Rare Disease

The orphan drug market continues its steady and inexorable growth even with increased scrutiny on the high prices of these lifesaving treatments.

The company did not recover from the 5 patient deaths associated with JCAR015 in 2016.

The National Organization for Rare Disorders has announced a new, yearlong campaign to raise public awareness of rare diseases. “Do Your Share for Rare” shines a light on people living with a rare disease and provides an opportunity for them to share their story.

The FDA has approved lenalidomide as maintenance therapy in patients with multiple myeloma following autologous stem cell transplant.

Population-level judgements on the value of therapies to treat multiple sclerosis don’t adequately depict the differences between how providers and payers view value compared with patients and how patients view value different from one another.

A new treatment option using stem cell transplants was shown to induce sustained remission of relapsing-remitting multiple sclerosis (MS). More than two-thirds of participants had no signs of progression of disability, relapse of MS symptoms, or new brain lesions after 5 years.

A new study has identified key mutations that can direct the conditioning regimen prior to an allogenic hematopoietic stem cell transplant (SCT) in patients with myelodysplastic syndrome (MDS), leading to a better response.

Children’s National Health System has announced the formation of the Children’s National Rare Disease Institute to advance the care of children and adults with rare genetic diseases.

A treatment for juvenile Batten disease has been tested in a mouse model and the results have shown reduced in symptoms and improved longevity. The results of the study were published in Nature Communications.

Reports explain that plans for Medicaid block grants or per capita caps overlook who really uses Medicaid. It's mostly children.

A review of 15 disease-modifying therapies (DMTs) for the treatment of relapsing-remitting and primary-progressive multiple sclerosis (MS) has found that prices for most of these drugs are not well-aligned with added value for patients.

Patients who have had at least 1 prior therapy or carry a 17p deletion or TP53 mutation are now eligible to be treated with ibrutinib (Imbruvica) in the United Kingdom.

A roundup of top healthcare news stories: January 27, 2017.

“Off-the-shelf” chimeric antigen receptor (CAR)-T cells, also known as universal donor cells, were used in 2 young infants with relapsed, refractory acute lymphoblastic leukemia resulted in molecular remission in 28 days in both infants.

Researchers from the Peter O’Donnell Jr. Brain Institute and Harold C. Simmons Comprehensive Cancer Center at the University of Texas Southwestern may have found the ultimate drivers of glioblastoma cell proliferation.

At the 58th annual meeting of the 58th American Society of Hematology, representatives from 2 national clinical trials, Beat AML and NCI-MATCH, detailed how they were incorporating genomic profiling to assign patients to different treatment arms.

A poster presented at the 58th Annual Meeting & Exposition of the American Society of Hematology compared the utilization patterns and associated costs for recently approved and older drugs in patients with relapsed/refractory multiple myeloma in the United States.

Patients with rare diseases often turn to the Internet for information on their illness, but an analysis of some of these websites found that their content often failed to meet important quality criteria and neglected key information categories.

The improved expansion of chimeric antigen receptor (CAR) T-cells indicates a deeper clinical response.

Despite the complexities associated with treating older patients diagnosed with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), patients in the RESONATE-2 trial continue to present a favorable response to single-agent ibrutinib at a follow-up of 29 months.

Among the 9 new orphan drugs approved by the FDA in 2016 were 3 treatments for rare diseases that, so far, had no approved treatments: Duchenne muscular dystrophy, spinal muscular atrophy, and severe hepatic veno-occlusive disease.

This study measured the value of survival gains attributable to the introduction of 3 novel therapies for myelodysplastic syndromes.

At the 58th American Society of Hematology Annual Meeting & Exposition, Jan Joseph Melenhorst, PhD, presented results of a study evaluating biomarkers of response to anti-CD19 CAR T-cell treatment in patients diagnosed with chronic lymphocytic leukemia (CLL).