Pharmaceutical Drug Approval

Decision to Approve a Drug

• The decision to approve a drug is based on its satisfactory balance of benefits and risks within the specified conditions in the product labeling.
• The decision is made based on the available information at the time of approval.

Safety Profile and Pharmacovigilance

• The safety profile of a product can change over time through expanded use, and new information is generated, which can impact the benefits or risks of the product.
• Evaluation of this information should be a continuous process, in consultation with regulatory authorities.
• Detailed evaluation of pharmacovigilance activities is essential to ensure the safe use of products.

Benefit-Risk Balance

• The benefit-risk balance can be improved by reducing risks to patients through effective pharmacovigilance.
• Pharmacovigilance enables information feedback to users of medicines in a timely manner.

Industry and Regulators

• Industry and regulators have identified the need for better and earlier planning of pharmacovigilance activities before a product is approved or licensed.
• The ICH guidance aims to encourage harmonization and consistency, preventing duplication of effort, and benefiting public health programs worldwide.

Epidemiology and Incidence Rates

• Discussions should include incidence, prevalence, mortality, and relevant co-morbidity, taking into account stratification by age, sex, and racial and/or ethnic origin.
• Differences in epidemiology across regions should be discussed, if available.
• Reviewing incidence rates of important adverse events among patients in whom the drug is indicated is useful.

Safety Specification and Pharmacovigilance Plan

• The safety specification should identify risks believed to be common to the pharmacological class.
• A summary of the safety specification should be provided, including important identified risks, important potential risks, and important missing information.

Pharmacovigilance Plan

• The pharmacovigilance plan should be based on the safety specification and updated as important information becomes available.
• The plan should include a summary of ongoing safety issues, routine pharmacovigilance practices, and an action plan for safety issues.

Routine Pharmacovigilance Practices

• Routine pharmacovigilance should include systems and processes to collect and collate adverse reaction information.
• Preparation of reports for regulatory authorities, including expedited adverse drug reaction reports and periodic safety update reports, is necessary.
• Continuous monitoring of the safety profile, including signal detection, issue evaluation, and updating of labeling, is required.

Action Plan for Safety Issues

• The plan for each important safety issue should be presented, including the safety issue, objective, proposed action, rationale, monitoring, and milestones.

Drug Event Monitoring and Registries

• Drug event monitoring is a method of active pharmacovigilance surveillance, involving the identification of patients from electronic prescription data or automated health insurance claims.
• Registries, such as disease registries or drug registries, can collect a battery of information using standardized questionnaires in a prospective fashion.