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2.
Front Pharmacol ; 10: 144, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-30906258

RESUMO

A host of challenges confront healthcare authorities worldwide. Topping the list is the demand for innovative new medicines to treat a range of both infectious and non-communicable diseases, while containing spiraling healthcare costs. The challenge is particularly great in therapeutic areas where, despite significant medical need and economic impact, the technical challenges and commercial risk of development serve as disincentives to drug sponsors. These areas include cardiovascular diseases as well as diseases and disorders of the central nervous system. Currently, the development and approval of new active substances, with its disproportionate focus on oncology, is not in alignment with healthcare needs in most geographic regions. In this article, we discuss the origins of this misalignment and suggest various approaches to address healthcare needs going forward.

3.
Clin Pharmacol Ther ; 105(4): 857-866, 2019 04.
Artigo em Inglês | MEDLINE | ID: mdl-30610746

RESUMO

Efficacy trials, designed to gain regulatory marketing approval, evaluate drugs in optimally selected patients under advantageous conditions for relatively short time periods. Effectiveness trials, designed to evaluate use in usual practice, assess treatments among more typical patients in real-world conditions with longer follow-up periods. In "efficacy-to-effectiveness (E2E) trials," if the initial efficacy trial component is positive, the trial seamlessly transitions to an effectiveness trial component to efficiently yield both types of evidence. Yet more time could be saved by simultaneously addressing efficacy and effectiveness in an "efficacy and effectiveness too (EE2) trial." Additionally, hybrids of the E2E and EE2 approaches with differing degrees of overlap of the two components could allow flexibility for specific drug development needs. In planning EE2 trials, each stakeholder's current and future needs, incentives, and perspective must be considered. Although challenging, the ultimate benefits to stakeholders, the health system, and the public should justify this effort.


Assuntos
Ensaios Clínicos como Assunto/legislação & jurisprudência , Aprovação de Drogas/legislação & jurisprudência , Desenvolvimento de Medicamentos/legislação & jurisprudência , Projetos de Pesquisa/legislação & jurisprudência , Análise Custo-Benefício/legislação & jurisprudência , Humanos , Marketing/legislação & jurisprudência , Seleção de Pacientes , Resultado do Tratamento
6.
Trans Am Clin Climatol Assoc ; 129: 279-300, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-30166723

RESUMO

Efficacy trials, which assess treatments in optimally selected patients under advantageous conditions for relatively short time periods, are necessary to gain regulatory approval for marketing. In contrast, effectiveness trials, which test treatments across a spectrum of patients in real-world conditions with follow-up periods that match typical treatment regimens, provide critical information on drug effects in those patients who may ultimately receive the treatment. We previously proposed a study design that integrates efficacy and effectiveness trials into a 2-component "efficacy-to-effectiveness (E2E) trial," in which if the initial efficacy trial component is positive, then the trial immediately and seamlessly transitions to the effectiveness trial component. However, we believe that total study duration could be even further shortened by simultaneously addressing efficacy and effectiveness too (EE2). An EE2 trial rigorously demonstrates efficacy, but uses broad inclusion characteristics of effectiveness trials. An example of a study using EE2 design, the IMMEDIATE (Immediate Myocardial Metabolic enhancement During Initial Assessment and Treatment in Emergency Care) trial, is provided.


Assuntos
Ensaios Clínicos como Assunto/métodos , Aprovação de Drogas , Determinação de Ponto Final , Projetos de Pesquisa , Síndrome Coronariana Aguda/sangue , Síndrome Coronariana Aguda/diagnóstico , Síndrome Coronariana Aguda/tratamento farmacológico , Administração Intravenosa , Esquema de Medicação , Glucose/administração & dosagem , Humanos , Insulina/administração & dosagem , Potássio/administração & dosagem , Fatores de Tempo , Resultado do Tratamento
7.
Clin Ther ; 40(7): 1056-1059, 2018 07.
Artigo em Inglês | MEDLINE | ID: mdl-30006070
8.
Clin Ther ; 39(7): 1409-1425.e20, 2017 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-28652015

RESUMO

PURPOSE: This study examines the complete timelines of translational science for new cardiovascular therapeutics from the initiation of basic research leading to identification of new drug targets through clinical development and US Food and Drug Administration (FDA) approval of new molecular entities (NMEs) based on this research. METHODS: This work extends previous studies by examining the association between the growth of research on drug targets and approval of NMEs associated with these targets. Drawing on research on innovation in other technology sectors, where technological maturity is an important determinant in the success or failure of new product development, an analytical model was used to characterize the growth of research related to the known targets for all 168 approved cardiovascular therapeutics. FINDINGS: Categorizing and mapping the technological maturity of cardiovascular therapeutics reveal that (1) there has been a distinct transition from phenotypic to targeted methods for drug discovery, (2) the durations of clinical and regulatory processes were significantly influenced by changes in FDA practice, and (3) the longest phase of the translational process was the time required for technology to advance from initiation of research to a statistically defined established point of technology maturation (mean, 30.8 years). IMPLICATIONS: This work reveals a normative association between metrics of research maturation and approval of new cardiovascular therapeutics and suggests strategies for advancing translational science by accelerating basic and applied research and improving the synchrony between the maturation of this research and drug development initiatives.


Assuntos
Fármacos Cardiovasculares , Aprovação de Drogas , Descoberta de Drogas , Humanos , Pesquisa Médica Translacional , Estados Unidos , United States Food and Drug Administration
12.
Am J Ther ; 22(2): 117-24, 2015.
Artigo em Inglês | MEDLINE | ID: mdl-23429165

RESUMO

Although most research professionals believe that protocol designs contain a growing number of unnecessary and redundant procedures generating unused data, incurring high cost, and jeopardizing study success, there are no published studies systematically examining this issue. Between November 2011 and May 2012, Tufts Center for the Study of Drug Development conducted a study among a working group of 15 pharmaceutical companies in which a total of 25,103 individual protocol procedures were evaluated and classified using clinical study reports and analysis plans. The results show that the typical later-stage protocol had an average of 7 objectives and 13 end points of which 53.8% are supplementary. One (24.7%) of every 4 procedures performed per phase-III protocol and 17.7% of all phase-II procedures per protocol were classified as "Noncore" in that they supported supplemental secondary, tertiary, and exploratory end points. For phase-III protocols, 23.6% of all procedures supported regulatory compliance requirements and 15.9% supported those for phase-II protocols. The study also found that on average, $1.7 million (18.5% of the total) is spent in direct costs to administer Noncore procedures per phase-III protocol and $0.3 million (13.1% of the total) in direct costs are spent on Noncore procedures for each phase-II protocol. Based on the results of this study, the total direct cost to perform Noncore procedures for all active annual phase-II and phase-III protocols is conservatively estimated at $3.7 billion annually, not including the indirect costs associated with collecting and managing Noncore procedure data and the ethical costs of exposing study volunteers to unnecessary risks associated with conducting extraneous procedures.


Assuntos
Ensaios Clínicos Fase II como Assunto/métodos , Ensaios Clínicos Fase III como Assunto/métodos , Indústria Farmacêutica/métodos , Ensaios Clínicos Fase II como Assunto/economia , Ensaios Clínicos Fase III como Assunto/economia , Coleta de Dados/economia , Coleta de Dados/métodos , Indústria Farmacêutica/economia , Humanos , Projetos de Pesquisa
13.
Clin Ther ; 36(10): 1349-55, 2014 Oct 01.
Artigo em Inglês | MEDLINE | ID: mdl-25444564

RESUMO

PURPOSE: Articles in peer-reviewed journals and the trade press presuppose that strategic outsourcing relationships have been formed to replace preexisting collaborative approaches with contract research organizations. They do not consider that large, fragmented pharmaceutical and biotechnology companies may be supporting competing and conflicting relationship models simultaneously. A recent Tufts Center for the Study of Drug Development study quantifies actual strategic outsourcing practices among drug development companies and sheds new light on why these relationships may be failing. METHODS: Tufts Center for the Study of Drug Development conducted an in-depth assessment of 43 Phase II and III clinical studies completed since 2012 to examine the outsourcing relationships used by 9 major pharmaceutical and biotechnology companies to support key functional areas. Descriptive statistics were assessed and t tests were performed to characterize outsourcing practices by function and to determine differences in study performance between transactional and strategic outsourcing relationships. FINDINGS: The results indicate that sponsor companies are using a variety of outsourcing relationship models to support their studies, mixing and matching the use of internal staff, and using traditional transactional and strategic outsourcing relationships simultaneously. Specifically, despite the fact that each sponsor company had entered into several strategic outsourcing relationships, in no instance did a single contract research organization manage all functional areas supporting an individual Phase II or III study. In addition, sponsor companies vary the types of outsourcing relationship models that they use on a study-by-study basis. IMPLICATIONS: The inability of pharmaceutical and biotechnology companies to consistently embrace and coordinate sourcing strategies is creating internal friction and inefficiency. As a result, the expected impact of strategic outsourcing relationships on drug development performance, quality, and cost remains elusive.


Assuntos
Biotecnologia/organização & administração , Indústria Farmacêutica/organização & administração , Eficiência Organizacional , Serviços Terceirizados , Ensaios Clínicos Fase II como Assunto , Ensaios Clínicos Fase III como Assunto , Comportamento Cooperativo , Humanos
14.
15.
Am J Ther ; 21(6): 442-6, 2014.
Artigo em Inglês | MEDLINE | ID: mdl-23011177

RESUMO

The Food and Drug Administration's MedWatch system--a voluntary surveillance program--received 600,000 adverse event reports on marketed drugs and devices in 2011. The Food and Drug Administration credits the MedWatch system with improving awareness, and expediting early detection, of drug and device risks and in illuminating the adoption of medical treatments. Reporting bias has been acknowledged as a limitation of the MedWatch system. No systematic assessment of the accuracy and completeness of adverse event reporting has been conducted, yet inaccurate adverse event reporting may lead drug safety professionals to draw incorrect conclusions, manufacturers may be wrongly forced to suspend and withdraw medications and interventions, health professionals may mistakenly alter their clinical practices, and patients may be denied safe and effective treatments. In 2011, the Tufts Center for the Study of Drug Development gathered and analyzed 10.2 million adverse event reports filed with the MedWatch system. Patient information was generally complete and accurate. Suspect product information, on the other hand, showed high levels of incomplete and inaccurate data. Start and end dates of suspect product use had 37% and 23% completion rates, respectively. Dosage level was completed only 31% of the time, and product lot numbers had only a 9% completion rate. More than 25% of the names of reported suspect products were inaccurate, and 31% of suspect product start dates were inaccurate. Higher levels of completion and accuracy were associated with reports filed closer to the date when the adverse event was observed. Implications of the results and suggested improvements are discussed.


Assuntos
Sistemas de Notificação de Reações Adversas a Medicamentos/normas , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/epidemiologia , Vigilância de Produtos Comercializados/métodos , Viés , Humanos , Vigilância de Produtos Comercializados/normas , Estados Unidos , United States Food and Drug Administration
16.
Ther Innov Regul Sci ; 47(6): 651-655, 2013 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-30235547

RESUMO

Pharmaceutical and biotechnology companies are actively seeking ways to optimize protocol design. An emerging approach has been the creation of a new governance mechanism designed to evaluate protocol feasibility and simplify design before final protocol approval. In late 2012, the Tufts Center for the Study of Drug Development conducted a qualitative in-depth assessment of 10 major pharmaceutical companies that have implemented these new governance mechanisms since 2009. Detailed profiles of each company's feasibility review mechanism are discussed including committee missions and objectives, positioning, composition, staffing models, reporting flow, implementation challenges, and measured impacts to date.

17.
Oncologist ; 18(1): 104-11, 2013.
Artigo em Inglês | MEDLINE | ID: mdl-23263289

RESUMO

BACKGROUND: Regulatory approval of oncology drugs is the cornerstone of the development process and approval characteristics shape eventual utilization. Approval trends and characteristics provide valuable information for drug developers and regulators and ultimately affect clinicians and patients. METHODS: Indication characteristics were tabulated for drugs approved by the U.S. Food and Drug Administration (FDA) for systemic therapy of malignancies from 1949 through October 2011. Variables included time to approval, initial/supplemental indication, tumor type, stage of disease, specification of protein expression or genetic information, drug class, trial design, concomitant agent, trial size, and endpoint. RESULTS: A total of 121 unique anticancer agents, including 242 unique indications, were approved. The number of trials for each indication has decreased; however, trial size has increased and more randomized controlled trials have been performed. Trial designs have increasingly used time-to-event endpoints and rarely have used symptom-based primary endpoints. Approvals have been primarily single agent, with less emphasis on palliative treatments and increasing emphasis on advanced disease stages and requirements for prior therapy. Molecular specifications in labels have increased, but they are present in less than 30% of recent indications and are not associated with shorter approval times. CONCLUSION: Approval of oncology agents is occurring in increasingly more challenging settings, suggesting gaps between eventual practice and development in potentially suboptimal indications. Molecular specifications promise to enhance development, yet widespread use in label indications has not yet been achieved.


Assuntos
Anticarcinógenos/uso terapêutico , Aprovação de Drogas/legislação & jurisprudência , Descoberta de Drogas , Neoplasias/tratamento farmacológico , Humanos , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência
19.
J Investig Med ; 60(7): 995-8, 2012 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-22918200

RESUMO

This article addresses current challenges facing pharmaceutical and biopharmaceutical developers, including the expiration of patents on many high-revenue-generating products, increasing competition in the marketplace, low public support, high regulatory hurdles, and the increasing time, cost, and risk of new product development. To meet these challenges, drug developers are looking to new models of innovation to improve efficiency, lower risk, and increase output. These new models include codevelopment agreements with small companies, multicompany consortia, and strategic partnerships with academic research centers. In the United States and the European Union, the government is supporting these efforts by creating incentives for academic centers to foster translational research and become more "commercially minded". The goal for all stakeholders is to reduce the barriers to product development and bring new medicines to market in a timely and cost-efficient manner.


Assuntos
Pesquisa Médica Translacional/organização & administração , Academias e Institutos , Comportamento Cooperativo , Indústria Farmacêutica/economia , Indústria Farmacêutica/organização & administração , Inovação Organizacional/economia , Fatores de Tempo , Pesquisa Médica Translacional/economia
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