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Aplastic Anemia and MDS International Foundation and NORD Launch New Natural History Study of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Aplastic Anemia and MDS International Foundation and NORD Launch New Natural History Study of Paroxysmal Nocturnal Hemoglobinuria (PNH) The worldwide registry will advance understanding of and spur innovation in treating PNH. News provided by Share this article Share this article BETHESDA, Md. and WASHINGTON, May 6, 2021 /PRNewswire/ The Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD®) today launched the largest-ever study to research Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare bone marrow failure disease. PNH is characterized by the destruction of red blood cells, blood clots and impaired bone marrow function. While there are treatments available to improve the patients quality of life, PNH currently has no cure.

CDISC and NORD Partner to Develop Data Standards for Rare Diseases

CDISC and the National Organization forRare Disorders (NORD ®) have announced a partnership to develop global data standards for rare diseases. The data standards will be released in a Therapeutic Area User Guide that will be available at no cost on the CDISC website for researchers to leverage in studies to maximize data’s full potential. According to the FDA, any disease, disorder, illness or condition affecting fewer than 200,000 people in the United States is considered rare. There are approximately 7,000 rare diseases and it’s estimated that 25-30 million Americans have rare diseases. More than 90% of rare diseases have no FDA-approved treatment. Globally, it is estimated that 350 million individuals are impacted by a rare disease.

New Report Finds Medical Treatments for Rare Diseases Account for Only 11% of US Drug Spending; Nearly 80% of Orphan Products Treat Rare Diseases Exclusively

New Report Finds Medical Treatments for Rare Diseases Account for Only 11% of US Drug Spending; Nearly 80% of Orphan Products Treat Rare Diseases Exclusively Majority of pharmaceuticals sales are driven by treatments for common conditions, not rare diseases News provided by Share this article Share this article WASHINGTON, March 4, 2021 /PRNewswire/ As US policymakers consider options to address rising health care costs while still meeting the needs of patients, a new report from IQVIA has found that rare diseases account for just 11% of medical invoice spending in the United States, and 79% of all orphan products treat only rare diseases. The report also highlights recent progress in drug development that has resulted in more people with rare diseases having access to critical treatments.

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