New approaches to product approval – Balancing early release with improved safety monitoring
14-15th June 2007, Sofitel Hotel, Washington DC, USA
Different approaches have been adopted worldwide to make new medicines available to patients more rapidly, including orphan drug programmes, conditional approvals and accelerated approval procedures. Earlier availability must, however, be balanced against appropriate safety monitoring during the post‑approval phase.
This Workshop consolidated earlier Institute discussions on changing development and review paradigms. It focused on faster access to new medicines through early release with follow‑up in real‑world patient populations, supported by enhanced post‑marketing surveillance, including the use of large‑scale electronic medical record (EMR) databases. It also considered whether early‑availability models should be extended beyond areas of acute unmet medical need.
Conclusions
Review models
After reviewing current standard and accelerated approval models, the Workshop concluded that efforts should focus on improving existing models rather than introducing radical new approval frameworks. This reflected a shared recognition that regulators and industry operate in an increasingly risk‑averse environment and must carefully balance benefit and risk.
Safety issues
Current safety criteria were considered adequate for standard approvals. However, if early‑release approaches are to be expanded, new and more robust post‑approval safety monitoring measures will be required in order to maintain public and professional confidence.
Population‑based EMR databases
Improved and more coordinated use of large‑scale population‑based electronic medical record databases was identified as a key enabler of enhanced post‑marketing surveillance. It was recommended that a dedicated forum be established to address database standards, develop guidance, align industry and regulatory expectations regarding database capabilities, and promote training and education in database technologies.
Communication
Effective communication was regarded as central to successful implementation of any changes to approval models or post‑marketing surveillance. The Workshop emphasised the need for coordinated, multidisciplinary efforts to improve how benefits and risks of medicines are communicated to all stakeholders, including healthcare professionals, patients, payers and policy makers.