Dataset Information

PCSK9 inhibitor access barriers-issues and recommendations: Improving the access process for patients, clinicians and payers.

ABSTRACT: The proprotein convertase subtilisin/kexin type 9 inhibitors or monoclonal antibodies likely represent the greatest advance in lipid management in 30 years. In 2015 the US Food and Drug Administration approved both alirocumab and evolocumab for high-risk patients with familial hypercholesterolemia (FH) and clinical atherosclerotic cardiovascular disease requiring additional lowering of low-density lipoprotein cholesterol. Though many lipid specialists, cardiovascular disease prevention experts, endocrinologists, and others prescribed the drugs on label, they found their directives denied 80% to 90% of the time. The high frequency of denials prompted the American Society for Preventive Cardiology (ASPC), to gather multiple stakeholder organizations including the American College of Cardiology, National Lipid Association, American Association of Clinical Endocrinologists (AACE), and FH Foundation for 2 town hall meetings to identify access issues and implement viable solutions. This article reviews findings recognized and solutions suggested by experts during these discussions. The article is a product of the ASPC, along with each author writing as an individual and endorsed by the AACE.

SUBMITTER: Baum SJ

PROVIDER: S-EPMC5412679 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

PCSK9 inhibitor access barriers-issues and recommendations: Improving the access process for patients, clinicians and payers.

Baum Seth J SJ Baum Seth J SJ Toth Peter P PP Underberg James A JA Jellinger Paul P Ross Joyce J Wilemon Katherine K

Clinical cardiology 20170322 4

The proprotein convertase subtilisin/kexin type 9 inhibitors or monoclonal antibodies likely represent the greatest advance in lipid management in 30 years. In 2015 the US Food and Drug Administration approved both alirocumab and evolocumab for high-risk patients with familial hypercholesterolemia (FH) and clinical atherosclerotic cardiovascular disease requiring additional lowering of low-density lipoprotein cholesterol. Though many lipid specialists, cardiovascular disease prevention experts, ...[more]

PMID: 28328015

Similar Datasets

Project description:ImportanceAlthough PCSK9 inhibitors (PCSK9i) were approved in 2015, their high cost has led to strict prior authorization practices and high copays, and use of PSCK9i in clinical practice has been low.ObjectiveTo evaluate patient access to PCSK9i among those prescribed therapy.Design, setting, and participantsUsing pharmacy transaction data, we evaluated 45 029 patients who were newly prescribed PCSK9i in the United States between August 1, 2015, and July 31, 2016.Main outcomes and measuresThe proportion of PCSK9i prescriptions approved and abandoned (approved but unfilled); multivariable analyses examined factors associated with approval/abandonment including payor, prescriber specialty, pharmacy benefit manager, out-of-pocket cost (copay), clinical diagnoses, lipid-lowering medication use, and low-density lipoprotein cholesterol levels.ResultsOf patients given an incident PCSK9i prescription, 51.2% were women, 56.6% were 65 years or older, and 52.5% had governmental insurance. Of the patients given a prescription, 20.8% received approval on the first day, and 47.2% ever received approval. Of those approved, 65.3% filled the prescription, resulting in 30.9% of those prescribed PCSK9i ever receiving therapy. After adjustment, patients who were older, male, and had atherosclerotic cardiovascular disease were more likely to be approved, but approval rates did not vary by patient low-density lipoprotein cholesterol level nor statin use. Other factors associated with drug approval included having government vs commercial insurance (odds ratio [OR], 3.3; 95% CI, 2.8-3.8), and those filled at a specialty vs retail pharmacy (OR, 1.96; 95% CI, 1.66-2.33). Approval rates varied nearly 3-fold among the top 10 largest pharmacy benefit managers. Prescription abandonment by patients was most associated with copay costs (C statistic, 0.86); with abandonment rates ranging from 7.5% for those with $0 copay to more than 75% for copays greater than $350.Conclusions and relevanceIn the first year of availability, only half of patients prescribed a PCSK9i received approval, and one-third of approved prescriptions were never filled owing to copay.

Project description:BackgroundCommunication serves several functions in pediatric oncology, but communication deficiencies persist. Little is known about the broad spectrum of barriers contributing to these deficiencies. Identifying these barriers will support new strategies to improve communication.MethodsThe authors performed 10 focus groups on perceived communication barriers with nurses, nurse practitioners, physicians, and psychosocial professionals across 2 academic institutions. They analyzed transcripts by adapting a multilevel framework from organizational psychology.ResultsThe authors identified 6 levels of barriers to communication from the clinicians' perspectives: individual, team, organization, collaborating hospital, community, and policy. Individual barriers were subdivided into clinician characteristics, family characteristics, or characteristics of the clinician-family interaction. Within each level and sublevel, several manifestations of barriers were identified. Some barriers, such as a lack of comfort with difficult topics (individual), cultural differences (individual), a lack of a shared team mental model (team), and time pressure (organization), manifested similarly across professions and institutions. Other barriers, such as a need for boundaries (individual), intimidation or embarrassment of family (individual), unclear roles and authority (team), and excessive logistical requirements (policy), manifested differently across professions. With the exception of collaborating hospitals, participants from all professions identified barriers from each level. Physicians did not discuss collaborating hospital barriers.ConclusionsNurses, nurse practitioners, physicians, and psychosocial professionals experience communication barriers at multiple levels, which range from individual- to policy-level barriers. Yet their unique clinical roles and duties can lead to different manifestations of some barriers. This multilevel framework might help clinicians and researchers to identify targets for interventions to improve communication experiences for families in pediatric oncology.Lay summaryClinicians and families experience many barriers to communication in pediatric oncology. The authors performed 10 focus groups with 59 clinicians who cared for children with cancer. In these focus groups, barriers to effective communication were discussed. In this article, the authors report on an analysis of the responses. Six levels of barriers to communication were found: individual, team, organization, collaborating hospital, community, and policy. With an understanding of these barriers, interventions can be developed to target these barriers in hopes of improving communication for parents and patients in pediatric oncology.

Project description:BACKGROUND:Prompt Mental Health Care (PMHC) is the Norwegian adaptation of Improving Access to Psychological Therapies (IAPT). Thus far, evaluations of PMHC have mostly focused on the effectiveness, rather than on contextual and implementation processes. Therefore, the objective of this study was to do a process evaluation and examine: 1) To what extent do the services follow guidelines provided by the Norwegian Directorate of Health (NDH), 2) what the therapists experienced as important barriers and facilitators in implementing the service, and 3) client treatment satisfaction and its associations with baseline variables. METHOD:The present study uses data from 526 clients who received PMHC treatment in the municipalities of Sandnes and Kristiansand. The therapists completed questionnaires about each client's course of treatment. We conducted semi-structured interviews with the therapists and analysed them using thematic analysis. Data from client questionnaires were used to report descriptive sample statistics including symptom severity and treatment satisfaction. Linear regression was adopted to examine the associations between client treatment satisfaction and baseline characteristics. RESULTS:Several aspects of PMHC were implemented in line with the guidelines provided by NDH. Importantly, both services reached out to the intended target group, and could further be characterized as low-threshold with relatively short waiting times (median waiting time between initial contact and treatment start was 27?days, IQR 18-39), no waiting lists, and frequent use of self-referral (33.3%). From the client perspective, results indicated a high degree of treatment satisfaction (Mean?=?3.93 (SD?=?.71, range 1-5)), and this was true across demographic characteristics and symptom severity at baseline (all p >?.05). Most notable challenges that came forward were; the low provision of guided self-help (received by only 1.0% of clients), the lack of focus on work participation (low to some degree of focus in 70.8% among sick-listed clients), the collaboration with other services (no collaboration in 85.3% of the clients), and some aspects regarding future development of the service. CONCLUSION:Both sites managed to implement key aspects of PMHC in line with the guidelines, but further development of the program is warranted. Discussion of challenges and future recommendations are presented.

Project description:ImportancePreliminary cost-effectiveness analyses of proprotein convertase subtilisin/kexin type 9 inhibitor (PCSK9i) were based on benefits estimated from reductions in low-density lipoprotein cholesterol that occurred in PCSK9i trials with variable results. The recent Further Cardiovascular Outcomes Research with PCSK9 Inhibition in Subjects with Elevated Risk (FOURIER) trial provides better information about the effectiveness of the drug.ObjectiveTo use the trial results to determine the cost-effectiveness of a PCSK9i and statin treatment strategy compared with a statin alone strategy.Design, setting, and participantsWe derived observed rates of events, outcomes, cost of care, and health insurance from existing literature for a theoretical cohort of patients designed to resemble the FOURIER PCSK9i trial population and created a Markov model during the time horizon of a full lifetime.Main outcomes and measuresWe evaluated the incremental cost-effectiveness ratio from a health system perspective, and the return on investment from a private payer perspective. For both measures, we assumed an annual PCSK9i drug price of $14 300, with a lapse in US patent protection that would reduce the price by 43% in year 12. Costs were reported in 2016 US dollars.ResultsThis study modeled 1000 hypothetical patients with attributes similar to those of the FOURIER trial cohort. At the current price, the incremental cost-effectiveness ratio of statin plus PCSK9i therapy was $337 729 per quality-adjusted life-year. Our probabilistic sensitivity analysis found that a statin plus PCSK9i strategy had a low probability (<1%) of being cost effective at the commonly accepted societal threshold of $100 000 per quality-adjusted life-year. Furthermore, PCSK9i produced a negative return on investment of 86% for private payers. In our threshold analysis, the price of PCSK9i would need to drop 62%, to $5459 per year, to reach $100 000 per quality-adjusted life year.Conclusions and relevanceAt current prices, the addition of PCSK9i to statin therapy is estimated to provide an additional quality-adjusted life year for $337 729 . Significant discounts are necessary to meet conventional cost-effectiveness standards.

Project description:BackgroundInclisiran (ALN-PCSsc) is a long-acting RNA interference (RNAi) therapeutic agent that inhibits the synthesis of proprotein convertase subtilisin-kexin type 9 (PCSK9), a target for the lowering of low-density lipoprotein (LDL) cholesterol.MethodsIn this phase 1 trial, we randomly assigned healthy volunteers with an LDL cholesterol level of at least 100 mg per deciliter in a 3:1 ratio to receive a subcutaneous injection of inclisiran or placebo in either a single-ascending-dose phase (at a dose of 25, 100, 300, 500, or 800 mg) or a multiple-dose phase (125 mg weekly for four doses, 250 mg every other week for two doses, or 300 or 500 mg monthly for two doses, with or without concurrent statin therapy); each dose cohort included four to eight participants. Safety, the side-effect profile, and pharmacodynamic measures (PCSK9 level, LDL cholesterol level, and exploratory lipid variables) were evaluated.ResultsThe most common adverse events were cough, musculoskeletal pain, nasopharyngitis, headache, back pain, and diarrhea. All the adverse events were mild or moderate in severity. There were no serious adverse events or discontinuations due to adverse events. There was one grade 3 elevation in the γ-glutamyltransferase level, which was considered by the investigator to be related to statin therapy. In the single-dose phase, inclisiran doses of 300 mg or more reduced the PCSK9 level (up to a least-squares mean reduction of 74.5% from baseline to day 84), and doses of 100 mg or more reduced the LDL cholesterol level (up to a least-squares mean reduction of 50.6% from baseline). Reductions in the levels of PCSK9 and LDL cholesterol were maintained at day 180 for doses of 300 mg or more. All multiple-dose regimens reduced the levels of PCSK9 (up to a least-squares mean reduction of 83.8% from baseline to day 84) and LDL cholesterol (up to a least-squares mean reduction of 59.7% from baseline to day 84).ConclusionsIn this phase 1 trial, no serious adverse events were observed with inclisiran. Doses of 300 mg or more (in single or multiple doses) significantly reduced levels of PCSK9 and LDL cholesterol for at least 6 months. (Funded by Alnylam Pharmaceuticals and the Medicines Company; ClinicalTrials.gov number, NCT02314442 .).

Dataset Information

PCSK9 inhibitor access barriers-issues and recommendations: Improving the access process for patients, clinicians and payers.

Publications

PCSK9 inhibitor access barriers-issues and recommendations: Improving the access process for patients, clinicians and payers.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets