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Research finds general public are positive about gene therapies

Polling conducted across six European countries, with over 6,000 respondents, finds high levels of excitement for gene therapy and medical innovation, particularly for the treatment of rare diseases.

Over the past ten years 11 gene therapies have received marketing authorisation from the European Medicines Agency (EMA) and it is anticipated that between 10 and 20 new transformative therapies will be approved each year from 2025. 

Yet according to a major new survey of 6,250 EU and British Citizens commissioned by biopharmaceutical company BioMarin, over half (54%) of respondents are concerned that research into breakthrough medical technologies may have been paused or deprioritised over the past two years during COVID-19. Only 43% of respondents are now confident their government or local health authority will make a gene therapy available once approved for use.

The survey explored the societal and economic benefits of gene therapies, with over seven in ten (73%) European and British respondents saying that gene therapies have the potential to transform the way diseases are treated in the future. Additionally, almost 7 in 10 (69%) respondents say they are positive and excited about the possibilities for gene therapies in the future, and their potential to target the underlying cause of life-limiting and life-threatening diseases.

Approximately two thirds (62%) of respondents agree “funding of gene therapies for rare diseases should be a priority” and 64% of respondents think “governments should be prepared to make changes to the way they pay for medicines to accommodate the costs of gene therapies” if the treatment:

Key highlights from the survey across Europe include:

The findings of the survey are timely as December 2021 saw the adoption of the first-ever UN resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families” to promote and protect the human rights of all persons, including the estimated 300 million rare disease patients around the world.

For people living with rare diseases, which may have few existing treatment options, gene therapy can address the root cause, offering patients the prospect of a disease-modifying treatment with just a single administration. The treatments have the potential to transform lives in a way not seen before, by fundamentally altering the course of certain diseases and the experience of living with them.

EURORDIS-Rare Diseases Europe, and the partners of RARE IMPACT initiative, have recently shared significant concerns that patients with rare diseases may not be able to access potentially transformative gene therapies, because too many barriers currently exist. Such barriers are reported to be across four identified areas in the accessibility, assessment, availability and affordability of gene and cell therapies across the European Union. To overcome these hurdles, the group have published a new RARE IMPACT report which sets out seven solutions to improve access to gene and cell therapies for people with rare diseases. 

As well as having a potentially transformative impact for patients, gene therapy may also be able to support healthcare systems to make smart decisions about the allocation of stretched resources. This is because some gene therapies may have the potential to lessen or eliminate the need for routine treatment, thereby allowing healthcare systems to reallocate their resources to help patients who need greater care.

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