However, not everyone supports the bill's provisions that promote the development of pediatric rare disease drugs through the "voucher" incentive.
The 21st Century Cures bill, which was unanimously approved by a House panel just a few weeks back, brings promise of faster drug development, and also offers hope to the rare disease advocacy groups. The bill includes provision to extend the "voucher" program for companies developing treatments for pediatric rare diseases.
Approximately 15 million children are diagnosed with rare diseases, which can result in nearly 35% of deaths in the first year of life.
Pharmaceutical companies are often cautious about venturing into the world of rare diseases for several reasons: fear of potential negative outcomes, limited returns, increased stringency with clinical trials in children, etc. Therefore, to motivate the pharmaceutical industry to invest efforts in developing theses drugs, the Congress initiated a voucher program in 2011 which allows the company to have another one of their drugs approved quicker than usual. The company can also sell this voucher if they so desire.
The Cures bill is seeking an extension on this voucher program, which is scheduled to expire next year.
However, not everyone is happy with this program. Read more at Kaiser Health News: http://bit.ly/1KXbSvK
Males With Hemophilia A Report Physical, Mental Burdens of Disease Regardless of Severity
April 25th 2024Self-reported data from adult and pediatric males with hemophilia A show that burden of the disease persists regardless of severity, highlighting a need for improved prophylactic treatment.
Read More
What We’re Reading: FDA Approves UTI Antibiotic; Ozempic, Wegovy Price Investigation; US Births Fall
April 25th 2024The FDA recently approved an antibiotic for the treatment of urinary tract infections (UTIs) in women; a Senate committee recently launched an investigation into the prices of Novo Nordisk’s diabetes and weight loss drugs; US births fell last year, resuming a national slide after a previous increase during the pandemic.
Read More