Rare Disease

Positive data from two Phase 3 clinical trials that show Dupixent (dupilumab) is effective in the treatment of moderate-to-severe atopic dermatitis was announced at the 25th European Academy of Dermatology and Venereology Congress in Vienna, Austria.

What we're reading, September 19, 2016: the government update rules on reporting clinical trial findings; hospitals say they aren't receiving discounts for Valeant's heart drugs; and the FDA approved the first treatment for Duchenne muscular dystrophy.

What we're reading, September 16, 2016: Donald Trump mentions using Medicaid to expand coverage and making birth control available without a prescription; medical marijuana could curb the opioid epidemic; and how rare diseases are becoming more common.

The use of orphan drugs for non-orphan diseases is driving up the cost of these already expensive medications. A new report from America’s Health Insurance Plans found that almost half of the utilization for 46 orphan drugs were used for non-orphan indications.

Health insurance status can have a big impact on a person’s health outcomes after they are diagnosed with cancer, according to 2 new studies published in CANCER.

AstraZeneca is trying to invoke the Orphan Drug Act to protect its blockbuster, on the grounds it is approved to treat a rare pediatric condition.

The lawsuit seeks an additional 7 years' protection for the popular cholesterol drug, which sold 20.3 million prescriptions last year.

Treatment for Duchenne muscular dystrophy remains in flux with the FDA delaying a decision on Sarepta's treatment and Biomarin discontinuing development on its own drug.

The earliest studies involving diabetes therapy for Alzheimer’s are recruiting patients.

Researchers in Germany have discovered that mitoxantrone-used to treat aggressive, relapsing, or progressive multiple sclerosis-can increase the risk of leukemia and colorectal cancer in those being treated.

The approval comes following the review of a phase 2 single-arm trial in 106 patients with chronic lymphocytic leukemia who carried the 17p deletion mutation and who had received at least 1 prior therapy for their condition.

This case study from the MS Center of Saint Louis examines how patients are impacted by high healthcare costs and addresses potential solutions to this problem.