Journal of managed care & specialty pharmacy
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J Manag Care Spec Pharm · Dec 2018
Comparative StudyDiscrepancies Between FDA-Required Labeling and Evidence that Payers Cite in Drug Coverage Policies.
FDA-required labeling summarizes certain data that the FDA relies on in its drug approval process. However, when determining coverage of specialty drugs, health care payers may consider dissimilar evidence. ⋯ This study was funded by an unrestricted grant from the Pharmaceutical Research and Manufacturers of America. The authors work with The Center for the Evaluation of Value and Risk in Health, which is partially supported through the CEA Registry Sponsorship program; the CEA Registry has received funding from the National Science Foundation, National Library of Medicine, Agency for Healthcare Research and Quality, Centers for Disease Control and Prevention, and a variety of pharmaceutical and device companies that subscribe to the data. Chambers reports personal fees from Health Advances, Ernst & Young, Magellan Health, Summit Therapeutics, and Sanofi-Aventis, unrelated to this study. Neumann reports past advisory board work with Amgen, Avexis, Axovant, Bayer, Bluebird, Congressional Budget Office, Janssen, Merck, Novo Nordisk, Pacira, Paratek, and Sage; consulting work for Boston Health Economics, GSK, Precision Health Economics, Veritech, and Vertex; speaker fees from AbbVie, Celgene, and Roche; and grants from the Alzheimer's Association, Amgen, Gates, Lundbeck, NIH, NPC, and Sage, all unrelated to this study. The other authors have nothing to disclose.
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J Manag Care Spec Pharm · Dec 2018
ReviewGene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease.
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease that, in most cases, involves homozygous deletion of the SMN1 gene. This causes a deficiency in survival motor neuron (SMN) protein, which plays a critical role in motor neuron development. SMA has a range of phenotype expression resulting in variable age of symptom onset, maximum motor strength achieved, and survival. Without intervention, infants with a more severe form of the disease (type 1 SMA) die before 2 years of age. Although it is rare, SMA is the most common fatal inherited disease of infancy, and until recently, treatment was primarily supportive. In 2016, a new agent, nusinersen, was approved by the FDA. Other treatments are in development, including a gene therapy, AVXS-101. These treatments are not only improving the lives of patients with SMA and their families, they are changing the disease phenotype. They have the greatest benefit when given early in the disease course. ⋯ Advances in clinical care have significantly extended the lives of individuals with SMA, and research into the genetic mechanisms leading to disease have revealed strategies for intervention that target the underlying cause of SMA. Nusinersen is now on the market, and other treatment options, such as AVXS-101, may soon be approved. This article provides an overview of SMA and the genetic mechanisms leading to SMN deficiency, then describes how new and emerging treatments work to overcome this deficiency and prevent associated nerve damage and disability. In addition, we discuss steps for incorporating AVXS-101 into hospital/health system formularies, along with barriers and concerns that may delay access, based in part on lessons learned with nusinersen.
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J Manag Care Spec Pharm · Dec 2018
Randomized Controlled Trial Observational StudyClinical Utility of Pharmacogenetic Testing and a Clinical Decision Support Tool to Enhance the Identification of Drug Therapy Problems Through Medication Therapy Management in Polypharmacy Patients.
In polypharmacy patients, medication therapy management (MTM) services provide a comprehensive review of current medications and future treatment goals. Pharmacogenetics (PGx) may further optimize the identification of potential drug therapy problems (DTPs); however, the clinical utility of PGx information with a clinical decision support tool (CDST) in an MTM setting in identifying DTPs has not been systematically assessed. ⋯ No funding was received for conducting the post hoc analysis presented in this study. Magness is employed by Magellan Health, which received funding from Genelex for costs to administrate the medication management program. The open-label randomized trial was sponsored by Genelex (Clinicaltrials.gov ID number NCT02428660). PGx tests were provided and laboratory analysis was performed by Genelex. Valerie Baron is an employee of YouScript, which created the clinical decision support tool used in this study and formerly was part of Genelex. The other authors have nothing to disclose.
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J Manag Care Spec Pharm · Dec 2018
Coverage of Novel Therapeutic Agents by Medicare Prescription Drug Plans Following FDA Approval.
Regulatory approval of novel therapies by the FDA does not guarantee insurance coverage requisite for most clinical use. In the United States, the largest health insurance payer is the Centers for Medicare & Medicaid Services (CMS), which provides Part D prescription drug benefits to over 43 million Americans. While the FDA and CMS have implemented policies to improve the availability of novel therapies to patients, the time required to secure Medicare prescription drug benefit coverage-and accompanying restrictions-has not been previously described. ⋯ Funding for this study was contributed by a student research grant awarded to Shaw and provided by the Yale School of Medicine Office of Student Research under National Institutes of Health training grant award T35DK104689. Ross reports research grants to Yale University from the U.S. Food and Drug Administration (U01FD005938, U01FD004585), Medtronic, Johnson & Johnson, Centers for Medicare & Medicaid Services (HHSM-500-2013-13018I), Blue Cross-Blue Shield Association, Laura and John Arnold Foundation, Agency for Healthcare Research and Quality (R01HS022882), and National Institutes of Health (R01HS025164), unrelated to this study. Dhruva has nothing to disclose. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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J Manag Care Spec Pharm · Dec 2018
Cost-Effectiveness of Niraparib and Olaparib as Maintenance Therapy for Patients with Platinum-Sensitive Recurrent Ovarian Cancer.
The recent approval of olaparib and niraparib as maintenance therapy can significantly affect the management of ovarian cancer. Clinical benefits, however, come with trade-offs in adverse events and costs. ⋯ This study was unfunded. The authors have nothing to disclose.