Regulatory Developments for Nonhematopoietic Stem Cell Therapeutics

Rosemann, Achim; Bortz, Gabriela Mijal; Vasen, Fedérico

doi:10.1016/b978-0-12-811920-4.00019-7

Cited by 5 publications

(11 citation statements)

References 41 publications

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“…In the previous section we defined new non-validated practice as the use of new, Rosemann, Bortz, & Vasen, 2018). We have called this concern "epistemic crowding out of sound research" in our previous work (Mastroleo and Holzer, 2018).…”

Section: Justification: What Is the Ethical Justification Of Innovatimentioning

confidence: 99%

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Innovative Care in Latin America: Definition, Justification and Ethical Principles

Mastroleo

2019

Controversies in Latin American Bioethics

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The term "innovation" or "innovative care" has recently gained attention in the context of the use of novel and not yet fully validated medical interventions and technologies. Most notably, there have been various incidences of medical activities insufficiently validated for its regular use in healthcare that fall into this category, such as stem cell treatments, genome sequencing for diagnostic purposes, or novel reproductive technologies. Latin American countries are among the places where new and non-validated medical activities take place, notably due to a lack of clear regulations and the poor support of authorities of existent legal and ethical guidelines, which is driven by "hidden battles" on the moral status of certain interventions. The increasing importance of innovative care underlines the importance of developing a general framework for these practices. Therefore, the present chapter scrutinizes this nascent field of inquiry in Latin America and offers a conceptual framework for innovation as well as its ethical justification. As we will argue, an important use of the term "innovation" or "innovative care" is best interpreted as "new non-validated practice" and not a research activity. Then, we will defend that responsible innovation understood as responsible new

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Section: Justification: What Is the Ethical Justification Of Innovatimentioning

confidence: 99%

“…(ACOG, 2015, pp. 4-5;Rosemann et al, 2018;Taylor, 2010). Hence, crowding out of sound research is not generated by one single use of a new non-validated intervention in isolation, but by the repeated use of new non-validated interventions in an unregulated context.…”

Section: Justification: What Is the Ethical Justification Of Innovatimentioning

confidence: 99%

Innovative Care in Latin America: Definition, Justification and Ethical Principles

Mastroleo

2019

Controversies in Latin American Bioethics

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“…The Commission has been aware that there is no universal gold standard of validation. Multi-phase randomized controlled trials seek to systematically identify risks and adverse effects and assure that treatments that are to be approved are shown to be safe and effective (Rosemann, Bortz, and Vasen 2019). However, the scientific community usually accepts that sufficient validation of an intervention is not obtained only through the multi-phase trials system (Diaz and Neuhauser 2005).…”

Section: Futile Practicementioning

confidence: 99%

“…The activity of innovative practice and the concept of innovation have gained importance in medical ethics guidelines (Rietsma and Moreno 2006;Brierley and Larcher 2009;ACOG 2015;ISSCR 2016), ethical literature (Taylor 2010;Sugarman 2012;Holzer & Mastroleo 2018, 2019a, 2019bEarl 2019) and current discussions of different "right-to-try" regulations in various countries (Mastroianni 2006;Miola 2015;Dresser 2016;Fins 2017;Savulescu 2017;Joffe and Fernandez Lynch 2018;Rosemann, Bortz, and Vasen 2019). The main background concern of this literature is seizing opportunity in medicine to offer new options for patients in desperate situations and the dilemmas around it.…”

Section: Introductionmentioning

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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“…The Commission knew that there is no universal gold standard of validation. Multi-phase randomized controlled trials seek to systematically identify risks and adverse effects and assure that treatments that are to be approved are shown to be safe and effective (Rosemann, Bortz, and Vasen 2019). However, the scientific community usually accepts that sufficient validation of an intervention is not obtained only through the multi-phase trials system (Diaz and Neuhauser 2005).…”

Section: Futile Practicementioning

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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Regulatory Developments for Nonhematopoietic Stem Cell Therapeutics

Cited by 5 publications

References 41 publications

Innovative Care in Latin America: Definition, Justification and Ethical Principles

Innovative Care in Latin America: Definition, Justification and Ethical Principles

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

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