Current Pathways For Rare Disease Drugs Are Not Optimal, US FDA’s Califf Says

Anticipating a ‘tsunami of therapies’ for rare diseases, commissioner says the agency will have to think of creative approaches and employ regulatory flexibility for them. FDA considers copying the oncology center’s Project Facilitate for expanded access to other diseases.

Creative pathway
Creative approach is needed for approval of rare disease treatments • Source: Shutterstock

The US Food and Drug Administration is aware of the difficulties of getting approval of rare disease treatments, Commissioner Robert Califf said, and the agency needs stakeholders help to develop creative approaches to overcome them.

Califf gave a closing keynote address at a recent conference sponsored by the New York University Grossman School of Medicine Working Group on Compassionate Use and Preapproval Access (CUPA). Appearing...

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 Conferences

EU Council Could Adopt Pharma Reform Package Next Week, But Disputes Risk A Year’s Delay

 

The Council of the EU wants to adopt its negotiating position on the reform of the general pharmaceutical legislation “sooner, rather than later,” but divergence between member states could push negotiations back another year, a European Commission policy officer said today.

EU HTA Regulation: ‘Stepping Beyond’ JCAs May Be Needed For Very Rare Disease Drugs

 

Cooperation between health technology assessment bodies across the EU will lead to a better joint clinical assessment process over time, but patients cannot afford a lengthy wait for improvements, speakers at a cell & gene therapy conference said.

DIA Korea: Medical Turmoil, Global Factors Blunt Korea’s Trial Edge

 
• By 

DIA Korea hears how multiple domestic and global factors are affecting the environment for clinical trials in South Korea, and what actions may be taken to address this.