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1.
Nat Rev Clin Oncol ; 17(3): 140-146, 2020 03.
Artigo em Inglês | MEDLINE | ID: mdl-32020042

RESUMO

The traditional regulatory drug approval paradigm comprising discrete phases of clinical testing that culminate in a large randomized superiority trial has historically been predominant in oncology. However, this approach has evolved in the current era of drug development, with multiple other development pathways now being utilized. Indeed, treatment approaches designed on the basis of an improved understanding of cancer biology have led to unprecedented responses in early phase trials, sometimes resulting in drug approvals in the absence of large-scale trials. At the same time, improved molecular diagnostic technologies have led to the identification of ever-smaller patient subgroups for molecularly targeted therapy. Moreover, new FDA regulatory paradigms have enabled the rapid review and accelerated approval of certain drugs in the absence of survival data. Regulatory approvals based on large-cohort trials with surrogate or intermediate clinical end points or on non-inferiority trials, as well as new tumour-agnostic indications, also set important precedents in the field. In this Viewpoint, we asked two leading oncologists involved in clinical drug development, an expert in regulatory science and prescription drug policy and a prominent patient advocate, to provide their opinions on the implications of these changes in regulatory practices for patient care.


Assuntos
Antineoplásicos/uso terapêutico , Aprovação de Drogas , Terapia de Alvo Molecular , Neoplasias/tratamento farmacológico , Desenvolvimento de Medicamentos , Humanos , Oncologia/tendências , Neoplasias/epidemiologia , Estados Unidos/epidemiologia , United States Food and Drug Administration
2.
Value Health ; 20(2): 283-285, 2017 02.
Artigo em Inglês | MEDLINE | ID: mdl-28237210

RESUMO

Recent scientific progress is, in some cases, leading to transformative new medicines for diseases that previously had marginal or even no treatment options. This offers great promise for people affected by these diseases, but it has also placed stress on the health care system in terms of the growing cost associated with some new interventions. Effort has been taken to create tools to help patients and health care providers assess the value of new medical innovations. These tools may also provide the basis for assessing the price associated with new medical products. Given the growing expenditures in health care, value frameworks present an opportunity to evaluate new therapeutic options in the context of other treatments and potentially lead to a more economically sustainable health care system. In summary, the contribution to meaningful improvements in health outcomes is the primary focus of any assessment of the value of a new intervention. A component of such evaluations, however, should factor in timely access to new products that address an unmet medical need, as well as the magnitude of that beneficial impact. To achieve these goals, value assessment tools should allow for flexibility in clinical end points and trial designs, incorporate patient preferences, and continually evolve as new evidence, practice patterns, and medical progress advance.


Assuntos
Descoberta de Drogas , Preferência do Paciente , Aquisição Baseada em Valor , Antineoplásicos , Humanos , Neoplasias/tratamento farmacológico
4.
Clin Cancer Res ; 21(7): 1514-24, 2015 Apr 01.
Artigo em Inglês | MEDLINE | ID: mdl-25680375

RESUMO

The Lung Master Protocol (Lung-MAP, S1400) is a groundbreaking clinical trial designed to advance the efficient development of targeted therapies for squamous cell carcinoma (SCC) of the lung. There are no approved targeted therapies specific to advanced lung SCC, although The Cancer Genome Atlas project and similar studies have detected a significant number of somatic gene mutations/amplifications in lung SCC, some of which are targetable by investigational agents. However, the frequency of these changes is low (5%-20%), making recruitment and study conduct challenging in the traditional clinical trial setting. Here, we describe our approach to development of a biomarker-driven phase II/II multisubstudy "Master Protocol," using a common platform (next-generation DNA sequencing) to identify actionable molecular abnormalities, followed by randomization to the relevant targeted therapy versus standard of care.


Assuntos
Biomarcadores Tumorais/genética , Carcinoma Pulmonar de Células não Pequenas/tratamento farmacológico , Carcinoma de Células Escamosas/tratamento farmacológico , Neoplasias Pulmonares/tratamento farmacológico , Medicina de Precisão/métodos , Projetos de Pesquisa , Antineoplásicos/uso terapêutico , Carcinoma Pulmonar de Células não Pequenas/genética , Carcinoma de Células Escamosas/genética , Humanos , Neoplasias Pulmonares/genética
5.
Nat Rev Drug Discov ; 12(10): 743-55, 2013 10.
Artigo em Inglês | MEDLINE | ID: mdl-24008432

RESUMO

As diagnostic tests become increasingly important for optimizing the use of drugs to treat cancers, the co-development of a targeted therapy and its companion diagnostic test is becoming more prevalent and necessary. In July 2011, the US Food and Drug Administration released a draft guidance that gave the agency's formal definition of companion diagnostics and introduced a drug-diagnostic co-development process for gaining regulatory approval. Here, we identify areas of drug-diagnostic co-development that were either not covered by the guidance or that would benefit from increased granularity, including how to determine when clinical studies should be limited to biomarker-positive patients, defining the diagnostically selected patient population in which to use a companion diagnostic, and defining and clinically validating a biomarker signature for assays that use more than one biomarker. We propose potential approaches that sponsors could use to deal with these challenges and provide strategies to help guide the future co-development of drugs and diagnostics.


Assuntos
Antineoplásicos/farmacologia , Técnicas de Diagnóstico Molecular , Terapia de Alvo Molecular , Neoplasias/tratamento farmacológico , Biomarcadores/análise , Biomarcadores/metabolismo , Aprovação de Equipamentos , Aprovação de Drogas , Desenho de Fármacos , Humanos , Neoplasias/diagnóstico , Neoplasias/patologia , Estados Unidos , United States Food and Drug Administration
6.
Clin Cancer Res ; 19(16): 4297-304, 2013 Aug 15.
Artigo em Inglês | MEDLINE | ID: mdl-23719260

RESUMO

In July 2012, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA to expedite clinical development of new, potential "breakthrough" drugs or treatments that show dramatic responses in early-phase studies. Using this regulatory pathway, once a promising new drug candidate is designated as a "Breakthrough Therapy", the U.S. Food and Drug Administration (FDA) and sponsor would collaborate to determine the best path forward to abbreviate the traditional three-phase approach to drug development. The breakthrough legislation requires that an FDA guidance be drafted that details specific requirements of the bill to aid FDA in implementing requirements of the Act. In this article, we have proposed criteria to define a product as a Breakthrough Therapy, and discussed critical components of the development process that would require flexibility in order to enable expedited development of a Breakthrough Therapy.


Assuntos
Oncologia , Neoplasias/terapia , Terapêutica/métodos , Terapêutica/normas , Humanos , Oncologia/normas , Estados Unidos , United States Food and Drug Administration
7.
Clin Cancer Res ; 19(14): 3722-31, 2013 Jul 15.
Artigo em Inglês | MEDLINE | ID: mdl-23665737

RESUMO

This study explores the historic use of different endpoints to support regular and accelerated approval of cancer drugs between 2002 and 2012. In the past 10 years, two thirds of oncology regular approvals were based on endpoints other than overall survival. More than three quarters of accelerated approvals were based on response rates. The accelerated approval program has been heavily used over this time period, with one third of all approved oncology indications receiving accelerated approval. At times, critics have characterized the agency as rigid and unpredictable. This research describes the degree of regulatory flexibility that U.S. Food and Drug Administration and drug sponsors have used over the past decade in the development of new treatments for cancer.


Assuntos
Aprovação de Drogas/estatística & dados numéricos , Neoplasias/tratamento farmacológico , Antineoplásicos/uso terapêutico , Ensaios Clínicos como Assunto , Humanos , Neoplasias/mortalidade , Resultado do Tratamento , Estados Unidos , United States Food and Drug Administration
9.
Health Aff (Millwood) ; 30(7): 1375-81, 2011 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-21680577

RESUMO

The US Food and Drug Administration is often criticized as inefficient compared to its European counterpart, the European Medicines Agency. This criticism is especially common in the field of oncology, where severely ill patients have few therapeutic options. We conducted a direct drug-to-drug comparison of the two regulatory agencies' approvals of new oncology drugs. We found that contrary to public assertions, the median time for approval for new cancer medicines in the United States was just six months--and that these new anticancer medicines are typically available in the United States before they are in Europe. Our findings reinforce the need for strong financial and public support of the Food and Drug Administration, so that such medicines can continue to be made available speedily to patients in need.


Assuntos
Antineoplásicos/farmacologia , Aprovação de Drogas/estatística & dados numéricos , Drogas em Investigação/uso terapêutico , Antineoplásicos/administração & dosagem , Ensaios Clínicos como Assunto , Estudos Transversais , Aprovação de Drogas/legislação & jurisprudência , Drogas em Investigação/farmacologia , Europa (Continente) , Humanos , Neoplasias/tratamento farmacológico , Controle de Qualidade , Fatores de Tempo , Estados Unidos , United States Food and Drug Administration
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