January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
Phase 2 Trial Produces Promising Results for Pemigatinib in Cholangiocarcinoma
October 8th 2019If approved, pemigatinib would be the first targeted therapy for patients with cholangiocarcinoma, a rare cancer that impacts the bile ducts. The disease is often diagnosed at a late or advanced stage.
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Dr David Snyder Outlines Use of Ruxolitinib After Approval of Fedratinib
October 8th 2019After the approval of fedratinib, familiarity may keep people using ruxolitinib to treat myelofibrosis or polycythemia vera, but it’s good to know there is a back-up if needed for patients who don’t respond adequately, said David Snyder, MD, associate chair of the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope.
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Variation in US Private Health Plans’ Coverage of Orphan Drugs
Health plans restrict orphan drug coverage less often than nonorphan drug coverage. However, the frequency of restrictions varies considerably across plans.
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Dr Naveen Pemmaraju Outlines the Symptoms of Myelofibrosis
October 5th 2019Myelofibrosis has a very heterogeneous disease presentation, which means patients with it can present to the clinic with a number of different symptoms, explained Naveen Pemmaraju, MD, associate professor in the Department of Leukemia at MD Anderson Cancer Center.
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Dr Scott Gottlieb: Reimbursement Models Play a Vital Role in the Development of Orphan Indications
September 27th 2019The development and access to orphan indications rely on reimbursement models that require regulatory action toward out-of-pocket costs for patients, said Scott Gottlieb, MD, former FDA commissioner (2017-2019).
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40-Year-Old RA Drug May Be Low-Cost Option for Patients With Myeloproliferative Neoplasms
September 18th 2019Patients with polycythemia vera and essential thrombocythemia may be able to reduce their symptoms through a low-cost drug used to treat rheumatoid arthritis (RA), according to a new study in British Journal of Haematology.
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Dr Ahmar Zaidi: How Social Determinants Could Limit Access to Gene Therapy for Sickle Cell
September 17th 2019The only thing that should matter in these patients is their genetic code, but often it’s your zip code that really restrains your ability to access the healthcare you deserve, explained Ahmar Zaidi, MD, pediatric hematologist-oncologist, Comprehensive Sickle Cell Center, Children's Hospital of Michigan, when discussing the possibility of a gene therapy for sickle cell.
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Orphan Drugs Are Driving Skyrocketing Drug Costs, AHIP Finds
September 12th 2019As orphan drugs account for an increasing share of drugs approved, they are driving up the cost of drug launches and drug prices. In a new paper, America's Health Insurance Plans analyzes these rising costs and the use of orphan drugs and asserts that policy makers need to revisit the Orphan Drug Act.
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Dr Neil Minkoff on Hopes, Payer Reactions If a Gene Therapy Is Approved for Sickle Cell Disease
September 6th 2019Neil Minkoff, MD, chief medical officer and vice president, EmpiraMed, discusses his hopes if the first gene therapy for sickle cell disease is approved and if he has any concerns with payer reactions.
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Dr Ahmar Zaidi on Efforts Needed to Address Complications of Sickle Cell Disease
August 29th 2019Ahmar Zaidi, MD, pediatric hematologist-oncologist, Comprehensive Sickle Cell Center, Children's Hospital of Michigan, discusses the need for more efforts from both the medical community and regulators to address the psychosocial complications of sickle cell disease.
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In a Surprise Decision, FDA Rejects Sarepta's Second Duchenne Drug
August 21st 2019In a highly anticipated decision, the FDA on Monday rejected accelerated approval for Sarepta Therapeutics’ second Duchenne drug, handing the drug maker a surprising blow that has some questioning the motivation behind the decision.
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FDA Approves Entrectinib for NTRK Fusion-Positive Solid Tumors and Rare Lung Cancer
August 16th 2019FDA's approval of entrectinib for neurotrophic tyrosine receptor kinase (NTRK) fusion-positive tumors represents a new paradigm in cancer treatment; It treats a cancer based on a common biomarker seen across different tumors rather than based on the location in the body where the cancer originated. The FDA also gave approval to entrectinib for the treatment of adults with metastatic non–small cell lung cancer whose tumors are ROS1 positive.
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NCCN Publishes Guidelines for Small Bowel Adenocarcinoma
August 2nd 2019The National Comprehensive Cancer Network (NCCN) has published a set of recommendations for the treatment of small bowel adenocarcinoma, representing the first treatment guidelines in the United States for the rare type of cancer.
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What We're Reading: Medicare Executive Order; Breast Implant Recall; Antibiotic Misuse
July 25th 2019President Trump is preparing an executive order that would slash prices on nearly all drugs sold to Medicare; Allergan has recalled certain breast implants following 573 cases of implant-associated anaplastic large cell lymphoma; a study has found 1 in 4 people intend to use antibiotics without a prescription.
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Senate Finance Committee Unveils Bipartisan Bill to Lower Drug Costs
July 24th 2019Spearheaded by Senate Finance Committee Chairman Chuck Grassley, R-Iowa, and Ranking Member Ron Wyden, D-Oregon, the bipartisan bill would lower out-of-pocket (OOP) costs for Medicare and Medicaid beneficiaries and save the government billions.
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Tezacaftor, Ivacaftor Combination Generally Safe, Well Tolerated in Children With CF Under Age 12
July 12th 2019Data from a phase 3 trial are supporting the use of tezacaftor in combination with ivacaftor for the treatment of cystic fibrosis (CF) in children aged 6 to 11 years, finding that the treatment combination was generally safe and well tolerated.
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Soliris Gains FDA Approval for Neuromyelitis Optica Spectrum Disorder
June 28th 2019The FDA approved a fourth indication for Alexion Pharmaceuticals’ brand-name eculizumab (Soliris) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
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