US FDA/NORD Partnership Will Further Increase Patient Involvement In Drug Development

Agency's agreement with rare disease advocacy group will facilitate patient meetings with review divisions, as well as enhance communication of safety information.

Two businessmen shaking hands during a meeting in the office, success, dealing, greeting & business partner concepts - soft light.

The US FDA is partnering with a prominent rare disease advocate to increase the patient voice in drug approval and development in what appears to be another evolution in the agency's patient-focused drug development efforts.

A new memorandum of understanding (MOU) with the National Organization for Rare Disorders will allow collaboration to plan workshops, training and listening sessions, as well as access to "patient...

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

US Most Favored Nations Policy: Higher Drug Spending In Germany Is ‘Difficult To Imagine’

 

Germany’s federal health care decision-making body, the G-BA, tells the Pink Sheet that German drug prices are fair and speculation of price rises in the market due to the US MFN policy is fear mongering.

Autolus’s Aucatzyl And GSK’s Blenrep Among 10 New Drugs To Get EMA Nod

 
• By 

The European Medicines Agency has OKd 10 new medicines for EU-wide approval, including a CAR-T therapy for treating acute lymphoblastic leukemia. Two drugs were, however, rejected.

Europe’s UPC Issues Landmark Ruling On Second Medical Use Claims

 

The verdict by the Unified Patent Court in the dispute between Sanofi/Regeneron Pharmaceuticals and Amgen explains what companies should look out for when deciding whether infringement has taken place when it comes to second medical use patents.