1. In Vivo
  2. >>Market Access

Re-Thinking Rare Disease

 
• By Melanie Senior

Germany is drastically squeezing orphan drug pricing while new US laws protect rare diseases drugs from price negotiations. These contrasting moves fuel the longstanding debate over whether orphans still deserve special incentives.  

Swan
Orphan drugs are classified as those destined for fewer than five in 10,000 people in Europe • Source: Shutterstock

In the last days of July 2022, Germany’s cabinet passed a swathe of drug pricing measures designed to curb pharmaceutical spending and help plug a €17bn healthcare funding gap in 2023. (Also see "Germany: Leaked Pricing Measures Could Have ‘Drastic’ Consequences For Orphans" - Pink Sheet, 7 July, 2022.) Weeks later, US Congress passed a law allowing Medicare to negotiate the prices of some drugs, potentially saving $100 billion over a decade. (Also see "Medicare Price ‘Negotiation’ Process Gets Broad Brush Treatment In New Law" - Scrip, 16 August, 2022.) But while Germany’s new laws include a drastic squeeze on how orphan drugs are assessed and priced, the US law protects the category – and may even provoke higher launch prices.

Payers everywhere are concerned over the growing collective cost of rare diseases drugs. They account for over half of FDA...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on In Vivo for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Market Access

Wide Of The Mark: ‘The Worst EU Medtech Predictions Have Not Come True’

 
• By Ashley Yeo

Jana Grieb, European regulatory and market access legal expert at McDermott Will & Emery, explains why the healthtech and pharma industries are warming to the new EU health commissioner as he faces calls to make the MDR more “user friendly.”

Crisis Or Opportunity? US MFN Policy Could Test Japan’s Appetite For Reforms

 
• By Lisa Takagi

While the adoption of most favored nation drug pricing in the US stands to affect Japanese biopharma firms now heavily reliant on this market, it might also present an opportunity for pricing and policy reforms at home.

AI In Health Delivery: Patients Most Confident When HCPs Are In Charge

 
• By Ashley Yeo

Annual survey of patients and professionals shows how attitudes to health system transformation are evolving and what stakeholders are demanding as acceptance of AI tools accelerates.

US Health System Redesign Critical, NAM’s Medical Experts Warn Trump Government

 
• By Ashley Yeo

The whirlwind back-and-forth on US tariffs and Robert F. Kennedy jr.’s plans to deregulate health care have become all-preoccupying, but the National Academy of Medicine was first to set out President Trump’s health administration priorities.

More from In Vivo

Wide Of The Mark: ‘The Worst EU Medtech Predictions Have Not Come True’

 
• By Ashley Yeo

Jana Grieb, European regulatory and market access legal expert at McDermott Will & Emery, explains why the healthtech and pharma industries are warming to the new EU health commissioner as he faces calls to make the MDR more “user friendly.”

This Belgian Biotech’s Drug Cocktail Could Help Reverse Muscle Aging

 
• By Jo Shorthouse

While big pharma pours billions into creating new anti-aging molecules, a Belgian startup has taken a different path: combining existing safe drugs with AI precision. The early results suggest it might be onto something revolutionary.

Rising Leaders 2025: Doxie Jordan, From UNC Graduate To Global Market Strategist

 
• By Jo Shorthouse

Bristol Myers Squibb executive Doxie Jordan discusses his path to global commercial leadership and the principles guiding pharmaceutical market strategy