Tony Hagen

Two possibly curative gene therapies for the disease, which disproportionately affects Black people, are under consideration by the FDA.

The National Bleeding Disorders Foundation (NBDF) has joined a petition demanding that Payer Matrix stop representing itself as a patient advocacy organization.

Despite positive safety and quality of life results for FEIBA, the emergency of Hemlibra created difficulties for the FEIBA study and resulted in it being halted.

Joe Pugliese, president and CEO of the patient advocacy group Hemophilia Alliance, discusses recent announcements by manufacturers of two ultra-expensive gene therapies for hemophilia that they will offer payers partial or full reimbursement if patients fail on their therapies.

BioMarin Pharmaceutical and CSL Behring are offering valu\ed-based contracts for their hemophilia gene therapies.

The British cost-effectiveness agency said the evidence from a pivotal clinical trial wasn't strong enough to clarify Hemgenix's effectiveness relative to other therapies or satisfy concerns about its long-term durability.

University of Pittsburgh’s Margaret Ragni, M.D., M.P.H., discussed fitusiran, the investigational small interfering RNA (siRNA) therapeutic, with Managed Healthcare Executive and also some of the possible limitations of gene therapy for hemophilia.

A yearlong study of the newly approved half-life extender showed no evidence of factor VIII inhibitors.

Emissions from healthcare facilities contribute to climate change, and climate change, in turn, is presenting new challenges for healthcare.

Promising results were reported in The Lancet and The Lancet Haematology.

Many companies are pursuing interchangeable status.

Hemophilia A is seen as a male-only disease, but this study shows the extent to which female carriers also suffer from bleeding and joint problems related to reduced levels of clotting factors VIII.

The researchers concluded that delayed prophylaxis start with intermediate dose intensity of factor VIII prevents bleeds, but at a cost of more joint damaged and reduced health-related quality of life.

FDA has announced evidence-based changes that will allow more individuals to donate blood without increasing the risk of HIV infection.

Bowing to political and public pressure, insulin makers have slashed their prices. So far, insulin biosimilars haven’t had much of an effect on prices.

How long the gene therapies for hemophilia will keep factor levels high is unclear, said the authors of a review in the Annual Review of Medicine. Patients also need to be counseled that they can receive only one adeno-associated viral (AAV) vector in their lifetime.

Nonexclusive licensing of discoveries made at academic institutions would work to lower prices, say the writers of a recent opinion piece published in the New England Journal of Medicine. CMS price negotiation under the Inflation Reduction Act will not have any immediate effect on gene therapy prices and may never have.

CSL Behring's Hemgenix may have some competition. At least three gene therapies for hemophilia B are in development.

STAQ Pharma has stepped in to supply desmopressin nasal spray for people with hemophilia A or Type 1 von Willebrand disease. The company is also producing albuterol for children’s hospitals.

STAQ, a 503B outsourcing facility, started making desmopressin nasal spray after Ferring Pharmaceuticals suspended production nearly three years ago.

Hemgenix for hemophilia B is priced to cost $3.5 million per treatment. Roctavian for hemophilia A may be approved by the FDA in July and is likely to be priced to cost between $2 million and $3 million per treatment.

Patient groups say the insurer is cutting off access to important medications. The Tennessee Blues plan say many of the drugs that it excluded from coverage have zero utilization.

Pharmacy benefit managers say they exclude high-cost drugs and have alternatives on their formularies. Critics say exclusions narrow patient options and may put effective medication financially out of reach.

It took less than a year to develop the first COVID-19 vaccine, but an epidemic preparation group has said even more speed could be added and proposed setting 100 days as a goal for the development of future vaccines,

Some research has shown an association between flooding, which is becoming more common with climate change, and Clostridioides difficile infections.

As many as 10 Humira biosimilars may be on the U.S. market before 2023 is over. AbbVie has taken several steps to protect its revenues, including the launches of Skyrizi and Rinvoq.

Pradaxa (dabigatran) and Coumadin (warfarin) came out even in this randomized study. The Brazilian researchers who conducted the study said one explanation for the result is that use of Coumadin was exceptionally well managed because the patients were enrolled a study.

Korean study shows that depression, insomnia and anxiety add to the risk of atrial fibrillation among those with diabetes, according to a Korean study. Those mental health condition may affect the autonomic nervous system that regulates the heart. Bipolar disorder and schizophrenia did not increase the risk.

Animal, human and epidemiologic studies point to an association between alcohol consumption and atrial fibrillation. The association is especially strong with binge drinking.