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5.
Curr Opin Infect Dis ; 33(4): 304-311, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32657967

RESUMO

PURPOSE OF THE REVIEW: Laboratory-developed tests (LDTs) are essential for the clinical care of immunocompromised individuals. These patients often require specialized testing not available from commercial manufacturers and are therefore dependent on the laboratory to create, validate, and perform these assays. Recent paradigm-shifting legislation could alter the way that LDTs are operationalized and regulated. RECENT FINDINGS: On March 5th, 2020 the Verifying Accurate and Leading-Edge In-Vitro Clinical Tests Development Act (VALID) was introduced in the US Congress. This statute would overhaul existing regulatory framework by unifying the oversight of LDTs and commercial in-vitro diagnostic tests (IVDs) through the FDA. If enacted, LDTs would be subject to regulatory requirements like those found in commercial submissions for market review. Stakeholders continue to discuss the details and scope of the proposed legislation in the setting of the Severe Acute Respiratory Syndrome Coronavirus 2 pandemic, where LDTs are integral to the national COVID-19 response. SUMMARY: Congressional lawmakers have introduced legislation to alter the regulatory framework governing LDTs. Moving forward, a balance must be struck to ensure the availability of safe and accurate testing without delays or overregulation that could be harmful to patients. The downstream implications of how VALID and other legislation will impact laboratories, clinicians, and patients warrant close examination.


Assuntos
Serviços de Laboratório Clínico/legislação & jurisprudência , Técnicas de Laboratório Clínico , Infecções por Coronavirus/diagnóstico , Hospedeiro Imunocomprometido , Laboratórios Hospitalares/legislação & jurisprudência , Pneumonia Viral/diagnóstico , Incerteza , United States Food and Drug Administration/legislação & jurisprudência , Betacoronavirus/patogenicidade , Congressos como Assunto , Pesquisa sobre Serviços de Saúde/legislação & jurisprudência , Humanos , Pandemias , Garantia da Qualidade dos Cuidados de Saúde , Estados Unidos
7.
Clin Ther ; 42(8): 1444-1450, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32651020

RESUMO

Against the backdrop of the COVID pandemic, the scientific and medical communities are working with all deliberate speed with state-of-the-art technologies to develop diagnostic and therapeutic products that can identify, treat, and prevent infection with SARS-CoV-2. These activities may only be legally conducted with the necessary statutes and regulations in place to facilitate the timely development, manufacturing, evaluation, and distribution of products that meet quality standards. The present regulatory landscape for medicinal and medical products for human use has been shaped by nearly 12 decades of statutory history that followed in reaction to disasters and tragedies. Five distinct, closely woven threads of statutory history have led to the regulatory infrastructure we have in place: (1) standardized processes for routine development of medicinal and medical device products for human use; (2) processes for expedited development to shorten time frames and expand patient populations; (3) mechanisms of Expanded Access to make medicinal products available to patients prior to approval of the US Food and Drug Administration; (4) Emergency Use Authorization during public health emergencies; and (5) the development of pathways for bringing generic drugs and biosimilar biologics to market. These mechanisms are being brought to bear to facilitate the defeat of infection with SARS-CoV-2.


Assuntos
Infecções por Coronavirus/diagnóstico , Infecções por Coronavirus/terapia , Aprovação de Drogas/legislação & jurisprudência , Legislação de Dispositivos Médicos , Pneumonia Viral/diagnóstico , Pneumonia Viral/terapia , United States Food and Drug Administration/legislação & jurisprudência , Antivirais/uso terapêutico , Betacoronavirus/imunologia , Técnicas de Laboratório Clínico , Infecções por Coronavirus/tratamento farmacológico , Infecções por Coronavirus/epidemiologia , Infecções por Coronavirus/prevenção & controle , Desenvolvimento de Medicamentos/legislação & jurisprudência , Humanos , Pandemias/prevenção & controle , Pneumonia Viral/epidemiologia , Pneumonia Viral/prevenção & controle , Estados Unidos
9.
Lancet Infect Dis ; 20(7): e159-e164, 2020 07.
Artigo em Inglês | MEDLINE | ID: mdl-32502431

RESUMO

Antimicrobial resistance is of growing concern. To encourage development of new treatments, some commentators have suggested regulators exercise increased flexibility on the clinical evidence required for approval. We examined all 1065 new drugs and biologics approved by the US Food and Drug Administration between 1984 and 2018 and recorded each drug's use of the Orphan Drug Act, fast-track, priority review, accelerated approval, and breakthrough therapy programmes, as well as dates of investigational new drug application, new drug application, and new drug approval, which were used to calculate clinical development and review times. There were 178 (17%) antimicrobial products, which were more likely than non-antimicrobial products to benefit from priority review (103 [58%] of 178 vs 402 [45%] of 887, p=0·0023), fast-track designation (58 [37%] of 157 vs 151 [19%] of 814], p<0·001), and accelerated approval (23 [18%] of 129 vs 67 [9%] of 711, p=0·0046), and less likely to have Orphan Drug Act designation (25 [14%] of 178 vs 267 [30%] of 887, p<0·0001). Median time from investigational new drug application to approval was shorter for antimicrobial than for non-antimicrobial drugs (5·9 years [IQR 4·6-7·3] vs 7·6 years [IQR 5·7-10·2], p<0·001). Except for Orphan Drug Act status, expedited clinical testing and review programmes have been used at least as frequently for antimicrobial products as for non-antimicrobial drugs. No evidence supported claims that antimicrobial progress through the regulatory approval process in the USA is more time-consuming than non-antimicrobial development.


Assuntos
Anti-Infecciosos , Produtos Biológicos , Aprovação de Drogas/legislação & jurisprudência , United States Food and Drug Administration/legislação & jurisprudência , Humanos , Estados Unidos
13.
PLoS One ; 15(4): e0230898, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32267884

RESUMO

PURPOSE: To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs). METHODS: Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descriptive and comparative statistics. RESULTS: Between 1983 and 2019, 124 orphan drug designations were granted by the FDA for compounds intended to treat 28 lysosomal storage diseases. Orphan drug designations focused on Gaucher disease (N = 16), Pompe disease (N = 16), Fabry disease (N = 10), MPS II (N = 10), MPS I (N = 9), and MPS IIIA (N = 9), and included enzyme replacement therapies, gene therapies, and small molecules, and others. Twenty-three orphan drugs were approved for the treatment of 11 LSDs. Gaucher disease (N = 6), cystinosis (N = 5), Pompe disease (N = 3), and Fabry disease (N = 2) had multiple approvals, CLN2, LAL-D, MPS I, II, IVA, VI, and VII one approval each. This is an increase of nine more approved drugs and four more treatable LSDs (CLN2, MPS VII, LAL-D, and MPS IVA) since 2013. Mean time between orphan drug designation and FDA approval was 89.7 SD 55.00 (range 8-203, N = 23) months. CONCLUSIONS: The drug development pipeline for LSDs is growing and evolving, with increased focus on diverse small-molecule targets and gene therapy. CLN2 was the first and only LSD with an approved therapy directly targeted to the brain. Newly approved products included "me-too"-enzymes and innovative compounds such as the first pharmacological chaperone for the treatment of Fabry disease.


Assuntos
Doenças por Armazenamento dos Lisossomos/tratamento farmacológico , Produção de Droga sem Interesse Comercial/estatística & dados numéricos , United States Food and Drug Administration , Estudos Transversais , Bases de Dados de Produtos Farmacêuticos , Descoberta de Drogas , Regulamentação Governamental , Humanos , Produção de Droga sem Interesse Comercial/legislação & jurisprudência , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência
15.
Adv Exp Med Biol ; 1248: 485-530, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32185723

RESUMO

Macromolecule drugs particularly antibody drugs are very powerful therapies developing rapidly in the recent 20 years, providing hopes for many patients diagnosed with "incurable" diseases in the past. They also provide more effective and less side effects for many afflicting diseases, and greatly improve the survival rate and life quality of patients. In the last two decades, the proportion of US Food and Drug Administration (FDA) approved macromolecules and antibody drugs are increasing quickly, especially after the discovery of immune checkpoints. To crown all, the 2017 Nobel prize in physiology or medicine was given to immunotherapy. In this chapter, we would like to summarize the current situation of macromolecule and antibody drugs, and what effort scientists and pharmaceutical industry have made to discover and manufacture better antibody drugs.


Assuntos
Anticorpos/uso terapêutico , Imunoterapia , Preparações Farmacêuticas , Pontos de Checagem do Ciclo Celular/efeitos dos fármacos , Pontos de Checagem do Ciclo Celular/imunologia , Aprovação de Drogas/legislação & jurisprudência , Indústria Farmacêutica , Humanos , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência
18.
Gastroenterology ; 158(2): 368-388, 2020 01.
Artigo em Inglês | MEDLINE | ID: mdl-31563626

RESUMO

Although colorectal cancer (CRC) screening has reduced the incidence of and mortality from CRC, chemoprevention strategies have the potential to further reduce CRC incidence and mortality. Chemoprevention agents might be used for average-risk as well as high-risk groups, and to prevent CRC recurrence after therapy. CRC chemoprevention agents that have been studied include aspirin, nonaspirin nonsteroidal anti-inflammatory drugs, statins, agents that target metabolic pathways, and vitamins and minerals. We review the prospect of chemoprevention of CRC, results from preclinical and human studies, challenges, and future directions.


Assuntos
Anti-Inflamatórios não Esteroides/uso terapêutico , Anticarcinógenos/uso terapêutico , Neoplasias Colorretais/prevenção & controle , Inibidores de Hidroximetilglutaril-CoA Redutases/uso terapêutico , Vitaminas/uso terapêutico , Animais , Neoplasias Colorretais/diagnóstico , Neoplasias Colorretais/epidemiologia , Modelos Animais de Doenças , Aprovação de Drogas , Avaliação Pré-Clínica de Medicamentos , Detecção Precoce de Câncer/estatística & dados numéricos , Humanos , Incidência , Programas de Rastreamento/estatística & dados numéricos , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Ensaios Clínicos Controlados Aleatórios como Assunto , Resultado do Tratamento , Estados Unidos/epidemiologia , United States Food and Drug Administration/legislação & jurisprudência
20.
Photochem Photobiol Sci ; 19(1): 66-70, 2020 Jan 22.
Artigo em Inglês | MEDLINE | ID: mdl-31845952

RESUMO

Photoprotection, including the use of sunscreen, has been shown to decrease the development of keratinocyte cancers and melanoma. Due to concerns about the environmental effects of some organic UVR filters, several locations across the world have begun to pass legislation banning the use of these ingredients in sunscreens. Furthermore, the health effects of several organic UVR filters have also been called into question and a recent proposal by the US FDA has resulted in public confusion about the safety of sunscreens. The aim of this article is to discuss FDA regulation of sunscreens and to review the environmental and health effects of oxybenzone and octinoxate. Ultimately, as dermatologists, our recommendations are to continue to encourage people to practice proper photoprotection including photoprotective clothing, staying in the shade while outdoors, and applying sunscreen to exposed areas. For those concerned about the potential environmental and health effects of organic UVR filters, inorganic/mineral UVR filter (namely, zinc oxide and titanium dioxide)-based sunscreens can be used.


Assuntos
Protetores Solares/legislação & jurisprudência , United States Food and Drug Administration/legislação & jurisprudência , Benzofenonas/farmacologia , Cinamatos/farmacologia , Humanos , Protetores Solares/farmacologia , Titânio/farmacologia , Estados Unidos , Óxido de Zinco/farmacologia
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