ACTION ALERT – Accelerated Approval to treatments for rare disease patients!
The RARE Project has been working with Rare Disease Legislative Advocates (RDLA) on legislative improvements that would help bring more drugs to rare disease patients more quickly. This legislation promotes the use a variety of scientific data to qualify a surrogate endpoint for use in a clinical trial, when the required historical clinical information on [...]
CALL TO ACTION !!
Rare Disease Language included in House draft PDUFA Bill RARE Project would like you to consider taking action in support of the new language (FAST Act) related to the Prescription Drug User Fee Act (PDUFA) in the following ways; The more support there is for the FAST Act, the better positioned the rare [...]
The Grassroots Movement Behind Ultra and Fast: Join the Movement!
A Guest Blog by Julia Jenkins: Director of Government Relations, EveryLife Foundation The Grassroots Movement Behind ULTRA-FAST & How you can join the Movement! We are very excited to share the news that rare disease Accelerated Approval language made it into the House draft PDUFA bill. The language that incorporates the goals of [...]
It’s a Wrap – World RARE Disease Day 2012
GLOBAL GENES PROJECT E-NEWS DANA POINT, Calif. – The 5th Annual World Rare Disease Day took place February 29, 2012 with a fanfare of activities worldwide! Sponsoring organizations like NORD and Eurordis engaged in more activities and awareness efforts, garnering increased attention and engagement than previous years. Once again, Global Genes Project™ helped support and [...]
Change is Needed at the FDA
“Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them.” - Andrew Von Eschenbach Mr. Von Eschenbach couldn’t have stated it any clearer. On February 14, the Wall Street Journal posted an on-line article by Mr. Eschenbach titled: Medical Innovation: How the U.S. Can Retain Its Lead, The FDA should approve drugs based on [...]
First-of-it’s-kind iPad Communication App
Gwendolyn Strong Foundation (theGSF) launches first ever iPad Communication App The demand for health information on the web continues to increase. With the utilization of digital products in healthcare, trends toward technological solutions in improving our health is gaining momentum. For those in the Rare Disease Community dealing with physical limitations precluding them from engaging [...]
FAST Act – Speeding RARE Disease Therapies
Last week over 80 RARE Disease advocates lobbied on Capitol Hill to enable better access to Accelerated Approval of therapies. We discussed H.R. 3737 “Unlocking Lifesaving Treatments for Rare-Diseases Act (ULTRA)” during our day on the hill. Today, an additional piece of bipartisan legislation, the FAST Act (HR 4132), was introduced in the House. Incorporating [...]
NIH Supports Global Genes Project and Jeans/Genes Awareness Efforts
Over the past two years, the Global Genes Project efforts have been highly visible at the NIH for the annual World Rare Disease Day events. In 2012, we presented Dr. Francis Collins and Dr. Steve Groft with a Rare Pictures of Hope™ photo book made by thousands of rare disease advocates. In 2011, Nicole Boice, [...]
Christian Billingsley: “Waiting for Medical Science to Catch Up”
On February 7, 2012, WWL-TV, the CBS affiliate in New Orleans, reported on Christian Billingsley, a 17-year old boy diagnosed with atypical hemolytic uremic syndrome (aHUS) at 3 months of age. Online Story Just Keep Him Alive Until Medical Science Catches Up (click on title/link above to see WWL-TV video) Doug Mouton / Northshore [...]
The RARE Blog Has A New Home
We want to welcome our Children’s Rare Disease Network followers to the new home of the RARE blog here at the RARE Project. Those of you who have been friends and followers for a long time know that we have been re-branding the RARE Project and re-focusing our Global Genes and Children’s Rare Disease Network [...]