Your browser doesn't support javascript.
loading
Mostrar: 20 | 50 | 100
Resultados 1 - 20 de 101
Filtrar
1.
Expert Opin Drug Discov ; 16(1): 23-38, 2021 01.
Artigo em Inglês | MEDLINE | ID: mdl-32877233

RESUMO

INTRODUCTION: The COVID-19 pandemic raises the question of strategic readiness for emergent pathogens. The current case illustrates that the cost of inaction can be higher in the future. The perspective article proposes a dedicated, government-sponsored agency developing anti-viral leads against all potentially dangerous pathogen species. AREAS COVERED: The author explores the methods of computational drug screening and in-silico synthesis and proposes a specialized government-sponsored agency focusing on leads and functioning in collaboration with a network of labs, pharma, biotech firms, and academia, in order to test each lead against multiple viral species. The agency will employ artificial intelligence and machine learning tools to cut the costs further. The algorithms are expected to receive continuous feedback from the network of partners conducting the tests. EXPERT OPINION: The author proposes a bionic principle, emulating antibody response by producing a combinatorial diversity of high q uality generic antiviral leads, suitable for multiple potentially emerging species. The availability of multiple pre-tested agents and an even greater number of combinations would reduce the impact of the next outbreak. The methodologies developed in this effort are likely to find utility in the design of chronic disease therapeutics.


Assuntos
Antivirais/uso terapêutico , Desenvolvimento de Medicamentos/métodos , Indústria Farmacêutica , Parcerias Público-Privadas/organização & administração , Algoritmos , Desenvolvimento de Medicamentos/economia , Avaliação Pré-Clínica de Medicamentos , Humanos , Internacionalidade , Aprendizado de Máquina , Parcerias Público-Privadas/economia , Bibliotecas de Moléculas Pequenas
5.
PLoS One ; 15(9): e0239118, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32946474

RESUMO

INTRODUCTION: In 2012, bedaquiline became the first new treatment from a novel class to be approved for tuberculosis in nearly five decades and is now a core component of the standard of care for multidrug-resistant tuberculosis. In addition to the originator pharmaceutical company, Janssen, a range of governmental and non-profit entities have contributed to the development of bedaquiline. MATERIALS AND METHODS: We identified various avenues of public investments in the development of bedaquiline: direct funding of clinical trials and a donation programme, tax credits and deductions, and revenues resulting from the priority review voucher (PRV) awarded to the originator. Data on investments were gathered through contact with study leads and/or funders; for non-responses, published average costs were substituted. The originator company's expenses were estimated by similar methods. Tax credits and deductions were calculated based on estimated originator trial costs and donation expenses. The value of the PRV was estimated by application of a published model. RESULTS: Public contributions through clinical trials funding were estimated at US$109-252 million, tax credits at US$22-36 million, tax deductions at US$8-27 million, administration of a donation programme at US$5 million, PRV revenues at US$300-400 million. Total public investments were US$455-747 million and originator investments were US$90-240 million (if capitalized and risk-adjusted, US$647-1,201 million and US$292-772 million, respectively). CONCLUSIONS: Estimating the investments in the development of a medicine can inform discussions regarding fair pricing and future drug development. We estimated that total public investments exceeded the originator's by a factor of 1.6-5.1.


Assuntos
Antituberculosos/economia , Diarilquinolinas/economia , Desenvolvimento de Medicamentos/economia , Financiamento Governamental/economia , Organizações sem Fins Lucrativos/economia , Antituberculosos/uso terapêutico , Ensaios Clínicos como Assunto/economia , Diarilquinolinas/uso terapêutico , Custos de Medicamentos , Indústria Farmacêutica/economia , Humanos , Tuberculose Resistente a Múltiplos Medicamentos/tratamento farmacológico
8.
Proc Natl Acad Sci U S A ; 117(24): 13386-13392, 2020 06 16.
Artigo em Inglês | MEDLINE | ID: mdl-32487730

RESUMO

Clinical research should conform to high standards of ethical and scientific integrity, given that human lives are at stake. However, economic incentives can generate conflicts of interest for investigators, who may be inclined to withhold unfavorable results or even tamper with data in order to achieve desired outcomes. To shed light on the integrity of clinical trial results, this paper systematically analyzes the distribution of P values of primary outcomes for phase II and phase III drug trials reported to the ClinicalTrials.gov registry. First, we detect no bunching of results just above the classical 5% threshold for statistical significance. Second, a density-discontinuity test reveals an upward jump at the 5% threshold for phase III results by small industry sponsors. Third, we document a larger fraction of significant results in phase III compared to phase II. Linking trials across phases, we find that early favorable results increase the likelihood of continuing into the next phase. Once we take into account this selective continuation, we can explain almost completely the excess of significant results in phase III for trials conducted by large industry sponsors. For small industry sponsors, instead, part of the excess remains unexplained.


Assuntos
Ensaios Clínicos como Assunto/economia , Ensaios Clínicos como Assunto/normas , Relatório de Pesquisa/normas , Pesquisa Biomédica/economia , Ensaios Clínicos como Assunto/estatística & dados numéricos , Desenvolvimento de Medicamentos/economia , Desenvolvimento de Medicamentos/organização & administração , Indústria Farmacêutica/economia , Humanos , Sistema de Registros , Apoio à Pesquisa como Assunto
10.
Am J Law Med ; 46(1): 55-88, 2020 03.
Artigo em Inglês | MEDLINE | ID: mdl-32460655

RESUMO

Patent rights are recognized as a property asset with an attendant right to exclude. However, recent policy developments highlight that the right to exclude is not inviolable. This paper explores two rapidly evolving exceptions to patent exclusivity, both of which take the form of compulsory licenses. First, under the international Agreement on Trade-Related Aspects of Intellectual Property Rights ("TRIPS"), national governments can compel patent owners to out-license technology in service of greater good. These egalitarian compulsory licenses improve access to technology but undermine patent value. Second, compulsory licenses are increasingly relied upon as an equitable remedy in U.S. patent litigation. Typically referred to as "ongoing royalties," these court-mandated compulsory licenses are a modern alternative to injunctions against adjudged infringers. TRIPS compulsory licenses and ongoing royalties arise under independent legal frameworks, but necessarily invoke parallel economic considerations. While the wisdom of each has been discussed at length by others, this paper explores principles of royalty determination employed in each context. Considering both frameworks, an analysis of where each succeeds and fails is provided, together with an exploration of optimized royalty frameworks.


Assuntos
Tecnologia Biomédica/economia , Tecnologia Biomédica/legislação & jurisprudência , Propriedade Intelectual , Patentes como Assunto/legislação & jurisprudência , Desenvolvimento de Medicamentos/economia , Desenvolvimento de Medicamentos/legislação & jurisprudência , Direito Internacional , Estados Unidos
13.
Value Health ; 23(2): 191-199, 2020 02.
Artigo em Inglês | MEDLINE | ID: mdl-32113624

RESUMO

BACKGROUND: Changes in the regulatory context enable faster approval of transformative medicines. They also lead to health technology assessment (HTA) agencies having to make decisions with less evidence. In response, HTA agencies have also initiated forms of conditional approval. When the evidence base for a new oncology treatment leaves substantial uncertainty, the new Cancer Drugs Fund allows the National Institute for Heath and Care Excellence to give the manufacturer two options: (1) offer a low price based on conservative assumptions and obtain immediate approval ("stick") or (2) wait until the evidence base has further matured before finalizing a potentially higher agreed price ("twist"). OBJECTIVES: The purpose of this article is to explain how, using the theoretical framework of the expected value of sample information, simulation methods can help inform a manufacturer's decisions when faced with the option to stick or twist. METHODS: We first summarize a general model to help frame the manufacturer's negotiating strategy. We then use a motivating case study, based on a hypothetical immunotherapy, to illustrate how manufacturers can use simulation methods to robustly characterize the uncertainty inherent to further data collection and incorporate this uncertainty within their decision making. RESULTS: Our approach allows us to estimate the commercial value of generating additional data (the difference between the estimated net present value of stick and twist). We test the sensitivity of the results to different assumptions via scenario analyses. CONCLUSIONS: This article shows that simulation methods can be used to help pharmaceutical managers make informed strategic decisions in contexts of uncertainty.


Assuntos
Antineoplásicos Imunológicos/economia , Antineoplásicos Imunológicos/uso terapêutico , Orçamentos , Contratos/economia , Tomada de Decisões , Técnicas de Apoio para a Decisão , Aprovação de Drogas/economia , Custos de Medicamentos , Desenvolvimento de Medicamentos/economia , Negociação , Avaliação da Tecnologia Biomédica/economia , Antineoplásicos Imunológicos/efeitos adversos , Simulação por Computador , Análise Custo-Benefício , Alocação de Recursos para a Atenção à Saúde/economia , Humanos , Modelos Econômicos , Modelos Estatísticos , Formulação de Políticas , Resultado do Tratamento , Incerteza
15.
Transl Res ; 220: 182-190, 2020 06.
Artigo em Inglês | MEDLINE | ID: mdl-32165059

RESUMO

While antibiotics are a key infrastructure underpinning modern medicine, evolution will continue to undermine their effectiveness, requiring continuous investment to sustain antibiotic effectiveness. The antibiotic R&D ecosystem is in peril, moving towards collapse. Key stakeholders have identified pull incentives such as Market Entry Rewards or subscription models as the key long-term solution. If substantial Market Entry Rewards or other pull incentives become possible, there is every reason to expect that for-profit companies will return to the antibiotic field. However, the political and financial will to develop such Market Entry Rewards or other similar incentives may be difficult to muster in the timeframes needed to prevent further diminishment of antibiotic research and development, especially if large drug companies are seen as substantial beneficiaries of these taxpayer-funded pull incentives. Bridging solutions are required from private actors in the interim. This article explores potential solutions led by private actors, including (1) traditional for-profit companies; (2) non-profit enterprises; and (3) public benefit corporations with lower profit expectations, akin to a public utility. All face similar commercial struggles, but nonprofits and public benefit corporations can accept lower profit expectations and might be more politically attractive recipients of pull incentives.


Assuntos
Antibacterianos/economia , Desenvolvimento de Medicamentos/economia , Indústria Farmacêutica/economia , Comércio
16.
JAMA Netw Open ; 3(3): e201737, 2020 03 02.
Artigo em Inglês | MEDLINE | ID: mdl-32219405

RESUMO

Importance: Sickle cell disease (SCD) and cystic fibrosis (CF) are severe autosomal recessive disorders associated with intermittent disease exacerbations that require hospitalizations, progressive chronic organ injury, and substantial premature mortality. Research funding is a limited resource and may contribute to health care disparities, especially for rare diseases that disproportionally affect economically disadvantaged groups. Objective: To compare disease-specific funding between SCD and CF and the association between funding and research productivity. Design, Setting, and Participants: This cross-sectional study examined federal and foundation funding, publications indexed in PubMed, clinical trials registered in ClinicalTrials.gov, and new drug approvals from January 1, 2008, to December 31, 2018, in an estimated US population of approximately 90 000 individuals with SCD and approximately 30 000 individuals with CF. Main Outcomes and Measures: Federal and foundation funding, publications indexed in PubMed, clinical trial registrations, and new drug approvals. Results: From 2008 through 2018, federal funding was greater per person with CF compared with SCD (mean [SD], $2807 [$175] vs $812 [$147]; P < .001). Foundation expenditures were greater for CF than for SCD (mean [SD], $7690 [$3974] vs $102 [$13.7]; P < .001). Significantly more research articles (mean [SD], 1594 [225] vs 926 [157]; P < .001) and US Food and Drug Administration drug approvals (4 vs 1) were found for CF compared with SCD, but the total number of clinical trials was similar (mean [SD], 27.3 [6.9] vs 23.8 [6.3]; P = .22). Conclusions and Relevance: The findings show that disparities in funding between SCD and CF may be associated with decreased research productivity and novel drug development for SCD. Increased federal and foundation funding is needed for SCD and other diseases that disproportionately affect economically disadvantaged groups to address health care disparities.


Assuntos
Anemia Falciforme/economia , Pesquisa Biomédica , Fibrose Cística/economia , Apoio à Pesquisa como Assunto , Anemia Falciforme/epidemiologia , Pesquisa Biomédica/economia , Pesquisa Biomédica/estatística & dados numéricos , Estudos Transversais , Fibrose Cística/epidemiologia , Desenvolvimento de Medicamentos/economia , Desenvolvimento de Medicamentos/estatística & dados numéricos , Fundações , Humanos , Apoio à Pesquisa como Assunto/economia , Apoio à Pesquisa como Assunto/organização & administração , Estados Unidos
17.
Value Health ; 23(2): 139-150, 2020 02.
Artigo em Inglês | MEDLINE | ID: mdl-32113617

RESUMO

Healthcare resource allocation decisions made under conditions of uncertainty may turn out to be suboptimal. In a resource constrained system in which there is a fixed budget, these suboptimal decisions will result in health loss. Consequently, there may be value in reducing uncertainty, through the collection of new evidence, to make better resource allocation decisions. This value can be quantified using a value of information (VOI) analysis. This report, from the ISPOR VOI Task Force, introduces VOI analysis, defines key concepts and terminology, and outlines the role of VOI for supporting decision making, including the steps involved in undertaking and interpreting VOI analyses. The report is specifically aimed at those tasked with making decisions about the adoption of healthcare or the funding of healthcare research. The report provides a number of recommendations for good practice when planning, undertaking, or reviewing the results of VOI analyses.


Assuntos
Orçamentos , Tomada de Decisões , Técnicas de Apoio para a Decisão , Custos de Medicamentos , Desenvolvimento de Medicamentos/economia , Alocação de Recursos para a Atenção à Saúde/economia , Pesquisa sobre Serviços de Saúde/economia , Avaliação da Tecnologia Biomédica/economia , Redução de Custos , Análise Custo-Benefício , Humanos , Reembolso de Seguro de Saúde/economia , Modelos Econômicos , Modelos Estatísticos , Formulação de Políticas , Seguro de Saúde Baseado em Valor/economia , Aquisição Baseada em Valor/economia
18.
JAMA ; 323(9): 834-843, 2020 03 03.
Artigo em Inglês | MEDLINE | ID: mdl-32125401

RESUMO

Importance: Understanding the profitability of pharmaceutical companies is essential to formulating evidence-based policies to reduce drug costs while maintaining the industry's ability to innovate and provide essential medicines. Objective: To compare the profitability of large pharmaceutical companies with other large companies. Design, Setting, and Participants: This cross-sectional study compared the annual profits of 35 large pharmaceutical companies with 357 companies in the S&P 500 Index from 2000 to 2018 using information from annual financial reports. A statistically significant differential profit margin favoring pharmaceutical companies was evidence of greater profitability. Exposures: Large pharmaceutical vs nonpharmaceutical companies. Main Outcomes and Measures: The main outcomes were revenue and 3 measures of annual profit: gross profit (revenue minus the cost of goods sold); earnings before interest, taxes, depreciation, and amortization (EBITDA; pretax profit from core business activities); and net income, also referred to as earnings (difference between all revenues and expenses). Profit measures are described as cumulative for all companies from 2000 to 2018 or annual profit as a fraction of revenue (margin). Results: From 2000 to 2018, 35 large pharmaceutical companies reported cumulative revenue of $11.5 trillion, gross profit of $8.6 trillion, EBITDA of $3.7 trillion, and net income of $1.9 trillion, while 357 S&P 500 companies reported cumulative revenue of $130.5 trillion, gross profit of $42.1 trillion, EBITDA of $22.8 trillion, and net income of $9.4 trillion. In bivariable regression models, the median annual profit margins of pharmaceutical companies were significantly greater than those of S&P 500 companies (gross profit margin: 76.5% vs 37.4%; difference, 39.1% [95% CI, 32.5%-45.7%]; P < .001; EBITDA margin: 29.4% vs 19%; difference, 10.4% [95% CI, 7.1%-13.7%]; P < .001; net income margin: 13.8% vs 7.7%; difference, 6.1% [95% CI, 2.5%-9.7%]; P < .001). The differences were smaller in regression models controlling for company size and year and when considering only companies reporting research and development expense (gross profit margin: difference, 30.5% [95% CI, 20.9%-40.1%]; P < .001; EBITDA margin: difference, 9.2% [95% CI, 5.2%-13.2%]; P < .001; net income margin: difference, 3.6% [95% CI, 0.011%-7.2%]; P = .05). Conclusions and Relevance: From 2000 to 2018, the profitability of large pharmaceutical companies was significantly greater than other large, public companies, but the difference was less pronounced when considering company size, year, or research and development expense. Data on the profitability of large pharmaceutical companies may be relevant to formulating evidence-based policies to make medicines more affordable.


Assuntos
Comércio/economia , Indústria Farmacêutica/economia , Renda/estatística & dados numéricos , Gastos de Capital/estatística & dados numéricos , Estudos Transversais , Custos de Medicamentos , Desenvolvimento de Medicamentos/economia , Indústria Farmacêutica/estatística & dados numéricos , Análise de Regressão , Tecnologia/economia , Estados Unidos
19.
JAMA ; 323(9): 844-853, 2020 Mar 03.
Artigo em Inglês | MEDLINE | ID: mdl-32125404

RESUMO

Importance: The mean cost of developing a new drug has been the subject of debate, with recent estimates ranging from $314 million to $2.8 billion. Objective: To estimate the research and development investment required to bring a new therapeutic agent to market, using publicly available data. Design and Setting: Data were analyzed on new therapeutic agents approved by the US Food and Drug Administration (FDA) between 2009 and 2018 to estimate the research and development expenditure required to bring a new medicine to market. Data were accessed from the US Securities and Exchange Commission, Drugs@FDA database, and ClinicalTrials.gov, alongside published data on clinical trial success rates. Exposures: Conduct of preclinical and clinical studies of new therapeutic agents. Main Outcomes and Measures: Median and mean research and development spending on new therapeutic agents approved by the FDA, capitalized at a real cost of capital rate (the required rate of return for an investor) of 10.5% per year, with bootstrapped CIs. All amounts were reported in 2018 US dollars. Results: The FDA approved 355 new drugs and biologics over the study period. Research and development expenditures were available for 63 (18%) products, developed by 47 different companies. After accounting for the costs of failed trials, the median capitalized research and development investment to bring a new drug to market was estimated at $985.3 million (95% CI, $683.6 million-$1228.9 million), and the mean investment was estimated at $1335.9 million (95% CI, $1042.5 million-$1637.5 million) in the base case analysis. Median estimates by therapeutic area (for areas with ≥5 drugs) ranged from $765.9 million (95% CI, $323.0 million-$1473.5 million) for nervous system agents to $2771.6 million (95% CI, $2051.8 million-$5366.2 million) for antineoplastic and immunomodulating agents. Data were mainly accessible for smaller firms, orphan drugs, products in certain therapeutic areas, first-in-class drugs, therapeutic agents that received accelerated approval, and products approved between 2014 and 2018. Results varied in sensitivity analyses using different estimates of clinical trial success rates, preclinical expenditures, and cost of capital. Conclusions and Relevance: This study provides an estimate of research and development costs for new therapeutic agents based on publicly available data. Differences from previous studies may reflect the spectrum of products analyzed, the restricted availability of data in the public domain, and differences in underlying assumptions in the cost calculations.


Assuntos
Desenvolvimento de Medicamentos/economia , Indústria Farmacêutica/economia , Pesquisa Farmacêutica/economia , Custos e Análise de Custo/estatística & dados numéricos , Custos de Medicamentos , Indústria Farmacêutica/estatística & dados numéricos , Estados Unidos , United States Food and Drug Administration
SELEÇÃO DE REFERÊNCIAS
DETALHE DA PESQUISA