Expedited drug review process: Fast, but flawed

Chary, Krishnan Vengadaragava

doi:10.4103/0976-500x.184768

Cited by 20 publications

(20 citation statements)

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“…On the other hand, it is also useful to distinguish between new and long-standing validated interventions to better understand the specific meaning of innovation in traditional research ethics. For instance, the drug imatinib for chronic myeloid leukemia is an exemplary case of new validated practice in the early 2000s after rational drug design and sufficient validation from expedited multiphase randomized controlled trials (Druker 2009, Chary 2016. Unlike the exemplary cases of innovation, imatinib did follow the linear model of basic research to clinical research and eventually to practice (Taylor 2010, p. 286).…”

Section: D Classification Of Medical Practice Characterized By Validmentioning

confidence: 99%

Seizing Opportunity at the Limits of Medicine: An Enhanced Definition of Innovative Practice

Mastroleo¹

2019

Preprint

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The activity of innovative practice relates to seizing opportunities now for patients in desperate situations and the ethical dilemmas around it. Sadly, the term innovation has multiple meanings, adding confusion and misunderstanding to an already difficult debate. This paper aims to enhance the definition of innovative practice in two ways. Fist, we propose to curb ambiguity by replacing the term "innovative practice" with the more precise of "new non-validated practice" and by distinguishing the specific meaning of innovation at stake. To identify this meaning, we analyse the traditional research ethics’ distinction between research, validated practice, and innovation. Second, we propose the following unified definition of "new non-validated practice", that is, the first or recent use of diagnostic, therapeutic or preventive interventions that introduce a significant change (new); with an insufficient level of evidence of safety or efficacy for regular healthcare (non-validated); and with the main aim to benefit patients (practice). To avoid objections to our definition, we analyse its three core elements (significant novelty, insufficient validation for, and patients' best interests) and we present a 2D classification of medical practice that follows from our definition. If sound, our enhanced definition of "new non-validated practice" allows comparing exceptional activities in different fields of medicine that previously have been considered unconnected (e.g. compassionate use of investigational drugs, humanitarian uses of devices, novel off-label uses, etc.).  In turn, it also provides a promising conceptual tool to inform empirical research, discuss responsible access to innovative care and evaluate the regulation of innovative practice (e.g. right-to-try laws).

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Section: D Classification Of Medical Practice Characterized By Validmentioning

confidence: 99%

Seizing Opportunity at the Limits of Medicine: An Enhanced Definition of Innovative Practice

Mastroleo¹

2019

Preprint

View full text Add to dashboard Cite

show abstract

“…On the other hand, it is also useful to distinguish between new and long-standing validated interventions to better understand the specific meaning of innovative practice for medicine. For instance, the drug imatinib for chronic myeloid leukemia is an exemplary case of new validated practice in the early 2000s after rational drug design and sufficient validation from expedited multiphase randomized controlled trials (Druker 2009, Chary 2016. Unlike the exemplary cases of innovation, imatinib did follow the linear model of basic research to clinical research and eventually to practice (Taylor 2010, p. 286).…”

Section: ) Table 2 2d Classification Of Medical Practice Charactmentioning

confidence: 99%

New Non-Validated Practice: An Enhanced Definition of Innovative Practice for Medicine

Mastroleo

2019

Preprint

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A significant part of the literature on innovative practice in medicine relates to seizing opportunities and curbing harms for patients in desperate situations. Unfortunately, the term innovation has multiple meanings and a rich rhetorical flourish that adds confusion and misunderstanding to an already difficult debate. This paper aims to enhance the current definition of innovative practice for medicine. First, we replace the term innovation with the more literal of new non-validated practice. To identify this meaning, we analyse the traditional research ethics’ distinction between research, validated practice, and innovation in the Belmont Report. Second, we propose the following explicit definition of new non-validated practice: the first or recent use of diagnostic, therapeutic or preventive interventions that introduce a significant change, with an insufficient level of evidence of safety or efficacy for regular healthcare, and with the main aim to benefit individual patients. This definition is a promising conceptual tool to inform empirical research, ethicists, and the harmonization of its regulation and legislation (e.g. right-to-try laws).

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“…Further, we did not think that our claim about Fast Track designation allowing therapeutic agents to be approved based on a single phase 2 study was controversial. Previous articles discussing Fast Track designation have included similar statements 15,16 and our review of the 1998 "Guidance for Industry: Fast While more recent guidance documents, including "Guidance for Industry: Expedited Programs for Serious Conditions -Drugs and Biologics", 18 do not include similar statements, neither do they establish that these prior standards are no longer acceptable.…”

Section: Clarifications and Commentsmentioning

confidence: 99%

The US Food and Drug Administration’s expedited approval programs: Addressing premarket flexibility with enhanced postmarket evidence generation

2018

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Expedited drug review process: Fast, but flawed

Cited by 20 publications

References 10 publications

Seizing Opportunity at the Limits of Medicine: An Enhanced Definition of Innovative Practice

Seizing Opportunity at the Limits of Medicine: An Enhanced Definition of Innovative Practice

New Non-Validated Practice: An Enhanced Definition of Innovative Practice for Medicine

The US Food and Drug Administration’s expedited approval programs: Addressing premarket flexibility with enhanced postmarket evidence generation

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