ResearchPUBMEDApr 5
Researchers looked at studies from 2014 to 2024 about the cost and value of special medicines for rare kidney diseases in poorer countries. They found that there isn't much research on whether these expensive medicines are worth the money in these areas. The study shows that doctors and governments need better information to decide if patients should have access to these treatments.
WHY IT MATTERSIf you have a rare kidney disease in a low- or middle-income country, this research highlights why your access to orphan drugs is limited — there's a major gap in evidence about whether these treatments are affordable and effective in your region.
AdvocacyPRESS RELEASEMar 26
The National Organization for Rare Disorders (NORD), a major patient advocacy group, announced new leadership positions focused on policy and government relations. These appointments aim to strengthen NORD's efforts to influence laws and policies that affect people with rare diseases. The moves show NORD is expanding its work to advocate for patients at the federal and global levels.
WHY IT MATTERSStronger policy leadership at NORD means patients with rare diseases will have more powerful advocates working directly with Congress and government agencies to push for faster drug approvals, better insurance coverage, and increased research funding.
AdvocacyRSSMar 26
The National Organization for Rare Disorders (NORD), a major patient advocacy group, announced new leadership positions to strengthen its work in rare disease policy. Michael J. Beard was appointed as Vice President of Federal and Global Public Affairs. These leadership changes are designed to help NORD better represent patients' interests in government and international discussions about rare diseases.
WHY IT MATTERSStronger NORD leadership in policy and advocacy can directly influence which rare diseases get research funding, how quickly new treatments are approved, and what insurance coverage looks like for rare disease patients.