Clinical efficacy and safety guidelines

The European Medicines Agency's scientific guidelines on the clinical efficacy and safety of human medicines help applicants prepare marketing authorisation applications. Guidelines reflect a harmonised approach of the EU Member States and the Agency on how to interpret and apply the requirements for the demonstration of quality, safety and efficacy set out in the Community directives.

The Agency's Committee for Medicinal Products for Human Use (CHMP) prepares scientific guidelines in consultation with regulatory authorities in the European Union (EU) Member States, to help applicants prepare marketing-authorisation applications for human medicines.

Guidelines reflect a harmonised approach of the EU Member States and the Agency on how to interpret and apply the requirements for the demonstration of quality, safety and efficacy set outin the Community directives.

The Agency strongly encourages applicants and marketing authorisation holders to follow these guidelines. Applicants need to justify deviations from guidelines fully in their applications at the time of submission. Before that, they should seek scientific advice, to discuss any proposed deviations during medicine development.

Clinical efficacy and safety guidelines are provided for:

• Alimentary tract and metabolism
• Blood and blood forming organs
• Blood products (including biotechnological alternatives)
• Cardiovascular system
• Dermatologicals
• Genito-urinary system and sex hormones
• Anti-infectives for systemic use
• Antineoplastic and immunomodulating agents
• Rheumatology/musculoskeletal system
• Nervous system
• Respiratory system
• Orally inhaled products
• Radiopharmaceuticals and diagnostic agents
• Allergy/Immunology
• Biostatistics
• General
• Real-world evidence