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 [...]
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 [...]
Anti-ACA Litigation, Extenders Act and Rare Disease Day 2011
A legislative update on anti-ACA litigation, the Medicare and Medicaid Extenders Act of 2010, and Rare Disease Day 2011. Oh, and HAPPY HOLIDAYS!
Do We Need a War on Rare Diseases?
By Amy Dockser Marcus, Wall Street Journal Is it time to declare a national “war on rare diseases” comparable to the famous “war on cancer” that began in the 1970s? The Institute of Medicine report released today stops short of recommending a full martial assault, but it does call for the creation of an aggressive [...]
Turning Science into Medicine – a Political Perspective
Our Government has an immense capability to help advance treatments and cures for rare diseases through incentives for industry, research grants, and improving regulatory conditions; however, our politicians often lack the public support to challenge the status quo and make good changes to the system. Our democratic system was created to be responsive to the [...]
Rare finds and reminders this week from a parent advocate.
Clearing my inbox, several worthwhile finds this week, including: (1) From Genetic Engineering and Biotechnology News, an article by Taralyn Tan reminding everyone of the importance and value in funding the little guys of research, the little guys of rare disease. See blog post here. (2) Just when I was about to scratch “gene therapy” off [...]
Clinical Trials for Rare Disease Patients – Legislation needs your support!
We wanted you to be aware of pending Federal legislation that may be significant for you and for our rare disease community. In June, legislation that enables individuals with rare diseases to participate in clinical trials without losing eligibility for public healthcare benefits was sponsored by Senators Ron Wyden (D-OR), Christopher Dodd (D-CT), Richard Durbin [...]
Save the Date! Conferences of interest to parent advocates!
Here are some upcoming conferences that parent advocates and advocacy groups might be interested in: Gov 2.0 Expo in Washington, DC on September 8, 2009. The FEAST Conference in New York City, NY on October 1, 2009. American Academy of Pediatrics in Washington, DC on October 17 – 20, 2009. Bone and Joint Decade Global [...]
Nominate Your Rock Star of Science!
Geoffrey Beene Gives Back®’s hip new campaign, “Rock Stars of Science” (a.k.a Rock S.O.S) is making its mark, and The Children’s Rare Disease Network is proud to be affiliated with this important initiative! Geoffrey Beene Gives Back® teamed up with GQ Magazine to create a philanthropic campaign pairing A-list rock stars and celebrities with the [...]