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

 
• By Derrick Gingery

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...

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Capricor Gets Complete Response For DMD Cell Therapy: Unconvincing Data Or New Attitude At FDA?

 
• By Joseph Haas

Seven weeks before its action date and a month after an advisory committee was hastily cancelled, Capricor gets a CRL for its cell therapy deramiocel in DMD-related cardiomyopathy citing efficacy and manufacturing concerns. It has a Phase III study nearing readout, though.

Bayesian Statistics In EU Clinical Trials: EMA Discusses Balancing Efficiency With Rigor

 
• By Eliza Slawther

Bayesian statistics could help clinical trial sponsors to include external information in their analyses, but concerns around bias and incorrect conclusions remain, the European Medicines Agency said.

Industry Groups Back EU Life Sciences Strategy As Commission Pledges Clinical Trial Investment

 
• By Eliza Slawther

Industry associations EFPIA, EUCOPE and EURORDIS have praised the European Commission’s newly launched life sciences strategy, which will see investment funnelled into improving the clinical trial ecosystem and supporting advanced therapy development.

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

 
• By Eliza Slawther

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.

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‘Seize the Moment:’ European Drug Regulators Called To Act On Changes In US

 
• By Francesca Bruce

European regulators must work hard to maintain and build vital but fragile trust in science and processes, said speakers at an event celebrating the European Medicines 30th anniversary.

US FDA To Advisory Committee Industry Reps: Don’t Talk Too Much

 
• By Sue Sutter

Nonvoting industry representative’s presence at Pediatric Advisory Committee meeting was required by statute, the agency said, a de facto acknowledgement that an April directive precluding industry reps from sitting on advisory committees has had little practical effect.

New US FDA Biologics License Pathway Could Be Missing Link To Reduce Animal Testing

 
• By Bridget Silverman

Akin to the 505(b)(2) NDA, a new biologic pathway could reduce the need for animal testing, FDA toxicologist says. Expanded use of generally accepted scientific knowledge (GASK), which few sponsors have attempted despite a 2023 draft guidance, would also help.