New England Journal of Medicine.
Terlipressin is an investigational product and its safety and effectiveness have not yet been established by the U.S. Food and Drug Administration (FDA) or Health Canada.
As previously announced, the Phase 3 CONFIRM study met its primary endpoint of Verified HRS Reversal, which is defined as renal function improvement, avoidance of dialysis and short-term survival. The main objective of the CONFIRM study was to assess the efficacy and safety of terlipressin, together with albumin, versus placebo in adults in the U.S. and Canada with cirrhosis and HRS-1. The trial met three of the four pre-specified secondary endpoints of the study including HRS reversal,
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
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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.
Kyowa Kirin Responds to the National Institute for Health and Care Excellence (NICE) Decision to Not Provide People Living With Certain Rare Blood Cancers Access to POTELIGEO (mogamulizumab)
two very rare forms of non-Hodgkin lymphoma will not have access to
an innovative systemic treatment following NICE s decision to not recommend mogamulizumab for routine use in the NHS
. Kyowa Kirin is disappointed by this decision but remains committed to finding a resolution with NICE.
The National Institute for Health and Care Excellence (NICE) today published its final appraisal document (FAD) for POTELIGEO
(mogamulizumab) announcing that POTELIGEO will not be made available on the NHS
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The hope for many companies developing drugs for rare diseases is this: The significant unmet need, given a dearth of treatment options, will drive demand and thus product uptake. Unfortunately, the reality is more challenging.
While we have seen a significant increase in the number of drugs for rare diseases, accurate diagnosis rates of rare diseases continue to trail behind those of more prevalent diseases.
The average time to diagnosis for a patient with a rare disease is 5 to 7 years. The delay is driven by:
Lack of awareness among health care professionals (HCPs) of symptoms and markers
Fulcrum Therapeutics Recognizes Rare Disease Day 2021 Theme of global unity highlights critical role of building communities of support for patients and families affected by rare diseases around the world
February 26, 2021 07:00 ET | Source: Fulcrum Therapeutics Fulcrum Therapeutics
CAMBRIDGE, Mass., Feb. 26, 2021 (GLOBE NEWSWIRE)
Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the company’s recognition of Rare Disease Day 2021.
“While COVID-19 has had an impact on most people around the world, it has put a new spotlight on the importance of connectivity and community for people affected by rare diseases who are at a higher risk of feelings of isolation and can face significant challenges in accessing information and healthcare services,” said Robert J. Gould, Ph.D., Fulcrum’s presiden