Your browser doesn't support javascript.
Mostrar: 20 | 50 | 100
Resultados 1 - 20 de 116
Filtrar
2.
Plast Reconstr Surg ; 144(2): 395-407, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31348350

RESUMO

BACKGROUND: Decision analysis allows clinicians to apply evidence-based medicine to guide objective decisions in uncertain scenarios. There is no comprehensive review summarizing the various decision analysis tools used. The authors aimed to appraise and review the decision analytic models used in hand surgery. METHODS: A search of English articles on the PubMed, Ovid, and Embase databases was performed. All articles, regardless of date of publishing, were considered. Two reviewers, based on strict inclusion criteria, independently assessed each article. RESULTS: The search resulted in 5525 abstracts, which yielded 30 studies that met inclusion criteria. Included studies were grouped according to medical indications, with scaphoid fractures (n = 6) and carpal tunnel syndrome (n = 5) being the most commonly reported. Included articles used decision analysis (n = 15) and/or economic analyses (n = 23) to discuss diagnostic strategies or compare treatments. The three most common outcomes reported were utility (n = 12), cost per quality-adjusted life-year (n = 16), and quality-adjusted life-years (n = 16). The decision analysis models compared diagnostic strategies, management options, and novel treatments. CONCLUSIONS: Decision analysis is increasingly popular in hand surgery. It is useful for comparing surgical strategies through evaluation of quality-of-life outcomes and costing data. The most common model was a simple decision tree. The quality of decision analysis models can be improved with the addition of sensitivity analysis. Surgeons should be familiar with the principles of decision analysis, so that complex decisions can be evaluated using rigorous probabilistic models that combine risks and benefits of multiple strategies.


Assuntos
Técnicas de Apoio para a Decisão , Mãos/cirurgia , Tratamento Conservador/economia , Análise Custo-Benefício , Humanos , Procedimentos Ortopédicos/economia , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Terapias em Estudo/economia
3.
Value Health ; 22(6): 677-683, 2019 06.
Artigo em Inglês | MEDLINE | ID: mdl-31198185

RESUMO

Payers are concerned that one-off "cures" bring great uncertainty with the consequential risk of incorrect adoption decisions, and significant budget impact from large one-off payments. Innovators worry about bias against "cures" in favor of repeat treatment, which is not in patients' interests. We find that even in the absence of a difference in uncertainty of outcomes, adverse pay-offs differ. The greater financial risk associated with a cure is related to the issue of treatment discontinuation, driven by irreversibility. This paper uses a stylized example to illustrate the need to separate three different elements of the issue: (i) one-off versus repeat or ongoing treatment, (ii) duration of treatment effect, and (iii) the potential role of financial arrangements or risk sharing to mitigate the financial risk to the payer. It concludes that: (i) prevalence and discontinuation issues mean that the impact on the payer of an incorrect decision is greater with a one-off treatment than a repeat therapy; (ii) with evidence collection this risk diminishes over time (a form of CED or OWR); and (iii) financial arrangements or risk sharing can eliminate differences for the payer as between one-off and repeat therapy. The impact of (iii) also addresses payer concerns about budget impact.


Assuntos
Gastos em Saúde/normas , Terapias em Estudo/normas , Incerteza , Análise Custo-Benefício , Gastos em Saúde/estatística & dados numéricos , Humanos , Terapias em Estudo/economia , Terapias em Estudo/métodos
6.
Value Health ; 22(2): 220-224, 2019 02.
Artigo em Inglês | MEDLINE | ID: mdl-30711067

RESUMO

OBJECTIVE: This paper constitutes the first attempt to draw lessons from the recent uptake of health economic evaluation of innovative drugs in the French regulatory framework. STUDY DESIGN: Taking the example of new direct-acting antivirals against hepatitis C virus, the paper asks whether and how the cost-effectiveness (CE) opinions issued by the French National Health Authority improve the information available to support the pricing decisions. METHODS: The analysis compares the assessment of these drugs based on three different sources: CE opinions, clinical opinions, and the published cost-utility analyses (CUA) available in the literature and identified through a systematic review. RESULTS: The results show that CE opinions bring to the fore three issues prone to impact the incremental cost utility ratio and those were not available to the decision maker through clinical opinions or published CUA: the stage of treatment initiation, the modeling of the disease progression, and the uncertainty around the efficacy rates. CONCLUSIONS: France has introduced the criterion of the cost per QALY gained in the pricing and regulation of innovative pharmaceuticals since 2013. Our analysis shows that the use of CUA does enhance the information available to the decision makers on the value of the treatments.


Assuntos
Antivirais/economia , Análise Custo-Benefício/métodos , Hepatite C/economia , Terapias em Estudo/economia , Antivirais/farmacologia , Antivirais/uso terapêutico , Economia Médica , França/epidemiologia , Hepatite C/tratamento farmacológico , Hepatite C/epidemiologia , Humanos , Imidazóis/economia , Imidazóis/uso terapêutico , Sofosbuvir/economia , Sofosbuvir/uso terapêutico , Terapias em Estudo/métodos
7.
J Manag Care Spec Pharm ; 25(2): 164-173, 2019 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-30698089

RESUMO

BACKGROUND: Preapproval information exchange (PIE) is the communication of clinical and health care economic information (HCEI) on therapies in development between U.S. population health decision makers (PHDMs) and drug manufacturers before regulatory approval. Early access to HCEI can help PHDMs plan budgets, inform formulary coverage decisions, and accelerate policy development to improve patient access to innovative health technologies. While recent FDA guidelines and proposed legislation aim to clarify definitions and execution of PIE, the level of U.S. PHDMs' awareness and preferences for early engagement with investigational therapies is unclear. OBJECTIVES: To (a) assess U.S. PHDMs' current knowledge and perceptions of PIE and (b) identify their preferences for PIE, in order to shape future development of related guidelines and policy. METHODS: An expert panel of 5 U.S. PHDMs representing national and regional payers from integrated health plans, pharmacy benefit management, and specialty pharmacy organizations participated in a 2-round modified Delphi process. A targeted literature review of PIE was used to develop a web-based survey administered to the panel. Survey responses were grouped by consensus, with ≥ 80% agreement or disagreement as the threshold in round 1. In round 2, content experts moderated an inperson meeting where panelists deliberated and then revoted on round 1 nonconsensus topics. RESULTS: In the round 1 survey, the panelists reached consensus on 35 of 54 (65%) multiple-choice questions. In the round 2 face-to-face discussion, 19 nonconsensus questions were debated. One question was removed due to duplication, and consensus was achieved on 16 additional questions, with 2 items of nonconsensus remaining. Overall, consensus was achieved on 51 of 53 topics (96%). There was full consensus by the panelists that PIE should encompass new molecular entities and new indications of marketed therapies. Panelists completely agreed on the need for a legislative "safe harbor" for PIE. Four of five panelists reported that the value of PIE was high to PHDMs, and they expressed a strong preference for peer-to-peer conversations with manufacturers' medical or outcomes liaisons for PIE. The main topic of nonconsensus was the optimal timing of PIE. CONCLUSIONS: This panel of U.S. PHDMs achieved consensus on the value of PIE to proactively budget, make informed formulary decisions, and develop pharmaceutical policy to facilitate patient access to new therapies. The PHDM panel's preferences for PIE should be considered in legislative discussions and planning for future PIE by PHDMs and manufacturers. The full contribution of PIE to improving the U.S. health system can best be realized under a safe harbor that allows U.S. PHDM and manufacturer experts to engage in robust scientific and economic discourse. Additional research and broad stakeholder engagement is needed to advance the development of formal U.S. PIE guidelines. DISCLOSURES: This study was funded by GlaxoSmithKline (GSK). Brixner, Oderda, and Biskupiak are principals of Millcreek Outcomes Group, a consultancy that received funding from GSK to conduct this study. Marciniak and Woodward are employees of GSK and own stock in GSK. Seifter was employed by GSK at the time of this study. Neumann served as external health policy advisor for this study and has consulted or served on advisory boards with Merck, Bayer, Pacira, Novo Nordisk, Amgen, Abbvie, Boston Health Economics, Vertex, Precision Health Economics, the Congressional Budget Office, CEA Registry Sponsors, Axovant, Veritech, Janssen, Parateck, Avexis, GSK, Celegene, Bluebird, Roche, Sage, Sarepta, Biogen, and Ipsen. Neumann also reports grants from Amgen, Lundbeck, Gates, NPC, Alzheimer's Association, and NIH.


Assuntos
Tomada de Decisões , Assistência à Saúde/economia , Acesso aos Serviços de Saúde , Terapias em Estudo/economia , Orçamentos , Consenso , Assistência à Saúde/organização & administração , Técnica Delfos , Aprovação de Drogas , Indústria Farmacêutica/economia , Indústria Farmacêutica/organização & administração , Guias como Assunto , Política de Saúde , Humanos , Inquéritos e Questionários , Estados Unidos
8.
Eur J Cancer ; 105: 33-40, 2018 12.
Artigo em Inglês | MEDLINE | ID: mdl-30384014

RESUMO

PURPOSE: Since 2011, significant progress was observed in metastatic melanoma (MM), with the commercialisation of seven immunotherapies or targeted therapies, which showed significant improvement in survival. In France, in 2004, the cost of MM was estimated at €1634 per patient; this cost has not been re-estimated since. This study provided an update on survival and cost in real-life clinical practice. METHODS: Clinical and economic data (treatments, hospitalisations, radiotherapy sessions, visits, imaging and biological exams) were extracted from the prospective MelBase cohort, collecting individual data in 955 patients in 26 hospitals, from diagnosis of metastatic disease until death. Survival was estimated by the Kaplan-Meier method. Costs were calculated from the health insurance perspective using French tariffs. For live patients, survival and costs were extrapolated using a multistate model, describing the 5-year course of the disease according to patient prognostic factors and number of treatment lines. RESULTS: Since the availability of new drugs, the mean survival time of MM patients has increased to 23.6 months (95%confidence interval [CI] :21.2;26.6), with 58% of patients receiving a second line of treatment. Mean management costs increased to €269,682 (95%CI:244,196;304,916) per patient. Drugs accounted for 80% of the total cost. CONCLUSION: This study is the first that evaluated the impact of immunotherapies and targeted therapies both on survival and cost in real-life conditions. Alongside the introduction of breakthrough therapies in the first and subsequent lines, MM has been associated with a significant increase in survival but also in costs, raising the question of financial sustainability.


Assuntos
Antineoplásicos/uso terapêutico , Melanoma/tratamento farmacológico , Terapias em Estudo/economia , Adulto , Idoso , Idoso de 80 Anos ou mais , Antineoplásicos/economia , Estudos de Coortes , Análise Custo-Benefício , Custos de Medicamentos , Feminino , França , Custos de Cuidados de Saúde , Custos Hospitalares , Humanos , Imunoterapia/economia , Imunoterapia/estatística & dados numéricos , Estimativa de Kaplan-Meier , Masculino , Melanoma/economia , Melanoma/mortalidade , Pessoa de Meia-Idade , Terapia de Alvo Molecular/economia , Terapia de Alvo Molecular/estatística & dados numéricos , Estudos Prospectivos , Taxa de Sobrevida , Terapias em Estudo/estatística & dados numéricos , Adulto Jovem
9.
J Clin Endocrinol Metab ; 103(11): 4043-4088, 2018 11 01.
Artigo em Inglês | MEDLINE | ID: mdl-30272171

RESUMO

Objective: To update the congenital adrenal hyperplasia due to steroid 21-hydroxylase deficiency clinical practice guideline published by the Endocrine Society in 2010. Conclusions: The writing committee presents updated best practice guidelines for the clinical management of congenital adrenal hyperplasia based on published evidence and expert opinion with added considerations for patient safety, quality of life, cost, and utilization.


Assuntos
Hiperplasia Suprarrenal Congênita/terapia , Endocrinologia/normas , Sociedades Médicas/normas , Hiperplasia Suprarrenal Congênita/diagnóstico , Hiperplasia Suprarrenal Congênita/economia , Hiperplasia Suprarrenal Congênita/genética , Análise Custo-Benefício , Feminino , Terapias Fetais/economia , Terapias Fetais/métodos , Terapias Fetais/normas , Aconselhamento Genético/economia , Aconselhamento Genético/métodos , Aconselhamento Genético/normas , Glucocorticoides/uso terapêutico , Humanos , Recém-Nascido , Assistência de Longa Duração/economia , Assistência de Longa Duração/métodos , Assistência de Longa Duração/normas , Triagem Neonatal/economia , Triagem Neonatal/normas , Segurança do Paciente/normas , Qualidade de Vida , Terapias em Estudo/economia , Terapias em Estudo/métodos , Terapias em Estudo/normas
10.
Clin Drug Investig ; 38(10): 967-976, 2018 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-30143953

RESUMO

BACKGROUND AND OBJECTIVE: Immuno-oncology therapies represent a new treatment opportunity for patients affected by metastatic melanoma. The purpose of this study was to estimate the costs of immune-related adverse events (irAEs) associated with the new anti-PD1 immuno-oncology therapies, with the anti-CTLA-4 immuno-oncology therapy and with the combined therapy (CTLA4 + anti-PD1) in patients affected by metastatic melanoma. MATERIALS AND METHODS: A probabilistic cost-of-illness (COI) model was developed to estimate the management costs of grade ≥ 3 adverse events associated with the new anti-PD1 therapies (pembrolizumab and nivolumab), the anti-CTLA-4 therapy (ipilimumab) and the combined therapy CTLA4 + anti-PD1 (nivolumab + ipilimumab) for the treatment of patients with metastatic melanoma from the National Health Service (NHS) perspective in Italy. Identification of the epidemiological and cost parameters was carried out through a systematic literature review (SLR). Univariate and probabilistic sensitivity analyses were performed to account for uncertainty and variation in the model results. RESULTS: The model estimated a cost associated with the management of grade ≥ 3 immune-related adverse events in patients with metastatic melanoma equal to €176.2 (95% CI 63.5-335.0) for anti-CTLA-4 therapy, €48.6 (95% CI 40.1-58.5) for the new anti-PDI therapies and €276.8 (95% CI 240.4-316.2) for the combined therapy. Among the innovative therapies for the considered metastatic melanoma, the combined therapy was the most expensive innovative treatment in terms of event management of immune-related grade ≥ 3 adverse events. CONCLUSION: This study may represent a useful tool to understand the economic burden associated with the management of irAEs associated with patients affected by metastatic melanoma.


Assuntos
Antineoplásicos Imunológicos/economia , Custos e Análise de Custo/métodos , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/economia , Melanoma/economia , Terapias em Estudo/economia , Anticorpos Monoclonais Humanizados/administração & dosagem , Anticorpos Monoclonais Humanizados/efeitos adversos , Anticorpos Monoclonais Humanizados/economia , Antineoplásicos Imunológicos/administração & dosagem , Antineoplásicos Imunológicos/efeitos adversos , Antígeno CTLA-4/administração & dosagem , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/epidemiologia , Humanos , Ipilimumab/administração & dosagem , Ipilimumab/efeitos adversos , Ipilimumab/economia , Itália/epidemiologia , Melanoma/tratamento farmacológico , Melanoma/epidemiologia , Nivolumabe/administração & dosagem , Nivolumabe/efeitos adversos , Nivolumabe/economia , Terapias em Estudo/efeitos adversos
14.
Appl Health Econ Health Policy ; 16(2): 157-165, 2018 04.
Artigo em Inglês | MEDLINE | ID: mdl-29470774

RESUMO

Many pharmaceuticals are effective in multiple indications and the degree of effectiveness may differ. A product-based pricing and reimbursement system with a single price per product is insufficient to reflect the variable values between different indications. The objective of this article is to present examples of actual pricing and reimbursement decisions using current value-based pricing in Sweden and to discuss their implications and possible solutions. The value of several cancer drugs was estimated for various indications based on a willingness-to-pay threshold of 1 million SEK (EUR 104,000) per QALY gained. For some drugs, the estimated value was higher than the drug acquisition cost in several indications, whilst in others, the estimated value was lower than the drug acquisition cost. Drugs used in combination present a special case. If a drug prolongs survival and consequently also a continued use of the anchor drug, the combination use may not be cost effective even at a zero price. In a product-based pricing and reimbursement system, patients may not get access to drugs or access may be delayed and manufacturers may be discouraged to invest in future indications. To overcome these issues, there are several approaches to link price and value. One approach is a "weighted-average" price based on an average of the value across all indications. Another is "multi-indication pricing," which enables price differentiation between indications. However, there are several barriers for applying multi-indication pricing and reimbursement schemes. One barrier is the lack of existing administrative infrastructure to track patients' indications.


Assuntos
Custos de Medicamentos , Quimioterapia Combinada , Melhoria de Qualidade , Mecanismo de Reembolso , Terapias em Estudo , Antineoplásicos/administração & dosagem , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Quimioterapia Combinada/economia , Quimioterapia Combinada/métodos , Humanos , Melhoria de Qualidade/economia , Melhoria de Qualidade/organização & administração , Mecanismo de Reembolso/economia , Mecanismo de Reembolso/organização & administração , Suécia , Terapias em Estudo/economia , Terapias em Estudo/métodos
15.
Health Policy ; 122(3): 217-229, 2018 03.
Artigo em Inglês | MEDLINE | ID: mdl-29223847

RESUMO

BACKGROUND: Advanced therapy medicinal products (ATMPs) are innovative therapies likely associated with high prices. Payers need guidance to create a balance between ensuring patient access to breakthrough therapies and maintaining the financial sustainability of the healthcare system. OBJECTIVE: The aims of this study were to identify, define, classify and compare the approaches to funding high-cost medicines proposed in the literature, to analyze their appropriateness for ATMP funding and to suggest an optimal funding model for ATMPs. RESULTS: Forty-eight articles suggesting new funding models for innovative high-cost therapies were identified. The models were classified into 3 groups: financial agreement, health outcomes-based agreement and healthcoin. Financial agreement encompassed: discounts, rebates, price and volume caps, price-volume agreements, loans, cost-plus price, intellectual-based payment and fund-based payment. Health outcomes-based agreements were defined as agreements between manufacturers and payers based on drug performance, and were divided into performance-based payment and coverage with evidence development. Healthcoin described a new suggested tradeable currency used to assign monetary value to incremental outcomes. CONCLUSION: With a large number of ATMPs in development, it is time for stakeholders to start thinking about new pathways and funding strategies for these innovative high-cost therapies. An "ATMP-specific fund" may constitute a reasonable solution to ensure rapid patient access to innovation without threatening the sustainability of the health care system.


Assuntos
Custos de Medicamentos/tendências , Indústria Farmacêutica/economia , Gastos em Saúde , Terapias em Estudo/economia , Comércio/economia , Comércio/métodos , Humanos , Terapias em Estudo/estatística & dados numéricos
17.
Med Sci (Paris) ; 33(12): 1121-1123, 2017 Dec.
Artigo em Francês | MEDLINE | ID: mdl-29261502

RESUMO

Inflated drug prices necessarily raise the issue of rational allocation of health care resources. The system operated by the NICE agency in the UK attempts to do this by calculating the cost per quality-adjusted life year gained (QALY) and recommending funding only for drugs whose cost per QALY falls under a certain threshold. The whole process is documented in detail and easily accessible, and often results in significant discounts on drug prices. Given that some kind of rationing of health care is inevitable, the rational and transparent process followed by NICE has a number of positive features.


Assuntos
Redução de Custos , Leucemia/economia , Leucemia/terapia , Terapias em Estudo/economia , Idade de Início , Criança , Controle de Custos/organização & administração , Controle de Custos/normas , Redução de Custos/economia , Redução de Custos/métodos , Redução de Custos/normas , Efeitos Psicossociais da Doença , Análise Custo-Benefício , Humanos , Leucemia/epidemiologia , Terapia de Alvo Molecular/economia , Administração em Saúde Pública/economia
SELEÇÃO DE REFERÊNCIAS
DETALHE DA PESQUISA