World Orphan Drug Congress 2023: solutions for market access and HEOR

World Orphan Drug Congress 2023 takes place in Barcelona, from October 31 to November 2. In this policy, access, value, and evidence update, Catherine Kielar, Executive Director and Practice Lead, and Sanjeev Gogna, Executive Director, share five access and HEOR challenges for orphan drug manufactures and invite you to meet them at the congress to start discussing solutions.

Global payer and policy landscapes are rapidly evolving, so it is important for orphan drug manufacturers to understand the new evidentiary requirements, economic considerations, and service implications related to their assets. 

Let’s meet at the World Orphan Drug Congress to talk through some of the latest market access and HEOR challenges and opportunities for orphan drugs, including: 

1. Political 

• Policies are more established for regulatory approval than for reimbursement. 
• Current value frameworks may not fully capture the value of treatments. 
   

2. Technological 

• Complex manufacturing and delivery mechanisms may require service redesign. 
• Accurate diagnosis may require access to diagnostic/genetic testing. 
   

3. Clinical 

• High level of clinical uncertainty due to small patient numbers, non-comparative trials, surrogate/novel endpoints, and lack of long-term follow-up. 
• Challenging to accurately model long-term outcomes. 
   

4. Economic 

• High cost due to manufacturing complexity and small patient populations 

• Potential for high up-front costs for once-only curative treatments. 
• Potential for innovative payment models and additional data collection, which may be administratively burdensome. 
• Significant pipeline activity across many indications, with potential for differential value across indications. 
   

5. Social 

• Greater role for patient advocacy and patient and caregiver perspectives. 

 
Creating connections across policy, access, value, and evidence for orphan drugs. 

We provide cutting-edge market access and pricing and reimbursement solutions for orphan drugs and regenerative medicines across a range of therapeutic areas and services. Our experts will work with you through the whole process, creating connections from early-stage development to evidence generation, evidence synthesis and communication. 

Our experience includes: 

• Mapping and understanding the policy landscape and payer requirements for orphan drugs globally. 
• Developing market access roadmaps. 
• Developing numerous value propositions, GVDs, and objections handlers for products indicated for rare diseases. 
• Facilitating in-depth interviews, panels, advisory boards, and mock HTA meetings with payers. 
• Support for obtaining early scientific advice from HTA agencies. 
• Understanding P&R opportunities and challenges, and innovative pricing strategies. 
• Conducting treatment patterns surveys and Delphi panels to understand treatment use and reach clinical consensus. 
• RWE research and strategy development  
• Early economic modeling and model conceptualization  
• PRO strategy, statistical analysis, and publications. 
• Conducting literature reviews, landscape assessments, and HTA reviews. 

Download our case studies: market access and HEOR solutions for orphan drugs. 

This collection of case studies illustrates some of the solutions to the market access and HEOR challenges faced by manufactures of orphan drugs. 

If you cannot meet us at World Orphan Drug Congress, this download is a great place to find out more about our global experience and best-in-class services.