The International Society for Cell and Gene Therapy (ISCT) has formed a working group to address concerns over the misuse of expanded access in the cell and gene therapy (CGT) sector.

As with many other sectors, the pandemic influenced the CGT space and expanded access. There was an increase in companies offering unproven CGT and declaring its benefits against Covid-19 as a preventative or post infection treatment.

While there are some legitimate reasons why CGT could help with Covid-19 infection, such advertising put many patients at risk, says Dr Aaron Levine, member of the ISCT Expanded Access Working Group, and bioethics vice-chair of the ISCT Committee on the Ethics of Cell and Gene Therapy (ECGT).

The ISCT Expanded Access Working Group aims to ensure that the development of CGT proceeds in an ethical manner and that the expanded access route is being used for its intended purpose.

Expanded access allows patients, who are terminally ill or have exhausted all available treatment options, to access preapproved investigational treatments outside clinical trials. The ISCT Working Group will investigate whether expanded access for CGT is being abused by companies for commercial gain by selling unsafe medications to patients.

The Working Group intends to help regulators, physicians, and patients navigate around potentially unsafe therapies by providing guidance and resources, identifying practical and ethical issues, and developing a code of ethics for the CGT stakeholders.

“I hope that the products of the Working Group help move CGT sector into the direction that maximises the ethical development and minimises the risks,” Levine says.

To an extent, expanded access is marketed as a way to promote therapies that are not proven, says Levine, who is also associate professor in the School of Public Policy at Georgia Institute of Technology. One of the key ethical issues is patient autonomy and informed consent. When seeking expanded access within the CGT sector, patients’ decision-making can be compromised by misleading and insufficient information about the potential risks.

Potential misuse hinders ongoing trials, complicates regulatory approval, and slows the development of CGT. Moreover, patients who choose to access unproven CGT are not eligible to participate in clinical trials later, thus hampering safety and dosing data that is needed for further research.

According to the ISCT, in 2018 the unproven CGT industry was worth up to $2.4 billion, with around 60,000 annual patients spending approximately $40,000 per treatment.

While ISCT primarily focuses on the United States, it intends to investigate the expanded access to CGT more globally in the future. An open discussion about the potential misuse, as well as advantages and disadvantages of expanded access in US context will help inform other countries, says Levine.

Clinical Trials Arena has previously reported on expanded access in the US.

Cell & Gene Therapy Coverage on Pharmaceutical Technology supported by Cytiva.

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