Pharmacovigilance and Risk Management Strategies Conference

For patients with serious illnesses and unmet medical need, access to innovative medicines as early as possible is important. Randomised controlled clinical trials are important for establishing efficacy of a medicine but may provide only limited evidence of how a medicine will perform in the real world. There is relatively little known about the true safety profile of a drug at the time of “normal” authorisation and some critics voice concerns about patient safety as an argument against regulatory pathways providing earlier access. Some stakeholders are wanting evidence of effectiveness before making new medicines available for patients. How do we balance all these conflicting needs and how to we plan a development pathway to satisfy all, or at least most, stakeholders?