness—the benefit the treatment produces in routine clinical practice.... Pragmatic trials measure effectiveness—the benefit the treatment produces in routine clinical practice Martin Roland, David J Torgerson, Understanding controlled trials: What are pragmatic trials? BMJ 316: 285 24 January, 1998 http://www.bmj.com/cgi/content/full/316/7127/285 Compare explanatory trials 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: Designed to be a stand- alone document that allows a periodic but comprehensive assessment of the worldwide safety data of a marketed drug or biological product. MJ Klepper, The periodic safety update report as a pharmacovigilance tool, Drug Safety 27(8): 569- 578, 2004 public policy and biotechnology: US President's Council on Bioethics http://www.bioethics.gov/topics/biotech_index.html randomized clinical trials RCTs: A study in which the participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively. At the time of the trial, it is not known which treatment is best. It is the patient's choice to be in a randomized trial. National Cancer Institute Dictionary of Cancer Terms http://www.cancer.gov/Templates/db_alpha.aspx?CdrID=45858 CONSORT Consolidated Standards of Reporting Trials, http://www.consort-statement.org/ registries: See under Large Streamlined Studies 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 risk ratio: The ratio of risk in the treated group (EER) to the risk in the control group (CER). This is used in randomised trials and cohort studies and is calculated as EER/CER. [Glossary of EBM [Evidence Based Medicine] Terms, Centre for Evidence Based Medicine, Mt. Sinai Hospital, 2000] http://www.cebm.utoronto.ca/glossary/ 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 6040.4 INDs: Screening INDS http://www.fda.gov/cder/mapp/6030-4.pdf side effect: Molecular medicine glossary Single Controlled Trial SCT: Since 1998, the FDA has allowed drug developers to use what is known as the single controlled trial (SCT) to support their drug approval applications. The SCT provision allows applicants to prove the effectiveness of new drugs by submitting data from only one controlled clinical study instead of multiple studies. SMO Site Management Organization: Why a SMO is not a CRO or is it? Stan Woollen, FDA 2001 http://www.fda.gov/oc/gcp/slideshows/smo2001/smo.ppt stratification- clinical trials: The FDA has been cautious |
