RESUMO
The National Center for Advancing Translational Sciences' virtual 2021 conference on gene-targeted therapies (GTTs) encouraged multidisciplinary dialogue on a wide range of GTT topic areas. Each of three parallel working groups included social scientists and clinical scientists, and the three major sessions included a presentation on economic issues related to their focus area. These experts also coordinated their efforts across the three groups. The economics-related presentations covered three areas with some overlap: (1) value assessment, uncertainty, and dynamic efficiency; (2) affordability, pricing, and financing; and (3) evidence generation, coverage, and access. This article provides a synopsis of three presentations, some of their key recommendations, and an update on related developments in the past year. The key high-level findings are that GTTs present unique data and policy challenges, and that existing regulatory, health technology assessment, as well as payment and financing systems will need to adapt. But these adjustments can build on our existing foundation of regulatory and incentive systems for innovation, and much can be done to accelerate progress in GTTs. Given the substantial unmet medical need that exists for these oft-neglected patients suffering from rare diseases, it would be a tragedy to not leverage these exciting scientific advances in GTTs.
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Doenças Raras , Humanos , Custos e Análise de CustoRESUMO
BACKGROUND: Employer-sponsored corporate wellness programs have spread despite limited evidence of effectiveness in improving health or reducing costs. Some programs have offered genetic testing as a benefit to employees, but little is known about this practice. METHODS: In December 2019, we conducted a systematic Google search to identify vendors offering corporate wellness programs involving genetics. We performed qualitative content analysis of publicly available information about the vendors' products and practices disclosed on their websites. RESULTS: Fifteen vendors were identified. Details regarding genetic testing offered within wellness programs were difficult to decipher from vendors' websites, including which specific products were included. No evidence was provided to support vendor claimed improvements in employer costs, employee health, and job performance. Only half offered health and genetic counseling services. Most vendors were ambiguous regarding data sharing. Disclaimer language was included in vendors' stated risks and limitations, ostensibly to avoid oversight and liability. CONCLUSION: We found a lack of transparency among corporate wellness program vendors, underscoring challenges that stakeholders encounter when trying to assess (a) how such programs are using genetics, (b) the potential benefits of such applications, and (c) the adequacy of protections to ensure scientific evidence support any health claims and genetic nondiscrimination.
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Comércio/estatística & dados numéricos , Testes Genéticos/estatística & dados numéricos , Promoção da Saúde/estatística & dados numéricos , Comércio/economia , Utilização de Instalações e Serviços/economia , Utilização de Instalações e Serviços/estatística & dados numéricos , Testes Genéticos/economia , Promoção da Saúde/economia , Humanos , Estados UnidosRESUMO
OBJECTIVES: Given the potential of real-world evidence (RWE) to inform understanding of the risk-benefit profile of next-generation sequencing (NGS)-based testing, we undertook a study to describe the current landscape of whether and how payers use RWE as part of their coverage decision making and potential solutions for overcoming barriers. METHODS: We performed a scoping literature review of existing RWE evidentiary frameworks for evaluating new technologies and identified barriers to clinical integration and evidence gaps for NGS. We synthesized findings as potential solutions for improving the relevance and utility of RWE for payer decision-making. RESULTS: Payers require evidence of clinical utility to inform coverage decisions, yet we found a relatively small number of published RWE studies, and these are predominately focused on oncology, pharmacogenomics, and perinatal/pediatric testing. We identified 3 categories of innovation that may help address the current undersupply of RWE studies for NGS: (1) increasing use of RWE to inform outcomes-based contracting for new technologies, (2) precision medicine initiatives that integrate clinical and genomic data and enable data sharing, and (3) Food and Drug Administration reforms to encourage the use of RWE. Potential solutions include development of data and evidence review standards, payer engagement in RWE study design, use of incentives and partnerships to lower the barriers to RWE generation, education of payers and providers concerning the use of RWE and NGS, and frameworks for conducting outcomes-based contracting for NGS. CONCLUSIONS: We provide numerous suggestions to overcome the data, methodologic, infrastructure, and policy challenges constraining greater integration of RWE in assessments of NGS.
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Tomada de Decisões , Medicina Baseada em Evidências/economia , Sequenciamento de Nucleotídeos em Larga Escala , Reembolso de Seguro de Saúde/economia , Avaliação da Tecnologia Biomédica , Farmacoeconomia , Sequenciamento de Nucleotídeos em Larga Escala/economia , Sequenciamento de Nucleotídeos em Larga Escala/tendências , Humanos , Oncologia/economia , Oncologia/tendências , Participação dos Interessados , Estados UnidosRESUMO
BACKGROUND: Clinical use of next-generation sequencing (NGS) tests has been increasing, but few studies have examined their economic value. Several studies have noted that there are methodological challenges to conducting economic evaluations of NGS tests. OBJECTIVE: Our objective was to examine key methodological challenges for conducting economic evaluations of NGS tests, prioritize these challenges for future research, and identify how studies have attempted solutions to address these challenges. METHODS: We identified challenges for economic evaluations of NGS tests using prior literature and expert judgment of the co-authors. We used a modified Delphi assessment to prioritize challenges, based on importance and probability of resolution. Using a structured literature review and article extraction we then assessed whether published economic evaluations had addressed these challenges. RESULTS: We identified 11 challenges for conducting economic evaluations of NGS tests. The experts identified three challenges as the top priorities for future research: complex model structure, timeframe, and type of analysis and comparators used. Of the 15 published studies included in our literature review, four studies described specific solutions relevant to five of the 11 identified challenges. CONCLUSIONS: Major methodological challenges to economic evaluations of NGS tests remain to be addressed. Our results can be used to guide future research and inform decision-makers on how to prioritize research on the economic assessment of NGS tests.
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Sequenciamento de Nucleotídeos em Larga Escala/economia , Sequenciamento de Nucleotídeos em Larga Escala/normas , Avaliação da Tecnologia Biomédica/métodos , Análise Custo-Benefício , Técnica Delphi , Sequenciamento de Nucleotídeos em Larga Escala/tendências , Humanos , Avaliação da Tecnologia Biomédica/economiaRESUMO
Genetic testing and spending on that testing have grown rapidly since the mapping of the human genome in 2003. However, it is not widely known how many tests there are, how they are used, and how they are paid for. Little evidence from large data sets about their use has emerged. We shed light on the issue of genetic testing by providing an overview of the testing landscape. We examined test availability and spending for the full spectrum of genetic tests, using unique data sources on test availability and commercial payer spending for privately insured populations, focusing particularly on tests measuring multiple genes in the period 2014-17. We found that there were approximately 75,000 genetic tests on the market, with about ten new tests entering the market daily. Prenatal tests accounted for the highest percentage of spending on genetic tests, and spending on hereditary cancer tests accounted for the second-highest. Our results provide insights for those interested in assessing genetic testing markets, test usage, and health policy implications, including current debates over the most appropriate regulatory and payer coverage mechanisms.
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Testes Genéticos/economia , Testes Genéticos/estatística & dados numéricos , Gastos em Saúde , Acessibilidade aos Serviços de Saúde/estatística & dados numéricos , Estudos de Coortes , Bases de Dados Factuais , Feminino , Previsões , Serviços em Genética/economia , Serviços em Genética/estatística & dados numéricos , Serviços em Genética/tendências , Humanos , Masculino , Estudos Retrospectivos , Estados UnidosAssuntos
Testes Genéticos/economia , Genômica/economia , Cobertura do Seguro , Humanos , Medicaid , Medicare , Estados UnidosRESUMO
OBJECTIVES: The aim of this study was to examine the evidence payers cited in their coverage policies for multi-gene panels and sequencing tests (panels), and to compare these findings with the evidence payers cited in their coverage policies for other types of medical interventions. METHODS: We used the University of California at San Francisco TRANSPERS Payer Coverage Registry to identify coverage policies for panels issued by five of the largest US private payers. We reviewed each policy and categorized the evidence cited within as: clinical studies, systematic reviews, technology assessments, cost-effectiveness analyses (CEAs), budget impact studies, and clinical guidelines. We compared the evidence cited in these coverage policies for panels with the evidence cited in policies for other intervention types (pharmaceuticals, medical devices, diagnostic tests and imaging, and surgical interventions) as reported in a previous study. RESULTS: Fifty-five coverage policies for panels were included. On average, payers cited clinical guidelines in 84 percent of their coverage policies (range, 73-100 percent), clinical studies in 69 percent (50-87 percent), technology assessments 47 percent (33-86 percent), systematic reviews or meta-analyses 31 percent (7-71 percent), and CEAs 5 percent (0-7 percent). No payers cited budget impact studies in their policies. Payers less often cited clinical studies, systematic reviews, technology assessments, and CEAs in their coverage policies for panels than in their policies for other intervention types. Payers cited clinical guidelines in a comparable proportion of policies for panels and other technology types. CONCLUSIONS: Payers in our sample less often cited clinical studies and other evidence types in their coverage policies for panels than they did in their coverage policies for other types of medical interventions.
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Tomada de Decisões , Testes Genéticos , Cobertura do Seguro/organização & administração , Reembolso de Seguro de Saúde/normas , Avaliação da Tecnologia Biomédica/organização & administração , Análise Custo-Benefício , Prática Clínica Baseada em Evidências/organização & administração , Humanos , Cobertura do Seguro/economia , Cobertura do Seguro/normas , Reembolso de Seguro de Saúde/economia , Guias de Prática Clínica como Assunto , Avaliação da Tecnologia Biomédica/normas , Estados UnidosRESUMO
Comparative effectiveness research (CER) in genomic medicine (GM) measures the clinical utility of using genomic information to guide clinical care in comparison to appropriate alternatives. We summarized findings of high-quality systematic reviews that compared the analytic and clinical validity and clinical utility of GM tests. We focused on clinical utility findings to summarize CER-derived evidence about GM and identify evidence gaps and future research needs. We abstracted key elements of study design, GM interventions, results, and study quality ratings from 21 systematic reviews published in 2010 through 2015. More than half (N = 13) of the reviews were of cancer-related tests. All reviews identified potentially important clinical applications of the GM interventions, but most had significant methodological weaknesses that largely precluded any conclusions about clinical utility. Twelve reviews discussed the importance of patient-centered outcomes, although few described evidence about the impact of genomic medicine on these outcomes. In summary, we found a very limited body of evidence about the effect of using genomic tests on health outcomes and many evidence gaps for CER to address.Genet Med advance online publication 13 April 2017.
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Pesquisa Comparativa da Efetividade/métodos , Medicina de Precisão/economia , Medicina Baseada em Evidências , Humanos , Avaliação de Resultados em Cuidados de Saúde/economia , Medicina de Precisão/métodos , Projetos de PesquisaRESUMO
Background: Hereditary cancer panels (HCPs), testing for multiple genes and syndromes, are rapidly transforming cancer risk assessment but are controversial and lack formal insurance coverage. We aimed to identify payers' perspectives on barriers to HCP coverage and opportunities to address them. Comprehensive cancer risk assessment is highly relevant to the Precision Medicine Initiative (PMI), and payers' considerations could inform PMI's efforts. We describe our findings and discuss them in the context of PMI priorities. Methods: We conducted semi-structured interviews with 11 major US payers, covering >160 million lives. We used the framework approach of qualitative research to design, conduct, and analyze interviews, and used simple frequencies to further describe findings. Results: Barriers to HCP coverage included poor fit with coverage frameworks (100%); insufficient evidence (100%); departure from pedigree/family history-based testing toward genetic screening (91%); lacking rigor in the HCP hybrid research/clinical setting (82%); and patient transparency and involvement concerns (82%). Addressing barriers requires refining HCP-indicated populations (82%); developing evidence of actionability (82%) and pathogenicity/penetrance (64%); creating infrastructure and standards for informing and recontacting patients (45%); separating research from clinical use in the hybrid clinical-research setting (44%); and adjusting coverage frameworks (18%). Conclusions: Leveraging opportunities suggested by payers to address HCP coverage barriers is essential to ensure patients' access to evolving HCPs. Our findings inform 3 areas of the PMI: addressing insurance coverage to secure access to future PMI discoveries; incorporating payers' evidentiary requirements into PMI's research agenda; and leveraging payers' recommendations and experience to keep patients informed and involved.
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Testes Genéticos/economia , Cobertura do Seguro , Reembolso de Seguro de Saúde/economia , Neoplasias/diagnóstico , Medicina de Precisão/economia , Acessibilidade aos Serviços de Saúde/economia , Humanos , Neoplasias/genética , Medicina de Precisão/métodos , Pesquisa Qualitativa , Medição de Risco/métodos , Estados UnidosRESUMO
BACKGROUND: New payment and care organization approaches, such as those of accountable care organizations (ACOs), are reshaping accountability and shifting risk, as well as decision making, from payers to providers, within the Triple Aim context of health reform. The Triple Aim calls for improving experience of care, improving health of populations, and reducing health care costs. OBJECTIVES: To understand how the transition to the ACO model impacts decision making on adoption and use of innovative technologies in the era of accelerating scientific advancement of personalized medicine and other innovations. METHODS: We interviewed representatives from 10 private payers and 6 provider institutions involved in implementing the ACO model (i.e., ACOs) to understand changes, challenges, and facilitators of decision making on medical innovations, including personalized medicine. We used the framework approach of qualitative research for study design and thematic analysis. RESULTS: We found that representatives from the participating payer companies and ACOs perceive similar challenges to ACOs' decision making in terms of achieving a balance between the components of the Triple Aim-improving care experience, improving population health, and reducing costs. The challenges include the prevalence of cost over care quality considerations in ACOs' decisions and ACOs' insufficient analytical and technology assessment capacity to evaluate complex innovations such as personalized medicine. Decision-making facilitators included increased competition across ACOs and patients' interest in personalized medicine. CONCLUSIONS: As new payment models evolve, payers, ACOs, and other stakeholders should address challenges and leverage opportunities to arm ACOs with robust, consistent, rigorous, and transparent approaches to decision making on medical innovations.
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Organizações de Assistência Responsáveis/organização & administração , Tomada de Decisões , Seguradoras/economia , Medicina de Precisão/métodos , Organizações de Assistência Responsáveis/economia , Análise Custo-Benefício , Humanos , Entrevistas como Assunto , Neoplasias/diagnóstico , Neoplasias/genética , Medicina de Precisão/economia , Avaliação da Tecnologia Biomédica/organização & administração , Estados UnidosRESUMO
PURPOSE: Cell-free DNA (cfDNA) prenatal screening tests have been rapidly adopted into clinical practice, due in part to positive insurance coverage. We evaluated the framework payers used in making coverage decisions to describe a process that should be informative for other sequencing tests. METHODS: We analyzed coverage policies from the 19 largest US private payers with publicly available policies through February 2016, building from the University of California San Francisco TRANSPERS Payer Coverage Policy Registry. RESULTS: All payers studied cover cfDNA screening for detection of trisomies 21, 18, and 13 in high-risk, singleton pregnancies, based on robust clinical validity (CV) studies and modeled evidence of clinical utility (CU). Payers typically evaluated the evidence for each chromosomal abnormality separately, although results are offered as part of a panel. Starting in August 2015, 8 of the 19 payers also began covering cfDNA screening in average-risk pregnancies, citing recent CV studies and updated professional guidelines. Most payers attempted, but were unable, to independently assess analytic validity (AV). CONCLUSION: Payers utilized the standard evidentiary framework (AV/CV/CU) when evaluating cfDNA screening but varied in their interpretation of the sufficiency of the evidence. Professional guidelines, large CV studies, and decision analytic models regarding health outcomes appeared highly influential in coverage decisions.Genet Med advance online publication 22 September 2016.
Assuntos
Ácidos Nucleicos Livres/genética , Sequenciamento de Nucleotídeos em Larga Escala/métodos , Diagnóstico Pré-Natal/métodos , Análise de Sequência de DNA/métodos , Trissomia/diagnóstico , Tomada de Decisão Clínica , Tomada de Decisões , Síndrome de Down/diagnóstico , Síndrome de Down/genética , Feminino , Testes Genéticos , Humanos , Cobertura do Seguro , Gravidez , Sistema de Registros , Trissomia/genética , Síndrome da Trissomia do Cromossomo 13/diagnóstico , Síndrome da Trissomia do Cromossomo 13/genética , Síndrome da Trissomía do Cromossomo 18/diagnóstico , Síndrome da Trissomía do Cromossomo 18/genéticaAssuntos
Pesquisa Comparativa da Efetividade/métodos , Técnicas de Diagnóstico Molecular , Medicina de Precisão/métodos , Ensaios Clínicos como Assunto , Análise Custo-Benefício , Humanos , Técnicas de Diagnóstico Molecular/economia , Técnicas de Diagnóstico Molecular/métodos , Técnicas de Diagnóstico Molecular/tendências , Medicina de Precisão/tendênciasRESUMO
Public and private payers face complex decisions regarding whether, when, and how to cover and reimburse for next generation sequencing (NGS)-based tests. Yet a predictable reimbursement pathway is critical both for patient access and incentives to provide the market with better clinical evidence. While preliminary data suggests that payers will use similar evidentiary standards as those used to evaluate established molecular diagnostic tests, the volume and complexity of information generated by NGS raises a host of additional considerations for payers that are specific to this technology.
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Genoma Humano , Sequenciamento de Nucleotídeos em Larga Escala/economia , Cobertura do Seguro , Seguro Saúde , Ciência de Laboratório Médico/economia , Mecanismo de Reembolso , Análise de Sequência de DNA , Humanos , Medicina de Precisão/economia , Estados UnidosRESUMO
PURPOSE: Insufficient evidence on the net benefits and harms of genomic tests in real-world settings is a translational barrier for genomic medicine. Understanding stakeholders' assessment of the current evidence base for clinical practice and coverage decisions should be a critical step in influencing research, policy, and practice. METHODS: Twenty-two stakeholders participated in a workshop exploring the evidence of genomic tests for clinical and coverage decision making. Stakeholders completed a survey prior to and during the meeting. They also discussed if they would recommend for or against current clinical use of each test. RESULTS: At baseline, the level of confidence in the clinical validity and clinical utility of each test varied, although the group expressed greater confidence for epidermal growth factor receptor mutation and Lynch syndrome testing than for Oncotype DX. Following the discussion, survey results reflected even less confidence for Oncotype DX, intermediate levels of confidence for [corrected] epidermal growth factor receptor mutation testing and stable levels of confidence [corrected] for Lynch syndrome testing. The majority of stakeholders would consider clinical use for all three tests, but under the conditions of additional research or a shared clinical decision-making approach. CONCLUSION: Stakeholder engagement in unbiased settings is necessary to understand various perspectives about evidentiary thresholds in genomic medicine. Participants recommended the use of various methods for evidence generation and synthesis.
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Neoplasias Colorretais Hereditárias sem Polipose/diagnóstico , Testes Genéticos/métodos , Genômica/métodos , Neoplasias da Mama/diagnóstico , Neoplasias da Mama/genética , Carcinoma Pulmonar de Células não Pequenas/diagnóstico , Carcinoma Pulmonar de Células não Pequenas/genética , Neoplasias Colorretais/diagnóstico , Neoplasias Colorretais/genética , Neoplasias Colorretais Hereditárias sem Polipose/genética , Tomada de Decisões , Receptores ErbB/genética , Feminino , Humanos , Mutação , Guias de Prática Clínica como Assunto , Reprodutibilidade dos TestesRESUMO
BACKGROUND: Systematic approaches to stakeholder-informed research prioritization are a central focus of comparative effectiveness research. Genomic testing in cancer is an ideal area to refine such approaches given rapid innovation and potentially significant impacts on patient outcomes. OBJECTIVE: To develop and pilot test a stakeholder-informed approach to prioritizing genomic tests for future study in collaboration with the cancer clinical trials consortium SWOG. METHODS: We conducted a landscape analysis to identify genomic tests in oncology using a systematic search of published and unpublished studies, and expert consultation. Clinically valid tests suitable for evaluation in a comparative study were presented to an external stakeholder group. Domains to guide the prioritization process were identified with stakeholder input, and stakeholders ranked tests using multiple voting rounds. RESULTS: A stakeholder group was created including representatives from patient-advocacy groups, payers, test developers, regulators, policy makers, and community-based oncologists. We identified 9 domains for research prioritization with stakeholder feedback: population impact; current standard of care, strength of association; potential clinical benefits, potential clinical harms, economic impacts, evidence of need, trial feasibility, and market factors. The landscape analysis identified 635 studies; of 9 tests deemed to have sufficient clinical validity, 6 were presented to stakeholders. Two tests in lung cancer (ERCC1 and EGFR) and 1 test in breast cancer (CEA/CA15-3/CA27.29) were identified as top research priorities. CONCLUSIONS: Use of a diverse stakeholder group to inform research prioritization is feasible in a pragmatic and timely manner. Additional research is needed to optimize search strategies, stakeholder group composition, and integration with existing prioritization mechanisms.
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Pesquisa Comparativa da Efetividade/organização & administração , Testes Genéticos , Neoplasias/genética , Pesquisa Comparativa da Efetividade/economia , Pesquisa sobre Serviços de Saúde , Humanos , Marketing de Serviços de Saúde , Guias de Prática Clínica como Assunto , PesquisaRESUMO
Economic evaluation provides health care decision makers with a powerful tool for resource allocation decisions because it offers a framework for comparing the costs and benefits of competing interventions or options. This paper reviews how economic analyses have been applied to the field of pharmacogenomics, both by the pharmaceutical industry to inform investment decisions and by payers to make coverage decisions. There is much anticipation that pharmacogenomic testing is likely to be cost-effective because it uses genomic information to improve drug effectiveness and reduce toxicity both in the drug development process and at the bedside. However, the demonstration of economic benefits first requires that pharmacogenomic testing show evidence of clinical effectiveness. This will only be achieved by greater participation of pharmacogenomics experts in comparative effectiveness research and additional emphasis on including costs in the determination of the relative value of pharmacogenomic testing to the health care system.
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Farmacogenética/economia , Análise Custo-Benefício , Atenção à Saúde/economia , Descoberta de Drogas , HumanosRESUMO
OBJECTIVE: To assess the effect of three-tier formulary adoption on medication continuation and spending among elderly members of retiree health plans. DATA SOURCES: Pharmacy claims and enrollment data on elderly members of four retiree plans that adopted a three-tier formulary over the period July 1999 through December 2002 and two comparison plans that maintained a two-tier formulary during this period. STUDY DESIGN: We used a quasi-experimental design to compare the experience of enrollees in intervention and comparison plans. We used propensity score methods to match intervention and comparison users of each drug class and plan. We estimated repeated measures regression models for each class/plan combination for medication continuation and monthly plan, enrollee, and total spending. We estimated logit models of the probability of nonpersistent use, medication discontinuation, and medication changes. DATA COLLECTION/EXTRACTION METHODS: We used pharmacy claims to create person-level drug utilization and spending files for the year before and year after three-tier adoption. PRINCIPAL FINDINGS: Three-tier formulary adoption resulted in shifting of costs from plan to enrollee, with relatively small effects on medication continuation. Although implementation had little effect on continuation on average, a small minority of patients were more likely to have gaps in use and discontinue use relative to comparison patients. CONCLUSIONS: Moderate cost sharing increases from three-tier formulary adoption had little effect on medication continuation among elderly enrolled in retiree health plans with relatively generous drug coverage.
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Custo Compartilhado de Seguro/economia , Formulários Farmacêuticos como Assunto/classificação , Cooperação do Paciente , Idoso , Idoso de 80 Anos ou mais , Uso de Medicamentos , Feminino , Humanos , Revisão da Utilização de Seguros , Masculino , Programas de Assistência Gerenciada/organização & administraçãoRESUMO
BACKGROUND: Medication guides (MG) and mandatory patient package inserts (MPPI) are required with some prescription medications. OBJECTIVE: We sought to determine how many patients receive, read, and understand these mandated materials. DESIGN AND PARTICIPANTS: A total of 3,620 patients were identified as filling prescriptions for isotretinoin or selected estrogen products from February 2004 to January 2005. Patients were surveyed to gauge receipt and understanding of the MG for isotretinoin and the MPPI for estrogen. MEASUREMENTS AND MAIN RESULTS: A total of 500 patients completed the survey, with 186 (93%) of the 200 isotretinoin patients and 258 (86%) of the 300 estrogen patients reporting receipt of the MG/MPPI with their most recent prescription. The majority of respondents reported confidence in their knowledge of their medication (86% for isotretinoin and 75% for estrogen). However, the mean score on 5 questions assessing recognition of medication risks was only slightly better than the score expected from guessing (3.1 vs 2.5, P < .01 for both isotretinoin and estrogen). CONCLUSIONS: Despite receiving the information and reporting confidence in medication knowledge, patients' understanding of major risks with these medications was poor. This finding highlights the need to develop better risk communication strategies to improve the safe and effective use of prescription medications.
Assuntos
Fármacos Dermatológicos/farmacologia , Rotulagem de Medicamentos , Estrogênios Conjugados (USP)/farmacologia , Estrogênios/farmacologia , Isotretinoína/farmacologia , Educação de Pacientes como Assunto , Adulto , Prescrições de Medicamentos , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Expenditures for medications used to treat attention-deficit/hyperactivity disorder (ADHD) in children have increased rapidly. Many employers and health plans have adopted 3-tier formularies in an attempt to control costs for these and other drugs. OBJECTIVE: To assess the effect of copayment increases associated with 3-tier formulary adoption on use and spending patterns for ADHD medications for children. DESIGN AND SETTING: Observational study using quasi-experimental design to compare effects on ADHD medication use and spending for children enrolled as dependents in an employer-sponsored plan that made major changes to its pharmacy benefit design and a comparison group of children covered by the same insurer. The plan simultaneously moved from a 1-tier (same copayment required for all drugs) to a 3-tier formulary and implemented an across-the-board copayment increase. The plan later moved 3 drugs from tier 3 to tier 2. PARTICIPANTS: An intervention group of 20 326 and a comparison group of 15 776 children aged 18 years and younger. MAIN OUTCOME MEASURES: Monthly probability of using an ADHD medication; plan, enrollee, and total ADHD medication spending; and medication continuation. RESULTS: A 3-tier formulary implementation resulted in a 17% decrease in the monthly probability of using medication (P<.001), a 20% decrease in expected total medication expenditures, and a substantial shifting of costs from the plan to families (P<.001). Intervention group children using medications in the pre-period were more likely to change to a medication in a different tier after 3-tier adoption, relative to the comparison group (P = .08). The subsequent tier changes resulted in increased plan spending (P<.001) and decreased patient spending (P = .003) for users but no differences in continuation. CONCLUSIONS: The copayment increases associated with 3-tier formulary implementation by 1 employer resulted in lower total ADHD medication spending, sizeable increases in out-of-pocket expenditures for families of children with ADHD, and a significant decrease in the probability of using these medications.