Rare Disease

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.

The authors review published evidence regarding associations between high cost sharing for specialty pharmaceuticals indicated for rheumatoid arthritis, multiple sclerosis, and cancer, and their utilization.

Just 2 days after being rejected by cost regulators in the UK, ibrutinib has been approved for the frontline treatment of patients with chronic lymphocytic leukemia in the United States.

Imbruvica, developed by Janssen for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia who are unsuitable to receive chemo-immunotherapy, has been rejected by UK’s National Institute of Health and Care Excellence.

Research presented during a health outcomes session at the recently concluded annual meeting of the American Society of Hematology evaluated the influence of patients’ insurance status on clinical outcomes of CML.

During the last 5 years of life, the cost of dementia eclipses that of other diseases, like cancer and heart disease, according to a study from the National Institutes of Health.

NCCN Evidence Blocks add affordability to Guidelines recommendations on safety, efficacy, quality, and consistency of regimens.

The PD-L1 inhibitor was granted orphan drug status just last month.

Women bear greater costs related to Alzheimer's disease (AD) compared with men for 2 reasons: they are at greater risk of developing the AD and cost Medicare and Medicaid more, and they are more likely to provide informal, unpaid care to family members with AD.

Parents and advocates for a slower timetable pointed to existing issues within Medi-Cal as proof that the most vulnerable should not be rushed into managed care. But other states are moving ahead with plans.

The diagnosis and management of patients with dementing illnesses can be challenging, but the cost of misdiagnosing dementia as Alzheimer's disease can be as high as $14,000 a year, according to a study published in Alzheimer's & Dementia.

By 2050 more than 28 million additional baby boomers will develop Alzheimer's disease and the cost of caring for them will account for nearly one-fourth of Medicare spending by 2040, according to research presented at the Alzheimer's Association International Conference.

Till Kalydeco was approved, cystic fibrosis patients primarily received only disease management support to extend their lives.

However, not everyone supports the bill's provisions that promote the development of pediatric rare disease drugs through the "voucher" incentive.

German health authorities are debating whether the price of Europe's first approved gene therapy is worth the $1 million price tag.

A majority of recent drug development has been in therapies that qualify for at least one special approval pathway that Congress has passed over the past few decades for important drugs that treat life-threatening or rare diseases.

As patents on some of the most lucrative medicines began to expire, many companies shifted money to rare-disease drugs, knowing that those medicines cost less to develop and will face limited competition.