December 21st 2023
Men with severe or moderately severe hemophilia B continue to display low bleeding rates and very little use of replacement therapies for at least three years following a single dose of the approved gene therapy hemgenix.
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Diversity Gaps in U.S. Rare Disease Trials and the Inequalities in Hemophilia Clinical Studies
November 17th 2023Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, spoke to MHE about the current disparities in diversity and ethnicity in rare disease clinical trials in the U.S., as well as what he's seeing in clinical trials for diseases like hemophilia, in specific.
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Gene Therapy for Hemophilia As a ‘Paradigm Shift’
September 28th 2023Roctavian for hemophilia A and Hemgenix for hemophilia B could spare some patients from treatment that involves intravenous infusions of clotting factors several times a week. But a hemophilia expert cautions that the high-priced gene therapies aren’t a cure and that the reprieve won’t last a lifetime.
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Safeguarding Hemostasis and Adherence in Hemophilia A: Specialty Pharmacy Strategies
August 30th 2023Specialty pharmacies can help patients with hemophilia A maintain hemostasis and ensure patient safety through medication therapy management, utilizing regional care coordinators and direct pharmacist outreach, and by maintaining consistent provider communication.
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Gene Therapy Performance Guarantees Will “Improve Access”
August 17th 2023Joe 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.
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Counseling Patients With Hemophilia A: Medication Storage and Administration Techniques
August 16th 2023Counseling for patients with hemophilia A should include medication storage requirements, the importance of maintaining sterile injections, and administration considerations, such as how to use blunt fill needles.
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Balancing Aspects That Influence Hemophilia A Treatment Decisions
August 16th 2023Diverse factors can influence treatment decisions for patients with hemophilia A, such as the evolving landscape of insurance coverage, challenges related to vein access and medication storage, and mode of administration.
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Fitusiran May Open Doors for Treatment of Multiple Women’s Bleeding Disorders
July 20th 2023University 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.
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FDA approves Roctavian, the First Gene Therapy for Hemophilia A
June 30th 2023The price tag of $2.9 million is typical of a new wave of gene therapies. The manufacturer, BioMarin Pharmaceutical, is offering arrangements that will link the price paid by payers to how well patients respond to the treatment.
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Gene Therapies for Hemophilia: Promising But There’s Room for Improvement, Say Reviewers
May 22nd 2023How 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.
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Academic Institutions Hold Key to Gene Therapy Price Reduction, Say Academic Experts
May 22nd 2023Nonexclusive 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.
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Gene Therapy for Hemophilia Is Becoming a Reality. Who Will Write the Checks to Pay for It?
March 27th 2023Hemgenix 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.
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