eBook: Rare Disease Biotechnology Landscape

  • This page as PDF

Summary

In an eBook, Avalere and Avalere Health experts describe the policy, market access, and evidence landscape of rare disease in the United States and globally.

Rare diseases are not so rare. This is a commonly used phrase in the rare disease community. There are more than 10,000 known rare conditions that affect more than 30 million Americans and more than 400 million people worldwide. This diverse and complex disease area poses challenges not only to patients but their caregivers, regulators, pharmaceutical manufacturers, policymakers, and other stakeholders. Considering that less than 10% of these conditions have any treatment options available, there is a significant unmet need in this space.  

In this eBook, Avalere and Avalere Health experts discuss issues and considerations associated with rare-disease drug development, the economic burden associated with these conditions, US and ex-US policy considerations, novel approaches to shortening the diagnostic odyssey, and clinical trial design.  

The eBook includes the following chapters: 

  • Manufacturer Considerations for Rare Disease Drug Development 
  • The Impact of European Union Policy and Advocacy on Progress in Rare Disease 
  • Assessing the Total Economic Burden of Rare Disease 
  • Shortening the Diagnostic Odyssey: Benefits, Barriers, and Solutions 
  • FDA Diversity Requirement: Impact on Rare Disease Drug Manufacturers 
  • Decentralized Clinical Trials for Rare Disease Treatments 
  • Case Studies 

Download the free Rare Disease eBook. 

Webinar: Decoding the CY 2025 Advance Notice Join Avalere’s healthcare policy experts as they dissect the CY 2025 Medicare Advantage Advance Notice and discuss the future trajectory of MA in Part D and potential outcomes, headwinds, and tailwinds for health plans. Learn More
Watch the Recording
From beginning to end, our team synergy
produces measurable results. Let's work together.
Back To Top