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Rare Disease Supporters Suggest Permanent PRV Program, Even If It May Concern FDA

 
• By Derrick Gingery

Non-profit biotech founder says permanent pediatric rare disease priority review voucher program will boost incentive, but FDA is concerned about resource drain vouchers cause.

Capitol House

Rare disease advocates want to add more certainty to the pediatric rare disease priority review voucher program, although that may put them at odds with the US FDA.

The pediatric rare disease priority review voucher program has generated hundreds of millions for sponsors, but the program will sunset...

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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.

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

 
• By Eliza Slawther

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

 
• By Sarah Karlin-Smith

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

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Clinical Trial Diversity Action Plan Guidance Must Return To US FDA Website, Court Says

 
• By Sue Sutter

HHS and its agencies violated the law by swiftly implementing “sweeping and poorly thought-through directives that ordered the bulk removal of healthcare resources,” including FDA draft guidances on diversity action plans and sex differences in clinical trials, a federal court said.

US Budget Bill Eases IRA Price Controls But Coverage Losses Will Impact Pharma

 
• By Cathy Kelly

The legislation expected to be signed by President Trump is a mixed bag for pharmaceutical manufacturers depending on their product mix and manufacturing situation.

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.