Launch is Just the Beginning: Rare Disease Care Requires Durability

Feature|Videos|July 7, 2026

Launch is Just the Beginning: Rare Disease Care Requires Durability

Author(s)Bansi Nagji, CEO, PANTHERx Rare

Key Takeaways

Understand why scientific innovation targets rare and orphan disease treatments
Learn what distinguishes a rare-only pharmacy model from traditional specialty pharmacy approaches
Recognize why launch is only the starting point for long-term therapy success

Bansi Nagji discusses the shifting science behind rare and orphan drug development, including what it takes for therapies to succeed well beyond launch

Bansi Nagji, CEO of PANTHERx Rare, explores how innovation is increasingly directed toward rare and orphan diseases, driven by targeted incentives, tax breaks, and a more streamlined FDA approval pathway. Nagji covers the operational realities that set rare disease therapies apart, including cold chain logistics, weight-based titration regimens, and the intensive patient interactions required before a single prescription is even filled. Learn why launch marks the beginning of a therapy’s journey rather than its conclusion, and why ongoing work is necessary for payer access and long-term adherence.

What's Hot

As Florida launches newborn genetic screening program, FSU institute leads statewide effort

GLP-1 Market Industry Trends and Opportunities

Recruiting Roundup: July 10

Launch is Just the Beginning: Rare Disease Care Requires Durability

As Florida launches newborn genetic screening program, FSU institute leads statewide effort

Missing just 80 minutes of sleep a night may lead to weight gain, Columbia study finds

Are the feds trying to kick sick people off Medicaid?

As Florida launches newborn genetic screening program, FSU institute leads statewide effort

GLP-1 Market Industry Trends and Opportunities

Recruiting Roundup: July 10

The Best Foods to Eat for Constipation

Opinion: The FDA must put biotech at its center or continue to cede early research to China

Inside Elevance’s digital chronic disease management strategy

Best, Worst States For Well

What do the Middle Ages tell us about mental health then and now? VCU historian Leigh Ann Craig has answers

Fitness

As Florida launches newborn genetic screening program, FSU institute leads statewide effort

GLP-1 Market Industry Trends and Opportunities

Recruiting Roundup: July 10

Health

Opinion: The FDA must put biotech at its center or continue to cede early research to China

Inside Elevance’s digital chronic disease management strategy

Best, Worst States For Well

Subscribe to Updates

What's Hot

Launch is Just the Beginning: Rare Disease Care Requires Durability

Launch is Just the Beginning: Rare Disease Care Requires Durability

Key Takeaways

Related Content

Access by Design: Embedding Market Access Thinking Across the Drug Development Lifecycle

Improving Global Pharma Operations with Data

What Specialty Access Looks Like When It’s Built to Scale

An End-to-End Approach to Specialty Patient Services

What’s Next in Access & Affordability: A Biopharma Perspective

Related Posts