Rare Disease

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.

An experimental drug for Alzheimer's disease sharply slowed the decline in mental function in a small clinical trial, researchers reported Friday, reviving hopes for an approach to therapy that until now has experienced repeated failures.

In it's report, "Clinical Cancer Advances 2015: ASCO's Annual Report on Progress Against Cancer," published in the Journal of Clinical Oncology, 4 newly approved treatments for CLL were predicted to have a dramatic impact on patients with the disease.

The FDA has provided Emergency Use Authorization, a pre-approval for use in bad outbreaks, for Roche's new Ebola test, the LightMix Ebola Zaire rRT-PCR Test.

Results from a large phase 3 prospective randomized open-label trial, comparing imatinib 400 mg with dasatinib 100 mg daily, were presented by Stephen O'Brien, MD, professor of hematology, Newcastle University Medical School, Newcastle upon Tyne, England.

A poster session on the second day of the 56th annual meeting of the American Society of Hematology, held December 6-9, San Francisco, was dedicated to trials evaluating therapeutic options in chronic myelogenous leukemia. Data presented included safety, efficacy, managing comorbidities, and biological differences that drive response to therapy.

On the second day at the Annual Meeting of the American Society of Hematology 2014, Francois-Xavier Mahon, MD, PhD, Bordeaux Hospital, INSERM 1035, Bordeaux, France, shared the results from the European LeukemiaNet Stop TKI (EURO-SKI) study; the trial used tyrosine kinase inhibitors (TKIs) in CML to define prognostic markers to increase the rate of patients in durable deep molecular response after stopping TKI.

Although there are no current cases of Ebola in the United States, an increased number of hospitals are now equipped to treat patients with the virus while minimizing risk to healthcare workers.

US officials trying to set up a network of hospitals in this country to care for Ebola patients are running into reluctance from facilities worried about steep costs, unwanted attention, and the possibility of scaring away other patients.

The leaders of the United Nations and the World Health Organization expressed renewed alarm on Friday about Ebola's tenacity in Africa and, in particular, its potential to ravage a fourth country, Mali, where they said hundreds of people had been exposed to an infected cleric who died last month.

Effective immediately, Karen B. DeSalvo, MD, MPH, will be stepping down as national coordinator for health information technology to assist HHS in the fight against Ebola.

While Ebola stokes public anxiety, more than 1 in six hospitals-including some top medical centers-are having trouble stamping out less exotic but sometimes deadly infections, federal records show.

The CDC has released guidelines with detailed information on the type of personal protective equipment and the procedure for donning and removing, for healthcare workers managing Ebola patients.

Brincidofovir is being developed by the company against several different viruses, including smallpox and Ebola.