R.A.R.E. Project's, Jonathan Jacoby to present at FDA Hearing on Rare Disease
On Tuesday, June 29, 2010, and Wednesday, June 30, 2010, FDA’s Office of Orphan Products Development will host a two-day public hearing and Webcast on the Development of Articles for Rare Diseases. This public hearing is intended to gain from health care providers, academia, industry, patients, and other interested persons their perspectives on various aspects [...]
FDA Hearing on Rare and Tropical (Neglected) Diseases
Just finished up watching the FDA hearing webcast from earlier this week. Loving Sen. Sam Brownback. If you have a rare disease, “God bless ya” because we “got no treatment for you.” The billion dollar per drug model doesn’t work for 7,000 rare diseases. We need a new different model – adjust the system so [...]
Prescription Drug User Fee Act (PDUFA)
The Prescription Drug User Fee Act (PDUFA) allows the FDA to collect fees from drug makers that produce specific human drug and biological products. The statutory authority for PDUFA expires in September 2012. At that time, new legislation will be required for FDA to continue collecting user fees for the prescription drug program (the renewal of this Act is called PDUFA V). The FDA will hold the first of many meetings on July 1 to gather input in advance of the next PDUFA, giving stakeholders an opportunity to weigh in on user fees the agency charges for drug review and how those fees are used by the agency.
Community Building Tools, a technology conference recap.
Before I forget everything I learned in early June at a technology conference, here goes. Use good content to build your audience (and encourage your members to share/make it easy for them to share) – you have to get out the “why” for your organization. Does your content touch people emotionally? Move people to action? [...]
Primary Care and HIT – What do they mean for you?
Howard Liebers of MarbleRoad asks questions about how efforts in both primary care and health information technology may impact families with rare disorders.
Rare Disease Caucus Announced
John Crowley announced the Rare Disease Caucus this week (link here). Leaders include Congressman Fred Upton and Congressman Joseph Crowley. This is exciting news for the rare disease community. Hoping great things come from this. // CC Recommend on Facebook Share on Linkedin share via Reddit Share with Stumblers Tweet about it Subscribe to the [...]
Wear Jeans, Help Sick Kids (Really)!
My son has a rare bone disease. I watch him suffer with bone pain – it really does get worse when it rains. He uses a wheelchair and walker. I would do absolutely anything for him to make it better – thing is though – the system is so broken there is not much that [...]
Make Your Voice Heard
Howard is collecting your stories, comments, recommendations, etc. for the FDA’s hearing on rare disease in June 2010. Howard will also be representing MarbleRoad at the NORD Partners in Progress 2010 Gala in Washington DC on May 18, 2010.
Quality of Life and the CLASS Act
Howard Liebers of MarbleRoad talks about the CLASS Act within Federal health reform legislation and other efforts to help families impacted by a rare disease diagnosis achieve a better quality of life.