US FDA’s START Program Might See Expanded Funding If Pilot Meets Speed Goals

The program bringing Operation Warp Speed-like treatment to rare disease product development could expand if successful, but will require more employees and thus funding commitments from Congress and industry, CBER Director Peter Marks said.

net present value
Peter Marks thinks the net present value of the START pilot program will support expansion. • Source: Shutterstock

An expansion of the Support for clinical Trials Advancing Rare disease Therapeutics (START) program beyond the pilot stage will require dozens of new employees for the US Food and Drug Administration, but Peter Marks believes the additional funding needed would be well-invested.

The START program is intended to provide Operation Warp Speed-like treatment to rare disease therapies. Instead of using the formal...

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.