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1.
Am J Obstet Gynecol ; 225(1): 21-32, 2021 07.
Artigo em Inglês | MEDLINE | ID: mdl-34215352

RESUMO

Obstetrical healthcare providers frequently field questions about the safety of medications recommended or prescribed to their pregnant patients. Most women use as least 1 medication during pregnancy; however, there is little information about the safety or appropriate dosing of many medications during this phase of life. In addition, the development of drugs for use in pregnant women trails behind the development of drugs intended for other sectors of the population. Our goal is to inform the obstetrics community about the US Food and Drug Administration authority and their role in approving drugs for marketing. We begin with the statutes that led to the creation of the Food and Drug Administration and its current organization. We then cover drug development and the Food and Drug Administration review process, including the role of the advisory committee. The different types of drug approvals are discussed, with some specific examples. Finally, we enumerate the drugs specifically approved for use in obstetrics and contrast them with drugs commonly used by pregnant women and drugs used "off-label" during pregnancy. The Food and Drug Administration is committed to protecting and advancing the public health of pregnant women by guiding the development and ensuring the availability of effective and safe therapeutics for obstetrical indications and for medical conditions during pregnancy. We hope this review will inspire more research addressing drug use during pregnancy.


Assuntos
Aprovação de Drogas , Gravidez , Medicamentos sob Prescrição , United States Food and Drug Administration , Animais , Ensaios Clínicos como Assunto , Aprovação de Drogas/legislação & jurisprudência , Aprovação de Drogas/estatística & dados numéricos , Feminino , Feto/efeitos dos fármacos , Humanos , Lactação , Complicações na Gravidez/tratamento farmacológico , Medição de Risco , Teratógenos , Estados Unidos
3.
AAPS PharmSciTech ; 22(5): 172, 2021 Jun 07.
Artigo em Inglês | MEDLINE | ID: mdl-34100150

RESUMO

Vaccination development and production was an essential question for the prevention and global control of COVID-19. The strong support from governing authorities such as Operation Warp Speed and robust funding has led to the development and authorization of the tozinameran (BNT162b2) vaccine. The BNT162b2 vaccine is a lipid nanoparticle-encapsulated mRNA that encodes for SARS-CoV-2 spike protein, the main site for neutralizing antibodies. Once it binds with the host cells, the lipid nanoparticles enable the transfer of the RNA, causing S antigens' expression of the SARS-CoV-2, conferring immunity. The vaccine is administered as a 2-dose regime 21 days apart for individuals 16 years and older. Pfizer-BioNTech's BNT162b2 vaccine was the first candidate to receive FDA-Emergency Use Authorization (EUA) on December 11, 2020. During phase 2/3 clinical trials, 95% efficacy was reported among 37,706 participants over the age of 16 who received the BNT162b2 vaccination; additionally, 52% efficacy was noted 12 days following the administration of the first dose of BNT162b2, reflecting early protection of COVID-19. The BNT162b2 vaccine has exhibited 100% efficacy in clinical trials of adolescents between the ages of 12 and 15. Clinical trials in pregnant women and children under the age of 12 are expected to also exhibit promising results. This review article encompasses tozinameran (BNT162b2) vaccine journey, summarizing the BNT162b1 and BNT162b2 vaccines from preclinical studies, clinical trial phases, dosages, immune response, adverse effects, and FDA-EUA.


Assuntos
Vacinas contra COVID-19/administração & dosagem , COVID-19/prevenção & controle , Ensaios Clínicos como Assunto/métodos , Aprovação de Drogas/métodos , SARS-CoV-2/efeitos dos fármacos , Animais , Anticorpos Neutralizantes/efeitos dos fármacos , Anticorpos Neutralizantes/metabolismo , COVID-19/epidemiologia , COVID-19/metabolismo , Vacinas contra COVID-19/efeitos adversos , Vacinas contra COVID-19/metabolismo , Ensaios Clínicos como Assunto/legislação & jurisprudência , Aprovação de Drogas/legislação & jurisprudência , Avaliação Pré-Clínica de Medicamentos/métodos , Exantema/induzido quimicamente , Feminino , Humanos , Masculino , SARS-CoV-2/metabolismo , Glicoproteína da Espícula de Coronavírus/efeitos dos fármacos , Glicoproteína da Espícula de Coronavírus/metabolismo , Vacinação/legislação & jurisprudência , Vacinação/métodos
5.
J Neurooncol ; 153(3): 375-381, 2021 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-34156585

RESUMO

OBJECTIVE: Contemporary management of patients with neuro-oncologic disease requires an understanding of approvals by the US Food and Drug Administration (FDA) related to nervous system tumors. To summarize FDA updates applicable to neuro-oncology practitioners, we sought to review oncology product approvals and Guidances that were pertinent to the field in the past year. METHODS: Oncology product approvals between January 1, 2020, and December 31, 2020, were reviewed for clinical trial outcomes involving tumors of the nervous system. FDA Guidances relevant to neuro-oncology were also reviewed. RESULTS: Five oncology product approvals described outcomes for nervous system tumors in the year 2020. These included the first regulatory approval for neurofibromatosis type 1: selumetinib for children with symptomatic, inoperable plexiform neurofibromas. Additionally, there were 4 regulatory approvals for non-central nervous system (CNS) cancers that described clinical outcomes for patients with brain metastases. These included the approval of tucatinib for metastatic human epidermal growth factor receptor 2 (HER2)-positive breast cancer including patients with brain metastases, brigatinib for anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC), and pralsetinib and selpercatinib for RET fusion-positive NSCLC. Finally, two FDA Guidances for Industry, "Cancer Clinical Trial Eligibility Criteria: Brain Metastases" and "Evaluating Cancer Drugs in Patients with Central Nervous System Metastases" were published to facilitate drug development for and inclusion of patients with CNS metastases in clinical trials. CONCLUSIONS: Despite the challenges of the past year brought on by the COVID-19 pandemic, progress continues to be made in neuro-oncology. These include first-of-their-kind FDA approvals and Guidances that are relevant to the management of patients with nervous system tumors.


Assuntos
Antineoplásicos/uso terapêutico , Neoplasias Encefálicas/tratamento farmacológico , Aprovação de Drogas/legislação & jurisprudência , Aprovação de Drogas/métodos , Humanos , Estados Unidos , United States Food and Drug Administration
6.
Paediatr Drugs ; 23(4): 403-409, 2021 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-34036533

RESUMO

Viloxazine (QELBREE™), a selective norepinephrine reuptake inhibitor, is being developed by Supernus Pharmaceuticals as a non-stimulant for the treatment of attention-deficit/hyperactivity disorder (ADHD) in pediatric and adult patients. This is a novel formulation of a pharmacological agent formerly marketed in Europe for the treatment of depression in adults. Viloxazine received its first pediatric approval in April 2021 in the USA for the treatment of ADHD in pediatric patients aged 6-17 years. Approval was based on positive results from a series of short-term phase III clinical trials in which viloxazine improved the severity of ADHD symptoms in children and adolescents with diagnosed ADHD. Viloxazine is available as extended-release capsules for once-daily oral administration. This article summarizes the milestones in the development of viloxazine leading to this first pediatric approval for ADHD.


Assuntos
Inibidores da Captação Adrenérgica/administração & dosagem , Transtorno do Deficit de Atenção com Hiperatividade/tratamento farmacológico , Aprovação de Drogas/métodos , Viloxazina/administração & dosagem , Administração Oral , Adolescente , Transtorno do Deficit de Atenção com Hiperatividade/diagnóstico , Transtorno do Deficit de Atenção com Hiperatividade/epidemiologia , Estimulantes do Sistema Nervoso Central/administração & dosagem , Criança , Ensaios Clínicos Fase III como Assunto/métodos , Aprovação de Drogas/legislação & jurisprudência , Humanos , Estados Unidos/epidemiologia
7.
Am J Obstet Gynecol ; 225(1): 33-42, 2021 07.
Artigo em Inglês | MEDLINE | ID: mdl-33887238

RESUMO

Pregnant and lactating women are considered "therapeutic orphans" because they generally have been excluded from clinical drug research and the drug development process owing to legal, ethical, and safety concerns. Most medications prescribed for pregnant and lactating women are used "off-label" because most of the clinical approved medications do not have appropriate drug labeling information for pregnant and lactating women. Medications that lack human safety data on use during pregnancy and lactation may pose potential risks for adverse effects in pregnant and lactating women as well as risks of teratogenic effects to their unborn and newborn babies. Federal policy requiring the inclusion of women in clinical research and trials led to considerable changes in research design and practice. Despite more women being included in clinical research and trials, the inclusion of pregnant and lactating women in drug research and clinical trials remains limited. A recent revision to the "Common Rule" that removed pregnant women from the classification as a "vulnerable" population may change the culture of drug research and drug development in pregnant and lactating women. This review article provides an overview of medications studied by the Obstetric-Fetal Pharmacology Research Units Network and Centers and describes the challenges in current obstetrical pharmacology research and alternative strategies for future research in precision therapeutics in pregnant and lactating women. Implementation of the recommendations of the Task Force on Research Specific to Pregnant Women and Lactating Women can provide legislative requirements and opportunities for research focused on pregnant and lactating women.


Assuntos
Desenvolvimento de Medicamentos , Lactação , Gravidez , Gestantes , COVID-19/tratamento farmacológico , COVID-19/prevenção & controle , Vacinas contra COVID-19 , Diabetes Gestacional/tratamento farmacológico , Aprovação de Drogas/legislação & jurisprudência , Desenvolvimento de Medicamentos/legislação & jurisprudência , Feminino , Feto/efeitos dos fármacos , Humanos , Trabalho de Parto Prematuro/tratamento farmacológico , Pré-Eclâmpsia/tratamento farmacológico , Gravidez/fisiologia , Complicações na Gravidez/tratamento farmacológico , Complicações na Gravidez/prevenção & controle , Complicações na Gravidez/virologia , SARS-CoV-2/imunologia , Teratogênese
11.
Pharmacol Res ; 166: 105472, 2021 04.
Artigo em Inglês | MEDLINE | ID: mdl-33592272

RESUMO

The coronavirus disease 2019 (COVID-19) has now rapidly spread around the world, causing an outbreak of acute infectious pneumonia. To develop effective and safe therapies for the prevention and treatment of COVID-19 has become the major global public health concern. Traditional medicine (TM)/herbal medicines (HMs) have been used to treat multiple epidemics in human history, which brings hope for the fight against COVID-19 in some areas. For example, in China, India, and South Korea with traditional medication history and theory, the governments issued a series of guidelines to support TM/HMs in the medication of COVID-19. In contrast, other countries e.g. North American and European governments are typically silent on these practices, unless to warn of possible harm and overselling. Such difference is due to the discrepancy in culture, history and philosophical views of health care and medication, as well as unharmonized policies and standards in the regulation and legalization of TM/HMs among different areas. Herein, we reviewed the responses and scientific researches from seven selected countries on the policies and legalization of TM/HMs to treat COVID-19, and also analyzed the major challenges and concerns to utilize the traditional knowledge and resource.


Assuntos
Antivirais/uso terapêutico , COVID-19/tratamento farmacológico , COVID-19/terapia , Terapias Complementares/legislação & jurisprudência , Aprovação de Drogas/legislação & jurisprudência , Saúde Global/legislação & jurisprudência , Medicina Tradicional , Preparações de Plantas/uso terapêutico , Disparidades em Assistência à Saúde/legislação & jurisprudência , Humanos , Formulação de Políticas
12.
Molecules ; 26(3)2021 Jan 25.
Artigo em Inglês | MEDLINE | ID: mdl-33504104

RESUMO

Although the pharmaceutical industry will remember 2020 as the year of COVID-19, it is important to highlight that this year has been the second-best-together with 1996-in terms of the number of drugs accepted by the US Food and Drug Administration (FDA). Each of these two years witnessed the authorization of 53 drugs-a number surpassed only in 2018 with 59 pharmaceutical agents. The 53 approvals in 2020 are divided between 40 new chemical entities and 13 biologic drugs (biologics). Of note, ten monoclonal antibodies, two antibody-drug conjugates, three peptides, and two oligonucleotides have been approved in 2020. Close inspection of the so-called small molecules reveals the significant presence of fluorine atoms and/or nitrogen aromatic heterocycles. This report analyzes the 53 new drugs of the 2020 harvest from a strictly chemical perspective, as it did for those authorized in the previous four years. On the basis of chemical structure alone, the drugs that received approval in 2020 are classified as the following: biologics (antibodies, antibody-drug conjugates, and proteins); TIDES (peptide and oligonucleotides); natural products; fluorine-containing molecules; nitrogen aromatic heterocycles; and other small molecules.


Assuntos
Aprovação de Drogas/legislação & jurisprudência , Indústria Farmacêutica , United States Food and Drug Administration/legislação & jurisprudência , História do Século XXI , Estados Unidos
14.
Expert Opin Drug Saf ; 20(3): 265-274, 2021 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-33455482

RESUMO

Introduction: Biosimilar medicines have transformed the healthcare landscape by providing improved access to life-saving medicines at a lower cost. Biosimilars are a distinct category of biologic therapeutics that enter the market after patent expiration of a reference molecule. Regulatory bodies worldwide have developed guidance to expedite the approval and entry of these drugs to the market. Biosimilar approval is based on a totality of the evidence approach, demonstrating similarity between the biosimilar and the originator in terms of physicochemical properties, quality characteristics, biological activity, safety, and efficacy.Areas covered: This article provides an overview of the biosimilar regulatory guidelines and discusses the importance and considerations of comparative clinical studies that are performed during biosimilar development. Two review assessment reports, one each from the EMA and the FDA, are presented.Expert opinion: The discussed case studies illustrate the importance of pharmacokinetic and pharmacodynamic studies in the regulatory approval of biosimilars. It is crucial for biosimilar developers to judiciously determine clinical parameters including biomarkers, endpoints, and acceptance criteria before executing clinical studies.


Assuntos
Produtos Biológicos/administração & dosagem , Medicamentos Biossimilares/administração & dosagem , Aprovação de Drogas/legislação & jurisprudência , Animais , Produtos Biológicos/efeitos adversos , Produtos Biológicos/farmacologia , Biomarcadores/metabolismo , Medicamentos Biossimilares/efeitos adversos , Medicamentos Biossimilares/farmacologia , Ensaios Clínicos como Assunto/métodos , União Europeia , Órgãos Governamentais , Humanos , Estados Unidos , United States Food and Drug Administration
15.
Clin Pharmacol Ther ; 109(2): 295-298, 2021 02.
Artigo em Inglês | MEDLINE | ID: mdl-33469964

RESUMO

A key goal of regulatory agencies for medical products is to make innovative products available to patients and the medical community in a timely manner in order to improve the quality of public health and health care. Thus, regulators must respond quickly to emerging technologies. It is a horizon scanning method, based on which the Japanese regulatory agency will have the expertise prior to review of forthcoming products of evolving technologies.


Assuntos
Desenvolvimento de Medicamentos/legislação & jurisprudência , Atenção à Saúde/legislação & jurisprudência , Aprovação de Drogas/legislação & jurisprudência , Órgãos Governamentais/legislação & jurisprudência , Humanos , Japão
16.
Prep Biochem Biotechnol ; 51(1): 1-8, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-32921222

RESUMO

Biosimilars are the biological drugs that are granted after the expiry of the patent of an affirmed innovator. Asia Pacific countries are characterized by significant demand as they account for majority of the world population and poor affordability due to low per capita income in most regions. Some of these countries offer potential to emerge as global suppliers of affordable, safe and efficacious biosimilars. This article highlights the prospects of biosimilars in the Asia Pacific market. Regulatory framework in the various countries is also discussed.


Assuntos
Medicamentos Biossimilares/economia , Medicamentos Biossimilares/provisão & distribuição , Aprovação de Drogas/economia , Aprovação de Drogas/legislação & jurisprudência , Indústria Farmacêutica/economia , Indústria Farmacêutica/legislação & jurisprudência , Ásia Sudeste , Ásia Ocidental , Extremo Oriente , Humanos , Marketing/economia , Marketing/legislação & jurisprudência
17.
Hum Vaccin Immunother ; 17(5): 1322-1325, 2021 05 04.
Artigo em Inglês | MEDLINE | ID: mdl-33270474

RESUMO

Vaccines have changed modern medicine, and are a mainstay in reducing morbidity and mortality from infections. Our research group recently published a study in which we found that vaccines approved by the US Food and Drugs Administration were safe with few clinically important post-approval adverse effects. The current COVID-19 pandemic presents regulators with the unprecedented challenge of balancing a public demand for the rapid development and approval of a safe and effective SARS-CoV-2 vaccine without compromising the strict pre-marketing requirements used for previous vaccines. Here, we review the approval process and safety profiles of FDA approved vaccines and discuss some of the challenges currently facing the FDA regarding the SARS-CoV-2 vaccine approval.


Assuntos
Vacinas contra COVID-19 , COVID-19/prevenção & controle , Segurança do Paciente , COVID-19/epidemiologia , Vacinas contra COVID-19/efeitos adversos , Aprovação de Drogas/legislação & jurisprudência , Humanos , Pandemias , SARS-CoV-2 , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência
18.
Expert Rev Pharmacoecon Outcomes Res ; 21(1): 145-157, 2021 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-32043904

RESUMO

Objective: Due to the impact of the Federal Joint Committee's (FJC) appraisal on following price negotiations, it is crucial to understand the underlying reasons of failure in early benefit assessment (EBA) of medicinal products in Germany. Methods: Medicinal products for which no added benefit was granted, the underlying reasons given by the FJC were extracted and grouped into respective categories according to predefined working definitions. Several reasons may hold for one subgroup. Furthermore, binomial proportion analysis was performed to gather proportions and their precision for each therapeutic area regarding possible failure. Results: 293/427 subgroups did not receive an added benefit. For 265/293 the following main formal reasons were stated: deviation of label to the pivotal studies (59%), wrong comparator (20.5%), and methodological deficiencies of indirect comparisons (12.3%). The proportion of failure in EBA is heterogeneous and therapeutic area depending (p = 0.0005). For most of the therapeutic areas, the confidence intervals of binomial proportions include 50%. Conclusion: Various different reasons led to the failure of EBAs in the past. Despite different objectives, a better alignment between the requirements and methods in the marketing authorization procedure and the EBA might facilitate the design of pivotal studies, which may be useful in both procedures.


Assuntos
Aprovação de Drogas/legislação & jurisprudência , Projetos de Pesquisa , Avaliação da Tecnologia Biomédica/legislação & jurisprudência , Alemanha , Humanos
19.
Pediatr Blood Cancer ; 68(2): e28828, 2021 02.
Artigo em Inglês | MEDLINE | ID: mdl-33245181

RESUMO

BACKGROUND: Pediatric anticancer drug development has numerous challenges. The Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) were passed to address pediatric drug development deficiencies in general. Until recently, the requirements for pediatric evaluation of most oncology products developed for adult cancers have been waived. Because children typically do not have the same type of cancers, which occur commonly in adults, or the indication or drug had been granted an orphan designation, PREA therefore has had no impact. Pediatric studies for labeling updates are largely done through BPCA by a written request (WR) issued by the Food and Drug Administration (FDA). Because the cancers that occur in pediatric and adult populations do not share the same etiology or natural history, there are limited opportunities to extrapolate adult efficacy and safety to the pediatric population. The characteristics of individual pediatric studies included in WRs have varied greatly over time. PROCEDURE: In this study, we searched WRs that were issued by the FDA since 2001. We found 40 such requests issued for oncology drugs and biologics, which had been accepted by sponsors. RESULTS: Clinical trials included in 23 of the WRs have been concluded, 19 have resulted in exclusivity, and three drugs that were studied have been approved for use in pediatric populations. Herein, we present the spectrum of WRs from a regulatory, study design, dosing, formulation, analysis plan, evidentiary standard of efficacy, and safety perspective. CONCLUSIONS: This provides information on requests issued in the past nearly 20 years and studies that are completed. As WRs have provided the only regulatory mechanism to assure pediatric cancer drug development, this can potentially provide insight on how pediatric cancer drug development may change in the future.


Assuntos
Antineoplásicos/uso terapêutico , Aprovação de Drogas/legislação & jurisprudência , Avaliação de Medicamentos/métodos , Neoplasias/tratamento farmacológico , Criança , Ensaios Clínicos como Assunto/estatística & dados numéricos , Humanos , Estados Unidos , United States Food and Drug Administration
20.
Cancer Invest ; 39(2): 120-123, 2021 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-33290099

RESUMO

Precision oncology has revolutionized the therapeutic landscape of oncology and is a goal for cancer drug development. However, lenient drug approvals by the United States Food and Drug Administration under the auspices of precision oncology are setting up this therapeutic approach to fail. In this commentary, I review two recent FDA drug approvals (pembrolizumab for tumor mutation burden-high solid tumors and olaparib for castration-resistant prostate cancer with deleterious homologous recombination repair mutations) where the FDA indication is broader than the studied population. I explain how these broad approvals stray from principles of precision oncology and can cause harm to patients.


Assuntos
Antineoplásicos/uso terapêutico , Legislação de Medicamentos/normas , Mutação , Neoplasias/tratamento farmacológico , Anticorpos Monoclonais Humanizados/uso terapêutico , Aprovação de Drogas/legislação & jurisprudência , Aprovação de Drogas/organização & administração , Humanos , Masculino , Neoplasias/genética , Ftalazinas/uso terapêutico , Piperazinas/uso terapêutico , Medicina de Precisão , Neoplasias de Próstata Resistentes à Castração/tratamento farmacológico , Neoplasias de Próstata Resistentes à Castração/genética , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência
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