Download the new resource here.

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 edition of the eBook, Avalere Health experts discuss issues and considerations associated with rare-disease drug development, ex-US policy considerations, US federal and state-level programs and initiatives, the economic burden associated with rare diseases, and opportunities for a cohesive quality strategy in these conditions.

The eBook includes the following chapters: 

• Manufacturer Considerations for Rare Disease Drug Development 
• The Impact of European Policy and Advocacy on Progress in Rare Disease 
• Newborn Screening: US Landscape and Rare Disease Development
• Rare Disease Advisory Councils: Opportunities for Engagement
• Assessing the Total Economic Burden of Rare Disease 
• The State of Quality in Rare Disease
• Case Studies 
• Every Patient Possible

Download the free Rare Disease eBook.

Want to learn more? Join our Rare Disease Day webinar: From patient empowerment to market impact on February 28 at 10AM ET. We will explore headwinds and tailwinds for shortening the diagnostic odyssey, what it means to live with a rare disease or be a caregiver to someone affected by it, and how manufacturers can navigate evolving market dynamics to ensure successful commercialization of treatments while securing patient access. Click here to register.