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1.
AAPS PharmSciTech ; 22(3): 128, 2021 Apr 09.
Artigo em Inglês | MEDLINE | ID: mdl-33835304

RESUMO

The adoption of Quality by Design (QbD) and Analytical Method Lifecycle Management (AMLM) concepts to ensure the quality of pharmaceutical products has been applied and proposed over the last few years. These concepts are based on knowledge gained from the application of scientific and quality risk management approaches, throughout method lifecycle to assure continuous improvement and high reliability of analytical results. The overall AMLM starts with the definition of the method's intended use through the Analytical Target Profile definition, including three stages: (1) Method Design, taking advantage of the well-known concept of QbD; (2) Method Performance Qualification; (3) Continued Method Performance Verification. This is intended to holistically align method variability with product requirements, increasing confidence in the data generated, a regulatory requirement that the pharmaceutical industry must follow. This approach views all method-related activities, such as development, validation, transfer, and routine use as a continuum and interrelated process, where knowledge and risk management are the key enablers. An increase in method robustness, cost reduction, and decreased risk failures are some of the intrinsic benefits from this lifecycle management. This approach is clearly acknowledged both by regulators and industry. The roadmap of the regulatory and industry events that mark the evolution of these concepts helps to capture the current and future expectation of the pharmaceutical framework.


Assuntos
Indústria Farmacêutica/normas , Preparações Farmacêuticas/análise , Química Farmacêutica , Desenho de Fármacos , Indústria Farmacêutica/tendências , Humanos , Controle de Qualidade
2.
AAPS PharmSciTech ; 22(3): 82, 2021 Feb 23.
Artigo em Inglês | MEDLINE | ID: mdl-33624199

RESUMO

Current trends in the pharmaceutical industry led to a demand for more flexible manufacturing processes with smaller batch sizes. Prepackaged nested vials that can be processed as a unit were introduced into the market to fulfill this need. However, vial nests provide a different thermal environment for the vials compared to a hexagonal packaging array and could therefore influence product temperature profiles, primary drying times, and product quality attributes. Polymer caps with the possibility of vial closure inside the freeze-drying chamber were developed to remove the risks and need of a crimping process. A general concern with the use of such caps is the possibility of an increase in resistance to water vapor flow out of the vial. This case study investigated the effect of the LyoSeal® and PLASCAP® polymer caps and EZ-fill® nests on the freeze-drying process. Amorphous and partially crystalline model formulations were freeze-dried. Process data and product quality attributes were compared for regularly stoppered vials and vials with polymer caps as well as vials in a hexagonal packaging array and nested vials. The results indicated no increased resistance or impeded water vapor flow by the polymer caps. Differences in the macro- and microscopic appearances of products and a trend towards lower product temperatures were observed for the investigated nest type compared to a regular hexagonal packaging array. Consequently, the polymer caps could be used as an alternative to regular stoppers without affecting freeze-drying process data or product quality attributes, while the different thermal environment of nested vials should be considered.


Assuntos
Indústria Farmacêutica/normas , Embalagem de Medicamentos/normas , Polímeros/normas , Dessecação/métodos , Indústria Farmacêutica/métodos , Embalagem de Medicamentos/métodos , Liofilização/métodos , Liofilização/normas , Temperatura
3.
Biochem Biophys Res Commun ; 545: 145-149, 2021 03 19.
Artigo em Inglês | MEDLINE | ID: mdl-33550095

RESUMO

In March 2013 it was reported by the World Health Organization (WHO) the first cases of human infections with avian influenza virus A (H7N9). From 2013 to December 2019, 1568 cases have been reported with 616 deaths. H7N9 infection has been associated with high morbidity and mortality rates, and vaccination is currently the most effective way to prevent infections and consequently flu-related severe illness. Developing and producing vaccines against pandemic influenza viruses is the main strategy for a response to a possible pandemic. This study aims to present the production of three industrial lots under current Good Manufacturing Practices (cGMP) of the active antigen used to produce the pandemic influenza vaccine candidate against A(H7N9). These batches were characterized and evaluated for quality standards and tested for immunogenicity in mice. The average yield was 173.50 ± 7.88 µg/mL of hemagglutinin and all the preparations met all the required specifications. The formulated H7N9 vaccine is poorly immunogenic and needs to be adjuvanted with an oil in water emulsion adjuvant (IB160) to achieve a best immune response, in a prime and in a boost scheme. These data are important for initial production planning and preparedness in the case of a H7N9 pandemic.


Assuntos
Subtipo H7N9 do Vírus da Influenza A/imunologia , Vacinas contra Influenza/biossíntese , Influenza Humana/prevenção & controle , Pandemias/prevenção & controle , Animais , Antígenos Virais/biossíntese , Antígenos Virais/imunologia , Composição de Medicamentos/métodos , Composição de Medicamentos/estatística & dados numéricos , Indústria Farmacêutica/normas , Feminino , Humanos , Vacinas contra Influenza/imunologia , Vacinas contra Influenza/isolamento & purificação , Influenza Humana/imunologia , Influenza Humana/virologia , Camundongos , Camundongos Endogâmicos BALB C , Vacinas de Produtos Inativados/biossíntese , Vacinas de Produtos Inativados/imunologia , Vacinas de Produtos Inativados/isolamento & purificação
4.
Drug Saf ; 44(1): 95-105, 2021 01.
Artigo em Inglês | MEDLINE | ID: mdl-33354753

RESUMO

INTRODUCTION: Evidence-based clinical data on coronavirus disease 2019 (COVID-19) pharmacotherapies are scarce. OBJECTIVE: This study documented and characterized COVID-19 cases reported in individuals receiving treatment with Pfizer pharmaceutical products and cases that reported use of Pfizer pharmaceutical products for COVID-19 treatment. METHODS: This retrospective observational review leveraged the Pfizer safety database containing adverse event data collected in association with use of Pfizer products between 1 October, 2019, and 25 June, 2020; the database includes worldwide adverse event data from various sources. Selected Medical Dictionary for Drug Regulatory Activities (MedDRA®) Preferred Terms and subsequent clinical review were used to characterize COVID-19 cases. RESULTS: Over 1500 relevant cases were identified over an 8-month period. In cases that reported COVID-19, immunosuppressant/immunomodulating agents, followed by anticoagulant/antithrombic agents and corticosteroids, were the most frequently reported agents. The frequent reporting of immunosuppressant/immunomodulating agents among cases of COVID-19 suggests increased vulnerability to infection among treated patients, either because of immunosuppressive effects of certain agents or the nature of the underlying treated condition. In cases involving off-label pharmacotherapy use for the treatment of COVID-19-related conditions, the most frequently reported therapeutic classes included antibiotics, antimalarial agents, antivirals/antiretroviral agents, immunosuppressant/immunomodulating agents, corticosteroids, anticoagulants, and immunoglobulin/interferons. The most frequently reported pharmacotherapeutic agents were azithromycin and chloroquine/hydroxychloroquine, followed by lopinavir-ritonavir, ceftriaxone, and tofacitinib. The most frequently reported clinical adverse events associated with azithromycin (as sole therapy or combined with chloroquine/hydroxychloroquine) include electrocardiogram QT prolonged, drug interaction, hepatitis, diarrhea, and hepatitis acute. Regarding cardiac-related events, 19% (120/645) of azithromycin cases reported events associated with QT prolongation/torsade de pointes (which included seven fatal cardiac events). The most frequently reported clinical adverse events associated with other commonly used agents are also presented. CONCLUSIONS: This pharmacovigilance surveillance study provides a unique characterization of cases in which a broad range of pharmaceutical products was reported in relation to COVID-19.


Assuntos
Sistemas de Notificação de Reações Adversas a Medicamentos/tendências , COVID-19/epidemiologia , Indústria Farmacêutica/tendências , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/epidemiologia , Saúde Global/tendências , Farmacovigilância , Sistemas de Notificação de Reações Adversas a Medicamentos/normas , Anticoagulantes/efeitos adversos , Antimaláricos/efeitos adversos , Antivirais/efeitos adversos , COVID-19/tratamento farmacológico , Bases de Dados Factuais/normas , Bases de Dados Factuais/tendências , Indústria Farmacêutica/normas , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/diagnóstico , Saúde Global/normas , Humanos , Imunossupressores/efeitos adversos , Estudos Retrospectivos
8.
PDA J Pharm Sci Technol ; 74(4): 468-494, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32467176

RESUMO

This article reviews currently available scientific literature related to the epidemiology, infectivity, survival, and susceptibility to disinfectants of Coronaviruses, in the context of the controls established to meet good manufacturing practice (GMP) regulations and guidance, and the public health guidance issued specifically to combat the COVID-19 pandemic. The possible impact of the COVID-19 pandemic on the pharmaceutical supply chain is assessed and recommendations are listed for risk mitigation steps to minimize supply disruption to pharmaceutical drug products. Areas addressed include a brief history of the COVID-19 viral pandemic, a description of the virus, the regulatory response to the pandemic, the screening of employees, the persistence of the virus on inanimate surfaces, cleaning and disinfection of manufacturing facilities, the use of GMP-mandated personal protective equipment to counter the spread of the disease, the role of air changes in viral clearance, and approaches to risk assessment and mitigation. Biological medicinal products have a great record of safety, yet the cell cultures used for production can be susceptible to viruses, and contamination events have occurred. Studies on SARS-CoV-2 for it ability to replicate in various mammalian cell lines used for biopharmaceutical manufacturing suggests that the virus poses a low risk and any contamination would be detected by currently used adventitious virus testing. The consequences of the potential virus exposure of manufacturing processes as well as the effectiveness of mitigation efforts are discussed. The pharmaceutical supply chain is complex, traversing many geographies and companies that range from large multinationals to mid- and small-size operations. This paper recommends practices that can be adopted by all companies, irrespective of their size, geographic location, or position in the supply chain.


Assuntos
Infecções por Coronavirus/prevenção & controle , Desinfecção/métodos , Pandemias/prevenção & controle , Preparações Farmacêuticas/provisão & distribuição , Pneumonia Viral/prevenção & controle , Animais , Betacoronavirus/isolamento & purificação , Infecções por Coronavirus/epidemiologia , Infecções por Coronavirus/virologia , Desinfetantes/química , Contaminação de Medicamentos/prevenção & controle , Indústria Farmacêutica/normas , Contaminação de Equipamentos/prevenção & controle , Humanos , Preparações Farmacêuticas/normas , Pneumonia Viral/epidemiologia , Pneumonia Viral/virologia , Gestão de Riscos/métodos , Replicação Viral
9.
Eur J Cancer ; 132: 100-103, 2020 06.
Artigo em Inglês | MEDLINE | ID: mdl-32335476

RESUMO

Direct-to-consumer (DTC) commercial companies offer genetic tests that are presented as allowing individuals the opportunity to increase their capacities to be in charge of their own healthcare managements. DTC companies deny performing medical tests, yet they provide data based on sequencing multigene panel or whole exome. This contradiction allows these companies to escape the requirements of a regulated medical practice that guarantees the quality of the tests, as well as the information and support for tested individuals. Herein, we illustrate the lack of such requirements by analysing the bad experience of a young man who dealt with DTC health genetic testing companies. There is an emergency for DTC testing to be either deprived of any medically relevant information, or carried out in a legally regulated medical framework.


Assuntos
Erros de Diagnóstico/prevenção & controle , Triagem e Testes Direto ao Consumidor/normas , Indústria Farmacêutica/normas , Testes Genéticos/normas , Disseminação de Informação/ética , Neoplasias/diagnóstico , Análise de Sequência de DNA/normas , Adulto , Triagem e Testes Direto ao Consumidor/legislação & jurisprudência , Indústria Farmacêutica/legislação & jurisprudência , Testes Genéticos/legislação & jurisprudência , Genoma Humano , Humanos , Achados Incidentais , Disseminação de Informação/legislação & jurisprudência , Masculino , Neoplasias/genética , Fatores de Risco
10.
Cardiovasc Drugs Ther ; 34(4): 579-584, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32318933

RESUMO

PURPOSE: Beginning in July of 2018, the FDA issued a voluntary recall regarding the presence of a contaminant found in the manufacturing of valsartan. What would ensue has become a largely unprecedented sequence of alarming events since the FDA began reporting public recalls, withdrawals and safety alerts on their website in 2016. Since then, the United States has been significantly impacted by drug recalls affecting angiotensin receptor blockers. This report arms clinicians with additional guidance and provides a framework for responding appropriately to future similar incidents and includes an overview of the angiotensin receptor blockers, and their effects and safety profiles. METHODS: This report includes a review of data from all pertinent clinical and scientific sources including information from the FDA's inspection documents and recall website. Additional information is provided on the specific bottles including all lot numbers, expiration dates, etc. RESULTS: The recalls/withdrawals are attributable to the presence of cancer-causing contaminants identified during the manufacturing process from drug manufacturers abroad. The root causes behind the recalls and subsequent shortage appear multifactorial, and stem to a certain extent from the outsourcing of medication manufacturing overseas and lack of quality checks and appropriate oversight. CONCLUSIONS: This inherent issue is not likely to resolve soon and has eroded the public trust of/in the healthcare system and the pharmaceutical industry. Patients and healthcare providers are significantly affected and should have a full understanding of the matter in order to guide appropriate response and actions.


Assuntos
Bloqueadores do Receptor Tipo 1 de Angiotensina II/análise , Carcinógenos/análise , Aprovação de Drogas , Contaminação de Medicamentos , Indústria Farmacêutica/normas , Recall de Medicamento , Controle de Qualidade , United States Food and Drug Administration/normas , Bloqueadores do Receptor Tipo 1 de Angiotensina II/efeitos adversos , Humanos , Segurança do Paciente , Medição de Risco , Fatores de Risco , Estados Unidos
12.
Biologicals ; 63: 97-100, 2020 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-31836276

RESUMO

Comparability is a key concept in the evaluation of both manufacturing changes and biosimilars. It constitutes a pragmatic and flexible approach which recognises that biologicals are inherently variable and that minor variations in quality attributes are often clinically irrelevant. In this discussion paper, we argue that comparability exercises rely on a number of pragmatic criteria. These criteria have been remarkably robust for 20 years of comparability exercises; however, the increased scrutiny of biosimilar applications provides an impetus for both codification and improvement of criteria for establishing comparability. Such a more rigorous, methodologically sound, approach towards comparability seems both feasible and beneficial.


Assuntos
Medicamentos Biossimilares/normas , Indústria Farmacêutica/normas
13.
Clin Ter ; 170(1): e66-e73, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-31850487

RESUMO

BACKGROUND: Cross-contamination and mix-ups are among the problems which could have a negative impact on the quality of the finished product during the production of highly active or sensitizing drugs with campaign manufacturing. Standardised, validated procedures ensure quality standards are maintained during production. In spite of this, the operating conditions and applicability of methods adopted by the various regulatory agencies manifest significant differences which could consequently compromise the safety of the finished product. This work has analysed and compared the GMP of various Regulatory Agencies to examine issues connected to campaign manufacturing highly active or sensitizing drugs. METHODS: The GMP of the following Regulatory Agencies have been studied: EMA, CFDA, COFEPRIS, FDA, Health Canada, ANVISA, CDSCO, PIC/S and WHO. The study was carried out for the purpose of understanding which agencies consent to the use of campaign manufacturing for the following categories of medicinal products: hormones, immunosuppressants, cytotoxic agents, highly active pharmaceutical ingredients (APIs), biological preparations, steroids, sensitizing pharmaceutical materials, antibiotics, cephalosporins, penicillins, carbapenems and beta-lactam derivatives. RESULTS: The GMP of Health Canada, EMA, PIC/S and FDA show a number of similarities, starting with the fact that they allow campaign manufacturing for similar categories of pharmaceutical products after an appropriate risk evaluation has been performed. CFDA, WHO, ANVISA authorise campaign manufacturing in "exceptional circumstances", though they do not always define what they mean by this. COFEPRIS authorises campaign manufacturing for certain classes of drugs, while there is no mention of campaign manufacturing in the CDSCO regulations. CONCLUSIONS: Quite a few significant differences have been found in the various regulations concerning the use of campaign manufacturing and the classes of drugs that can be produced with this method. In the light of this, it is obvious that efforts to harmonise legislation internationally have not yet been successful: currently, states can adopt different quality standards. The pharmaceutical industry could use this situation to its advantage by delocalising production on the basis of existing standards. The need to harmonise GMPs is a priority which must be achieved as soon as possible.


Assuntos
Indústria Farmacêutica/legislação & jurisprudência , Indústria Farmacêutica/normas , Saúde Global/legislação & jurisprudência , Saúde Global/normas , Guias como Assunto , Medicamentos sob Prescrição/normas , Controle de Qualidade , Humanos
14.
Aliment Pharmacol Ther ; 51(1): 90-109, 2020 01.
Artigo em Inglês | MEDLINE | ID: mdl-31762074

RESUMO

BACKGROUND: Improved knowledge of the molecular pathophysiology and immunopathogenesis of cholestatic liver diseases in recent years has led to an increased interest in developing novel therapies. Patients with cholestatic liver disease often require different approaches to assessment and management of suspected drug-induced liver injury (DILI) compared to those with healthy livers and those with parenchymal liver diseases. At present, there are no regulatory guidelines or society position papers, that systematically address best practices pertaining to detection of DILI in these patients. AIMS: To outline best practices for detection, assessment and management of suspected acute DILI during clinical trials in adults with the cholestatic liver diseases - Primary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC). METHODS: This is one of the several papers developed by the IQ DILI Initiative, which is comprised of members from 16 pharmaceutical companies, in collaboration with DILI experts from academia and regulatory agencies. The contents are the result of an extensive literature review, as well as in-depth discussions among industry, regulatory and academic DILI experts, to achieve consensus recommendations on DILI-related issues occurring during clinical trials for cholestatic liver diseases. RESULTS: Recommended best practices are outlined pertaining to hepatic eligibility criteria, monitoring of liver tests, approach to a suspected DILI signal, and hepatic discontinuation rules. CONCLUSIONS: This paper provides a framework for the approach to detection, assessment and management of suspected acute DILI occurring during clinical trials in adults with cholestatic liver disease.


Assuntos
Doença Hepática Induzida por Substâncias e Drogas/diagnóstico , Doença Hepática Induzida por Substâncias e Drogas/terapia , Colestase/tratamento farmacológico , Ensaios Clínicos como Assunto , Consenso , Cirrose Hepática Biliar/tratamento farmacológico , Adulto , Doença Hepática Induzida por Substâncias e Drogas/patologia , Colestase/patologia , Doença Crônica , Ensaios Clínicos como Assunto/estatística & dados numéricos , Indústria Farmacêutica/organização & administração , Indústria Farmacêutica/normas , Humanos , Fígado/efeitos dos fármacos , Fígado/patologia , Fígado/fisiopatologia , Cirrose Hepática Biliar/patologia , Testes de Função Hepática , Sociedades Farmacêuticas/normas
15.
Eur J Pharm Biopharm ; 147: 19-37, 2020 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-31862299

RESUMO

Quality by Design (QbD) was originated in the broad domain of Quality Management and was recently adapted and formalized in specific terms for assisting pharmaceutical companies efforts towards market and operational excellence. However, despite some impressive success stories, the pharmaceutical industry have not yet fully embraced QbD, particularly in routine commercial manufacturing (Rantanen and Khinast, 2015; Puñal Peces et al., 2016). In this review, we aim to analyse the current state of implementation of QbD methodologies and tools in the pharmaceutical industry, extracting patterns and trends and identifying gaps and opportunities that may be considered to improve QbD adoption. For this purpose, a critical analysis of 60 research papers was performed, whose contents were classified, compared and summarized at different abstraction levels. Our analysis reveals the following tools as the frequently adopted for conducting each activity: Risk Assessment (RA) - Ishikawa Diagram, Failure Mode and Effects Analysis (FMEA) and Risk Estimation Matrix (REM); Screening Design of Experiments (DoE) - 2-level Full and Fractional Factorial Designs; Optimisation DoE - Central Composite Design (CCD). Emerging trends include the growing interest in quantifying and managing the impact of raw materials' attributes variability on process and product, as well as the development of Retrospective QbD approaches (rQbD) in complement to standard QbD.


Assuntos
Indústria Farmacêutica/normas , Pesquisa/normas , Tecnologia Farmacêutica/métodos , Indústria Farmacêutica/métodos , Humanos , Preparações Farmacêuticas/química , Medição de Risco/métodos
16.
Ann Pharmacother ; 54(3): 283-286, 2020 03.
Artigo em Inglês | MEDLINE | ID: mdl-31615266

RESUMO

Food and Drug Administration (FDA) rules for the production of prescription drugs are very rigorous and, if followed, guarantees a safe drug supply. For many years, foreign manufacturers have produced substandard generic products and active pharmaceutical ingredients and shipped them into the United States. If the FDA had inspected them with the same rigor as they do domestic manufacturers, they would have found many of these egregious deviations from ethical manufacturing much earlier. Although the FDA is finally stepping up the number of inspections, their current processes still rely on preannounced inspections with long time horizons, so quality issues can be temporarily corrected and documents altered or destroyed.


Assuntos
Medicamentos Genéricos , Medicamentos sob Prescrição , Indústria Farmacêutica/normas , Indústria Farmacêutica/tendências , Medicamentos Genéricos/efeitos adversos , Medicamentos Genéricos/normas , Medicamentos Genéricos/provisão & distribuição , Humanos , Cooperação Internacional , Serviços Terceirizados/normas , Serviços Terceirizados/tendências , Estados Unidos , United States Food and Drug Administration
17.
Regul Toxicol Pharmacol ; 111: 104542, 2020 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-31756353

RESUMO

The Standard for Exchange of Nonclinical Data (SEND) identifies an approach for representing nonclinical data in a structured format which has been widely adopted by the pharmaceutical industry as it is required for data submission to the United States Food & Drug Administration (US FDA). The SEND Implementation Guide (SENDIG) allows for considerable flexibility in how data is represented; interpretation of these guidelines has led to significant variability in the approach to SEND dataset creation. The purposes of this manuscript are to identify common variability in certain SEND domains and to describe how variability can be managed to enable valuable cross-study analysis use cases. The example of extracting a commonly used data point, animal age, is used to illustrate the complexity and variability of SEND datasets. Developing a solution framework to the variability problem that includes all stakeholders involved in the creation and use of SEND datasets may enable future, routine analysis of warehoused SEND data. Harmonizing the implementation and use of SEND is expected to benefit all involved stakeholders and to ultimately contribute to the goal of increased productivity in nonclinical research.


Assuntos
Bases de Dados Factuais/normas , Indústria Farmacêutica/normas , Estudos Transversais , Humanos , Estados Unidos , United States Food and Drug Administration
18.
Bull World Health Organ ; 97(12): 797-798, 2019 Dec 01.
Artigo em Inglês | MEDLINE | ID: mdl-31819287

RESUMO

Ahd Hamidi talks to Gary Humphreys about technology transfer and the implications of new vaccine production methods for local production.


Assuntos
Indústria Farmacêutica/organização & administração , Vacinas/provisão & distribuição , Indústria Farmacêutica/normas , Saúde Global , Humanos
19.
Value Health ; 22(11): 1275-1282, 2019 11.
Artigo em Inglês | MEDLINE | ID: mdl-31708064

RESUMO

BACKGROUND: Despite increasing informal and formal use of unmet medical need (UMN) in drug development, regulation, and assessment, there is no insight into its definitions in use. This study aims to provide insight into the current definitions in use and to provide a starting point for a multi-stakeholder discussion on alignment. METHODS: A scoping and a gray literature review were performed to locate definitions of UMN in literature and on stakeholder websites. These definitions were categorized and then discussed among the multi-stakeholder author group via semistructured group discussions and open session workshops with a broader stakeholder audience. Issues with the formation of a common definition and mechanisms for use were discussed. RESULTS: The reviews yielded 16 definitions. Differences were evident, but all included 1 or more of the following elements: (adequacy of) available treatments (16 of 16: 100%), disease severity or burden (6 of 16: 38%), and patient population size (1 of 16: 6%). The stakeholder discussions led to a suggestion for a definition including the first 2 items and, depending on context, population size. The discussions also showed that quantification of UMN is highly dependent on the scope and the value framework in which it is used based on different stakeholder preferences and responsibilities. CONCLUSION: We encourage stakeholders that want to promote alignment on the concept of UMN to prospectively discuss the scope in which they want to apply the concept, what elements they find important for consideration in each case, and how they would measure UMN within the broader regulatory or value framework applicable.


Assuntos
Indústria Farmacêutica/organização & administração , Determinação de Necessidades de Cuidados de Saúde/normas , Indústria Farmacêutica/normas , Controle de Medicamentos e Entorpecentes/métodos , Humanos , Reembolso de Seguro de Saúde/normas , Índice de Gravidade de Doença , Estados Unidos
20.
BMC Med Res Methodol ; 19(1): 204, 2019 11 05.
Artigo em Inglês | MEDLINE | ID: mdl-31690260

RESUMO

BACKGROUND: Clinical study reports (CSRs) have been increasingly utilised within academic research in recent years. European Medicines Agency (EMA) Policy 0070 'Phase 1,' which came into effect in January 2015, requires the publication of regulatory documents such as CSRs from central applications in an anonymised format. EMA Policy 0070 requires sponsors to demonstrate careful consideration of data utility within anonymised CSRs published within the scope of the policy, yet the concept of data utility is not clearly defined in the associated anonymisation guidance. OBJECTIVE: To review the use of data from CSRs in published academic research and to hypothesise the potential data utility of CSRs anonymised under the objectives of EMA Policy 0070 for future academic research. METHODS: Review of the objectives, research methodologies and findings of academic research reports using unpublished data from CSRs (prior to EMA Policy 0070). Semi-structured interviews with authors of academic research reports, including questions related to data utility of anonymised CSRs published under EMA Policy 0070. RESULTS: Thirteen academic research reports were identified and reviewed. The research purposes ranged from assessment of reporting bias, comparison of methods and results with published data sources, detailed evaluation of harms and adverse events, re-analysis and novel analyses including systematic reviews and meta-analysis. All of the examples identified required access to the methods and results sections of CSRs (including aggregated summary tables) and research purposes relating to evaluation of adverse events also required access to participant narratives. Retaining anonymised participant narratives relating to interventions, findings and events, while maintaining an acceptably low risk of participant re-identification, may provide an important gain in data utility and further understanding of drug safety profiles. CONCLUSIONS: This work provides an initial insight into the previous use of CSR data and current practices for including regulatory data in academic research. This work also provides early guidance to qualitatively assess and document data utility within anonymised CSRs published under EMA Policy 0070.


Assuntos
Pesquisa Biomédica/normas , Ensaios Clínicos como Assunto/normas , Relatório de Pesquisa/normas , Avaliação da Tecnologia Biomédica/métodos , Pesquisa Biomédica/métodos , Indústria Farmacêutica/organização & administração , Indústria Farmacêutica/normas , Europa (Continente) , Humanos , Publicações Periódicas como Assunto/normas , Viés de Publicação , Avaliação da Tecnologia Biomédica/organização & administração
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