Publications Glossary

ABPI
Association of the British Pharmaceutical Industry

Accelerated approval
FDA program 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

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

ADE
adverse drug event; adverse drug effect

ADME
absorption, distribution, metabolism, and excretion (used to describe pharmacokinetic processes)

ADR
adverse drug reaction

AERS
Adverse Event Reporting System (FDA)

AHRQ
Agency for Healthcare Research and Quality

AMCP
Academy of Managed Care Pharmacy

ANDA
abbreviated new drug application (FDA)

Antibody
complex protein–based molecule produced by B–lymphocytes that binds to and helps eliminate foreign and infections agents

Approval phase time
time from date of submission of an NDA or BLA to date of U.S. FDA approval

Approval Success Rate
the percentage of NCEs in clinical development that eventually obtains FDA marketing approval

Attrition Rate
the percentage of NCEs that drop out of testing in a clinical phase

BIO
Biotechnology Industry Organization

Biopharmaceutical
any therapeutic biological compound, including recombinant proteins, monoclonal and polyclonal antibodies, antisense oligonucleotides, therapeutic genes, and recombinant and DNA vaccines.

BLA
biologics license application, which is an application to FDA for a license to market a new biological product in the United States

Capitalized costs
sum of out-of-pocket and time costs

CBER
FDA's Center for Biologics Evaluation and Research

CDER
FDA's Center for Drug Evaluation and Research

CDISC
Clinical Data Interchange Standards Consortium (formerly a DIA special interest group called the Clinical Data Interchange Standards Committee)

CDRH
FDA's Center for Devices and Radiological Health

CFR
Code of Federal Regulations

CHMP
EMEA’s Committee for Human Medicinal Products reviews product applications through a centralized procedure and forwards a scientific opinion on each to the European Commission for final marketing authorization.

Clinical approval success rate
percentage of investigational new compounds entering clinical testing that eventually obtain FDA marketing approval

Clinical phase time
time from filing of an IND, which is necessary to begin testing of a new compound in human subjects, to the submission of an NDA or BLA

Clinical studies
studies done on human subjects, such as randomized controlled trials, registry studies, and pediatric PK/PD studies.

CMC
chemistry, manufacturing and controls

CMS
Centers for Medicare & Medicaid Services (formerly Health Care Financing Administration)

CPMP
Committee for Proprietary Medicinal Products (EU)

CRADA
cooperative research and development agreement (with NIH)

CRF
case report form (sometimes case record form)

CRO
contract research organization. An organization contracted by a drug sponsor to manage various steps in the drug or biologic development process, including study design to trial execution, data management, analysis, and medical writing.

CTD
common technical document

DESI
Drug Efficacy Study Implementation notice. IOM initiative to assess the efficacy of drugs in use before 1962.

Development Cycle Time
time from filing of an investigational new drug application (IND), which is necessary to begin testing of a new compound in human subjects, to submission of a new drug application (NDA) or biologics license application (BLA).

Disease management
a continuous, coordinated health care process that seeks to manage and improve the health status of a carefully defined patient population over the entire course of a disease. The patient populations targeted are high–risk, high–cost patients with chronic conditions that depend on appropriate pharmaceutical care for proper maintenance. Disease management services include disease prevention efforts as well as patient management once the disease states have developed.

DME
drug metabolizing enzyme

DMO
disease management organizations

DPC-PTR Act
Drug Price Competition and Patent Term Restoration Act of 1984 (also Waxman–Hatch or Hatch–Waxman Bill)

DTC
direct-to-consumer (drug advertising)

EC
European Commission

ECG or EKG
electrocardiogram

EDC
electronic data capture/collection

EDL
Essential Drug List of the World Health Organization (WHO). Drugs deemed by WHO to be essential to address the health needs of any developing nation. Each drug on the list is recommended for one or more associated indications.

Efficacy
refers to whether or not the mechanism or compound treats the disease, and whether or not it is able to differentiate over existing treatments

EMEA
European Agency for the Evaluation of Medicinal Products. Similar to the FDA, the EMEA is responsible for protecting public health by mobilizing the best scientific resources within the European Union, promoting health care through the effective regulation of new pharmaceuticals, and supporting the pharmaceutical research and development industry by developing efficient, effective and responsive operating procedures

EU
European Union

Exceptional Circumstances
EMEA designation allowing products to be approved in cases where comprehensive quality, safety, and efficacy data cannot be provided. The applicant must agree to conduct post-approval studies, and labeling must reflect the lack of adequate data..Analogous to the FDA’s accelerated approval designation, which allows a drug to be approved on the basis of a surrogate endpoint.

Fast track
FDA designation for a therapeutic development program intended for treating a serious or life–threatening condition and which has the potential to address unmet medical needs for the condition

FDA
Food and Drug Administration. The U.S. federal agency charged with promoting and protecting the public health by helping safe and effective products reach the market in a timely way and monitoring products for continued safety after they are in use.

FDAMA
FDA 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

Follow–on drug or Me–too drug
new entrant to a therapeutic class defined by another drug entity that was the first to receive regulatory approval for marketing.

Follow–on indication
any indication approved by FDA after an originally approved indication.

Formulary
a list of selected pharmaceuticals and dosages recommended by health insurers. Formularies are developed by Pharmacy and Therapeutics Committees, which consider drug safety, efficacy, quality, and cost–effectiveness in the decision whether or not to include a drug in a formulary.

FPFV
first patient, first visit

Fulfilled studies
Postmarketing commitments that have been submitted and which have received a determination from the FDA that the commitment has been fulfilled

GCP
good clinical practice

GLP
good laboratory practice

GMP
good manufacturing practice

HHS
U.S. Department of Health and Human Services (also called DHHS)

HIPAA
Health Insurance Portability and Accountability Act of 1996. Established to improve portability and continuity of health insurance coverage in group and individual markets, combat waste, fraud, and abuse in health insurance and health care delivery, improve access to long–term care services and coverage, and simplify the administration of health insurance, among other goals.

ICH
International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use

IDM
independent disease manager. An entity that develops and operates care management systems, usually on behalf of a healthcare provider network or insurance company, for patients with chronic conditions.

IND
investigational new drug application. Notification by a drug sponsor to the FDA of its intention to conduct clinical studies on human subjects.

IRB
institutional review board; independent review board

List A
EMEA designation for products developed by means of recombinant DNA technology, controlled expression of genes coding for biologically active proteins in prokaryote and eukaryote cells, including seven transformed mammalian cells, and hybridoma and monoclonal antibody methods.

List B
EMEA designation for optional submissions to the centralized procedure, which includes new and innovative medicinal products not included in List A.

LPFV
last patient, first visit

LPLV
last patient, last visit

M&A
mergers and acquisitions

mAb
monoclonal antibody

MCO
managed care organization. An entity that combines the financing and delivery of health care into one organization, such as a Health Maintenance Organization or Preferred Provider Organization.

MedDRA
Medical Dictionary for Regulatory Activities. New global standard medical terminology designed to supersede other terminologies used in the medical product development process, including COSTART, ICD9, and others.

MHLW
Ministry of Health, Labor and Welfare (Japan)

Monoclonal antibody (mAB) types
murine mAbs are derived from mouse genes; human mAbs are derived from human genes; chimeric and humanized mAbs are each derived from varying amounts of mouse and human genes, with the humanized products containing more human protein sequence than the chimeric versions

MOU
Memorandum of Understanding (e.g., an MOU between FDA and a regulatory agency in another country allows mutual recognition of inspections)

Multi–tier cost sharing
graduated system of co–payments mandated by health insurers in which patients share in the cost of the prescription medicines. Typically, lower tiers with smaller co–payments apply to generic drugs, and higher tiers with higher co–payments apply to brand name drugs.

NAS
new active substance (UK)

NCE
new chemical entity. Any new molecular compound (excluding diagnostic agents, vaccines, and other biologic compounds) not previously approved for human use by FDA. Also excluded are new salts, esters, and dosage forms of previously approved compounds.

NCI
National Cancer Institute

NDA
new drug application. An application to FDA for a license to market a new drug in the United States.

NIH
National Institutes of Health

NME
new molecular entity

Non–clinical studies
studies that were not conducted on human subjects, such as in vitro studies, animal studies, and studies on product quality.

OHRP
Office for Human Research Protections

Orphan Drug
drugs developed for rare diseases and conditions which, in the U.S., affect fewer than 200,000 people or, in the EU, 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.

OTC
over–the–counter (refers to nonprescription drugs)

Out–of–pocket costs
cash outlays incurred developing new biopharmaceuticals or pharmaceuticals

Overall cycle time
total time from IND filing to NDA or BLA approval.

Patient compliance with prescribed drug regimens
the degree of correspondence between the patient’s actual dosing history and the prescribed dosing regimen, the result of a comparison of two time–series

PBM
pharmacy benefit manager. An entity that processes pharmacy claims, manages drug formularies, and negotiates manufacturer rebates and pharmacy discounts on behalf of its clients.

PD
pharmacodynamics

PDUFA
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.

Pediatric study
clinical research undertaken by firms, in response to incentives provided by the Best Pharmaceuticals for Children Act, to develop appropriate formulations as well as prescribing information for drugs (biologics were not eligible for the program) used to treat children.

Persistence
the length of time elapsed between the first–taken and the last–taken dose in a prescribed dosing regimen

Pharmacodynamics
study of the biochemical and physiological effects of drugs on the organism and the relationship between drug concentration and effect, i.e. what a drug does to the body.

Pharmacogenetics
the genetic determinants of how individuals metabolize drugs

Pharmacogenomics
study of how an individual's genetic inheritance (genome) affects the body's response to drugs, and the use of genetic testing in conjunction with drug therapy

Pharmacokinetics
the analysis of the course of substances in the body and their relationship with an organism or system over time. A pharmacokinetic study is a systematic assessment of what the body does to a drug after it has been introduced into the body.

Pharmionics
the discipline concerned with measuring and understanding ambulatory patients’ use of prescription drugs: ‘what the patient does with the drug’

Phase I Study
typically conducted in healthy volunteers and designed to determine the pharmacokinetic and pharmacologic actions of the drug in human subjects, the side effects associated with increasing doses, and, if possible, early evidence of effectiveness

Phase II Study
helps determine the scientific validity of the drug by obtaining data on the effectiveness of the drug for a particular indication or indications in patients with the disease or condition, and helps determine the common short–term side effects and risks associated with the drug

Phase III Study
involves greater numbers of patients than in phase II, aimed at gaining additional data about effectiveness and safety needed to evaluate the benefits and risks of the drug. Results yield data that will provide information that eventually will go on physician labeling.

Phase transition probability
The likelihood that an investigational new compound that begins a particular development phase will transition to the next development phase

PhRMA
Pharmaceutical Research and Manufacturers of America

PHS
Public Health Service (U.S.)

PI
FDA–regulated U.S. principal investigator

PK
pharmacokinetics

PLA
product license application

PMA
pre–market approval application

PMCs
postmarketing study commitments. Post-approval studies that are requested or required by the FDA at the time of product approval, and which the sponsor agrees to conduct.

PMS
postmarketing surveillance

PPI
patient package insert

Preclinical R&D
all research and development conducted prior to the initiation of clinical testing, including discovery, in vitro testing, and animal studies.

Priority and standard NCEs
priority NCEs are considered by CDER to offer high therapeutic value and earmarked for priority review status. Standard NCEs are viewed as offering little or no therapeutic advantages over existing therapies and receive lower review status. CBER also grants priority review status, but the criteria are somewhat different than CDER; the products must also be for serious or life–threatening illness.

Priority drugs
those considered by the FDA to offer high therapeutic value and are earmarked for priority review

QALY
quality adjusted life year. Measure that takes into account both quantity and quality of life generated by health care interventions. It is the product of life expectancy and a measure of the quality of remaining life years.

RCT
randomized controlled trial

Regulatory cycle time
time from the date of NDA or BLA submission to FDA approval.

Rx–to–OTC switch
change in legal marketing status of a drug from prescription-only to over-the-counter status

Significant biologic
newly developed biologically-derived product not previously approved in the U.S and not classified as an NME, such as most therapeutic monoclonal antibodies, recombinant proteins, and vaccines.

SNDA
supplemental new drug application

SNP
single nucleotide polymorphism

Standard drugs
viewed as offering little or no therapeutic advantage over existing therapies and receive lower review status by the FDA

Submitted studies
postmarketing commitments that have been concluded or terminated by the sponsor, and final study reports have been submitted, but FDA has not made final determination that commitment has been fulfilled.

Subpart E
program launched in 1987 that was intended to speed access to AIDS drugs by allowing drug sponsors to skip phase III clinical trials and commit to conduct specific phase IV post–approval studies

Success rate
the percentage of products beginning clinical testing that eventually receive FDA marketing approval

Tier
level of co–payment an insurer asks its insured to bear for services and drugs

Time cost
earnings that investors forego on funds used to support R&D activities before any return can be earned

TPP
Canada's Therapeutic Product Programme

User fee era
period following passage of the Prescription Drug User Fee Act of 1992 (PDUFA), authorizing the FDA to collect user fees for regulatory review of new drug applications

USP
United States Pharmacopoeia. The official public standards-setting authority for therapeutic classification of all prescription and over-the-counter medicines, dietary supplements, and other health care products manufactured
and sold in the United States.

Vaccine
preparation that contains an antigen, consisting of whole disease-causing organisms (killed or weakened) or parts of such organisms. Vaccine preparations can be natural, synthetic, or derived by recombinant DNA technology, and they may be therapeutic or prophylactic.

WHO
World Health Organization

Written request
issuance of a written request by the FDA, which initiates the process of undertaking a pediatric study program in exchange for exclusivity. The request details the agency’s interest in a particular active ingredient. This is followed by submission of studies that may result in an award of pediatric exclusivity and, ultimately, labeling change.

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