Approvals 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.
The 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.
The 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 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.
In 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
As the numbers of orphan drugs increase as well as their costs, patients are facing growing challenges accessing orphan drugs, according to a study from the Tufts Center for the Study of Drug Development (CSDD) at Tufts University.