Rare Disease Challenges At US FDA: CDER Director Says More Staff, Not Reorganization, Will Fix Issues

Rather than changing the agency's structure to create more focus on rare diseases, Patrizia Cavazzoni wants funding for people to do the necessary work.

company reorganization
Advocates continue pushing for a Rare Disease Center of Excellence at the FDA, despite agency opposition. • Source: Alamy

Hiring, not reorganization, is the answer to rare disease expertise issues at the US Food and Drug Administration’s Center for Drug Evaluation and Research, Director Patrizia Cavazzoni said.

Advocates and stakeholders have argued for years that the FDA could better handle rare disease applications and drug development with...

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

EU Pharma Reform: Council Wants Strengthened Member State Powers On Drug Supply

 

The Council of the EU’s vision for the pharma reform package includes a stronger role for member states regarding the supply of medicines and the prevention of indication-stacking for orphan drugs.

EMA Nod For First MASH Drug Rezdiffra, Cell Therapy Zemcelpro And 11 Others

 

Madrigal Pharmaceuticals’ Rezdiffra is on track to become the first medicine approved in the EU for non-cirrhotic metabolic dysfunction-associated steatohepatitis after the European Medicines Agency recommended that it be granted conditional marketing authorization.

Woodcock: Do Not Do The Wrong Study, Even If Against US FDA Advice

 

The former CDER director said she tells sponsors not to conduct an FDA-recommended study design or randomized trial if it will not work.

BIO Notebook: MFN Pricing, Obesity R&D, US FDA’s Rare Disease Hub And Reaction To Review Program

Highlights from Day Three of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for the FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.

More from Pink Sheet

Industry Criticizes Dutch Decision To Slash PARP Inhibitor Reimbursement

 

ZIN, the Dutch health technology assessment body, estimates that the number of patients eligible for treatment with a PARP inhibitor will be halved following a reassessment of drugs in the class.

EU Biosimilar Filings, Opinions And Approvals

 

A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.

US FDA’s ADHD Drug Relabeling Advances A MAHA Priority, Reviving Data Dispute

 

Extended-release stimulants will add a new ‘Limitation of Use’ about weight loss in children under 6 years, reflecting concerns about overuse from the Make America Healthy Again commission, a concern multiple studies did not find valid.