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
Paul Melmeyer: Value of Rare Disease Therapies to Caregivers is Often Left Out of Value Assessments
June 24th 2019Paul Melmeyer, director of Federal Policy, National Organization for Rare Disorders, discusses factors that have historically been underrepresented in value assessment frameworks for rare disease therapies.
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Adding Hydroxyurea to Ruxolitinib Yields Higher Clinical Response in Myelofibrosis
June 20th 2019Adding hydroxyurea to the selective inhibitor of Janus kinase 1 and 2 could elicit a high clinical response and increased ruxolitinib exposure for patients with hyperproliferative forms of myelofibrosis.
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Attending to Psychosocial Concerns for Those With Epidermolysis Bullosa
June 14th 2019A recent literature review, involving an international multidisciplinary panel of social and healthcare professionals and people living with epidermolysis bullosa (EB), was conducted to create evidence-based guidelines to optimize psychosocial well-being in EB, given that there is no cure or treatment.
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Starting 2020, employees will be able to use health reimbursement arrangements (HRAs) to purchase individual coverage; New York has enacted legislation that ends nonmedical exemptions for school vaccination requirements; the FDA is being sued by Catalyst Pharmaceuticals over its approval of a similar orphan drug.
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Paul Melmeyer on Willingness to Pay for High Cost of Rare Disease Therapies Among Stakeholders
June 10th 2019There is generally a greater willingness to pay among just about everybody for rare disease therapies, explained Paul Melmeyer, director of Federal Policy, National Organization for Rare Disorders.
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Frequency, Severity of Sickle Cell Crises Linked to Productivity Losses at Work
June 8th 2019A recent study found that patients with sickle cell who had more frequent or severe vaso-occlusive crises (VOCs) had greater absenteeism, overall productivity loss, and activity impairment than patients with less frequent or severe VOCs.
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Paul Melmeyer on How Therapies for Rare Diseases Present Challenges to Value Assessment
May 29th 2019Therapies for rare diseases, such as gene therapy, pose very unique challenges to value assessments in a variety of different ways, explained Paul Melmeyer, director of Federal Policy, National Organization for Rare Disorders.
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