Topic

Drug development and regulation

Before a medicine can be priced or appraised it must be approved, and regulators continue to monitor its safety once it is in use. Concepts here include accelerated approval, the Abbreviated New Drug Application for generic medicines, advanced therapy medicinal products, and risk evaluation and adverse event reporting after launch.

Concepts in this topic

• Abbreviated New Drug ApplicationA streamlined US regulatory application letting a generic manufacturer gain approval by demonstrating bioequivalence to an approved reference drug, without repeating full trials.
• Accelerated ApprovalAccelerated approval is an FDA pathway that approves medicines for serious conditions on a surrogate endpoint, with confirmatory trials to verify benefit.
• Advanced Therapy Medicinal ProductAn advanced therapy medicinal product (ATMP) is an EU-regulated gene, cell or tissue-based medicine, often given once, whose lasting value HTA must judge.
• Adverse Drug ReactionAn adverse drug reaction (ADR) is a noxious, unintended response to a medicine, where a causal link to the drug is at least a reasonable possibility.
• Adverse Event ReportingAdverse event reporting sends suspected drug reactions and safety incidents to regulators or national systems, giving safety signals but not event rates.
• Biological TherapyTreatment using a medicinal product made from or derived from a biological source, assessed for a specific indication, delivery pathway and patient outcome.
• Precision MedicineAn approach to prevention, diagnosis or treatment that uses relevant individual or disease characteristics to select a more appropriate care strategy.
• Risk EvaluationA formal assessment of a drug's potential safety risks, sometimes resulting in a required mitigation strategy such as restricted distribution or mandatory counselling.