How do we encourage the development of new cell and gene therapies without compromising our social ideals or the wellbeing of individuals? In my view, there are two significant ethical issues that must be addressed with advanced medicines: i) informed consent and ii) the cost of therapies.
Given that cell and gene therapies rely on manipulated genetic materials, there are fears that the industry lacks the knowledge to predict long-term impacts (1), which creates challenges for informed consent. Informed consent requires patients to be given all possible knowledge of what the treatment involves, including benefits, risks, and alternatives. In a sector that is still developing and changing its knowledge rapidly, such risk assessments are difficult to accurately make, and can quickly become outdated with new patient outcomes and results. For instance, the Francis Crick Institute’s attempt to develop a new germline therapy found that 16 percent of altered embryos in the lab saw mutations that would have been undetectable by conventional methods (2).
The European Commission has been pushing to overhaul current regulations to ensure they remain fit for purpose for innovative medicines and to provide clearer standards for industry leaders to assess their peer’s ethical adherence (3). Separately, the FDA released a new guideline document – “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products” – in 2022 as part of their own regulatory upheaval. The document offers a codified guideline, based on discussion from leading experts, on how CAR T focused on cancer treatments can be made safe.
Informed consent in cell and gene therapies is a topic that I expect to remain complex for the immediate future. However, regulatory reform championed through sector collaboration – and supported by leading biopharmaceutical professionals and other companies – will, in time, help address ongoing concerns and inform future developments.
Improving standards of communication and regulation within the biologics industry should also help address another crucial ethical concern with cell and gene therapies; how to ensure fair access.
Analysis from the Institute of Clinical and Economic Review indicates that the average price of a new gene therapy is $1-2 million per dose (4), and there are fears that such expenses will increase current healthcare inequalities. Without funding policies in place, treatments may only be accessible to the wealthiest sections of society.
Cost–benefit analysis is a fundamental component of new medical developments, but this is particularly resonant in regard to therapies at the cutting edge of scientific developments with very high manufacturing costs. The high prices are understandable, given the technical and specialist knowledge that underpins these developing therapies, but what if those who would benefit most from the therapies have the worst access to them? Such a concern first became apparent in 2015 when Glybera, the first gene therapy approved in Europe, was priced at €1 million per dose.
Regulatory bodies and industry associations can encourage more equitable access. In the UK, for example, the Bioindustry Association has advocated for the NHS and other healthcare organizations to implement new payment models to defer the large upfront costs of treatments through payment plans and discounts (5). The success of such an approach, however, will depend on collaboration and communication across the entire value chain – from government departments to healthcare bodies and manufacturers. Together, we must aim to create a system that balances financial motivations with ethical adherence.
An evolving biologics sector needs to ensure that its ethical regulations remain robust, while striving to distribute them fairly – all without limiting future development. Striking this balance between innovation and ethics is complex, but I am certain that greater industry cooperation is the foundation of a satisfactory answer to such questions.
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References
L Riva C Petrini, “A few ethical issues in translational research for gene and cell therapy,” Journal of Translational Medicine, 17 (2019). DOI: 10.1186/s12967-019-02154-5
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The Francis Crick Institute, “Researchers call for greater awareness of unintended consequences of CRISPR gene editing,” (2021). Available here
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Pinsent Masons, “Cell and gene therapies: resilience the aim in 2023 amidst regulatory flux,” (2023). Available here
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Institute for Clinical and Economic Review, “Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value,” (2017). Available here
- BioIndustry Association, “Ensuring patient access to cell and gene therapies: The case for an innovative payment model,” (2021). Available here
