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 vital role in ensuring that rare diseases are detected and can be better treated. Read more
As an advocate for patients with rare diseases you are a very important part of the legislative process. You can make the difference as you are the voices your legislators and congressmen want, or in some cases do not want to hear. Please complete the form below to take action and contact your Member of Congress
After you take action your job is not done! The final step is to share the action alert with your family, friends, co-workers and any other people that might be interested in taking action on behalf of the rare disease community.
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 House, the bill has stalled in the Senate. Your Senator, the Honorable Bob Casey (D-PA) is a key decision-maker on this vital legislation. Please take a moment to call his office to ask that they support the bipartisan OPEN ACT, co-sponsored by Sens. Hatch (R-UT) and Klobuchar (D-MN). You can find suggested talking points below: Read more
On behalf of the Lupus and Allied Diseases Association (LADA), in conjunction with the International Foundation for Autoimmune Arthritis (IFAA), we are sharing the position paper, “The Ethics of Step Therapy and Autoimmune Disease: Next Steps.” This important investigation into step protocol was led by IFAA, in consultation with bioethicists and LADA, and was recently presented at a Congressional Briefing where an expert group of panelists joined the launch. Read more
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 pediatric cancers. EveryLife Foundation, NORD, Global Genes, Genetic Alliance and an additional 155 patient organizations support this bipartisan legislation.
In July, the House passed the 21st Century Cures Act (HR 6), historic legislation that holds the potential to dramatically advance rare disease research and treatments. The Senate’s companion bill to 21st Century Cures, titled “Innovation for Healthier Americans“, has stalled, and we need your help to ensure the bill is acted upon TODAY. Patients can’t wait, as a delay in legislation is a delay in treatment.
Invite your U.S. Senators to attend a briefing on the Advancing Research for Neurological Diseases Act (S. 849) on September 16 from 11:30 to 12:30 in the Senate Russell Building, room 485. The briefing is sponsored by the American Academy of Neurology, American Brain Coalition, Brain Injury Association of America, Epilepsy Foundation, International Essential Tremor Foundation, National Multiple Sclerosis Society, Parkinson’s Action Network, Rare Disease Legislative Advocates, Research!America, Tourette Association of America, and United Spinal Association.
For more information and to contact your Representative, click here.
The House passed historic bipartisan legislation called the 21st Century Cures Act HR 6, and they deserve our thanks for ensuring that the voice of the rare disease community was heard. Let’s show every member who supported 21st Century Cures how much their hard work means to us, and how eager we are to see this meaningful legislation make its way through the Senate. Please take a moment to thank your representative for supporting the 21st Century Cures Act and the rare disease community.
Sequestration poses significant risk to the funding NIH and FDA receives – agencies which are critical to advancing our understanding of and treatments for rare diseases. Help end sequestration and the threat of cutting billions of dollars from our most crucial facilities by getting your organization to sign-on to the letter linked below: Read more
From The California Healthcare Institute (CHI): We are working to protect critical funding for the drug review divisions at FDA, which are under threat of budget cuts that could greatly hamper the drug approval process. Organizations are being asked to endorse legislation, FDA SOS Act HR 1078, to protect these vital funds.
After hundreds of Hill meetings, emails and calls to Congress, we are seeing the true impact of Rare Disease Patient Advocacy on the 21st Century Cures Legislation. The latest draft released this week includes many priorities for the rare disease community – including 3 out of the 4 pieces of legislation advocates lobbied for during Rare Disease Week 2015:
- HR 971, Orphan Product Extensions Now Accelerating Cures and Treatments Act(OPEN ACT)
- HR 292, Advancing Research for Neurological Diseases Act
- HR 909, Compassionate use Reform & Enhancement (CURE) Act
Other important provisions benefiting the rare disease community include, making the Pediatric Priority Review voucher program permanent, the NIH Innovation Fund, language on Precision Medicine for Orphan diseases, and patient focused drug development.