FDA Orphan Approval Flexibility Remains Steady As Pressure Increases

Frank Sasinowski updates his 2012 landmark analysis and finds that FDA maintained its rate of flexibility in the evidentiary standards for approval of orphan products.

It appears FDA has not wavered from, or increased, its rate of using flexibility toward orphan drugs four years after a landmark analysis confirmed the long-held agency belief.

Frank Sasinowski, a director at Hyman, Phelps and McNamara, and colleagues showed that from July 2010 to June 2014, about...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet 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

More from United States

Repurposed Generics For Rare Diseases: Janet Woodcock Proposes Shortcut

 
• By 

A Duke-Margolis working group is developing ideas to enable non-profit firms to seek US FDA approval for new indications for off-patent medicines. The former acting commissioner thinks citizen petitions might be the fastest route.

NDA User Fees Will Grow 8.6% Next Year As FDA Projects More Applications But Fewer Sponsor Meetings

 

The ups – and some downs – of the US FDA’s prescription, biosimilar, and generic user fees for FY 2026 are tabulated by the Pink Sheet.

Vinay Prasad Out At US FDA

 

The removal of CBER Director Prasad comes after a wave of right-wing backlash to his appointment over the past few days that was also critical of his decision to ask Sarepta to suspend all sales of the DMD gene therapy Elevidys. FDA reversed its decision the day before Parsad's departure.

Merck, AstraZeneca Downplay Impact of European Tariffs Despite Timing Uncertainties

 
• By 

Meanwhile, companies continue discussions with the Trump Administration over ways of increasing what European nations pay for drugs as an alternative to imposing a Most Favored Nation drug pricing policy in the US.

More from North America

EU 15% Tariff Deal Still Leaves Questions For Pharma On Possible Grace Period, Generic Exemption

 

While the 15% rate is a substantial blow for pharmaceutical firms, the US-EU deal provides a framework for trade agreements with other countries that could see the industry avoiding higher tariffs elsewhere.

Medicare Part B ASP Proposal Sets ‘Guardrails’ Around Bona Fide Service Fee Reporting

 
• By 

Average sales prices, and Medicare spending on some drugs, would be expected to be lower, CMS says in the Physician Fee Schedule proposed rule.

A Record Year For US FDA Approvals? Commissioner Says It’s Possible

 

FDA Commissioner Martin Makary suggested 60 new molecular entities could be approved in 2025, which appears possible, but can the strained staff deliver?