Patients cannot be mere spectators in decisions that affect their health—they must be active participants in the process. Across Europe, patient advocacy groups (PAGs) are increasingly recognized as key players in ensuring that patient voices are heard at every stage of a drug’s lifecycle, from research and development to approval, pricing, and reimbursement.
Patients bring unique firsthand insights, which are particularly valuable in rare diseases where traditional data sources may be limited. Structured dialogue with patient organizations enhances the quality of regulatory decisions, making healthcare systems more equitable, transparent, and patient-centered.
The Role of PAGs in European Decision-Making Processes
In several European countries, patient organizations are playing an increasingly crucial role in Health Technology Assessment (HTA) and regulatory decision-making:
– European Union: The new HTA Regulation, set to take effect in 2025, encourages patient associations to participate in Joint Scientific Consultations and Joint Clinical Assessments, fostering direct interaction with regulatory agencies and pharmaceutical developers.
– France, Germany, United Kingdom: National healthcare authorities have established formal frameworks for periodic consultations with PAGs, ensuring that patient perspectives are integrated throughout the HTA process.
– Italy: The new AIFA regulation allows the Scientific and Economic Commission (CSE) to summon PAGs for consultations. Additionally, the InPagsNetwork initiative, bringing together over 50 patient associations, is working to strengthen the dialogue between PAGs and AIFA, ensuring that patient input has a tangible impact on regulatory decisions.
When should pharma companies engage with PAGs?
Patient engagement should start as early as possible. True collaboration is not just about securing support during the approval phase—it begins much earlier, at the very start of drug development. Engaging patients from the outset is not only a way to build trust but also a win-win strategy to develop therapies that truly meet the needs of the patient community.