orphan drugs

FDA Fast-Tracks Orphan Drugs
FDA Fast-Tracks Orphan DrugsThe agency is trying to deliver on its goal of a 90-day response time.
What’s in the Specialty Pharmacy Pipeline?
What’s in the Specialty Pharmacy Pipeline?Biosimilars, cancer drugs, and orphan drugs are heading towards FDA approval in 2017.
How Did You Participate in Rare Disease Day 2017?Rare Disease Day is held every year on the last day of February to help spread awareness about rare diseases and the people they impact.
Watch list 2017: Top therapeutic areas
Watch list 2017: Top therapeutic areasFind out about the latest treatment and drug pipeline developments
Five specialty pharmaceutical trends to watchApprovals of specialty pharmaceuticals have far outpaced traditional drugs, and that trend will continue. But that’s not the only specialty medication trend that healthcare executives should have on their radar.
Three drug trends impacting specialty pharmacyAMCP session, “Specialty Pharmaceutical in Development,” highlighted critical trends that healthcare executives should be watching
FDA approves drug for rare lung diseaseThe FDA recently granted orphan drug approval to Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation.
FDA approves orphan drug for bleeding disorderThe FDA this week approved Coagadex, Coagulation Factor X (Human) for hereditary Factor X (10) deficiency. Until the new orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.
Judge rules: Orphan drugs not subject to 340B discountsJudge Rudolph Contreras ruled against the Department of Health and Human Services by saying that pharma companies do not have to offer rural and cancer hospitals that serve low-income populations 340B discounts on orphan drugs.
Patient access to orphan drugs faces new set of challengesIn 1983, the United States enacted the Orphan Drug Act (ODA). An analogous law was passed in Europe in 2000. Both pieces of legislation are considered major successes in terms of spurring the development of orphan drugs. To illustrate, in the decade prior to 1983 only 34 orphan products were marketed, whereas in the past year alone 9 orphan drugs were launched. In the past 5 years, 39 orphan drugs were launched in the US across numerous therapeutic categories, including multiple myeloma, chronic myeloid leukemia, metastatic non-small cell lung cancer, hemophilia, tuberculosis, homozygous familial hypercholesterolemia, and cystic fibrosis.1