Rare Diseases and Orphan Products Accelerating Research and Development (2010) / Chapter Skim
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Appendix D: Glossary, Abbreviations, and Public Laws
Appendix D: Glossary, Abbreviations, and Public Laws
Pages 345-364
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Active treatment concurrent control. In a clinical trial, "the test drug is compared with known effective therapy." (21 CFR 314.126)
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(see also Clearance; Humanitarian Device Exemption; New Drug Application; Premarket Approval application)
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A"network of blood vessels with closely spaced cells that makes it difficult for potentially toxic substances (such as anticancer drugs) to penetrate the blood vessel walls and enter the brain." (http://www.
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is 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) is comprised of 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; or 3)
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A systematic evaluation of the evidence on the outcomes of different drugs or other options for treating, preventing, or diagnosing a medical condition Compound library. A collection of small organic molecules organized in a format that facilitates drug discovery and biomedical research, also known as a chemical library.
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: "(A) articles recognized in the official United States Pharmacopoeia, official Homoeopathic Pharmacopoeia of the United States, or official National Formulary, or any supplement to any of them; and (B)
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The human genome consists of 23 pairs of chromosomes in the cell nucleus and a small chromosome found in the mitochondria of cells.
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Genotyping. Testing to identify specific genetic sequences (see Allele)
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In a clinical trial, "the results of treatment with the test drug are compared with experience historically derived from the adequately documented natural history of the disease or condition, or from the results of active treatment, in comparable patients or populations." (21 CFR 314.126) Homozygous.
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Medical device. "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 of 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
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When a sample containing DNA or RNA is placed in contact with the gene chip, base pairing between the expressed sequences in the sample and complementary sequences on the gene chip produces light, which allows the expressed sequences in the sample to be identified. (http://www.genome.gov/glossary/ index.cfm?
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Form used by drug sponsors to request FDA approval to market a new pharmaceutical in the United States based on information about its safety and effectiveness and other requirements. No treatment concurrent control.
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In a clinical trial, the "test drug is compared with an inactive preparation designed to resemble the test drug as far as possible." (21 CFR 314.126) Pluripotent stem cell.
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Rare disease. In the Orphan Drug Act, a disease or condition that affects fewer than 200,000 people in the United States (21 USC 360bb)
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A stem cell transplant may be autologous (using a patient's own stem cells that were saved before treatment) , allogeneic (using stem cells donated by someone who is not an identical twin)
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Use of a drug or medical device for a purpose, patient group, or other use that is not specifically approved by the Food and Drug Administration for use as indicated on the product's label. Such use by physicians is considered part of the practice of medicine, which FDA -- by statute -- does not regulate.
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Washington, DC: The National Academies Press. ABBREVATIONS ACMG American College of Medical Genetics AHRQ Agency for Healthcare Research and Quality ANDA Abbreviated New Drug Application ATSDR Agency for Toxic Substances and Disease Registry BLA Biologics License Application CAN Cures Acceleration Network CBER Center for Biologics Evaluation and Research CDC Centers for Disease Control and Prevention CDER Center for Drug Evaluation and Research CDRH Center for Devices and Radiological Health CFF Cystic Fibrosis Foundation CLIA Clinical Laboratory Improvement Amendments of 1988 CML chronic myelogenous leukemia CMS Centers for Medicare and Medicaid Services COG Children's Oncology Group C-Path Critical path Institute CTSA Clinical and Translational Science Awards CTX cerebrotendinous xanthomtosis EMEA European Medicines Agency FDA Food and Drug Administration FEV1 forced expiratory volume FMF familial Mediterranean fever FOP fibrodysplasia ossificans progressiva
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RARE DISEASES AND ORPHAN PRODUCTS GAO Government Accountability Office GenTAC National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions HDE Humanitarian Device Exemption HHS U.S. Department of Health and Human Services ICD International Classification of Diseases ICD implantable cardioverter defibrillator IDE Investigational Device Exemption IND Investigational New Drug IOM Institute of Medicine IRB Institutional Review Board MedPAC Medicare Payment Advisory Commission NAGS N-acetylglutamate synthase NCGC NIH Chemical Genomics Center NCI National Cancer Institute NDA New Drug Application NHLBI National Heart, Lung, and Blood Institute NIH National Institutes of Health NORD National Organization for Rare Disorders OMIM Online Mendelian Inheritance in Man OOPD Office of Orphan Products Development ORDR Office of Rare Diseases Research PHS Public Health Service PMA Premarket Approval application RAID Rapid Access to Interventional Development RDCRN Rare Diseases Clinical Research Network REMS Risk Evaluation and Mitigation Strategy SEER Surveillance, Epidemiology, and End Results SSDI Social Security Disability Insurance TRND Therapeutics for Rare and Neglected Diseases VEPTR Vertical Expandable Prosthetic Titanium Rib WHO World Health Organization
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101-629 Safe Medical Devices Act of 1990 P.L. 102-571 Prescription Drug User Fee Act of 1992 P.L.
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Key Terms
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