Policy Update – PDUFA
We’re continuing to work on improving PDUFA to allow the FDA additional authority to include RARE disease in the Accelerated Approval Program. You can learn more here from last week’s public call for support and our other public policy blogs. After a very successful response from the patient community last week, today we are mobilizing organizations [...]
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 [...]
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 [...]
Managed Named or Compassionate Access to Drugs Still in Clinical Trial
I am participating in an interview tomorrow on early access to drugs that are in Clinical Trial under Managed/Named/Compassionate Access programs (it appears the names are different but the programs are similar). If you have experiences you wish to share about acces during Phase III Trials I would be interested in learning about them – [...]
Rare Disease Bloggers Unite With “Blog Hop” to Raise Awareness for World Rare Disease Day 2012 and 1 Million For Rare™ Campaign
PRESS RELEASE Jan. 30, 2012, 5:29 p.m. EST Rare Disease Bloggers Unite With “Blog Hop” to Raise Awareness for World Rare Disease Day 2012 and 1 Million For Rare† Campaign DANA POINT, Calif., Jan. 30, 2012 /PRNewswire via COMTEX/ — The R.A.R.E. Project(TM) ( www.rareproject.org ) and The Global Genes Project(TM) ( www.globalgenesproject.org ), leading [...]