Off-Label Drug Use (OLDU) Concepts and Definitions

Flashcards covering key terminology, medical concepts, legal precedents, trial phases, and legislative background related to off-label drug use as presented in the article.

Off-label drug use (OLDU)

The practice of prescribing currently available and marketed medications for an indication, patient population, dosage, or dosage form that has not received approval from the US Food and Drug Administration (FDA).

Food, Drug, and Cosmetic Act of 1938

A US statute that historically required new medications to be safe before commercial availability and granted the FDA authority to regulate drug promotional materials.

Kefauver-Harris Amendment

A 1962 amendment to the Food, Drug, and Cosmetic Act mandating that FDA-approved new drugs must demonstrate evidence of efficacy in addition to safety.

On-label prescribing

Prescribing FDA-approved medications specifically for the indications, populations, doses, and forms for which they have been demonstrated to be safe and effective.

FDA Modernization Act of 1997

Legislation that created economic incentives, including exclusive marketing rights and patent extensions, for pharmaceutical companies to test medications in pediatric populations.

Indomethacin

A nonsteroidal anti-inflammatory agent used off-label as a medical therapy to close a persistent, symptomatic patent ductus arteriosus in newborns to avoid surgery.

Canterbury v Spence

A landmark 1972 legal case regarding medical informed consent that established the test for disclosing a particular peril based on its materiality to the patient's decision.

Material risk

A risk to which a reasonable person, in the patient's position as known by the physician, would likely attach significance when deciding whether to undergo or forego a proposed therapy.

Medical malpractice

A broad legal category including negligence, which requires proving four elements: a physician's duty to the patient, a breach of that duty, an injury requiring compensation, and a causal link between the breach and the injury.

Duty of care

The legal standard defining the degree of care a prescribing physician must provide, set as the same degree of care provided by other physicians practicing under similar circumstances.

Phase 0 clinical trial

Preliminary clinical trials involving pharmacokinetic and pharmacodynamic studies of subtherapeutic drug doses in small numbers of human subjects.

Phase 1 clinical trial

Initial clinical studies evaluating drug pharmacodynamic properties and safety in small groups of healthy human volunteers.

Phase 2 clinical trial

Clinical studies conducted to evaluate drug pharmacology, safety, and efficacy in larger groups of human volunteers and patients.

Phase 3 clinical trial

Large, randomized, multicenter clinical trials evaluating drug safety and efficacy in comparison to a placebo or an existing standard treatment.

Phase 4 clinical trial

Post-marketing clinical studies completed after FDA approval to further delineate a drug's long-term effects and adverse reactions.

Seeding reports

Articles or clinical trials primarily designed to support a commercial marketing angle for a drug proprietor rather than to address genuine scientific questions.

Misbranding

The prohibited practice of labeling or distributing a medication with false or misleading information, including unapproved or off-label indications.

Bad Ad Program

An FDA initiative officially named the Truthful Prescription Drug Advertising and Promotion Program, launched in 2010 to allow health care professionals and patients to report illicit off-label marketing.

Orphan drugs

Medications developed and used to treat rare diseases (orphan diseases) that typically affect small patient populations and offer limited commercial profitability.

Orphan Drug Act

A 1983 federal law offering financial incentives—such as tax breaks, exclusive marketing rights, and reduced application fees—to encourage pharmaceutical manufacturers to develop drugs for rare diseases.