Do you know anyone who has gone above and beyond to become a rare disease policy leader or advocate in a state or in our nation? Rare Disease Legislative Advocates (RDLA) is seeking nominations for the RareVoice Awards to be given on November 16th in Washington, DC.
On April 6th, the Senate Health, Education, Labor and Pensions (HELP) Committee held the last of three markups to discuss biomedical innovation legislation. The legislation considered and approved in those mark-ups will be conferenced with the 21st Century Cures Act which passed the House of Representatives with broad bipartisan support last summer. In addition to approving the five bills under consideration, members of the Committee offered several amendments of particular interest to the rare disease community.
House Energy and Commerce Committee Chairman Fred Upton (R-MI), Ranking Member Frank Pallone, Jr. (D-NJ), and Rep. Diana DeGette (D-CO) issued the following statement following a meeting at the White House with Vice President Joe Biden on H.R. 6, the 21st Century Cures Act. Senate HELP Committee Chairman Lamar Alexander (R-TN) and Ranking Member Patty Murray (D-WA) also attended this afternoon’s meeting.
The EveryLife Foundation is working to expand the lifesaving potential of newborn screening in California. Last month, SB 1095 was introduced by pediatrician and California State Senator Dr. Richard Pan. This bill would help eliminate the legislative delay in newborn screening and ultimately ensure that patients are treated as early as possible. Early screening plays a […]
Dear Advocate, As you may know, the House passed the Orphan Products Extension Now, Accelerating Cures & Treatments or OPEN ACT in July as part of a broader legislative package called the 21st Century Cures Act. The OPEN ACT has the potential to double the number of treatments available to rare disease patients. Since passing […]
The Senate Health Committee will be turning its attention to a medical innovation bill now that it has completed the overhaul of No Child Left Behind, Chairman Lamar Alexander (R-Tenn.) said. The idea behind both bills is to speed up the Food and Drug Administration’s approval process for new drugs and boost funding for research at the National Institutes of Health (NIH). The 21st Century Cures bill passed the House on a bipartisan vote in July, but since then the momentum has slowed.
Take action to support the OPEN ACT – Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 971/S 1421). The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to “repurpose” therapies for the treatment of life-threatening rare diseases and […]
Coalition Support Letter for the Advancing Targeted Therapies for Rare Diseases Act of 2015
PFIA Senate Sign On Letter, June 2015 Read more about the Patient-Focused Impact Assessment below: 1597: The Patient-Focused Impact Assessment Act Overview The Food and Drug Administration Safety and Innovation Act (FDASIA) and its related user fee agreement contained a number of provisions intended to strengthen the meaningful engagement of the patient and other […]
Taylor’s Tale, a Charlotte-based advocacy group in the fight against Batten disease and other rare diseases, spearheaded new legislation in North Carolina to create a new advisory council housed within the UNC-Chapel Hill School of Medicine to work towards finding more treatments for rare diseases. North Carolina House Bill 823 will establish the council that will […]