Michelle Obama’s Video Challenge: Vote for Prader-Willi Syndrome and Rare Disease
Taking Rare Disease To the White House The Foundation for Prader-Willi Research (FPWR) and the Prader-Willi Syndrome Association (PWSAUSA) have entered a video into Michelle Obama’s video challenge to fight childhood obesity. You can view their video submission and help to spread the word about rare disease by casting your VOTE (everyday) at “Take One SMALL [...]
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 [...]
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 [...]
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 [...]
How A Bill Becomes a Law … It’s Complicated
We are often asked why we can’t quickly change the laws related to RARE disease therapy approval. The answer is that not only are politics always involved, it’s that the process is very complicated. This cartoon depicts what often happens in this process … what is needed seems very clear to us, but what is [...]
RARE Disease Drug Development … it’s complicated!
The development of any drug is a very complicated process … for RARE diseases the task is especially difficult due to the limited patient populations. Typically the disease is less well understood and there are fewer patients to access for testing to prove the drug works. We’re hoping this simplified overview of the process will [...]
RARE But Aware: What You Should Know About World Rare Disease Day
RARE Disease Day Blogs and Amylin Pharmaceuticals Since 2010, Amylin Pharmaceuticals, Inc.’s blog, Building Blocks, has continued to bring science, and dedication to changing lives through communication. In Building Blocks - Amylin’s Blog explores top discussions in healthcare, policy, patient commitment and latest scientific advancements in managing diabetes and obesity. Recently, our very own Nicole Boice, [...]
February 29 – A Celebration of Unity
The Month of February During the month of February, much of the American population will be celebrating historical events, special interest groups and campaigns. Some of these include Black History, American History, Children’s Dental Health, and American Heart Month. Internationally recognized events in February include National Embroidery month (my favorite), Wild Bird Feeding, Cherry and [...]