In the past and, perhaps to some extent, today, the specialized care and medications required for the appropriate management of patients with rare diseases, such as hemophilia and SLE, was neither readily accessible nor affordable. A collaborative effort for advocacy by patients and physicians has gradually pushed the evolution of legislation and regulations to improve access to optimal management. Two such legislation are the Orphan Drug Act of 1983 and the Rare Diseases Act of 2002, which provided the incentives and means necessary to drive the pharmaceutical research and development to grow a flourishing drug market and pipeline for rare diseases. As a result, managed care policies continue to evolve in many ways, implementing mechanisms and policies to balance treatment access and costs with appropriate medication use and patient outcomes. Patient and physician advocacy for rare diseases prompted a wave of change, but the momentum will need to be maintained.
Perioperative Nivolumab Boosts NSCLC Survival: CheckMate 77T Trial
May 16th 2024This interim analysis of the CheckMate 77T trial, outcomes were compared between adult patients receiving neoadjuvant nivolumab plus chemotherapy or neoadjuvant chemotherapy plus placebo for resectable non–small cell lung cancer (NSCLC).
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Frameworks for Advancing Health Equity: Urban Health Outreach
May 9th 2024In the series debut episode of "Frameworks for Advancing Health Equity," Mary Sligh, CRNP, and Chelsea Chappars, of Allegheny Health Network, explain how the Urban Health Outreach program aims to improve health equity for individuals experiencing homelessness.
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Covering antiobesity medications like semaglutide could save Medicare around $500 million annually; preliminary CDC data showed a 3% decline in the number of US overdose deaths last year; the Biden administration recently announced the first national maternal mental health strategy.
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Decitabine, Venetoclax Combo Boosts Survival in Patients With AML and MDS
May 16th 2024Study findings demonstrate that combining decitabine with venetoclax enhances molecular response rates in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), correlating strongly with improved clinical outcomes and overall survival.
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