Assessment of Regulatory and Access Opportunities for Rare Pediatric Disease Therapy

The client sought to gauge the options for its rare disease assets to enter the market of Georgia. Different access scenarios, both standard and early (pre-authorization), and pricing and reimbursement implications were thoroughly analyzed based on and tailored to versatile treatments' characteristics, clinical trial design and available real-world evidence of the therapy of interest.

• Overview of pre-approval mechanisms to support successful subsequent marketing authorization of the drug.
  
• Analysis of market entry strategies for rare disease assets already presented in the country.
  
• Marketing authorization and reimbursement implications of competitive orphan therapies.
  
• Overview of Pricing&Reimbursement and HTA landscape and recommendations for optimal pathways.

A detailed report with tables and schemes outlining key information related to early access, marketing authorization, regulator's approach to evaluation of available clinical data, P&R landscape, main stakeholders and decision-making style, as well as a set of recommendations for the most relevant options of market access for the Client.