Patient advocacy groups (PAGs) are playing an ever-widening role across disease areas, from supporting individual patients to driving shifts in therapy development, market access and policy. Nowhere is this potentially more significant than in rare diseases where the awareness and understanding of patients’ conditions and needs are lower, regardless of treatment availability.
As industry evolves to become more patient-centric, effective collaboration with PAGs may provide a competitive advantage. This is particularly relevant in the rare disease space where patients play an even more important role in influencing treatment choices and access to therapies.
In this paper, we explore recommendations for more impactful collaborations with PAGs in the rare disease space. We look at where PAGs have been effective in influencing decisions and processes, and the impact of pharma/PAG collaboration in rare diseases.
France’s temporary authorisation (ATU) programme: reform implications
The temporary authorisation for use (Autorisation Temporaire d’Utilisation, ATU) programme in France is intended to provide early access to medicines for...