Orphan Drug Act: Congressional, FDA, NORD Reviews Come Amid Pricing Debate

Republican Senators request US Government Accountability Office quantify law’s impact and assess whether regulatory or legislative changes are needed. FDA plans ‘unbiased’ look at outcomes from law’s incentives, while NORD is developing data on total percentage of drug spending represented by orphan indications.

fire embers

Congress and the US FDA are carrying out separate but seemingly parallel evaluations of whether the Orphan Drug Act is working to spur rare disease research and whether its incentives have been abused.

Three Republican senators have requested the Government Accountability Office (GAO) quantify the impact that the law – which provides tax credits, marketing exclusivity and other incentives – has had on...

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 Rare Diseases

ICH Targets RWE, Rare Diseases, Biosimilars, ATMPs In New Guideline Push

 
• By 

The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.

US FDA Rare Disease Case Studies Provide Development Models For Sponsors

 

Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices.

House Budget Bill Includes Delayed Orphan Fix, Also Risks Downstream Cell and Gene Coverage

 

The first two classes of negotiated drugs under the Inflation Reduction Act would not benefit from a rare disease adjustment House Republicans included in their reconciliation package.

Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

More from Pink Sheet

EMA Touts Faster Global Post-Approval Changes Via Regulatory Reliance

 

The European Medicines Agency says that a regulatory reliance pilot that it is supporting to speed up assessments of post-approval changes appears to be accelerating evaluation timelines in non-EU countries.

UK And US Regulators In Sync On RWD External Control Arms

 
• By 

The UK regulator’s draft guideline on the use of external control arms based on real-world data reflects concepts similar to those outlined by the US Food and Drug Administration.

New EU Approvals

 

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Vyjuvek, Krystal Biotech's advanced therapy for treating dystrophic epidermolysis bullosa.