Developing a strategy for rare diseases in France requires a clear understanding of the care ecosystem, key opinion leaders and regulatory requirements
Pharma companies face several strategic challenges:
- How to identify eligible patients and improve their diagnostic and referral pathway?
- How to navigate the regulatory and market access environment to secure timely and sustainable reimbursement?
- How to engage effectively with expert centers, rare diseases networks and patient advocacy groups, while generating the evidence needed to demonstrate long-term value?
This new paper is a synthetic review of the situation analysis, the key trends and the implications for pharma companies