Takeaway 1

Are we ready? It depends who you ask

Simone Boselli- Patient perspective

Quick summary

Patients, regulators and clinicians are ready for gene therapies. HTA agencies are gaining experience. Policy makers and payers are not ready. Some people may be anti-gene therapy because of a fear of this new technology.

Gene therapies that target the root causes represent a great hope for people living with rare diseases.

Simone Boselli- Patient Perspective

Patients and clinicians are readyPatients are ready for gene therapies. They have high expectations and they are empowered to seek transformative therapies. Patients are interested in reducing delays to accessing gene therapies Regulators and HTA bodies are gaining expertiseA number of gene therapies have been approved and reimbursed in recent years. Through this process regulators and HTA bodies have gained expertise. This expertise will be useful in the future as new gene therapies come to market.

The "political class" are not readyPoliticians and policymakers are risk averse and have many other concerns. This group also focus on short-term budget cycles and they are not used to considering opportunity costs or the potential cost savings a technology may bring over the long-term.There is also a lack of understanding of gene therapies among policymakers and politicians.

We don’t have unlimited resources so it is always about making choices, and if you do A you can’t do B.

Mary Harney- Policy Perspective

Some people may be anti-gene therapyPeople working closely with gene therapies, who understand the technology and its limitations could easily forget that for complicated and invasive technologies, public opinion could sour.Similar to anti-vax and anti-GM crop movements, there is a potential for a backlash against gene therapy if cautiousness around safety morphs into disinformation and speculation.

Have your say. Are we ready for gene therapies?

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