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2.
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
4.
PLoS One ; 15(1): e0227216, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-31914156

RESUMO

OBJECTIVES: Mifepristone was approved for use in medical abortion by Health Canada in 2015. Approval was accompanied by regulations that prohibited pharmacist dispensing of the medication. Reproductive health advocates in Canada recognized this regulation would limit access to medical abortion and successfully worked to have this regulation removed in 2017. The purpose of this study was to assess the leadership involved in changing these regulations so that the success may be replicated by other groups advocating for health policy change. METHODS: This study involved a mixed methods instrumental design in the context of British Columbia, Canada. Our data collection included: a) interviews with seven key individuals, representing the organizations that worked in concert for change to Canadian mifepristone regulations, and b) document analysis of press articles, correspondence, briefing notes, and meeting minutes. We conducted a thematic analysis of transcripts of audio-recorded interviews. We identified strengths and weaknesses of the team dynamic using the Develop Coalitions, Achieve Results and Systems Transformation domains of the LEADS Framework. RESULTS: Our analysis of participant interviews indicates that autonomy, shared values, and clarity in communication were integral to the success of the group's work. Analysis using the LEADS Framework showed that individuals possessed many of the capabilities identified as being necessary for successful health policy leadership. A lack of post-project assessment was identified as a possible limitation and could be incorporated in future work to strengthen dynamics especially when a desired outcome is not achieved. Document analysis provided a clear time-line of the work completed and suggested that strong communication between team members was another key to success. CONCLUSIONS: The results of our analysis of the interviews and documents provide valuable insight into the workings of a successful group committed to a common goal. The existing collegial and trusting relationships between key stakeholders allowed for interdisciplinary collaboration, rapid mobilization, and identification of issues that facilitated successful Canadian global-first deregulation of mifepristone dispensing.


Assuntos
Abortivos Esteroides , Aborto Induzido , Mifepristona , Abortivos Esteroides/provisão & distribução , Aborto Induzido/legislação & jurisprudência , Colúmbia Britânica , Aprovação de Drogas/legislação & jurisprudência , Feminino , Política de Saúde , Humanos , Liderança , Mifepristona/provisão & distribução , Gravidez , Saúde Reprodutiva/legislação & jurisprudência
6.
JAMA ; 323(2): 164-176, 2020 01 14.
Artigo em Inglês | MEDLINE | ID: mdl-31935033

RESUMO

Importance: US law requires testing of new drugs before approval to ensure that they provide a well-defined benefit that is commensurate with their risks. A major challenge for the US Food and Drug Administration (FDA) is to achieve an appropriate balance between rigorous testing and the need for timely approval of drugs that have benefits that outweigh their risks. Objective: To describe the evolution of laws and standards affecting drug testing, the use of new approval programs and standards, expansions of the role and authority of the FDA, and changes in the number of drugs approved from the 1980s to 2018. Evidence: Sources of evidence included principal federal laws and FDA regulations (1962-2018) and FDA databases of approved new drugs (1984-2018), generic drugs (1970-2018), biologics (1984-2018), and vaccines (1998-2018); special development and approval programs (Orphan drug [1984-2018], Fast-Track [1988-2018], Priority Review and its predecessors [1984-2018], Accelerated Approval [1992-2018], and Breakthrough Therapy [2012-2018]); expanded access (2010-2017) and Risk Evaluation and Mitigation Strategies (2008-2018); and user fees paid to the FDA by industry (1993-2018). Findings: From 1983 to 2018, legislation and regulatory initiatives have substantially changed drug approval at the FDA. The mean annual number of new drug approvals, including biologics, was 34 from 1990-1999, 25 from 2000-2009, and 41 from 2010-2018. New biologic product approvals increased from a median of 2.5 from 1990-1999, to 5 from 2000-2013, to 12 from 2014-2018. The median annual number of generic drugs approved was 136 from 1970 to the enactment of the Hatch-Waxman Act in 1984; 284 from 1985 to the enactment of the Generic Drug User Fee Act in 2012; and 588 from 2013-2018. Prescription drug user fee funding expanded from new drugs and biologics in 1992 to generic and biosimilar drugs in 2012. The amount of Prescription Drug User Fee Act fees collected from industry increased from an annual mean of $66 million in 1993-1997 to $820 million in 2013-2017, and in 2018, user fees accounted for approximately 80% of the salaries of review personnel responsible for the approval of new drugs. The proportion of drugs approved with an Orphan Drug Act designation increased from 18% (55/304) in 1984-1995, to 22% (82/379) in 1996-2007, to 41% (154/380) in 2008-2018. Use of Accelerated Approval, Fast-Track, and Priority Review for new drugs has increased over time, with 81% (48/59) of new drugs benefiting from at least 1 such expedited program in 2018. The proportion of new approvals supported by at least 2 pivotal trials decreased from 80.6% in 1995-1997 to 52.8% in 2015-2017, based on 124 and 106 approvals, respectively, while the median number of patients studied did not change significantly (774 vs 816). FDA drug review times declined from more than 3 years in 1983 to less than 1 year in 2017, but total time from the authorization of clinical testing to approval has remained at approximately 8 years over that period. Conclusions and Relevance: Over the last 4 decades, the approval and regulation processes for pharmaceutical agents have evolved and increased in complexity as special programs have been added and as the use of surrogate measures has been encouraged. The FDA funding needed to implement and manage these programs has been addressed by expanding industry-paid user fees. The FDA has increasingly accepted less data and more surrogate measures, and has shortened its review times.


Assuntos
Aprovação de Drogas/legislação & jurisprudência , Regulamentação Governamental , Legislação de Medicamentos/tendências , Preparações Farmacêuticas/normas , United States Food and Drug Administration , Aprovação de Drogas/economia , Aprovação de Drogas/estatística & dados numéricos , História do Século XX , Legislação de Medicamentos/história , Estados Unidos
12.
BMJ ; 367: l5766, 2019 10 23.
Artigo em Inglês | MEDLINE | ID: mdl-31645328

RESUMO

OBJECTIVE: To determine the extent to which late stage development of new drugs relies on support from public funding. DESIGN: Cohort study. SETTING: All new drugs containing one or more new molecular entities approved by the US Food and Drug Administration (FDA) between January 2008 and December 2017 via the new drug application pathway. MAIN OUTCOME MEASURES: Patents or drug development histories documenting late stage research contributions by a public sector research institution or a spin-off company, as well as each drug's regulatory approval pathway and first-in-class designation. RESULTS: Over the 10 year study period, the FDA approved 248 drugs containing one or more new molecular entities. Of these drugs, 48 (19%) had origins in publicly supported research and development and 14 (6%) originated in companies spun off from a publicly supported research program. Drugs in these groups were more likely to receive expedited FDA approval (68% v 47%, P=0.005) or be designated first in class (45% v 26%, P=0.007), indicating therapeutic importance. CONCLUSIONS: A review of the patents associated with new drugs approved over the past decade indicates that publicly supported research had a major role in the late stage development of at least one in four new drugs, either through direct funding of late stage research or through spin-off companies created from public sector research institutions. These findings could have implications for policy makers in determining fair prices and revenue flows for these products.


Assuntos
Ensaios Clínicos como Assunto/economia , Aprovação de Drogas/economia , Setor Público/economia , Pesquisa Médica Translacional/economia , Ensaios Clínicos como Assunto/estatística & dados numéricos , Estudos de Coortes , Aprovação de Drogas/legislação & jurisprudência , Aprovação de Drogas/estatística & dados numéricos , Humanos , Patentes como Assunto/estatística & dados numéricos , Setor Público/estatística & dados numéricos , Pesquisa Médica Translacional/estatística & dados numéricos , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência , United States Food and Drug Administration/estatística & dados numéricos
17.
Pediatr Blood Cancer ; 66(8): e27809, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31144772

RESUMO

It is well appreciated that the number of anticancer drugs approved for use in children is a fraction of the number approved for use in cancers that occur in adults. We address this fact by summarizing the relevant U.S. legislation that provides the framework for the evaluation and approval of drugs used to treat children with cancer. In total, the Food and Drug Administration (FDA) has approved 38 new drug applications for pediatric oncology indications, 12 of which were new molecular entities. FDA continues to collaborate with multistakeholders regarding the development of products intended for pediatric cancer and encourages the submission of marketing applications.


Assuntos
Antineoplásicos/uso terapêutico , Aprovação de Drogas/legislação & jurisprudência , Aprovação de Drogas/estatística & dados numéricos , Neoplasias/tratamento farmacológico , Criança , Humanos , Estados Unidos , United States Food and Drug Administration
20.
J Am Acad Dermatol ; 81(3): 867-877, 2019 09.
Artigo em Inglês | MEDLINE | ID: mdl-31103566

RESUMO

The Orphan Drug Act of 1983 (ODA) put in place a set of financial and marketing incentives to stimulate the development of drugs to treat rare diseases, and since its passage, more than 600 orphan drug and biologic products have been brought to market in the United States. Rapid growth in orphan drug approvals in conjunction with high orphan drug prices have triggered concern that drug makers are exploiting certain aspects of the ODA for financial gain and that some pharmaceutical drugs are receiving orphan status where it is not warranted. The landscape of approved therapies for rare skin diseases has not been well described. In this article, we provide a descriptive analysis of the United States Food and Drug Administration-approved orphan drugs for the treatment of rare dermatologic conditions and skin-related cancers since the enactment of the ODA. We discuss policy issues that emerge from the analysis and suggest areas for future research. Next, we elucidate ODA loopholes using dermatologic drugs as examples and propose potential reforms. Finally, we consider future directions for orphan drug development in the field of dermatology.


Assuntos
Fármacos Dermatológicos/uso terapêutico , Aprovação de Drogas/estatística & dados numéricos , Produção de Droga sem Interesse Comercial/estatística & dados numéricos , Doenças Raras/tratamento farmacológico , Neoplasias Cutâneas/tratamento farmacológico , Aprovação de Drogas/legislação & jurisprudência , Humanos , Motivação , Produção de Droga sem Interesse Comercial/legislação & jurisprudência , Políticas , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudência
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