Moving somatic gene editing to the clinic: routes to market access and reimbursement in Europe

Rigter, Tessel; Klein, David; Weinreich, S.; Cornel, Martina C.

doi:10.1038/s41431-021-00877-y

Cited by 8 publications

(6 citation statements)

References 14 publications

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“…This renders the generation of evidence of the product’s safety and efficacy more difficult. Personalised technologies may need to be evaluated—and regulated—not as medicines, but as health services ( 17 ). In a services-based regulatory model, it would not be the product, but rather the service that is evaluated and approved for use.…”

Section: Panel Discussionmentioning

confidence: 99%

Ethics of Early Clinical Trials of Bio-Artificial Organs

2022

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Regenerative medicine is the new frontier in the field of organ transplantation. Research groups around the world are using regenerative medicine technologies to develop bio-artificial organs for transplantation into human patients. While most of this research is still at the preclinical stage, bio-artificial organ technologies are gearing up for first-in-human clinical trials in the not-too-distant future. What are the ethical conditions under which early-phase clinical research of bio-artificial organs can be conducted safely and responsibly? What lessons can be learned from prior experiences with early-phase clinical trials in adjacent fields of research? This is a Meeting Report of an online international workshop organised in the context of the Horizon 2020-funded VANGUARD project, which is developing a bio-artificial pancreas for the treatment of patients with type 1 diabetes.

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Section: Panel Discussionmentioning

confidence: 99%

Ethics of Early Clinical Trials of Bio-Artificial Organs

2022

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“…There are multiple alternative medicine-to-patient routes in the European regulatory framework, to which varying national rules apply, particularly regarding reimbursement (Rigter et al, 2021). Hence, it is imperative that the relevant national rules should be reviewed and, if applicable, corresponding organizations should be engaged from the onset to ensure patient access, e.g.…”

Section: Alternative Routes and Reimbursementmentioning

confidence: 99%

Access to medicines for rare diseases: A European regulatory roadmap for academia

et al. 2023

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Background: Novel or repurposed medicines for rare diseases often emerge from fundamental research or empirical findings in academia. However, researchers may be insufficiently aware of the possibilities and requirements to bring novel medicinal treatment options to the patient. This paper aims to provide an easily applicable, comprehensive roadmap designed for academic researchers to make medicines for rare diseases available for patients by addressing the relevant regulatory frameworks, including marketing authorization and alternative routes.Methods: Key points of the regulatory chapters “Placing on the Market” and “Scope” of Directive 2001/83/EC relating to medicinal products for human use were summarized. Provisions in EU directives regarding blood products, radiopharmaceuticals, and herbal and homeopathic medicinal products were excluded. Cross-referencing to other provisions was included. European case-law was retrieved from the InfoCuria database to exemplify the implications of alternative routes.Results: Medicines may only be placed on the market with a valid marketing authorization. To obtain such authorization in Europe, a “Common Technical Document” comprising reports on quality and non-clinical and clinical studies must be submitted to a “competent authority”, a national medicine agency or the European Medicines Agency. Timely interaction of academic researchers with regulators via scientific advice may lead to better regulatory alignment and subsequently a higher chance for approval of academic inventions. Furthermore, reimbursement by national payers could be essential to ensure patient access. Apart from the marketing authorization route, we identified multiple alternative routes to provide (early) access. These include off-label use, named-patient basis, compassionate use, pharmacy compounding, and hospital exemption for Advanced Therapy Medicinal Products.Discussion: Aligning academic (non-)clinical studies on rare diseases with regulatory and reimbursement requirements may facilitate fast and affordable access. Several alternative routes exist to provide (early) pharmaceutical care at a national level, but case-law demonstrates that alternative routes should be interpreted strictly and for exceptional situations only. Academics should be aware of these routes and their requirements to improve access to medicines for rare diseases.

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“…Clinical trials are also expensive, and many products do not make it through to approval. While CRISPR is sometimes described as ‘efficient, simple and cheap,’ the cost of taking a single genome-edited product through these research and clinical trials phases will be considerable – at least many millions of dollars – and sponsor-manufacturers seek to recover these costs, and to profit from that investment, when pricing the end product (Rigter et al, 2021 ). As a result, unless there is government support, the price of the product makes it out of reach for most people.…”

Section: Regulatory Issues For Human Genome Editingmentioning

confidence: 99%

Points to consider in the development of national human genome editing policy

Nicol¹,

Niemeyer²,

Paxton³

et al. 2023

Camb. prisms Precis. med.

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CRISPR and other genome editing technologies have the potential to transform the lives of people affected by genetic disorders for the better. However, it is widely recognised that they also raise large ethical and policy questions. The focus of this article is on how national genome editing policy might be developed in ways that give proper recognition to these big questions. The article first considers some of the regulatory challenges involved in dealing these big ethical, social questions, and also economic issues. It then reviews the outcomes of a series of major reports on genome editing from international expert bodies, with particular focus on the work of the World Health Organisation's expert committee on genome editing. The article then summarises five policy themes that have emerged from this review of the international reports together with a review of other literature, and the authors' engagement with members of the Australian public and with a wide range of experts across multiple disciplines. Each theme is accompanied by one to three pointers for policy makers to consider in developing genome editing policy.

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Moving somatic gene editing to the clinic: routes to market access and reimbursement in Europe

Cited by 8 publications

References 14 publications

Ethics of Early Clinical Trials of Bio-Artificial Organs

Ethics of Early Clinical Trials of Bio-Artificial Organs

Access to medicines for rare diseases: A European regulatory roadmap for academia

Points to consider in the development of national human genome editing policy

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