Urgent action needed to reduce high costs of gene therapies

By Jane Byrne

- Last updated on GMT

© GettyImages/ipopba
© GettyImages/ipopba

Related tags Gene editing CRISPR

The extremely high costs of gene therapies are unsustainable, and a global commitment to affordable, equitable access to these treatments is urgently needed, concluded the organising committee of a conference on human genome editing.

The Third International Summit on Human Genome Editing took place on 6-8 March 2023 at the Francis Crick Institute, London UK.

Building on previous events held in Washington, DC (2015) and Hong Kong (2018), the London meeting continued the global dialogue on somatic and germline human genome editing. Major themes for discussion included developments in clinical trials and genome editing tools such as CRISPR/Cas9, as well as social, ethical and accessibility considerations these scientific developments entail.

The event was organised by the Royal Society, the UK Academy of Medical Sciences, the US National Academies of Sciences and Medicine and The World Academy of Sciences.

The summit’s organising committee, chaired by Professor Robin Lovell-Badge, released a statement summarizing the discussions at the meeting.

They said remarkable progress has been made in somatic human genome editing, demonstrating it can cure once incurable diseases. But to realise its full therapeutic potential, research is needed to expand the range of diseases it can treat, and to better understand risks and unintended effects.

As interventions based on somatic genome editing become more widespread, a commitment to equitable, financially sustainable, and accessible treatments becomes more urgent, argued the organizers.

“In many cases, costs and infrastructure needs of current gene therapy treatments are not manageable for either patients or healthcare systems. Correcting this will require appropriate planning from the earliest stages of the research and development for each potential application.”

And gene therapy research needs to include more genetically diverse populations and the range of those who conceive and conduct the research also should be expanded to achieve more equitable outcomes, said the experts. 

By the end of 2023, innovative cell or gene therapies may have received approval for use in Europe, the US, or both.

December last year saw the US Food and Drug Administration (FDA) green lgiht the first gene therapy for patients with a high-risk form of bladder cancer, following the US regulatory body's approval of the first gene therapy for hemophilia B​ ​in November 2022.

And there are huge number of others in development, supporting the FDA’s forecast that it would be approving 10-20 new therapies annually by 2025.

Although authorities in the US and Europe are moving forward with reforming their regulatory frameworks in response to scientific advancements, barriers to patient access remain a major issue in both regions, reads a market research​ report.

The EU will update its pharmaceuticals legislation; the proposal is anticipated this month, and it could have significant impact on whether European patients have access to advanced therapy medicinal products (ATMPs) for years to come by balancing affordability and access, among other issues.

Promising results

Numerous clinical trials using somatic human genome editing are in progress or soon to be initiated, with preliminary but encouraging results that point to future therapies.

“The dramatic improvement following CRISPR-based research interventions for sickle cell disease offers hope for patients. Many techniques, including base, prime, and epigenetic editing, may also prove to be useful interventions for a broad range of both genetic and acquired diseases and disorders.

“However, as with other gene therapies, extended long-term follow-up is essential to fully understand the consequences of an edit and to identify any unanticipated effects, should they occur. Improved techniques have enhanced the efficiency, precision, and accuracy of the editing process, yet effective delivery and editing remains difficult for many tissues of the body.

“Further research to diversify and increase the efficiency, specificity, and safety of editing-delivery systems is essential for improving potential treatment options and promoting equitable access.”

Somatic human genome editing refers to the editing of somatic cells, which are non-reproductive cells, and changes made in these cells affect only the person who receives the genome editing.

Heritable human genome editing

As regards heritable human genome editing, they said this technology remains unacceptable at this time.

“Public discussions and policy debates continue and are important for resolving whether this technology should be used. Governance frameworks and ethical principles for the responsible use of heritable human genome editing are not in place. Necessary safety and efficacy standards have not been met.”

The scientists stressed that governance mechanisms for human genome editing need to protect ongoing, legitimate research, while preventing clinics or individuals from offering unproven interventions in the guise of therapies or ways to avoid disease.

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