Overcoming Challenges Facing Advanced Therapies in the EU Market

Abou‐El‐Enein, Mohamed; Elsanhoury, Ahmed; Reinke, Petra

doi:10.1016/j.stem.2016.08.012

Cited by 116 publications

(115 citation statements)

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“…For example, when delivered through viral mechanisms they can be tumorigenic and can give rise to proliferation in tissues that have not been intentionally targeted [12]. They can also stimulate immune reactions, requiring immunotherapy, adding to overall risks [13].…”

Section: Safety Evidencementioning

confidence: 99%

Gene therapy: evidence, value and affordability in the US health care system

et al. 2018

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Aims:To explore the challenges presented by gene therapies, discuss potential solutions, and present policy recommendations. Methods: A review of the literature and series of expert interviews were conducted and discussed at a Policy Forum convened by The Institute for Clinical and Economic Review (ICER). The Policy Forum was attended by independent experts and senior representatives from 20 payer organizations and life sciences companies. Results: Three categories of challenges are identified: evidence generation, assessing value and affordability. Possible solutions and policy recommendations are presented for each of the three main categories of challenges. Conclusions: Gene therapies present exciting opportunities, but also pose major challenges. Dialogue between manufacturers and payers around the issues and possible solutions is crucial.

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Section: Safety Evidencementioning

confidence: 99%

Gene therapy: evidence, value and affordability in the US health care system

et al. 2018

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“…Institutional interests are also likely to be important: existing studies have illustrated that some organizations have strategically positioned themselves as 'pioneers' in RM [25]. And, uncertainties surrounding evidence of clinical and cost-effectiveness have already hindered the adoption of some advanced therapies (specifically ChondroCelect R and MACI, competing products for treating cartilage defects) [25,38]. The complexity and novelty of RM products and techniques, however, presents an additional set of more specific adoption challenges.…”

Section: Specific Adoption Challenges Relating To Rmmentioning

confidence: 99%

Anticipating the Clinical Adoption of Regenerative Medicine: Building Institutional Readiness in the UK

2018

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This perspective paper examines the challenges of implementing regenerative medicine (RM) therapies within hospitals and clinics. Drawing on recent work in the social sciences, the paper highlights dynamics within existing healthcare systems that will present both hindrances and affordances for the implementation of new RM technologies within hospitals and clinics. The paper argues that identifying suitable locations for cell-and gene-therapy treatment centers requires an assessment of their institutional readiness for RM. Some provisional criteria for assessing institutional readiness are outlined, and the paper will suggest that it is necessary to begin developing a program for the phased introduction of RM in the longer term. Regenerative medicine (RM) therapies, particularly those that are commonly referred to as advanced cell and gene therapies, are in many ways significantly different from existing, conventional therapeutic products and techniques [1]. Cell, tissue or gene-based technologies have the potential to provide therapeutic relief for a range of disorders for which there is currently severe unmet clinical need [2], but the sensitivity and complexity of these technologies presents a range of innovation challenges [3], and commentators have suggested that promising RM technologies are particularly susceptible to the 'valley of death' [4]. Many of these challenges relate to upscaling and manufacturing, regulatory requirements, reimbursement and commissioning [5]. These have been well characterized in recent reviews of the RM field [6], and government-supported initiatives such as the UK's Cell and Gene Therapy Catapult have been launched to mitigate these and facilitate commercialization [7]. Recent reports, however, have identified an additional area of concern: the clinical adoption of RM therapies within existing healthcare systems [8,9]. Hospitals and clinics within contemporary healthcare systems have developed systems to accommodate drug-and device-based therapies and surgical interventions, and they may, then, be poorly suited to implementing cell-, tissueand gene-based treatments. There is, in other words, a degree of incommensurability between existing healthcare providers and the exigencies of providing RM therapies [10].A consequence of this mismatch is that potential investors have difficulties envisaging a clear pathway to commercial and clinical success [11]. This uncertainty represents a risk to the momentum that currently characterizes RM in the UK, Canada, various US states and elsewhere. Hence, while the majority of promising RM therapies are still in the early phases of clinical development [12], commentators have argued that it is necessary to address the potential challenges of clinical delivery in the immediate future [8,9,11]. In this vein, this paper examines the challenges of implementing RM technologies within existing systems of delivery, and it offers some guiding reflections on how such challenges may responsibly be managed. RM represents a diverse range of technologies a...

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“…Glybera, the first gene therapy approved in Europe, was introduced at a cost of €1.1 million per patient, making it the world’s most expensive medicine and resulting in disputes over insurance reimbursement. The second, Strimvelis, cost €594,000 (Abou-El-Enein et al, 2016). Although RCD for embryos would not necessarily be as expensive, it would have to be performed alongside one or more cycles of IVF ( in vitro fertilization), incurring further medical, economic, and social costs.…”

Section: Why Is the Classification Of “Enhancement” Significant?mentioning

confidence: 99%

Disease Resistance and the Definition of Genetic Enhancement

Kleiderman

Touré

et al. 2017

Front. Genet.

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Recent gene editing experiments carried out in human embryos have raised the question of whether interventions like the introduction of a CCR5-Δ32 deletion, which could provide heritable resistance to HIV infection, ought to be considered enhancements. Many authors have used the term “enhancement” in different ways, some based on patients’ biomedical outcomes and others on their social context. These classifications are often considered overly imprecise. Nevertheless, the concept of “enhancement” could affect the ways in which these applications are regulated in different jurisdictions, the availability of coverage by insurers or public health care, and the force of public opinion in shaping future policy on gene editing. In order to ethically situate resistance to communicable disease with reference to other techniques, this article provides an overview of its similarities and differences with disease gene therapy in embryos, gene therapy in consenting adults, and vaccination. In discussing key ethical features of CCR5-Δ32 deletion (including its frequency in various populations, biological mechanism, benefits for individuals, and use in previous clinical trials) we offer some potential guideposts for the continuing discussion on how to classify “enhancements” in the age of CRISPR gene editing.

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Overcoming Challenges Facing Advanced Therapies in the EU Market

Cited by 116 publications

References 10 publications

Gene therapy: evidence, value and affordability in the US health care system

Gene therapy: evidence, value and affordability in the US health care system

Anticipating the Clinical Adoption of Regenerative Medicine: Building Institutional Readiness in the UK

Disease Resistance and the Definition of Genetic Enhancement

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