European Medicines Agency [EMA]

Biologic medicines, or protein-based medicines such as insulin, are designed to interact with specific targets in the patient’s body. A more targeted mechanism of action should lead to a greater chance of the medicine having the desired effect against the disease and should result in fewer side effects than traditional medicines.

Nanoparticles are tiny objects, available only as intravenous formulations, that help medicines get to difficult-to-reach cells and tissue. They can also help protect molecules and control the targeted release of medicines.

Small populations present ethical and practical challenges to organising clinical trials. Small populations include groups with rare diseases or specific sub-types of common diseases, children, and the elderly.

Non-clinical studies can be performed at any time during the life-cycle of the product, testing the compound outside of humans. Non-clinical studies aim to identify candidate therapies with the greatest probability of success, assess safety, and build solid scientific foundations before the transition to clinical development phases.

Data from non-clinical trials is important as a predictor and decision-making tool in the medicines development process. Adequate and satisfactory non-clinical results are required before a medicine may be administered to healthy human volunteers, and non-clinical data is closely reviewed before a medicine is allowed to enter the market.