NORD State Report Card Grades States on Policy Issues Critical to Rare Disease Patients
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WASHINGTON, Jan. 27, 2021 /PRNewswire/ Today, the National Organization for Rare Disorders (NORD®) and its Rare Action Network (RAN™) published the 6
th Edition of the State Report Card, the annual report rating each state and Washington, DC on the most important issues directly affecting more than 25 million Americans living with rare diseases. Despite a year marked by devastating impacts from the COVID-19 pandemic, the State Report Card demonstrates that progress was made in many states on newborn screening, step therapy, Rare Disease Advisory Councils and other key policies. The report also notes the need for states to take additional steps to address out-of-pocket prescription drug costs for rare disease patients and to protect patient access to affordable, comprehensive health care services.
CALGARY Alberta Health Minister Tyler Shandro announced funding Wednesday for the gene therapy drug Zolgensma, which is used to treat toddlers with spinal muscular atrophy (SMA). The drug was approved by the federal government in December. Before that, patients could only access it through a federal special access program at a cost of roughly $2.8-million. Funding from the province will be done on a case-by-case basis, said Shandro. There is no budget cap, what we re looking for is focusing on kids not falling through the cracks as we re waiting for the rest of the processes that need to be done for drugs to become approved and listed in the formulary here in Alberta, he said.
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WASHINGTON, Jan. 26, 2021 /PRNewswire/ In an effort to provide clear, useful, authoritative information to rare disease patients and caregivers on the COVID-19 vaccines, on January 15 the National Organization for Rare Disorders (NORD®), along with The ALS Association, Cystic Fibrosis Foundation and Muscular Dystrophy Association, hosted a special webinar with leaders from the US Food and Drug Administration (FDA) and the Centers for Disease Control and Prevention (CDC). In December 2020 FDA granted Emergency Use Authorization (EUA) for the Pfizer-BioNTech and Moderna vaccines against COVID-19, which are now being distributed.
The webinar provided several key takeaways and included discussion of the expedited clinical trials that resulted in FDA s issuing EUAs for the Pfizer-BioNTech and Moderna vaccines, the vaccines high rates of effectiveness in combating COVID-19, as well as the imminent arrival of more vaccines, including a one do
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As my colleague Tom wrote about in a recent post, the Centers for Medicare & Medicaid Services (CMS) has finally responded to a growing chorus of stakeholders that government price reporting requirements, particularly Medicaid Best Price (BP), are stifling innovative value-based contracting arrangements (VBAs). As the proverbial wisdom goes, “nothing changes if nothing changes,” and CMS’ recently finalized proposal to allow manufacturers to report multiple BPs is meant to stimulate innovative changes in contracting for drugs and biologicals.
In this post, we want to take a closer look at the proposal and consider some potential implications in a bit more detail, especially as they relate to state Medicaid programs’ ability to leverage VBAs.
A parasite found in undercooked meat and contaminated water may be linked to rare brain cancers Canela López Toxoplasma gondii, a parasite commonly found in undercooked or raw meat, could be linked with certain brain cancers. According to the study, 20% to 50% of the global population has been exposed to T. gondii, often spreading through contaminated water and food. Researchers say the study suggests people who are exposed to T. gondii are more likely to develop glioma.
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Researchers studied blood samples from 111 people in a US database and 646 in Norway. The results showed those with larger amounts of T. gondii antibodies appeared to have a higher risk of developing glioma, an aggressive form of brain cancer that makes up 80% of malignant tumours.