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Orphan Drug Development May Be Well-Suited To Digital/Wearables

 
• By Cole Werble

Pfizer feels that orphan products may present a particularly good area for testing wearable monitors and other digital devices as research tools. The small patient populations and fine distinctions in functional improvement or delay of disease progression may be ideally suited for the digital collection of data.

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Pfizer Inc. is investigating wearables to improve, expand and accelerate the capture of data in the small population of rare diseases. The company demonstrated its interest in digital monitoring in a recent presentation at a patient advocacy meeting in Friedreich’s Ataxia in Hyattsville, MD.

The meeting, hosted by the Friedreich’s Ataxia Research Alliance (FARA), was one of the new wave of “patient-focused drug development” meetings held outside FDA, with participation by the agency. FDA is encouraging the meetings as a way to expand the effort and format created by the agency under the PDUFA V-chartered meetings organized by the agency

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US FDA Rare Disease Case Studies Provide Development Models For Sponsors

 
• By Laura Helbling

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

 
• By Sarah Karlin-Smith

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

 
• 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.

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HHS Negotiating With Manufacturers On Most Favored Nation Drug Pricing

 
• By Cathy Kelly

MFN policy would would apply to all single source drugs in all insurance markets, according to HHS release. Secretary Robert F. Kennedy Jr. said the department is discussing the plan with drug sponsors.

Spain Slashes Wait Between EU Drug Authorization And Financing Decisions

 
• By Francesca Bruce

Spanish authorities say that that timelines for drug reimbursement decisions can be improved further with the right submissions from companies.

Spain Beats England To Tevimbra Reimbursement Recommendations

 
• By Francesca Bruce

Spanish authorities have published more pricing and reimbursement reports as part of a new drive to increase transparency.