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

 
• By Brenda Sandburg

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)

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More from Rare Diseases

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

 
• By Sarah Karlin-Smith

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.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
• By Kate Rawson

Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.

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

 
• By Francesca Bruce

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.

US FDA Not Industry’s ‘Sock Puppet,’ Ultragenyx’s Emil Kakkis Says

 
• By Sue Sutter

The biotech CEO said the Trump Administration’s treatment of FDA employees, including the HHS secretary’s comments about industry capture, are insulting to agency staff, but he is encouraged by FDA Commissioner Martin Makary’s talk of a new conditional approval pathway.

More from Conferences

Trump’s Tariffs Will Lead To ‘Instability’ And ‘Less Investment’ In UK

 
• By Eliza Slawther

While the pharma industry appears to be exempt from US tariffs imposed by President Trump, a member of the UK House of Lords says the details are unknown and warned that uncertainty “leads to less investment” in business as a whole.

UK Health Secretary Admits VPAG ‘More Expensive’ Than Expected & Needs To Be ‘Resolved’

 
• By Eliza Slawther

The UK government has listened to industry concerns about high clawback rates under the voluntary scheme and will review it in June in a bid to resolve the issue and “move on to bigger and more important things,” health secretary Wes Streeting says.

England’s NICE Wants Industry Involvement In HTA Sandbox Projects

 
• By Eliza Slawther

Pharmaceutical companies are being encouraged to reach out to NICE in relation to its HTA Innovation Lab, which provides a sandbox environment in which the health technology assessment body can test new methods of evaluating “innovative and disruptive” therapies.