We need a policy for making ‘breakthrough’ cell and gene therapies available to patients, writes Bernard Mallee
As if Covid-19 wasn’t enough, the health service was hit by a ransomware attack, delaying care for patients across the country. Hackers exposed the vulnerabilities of digital systems. As the deployment of the decryption tool continues, there is hope for the full restoration of the care pathway. When that happens, one missing piece will be cell and gene therapies. These breakthrough treatments are not yet available to patients in Ireland. We hope that can soon change.
Cell therapy replaces diseased, broken or missing cells with healthy versions. Gene therapy replaces faulty DNA to cure genetic diseases. These treatments treat, prevent and, potentially, cure diseases, whether genetic or acquired. With data so far showing transformative results, they are game-changers for patients.
Across the world, cell and gene therapies are either in use or in research for the treatment of a range of disease areas, including cancer, blood disorders, ophthalmology, neurology, and musculoskeletal, metabolic and endocrinological conditions. In the US, the medicines regulator, the Food and Drug Administration, says it expects to approve between 10 and 20 new cell and gene therapies between now and 2025. Europe’s medicines regulator, the European Medicines Agency, has approved cell and gene therapies for diseases affecting the eyes, for gastroenterology, for spinal muscular atrophy, for metachromatic leukodystrophy and for cancer.
CAR-T, a form of cell therapy, is used to treat leukaemia, lymphoma and multiple myeloma. Two CAR-Ts for three indications in cancer are, so far, available to patients in more than 15 EU countries. Irish patients are travelling to the UK to receive CAR-T treatment on the Treatment Abroad Scheme. The scheme covers the cost of treatments not available locally or where there is a delay in getting a treatment. In 2019, it cost the State €54 million.
Cell and gene therapies can add months, sometimes years, to a patient’s life, replacing a lifetime of treatment. In some cases, these treatments can save lives, especially if the intervention happens early. The treatments are expensive because research and development costs are high, with specialised production and logistics operations needed to get them from bench to bedside. Often, patient populations are small because the conditions they treat are rare. The treatments, usually one-time rather than stretched out across a patient’s life, dramatically reduce the cost of chronic care. That means they make economic, as well as clinical, sense.
Take haemophilia, an inherited bleeding disorder where a missing factor means that the blood does not clot properly. Standard treatment since the 1970s has been intravenous infusions of the missing clotting factor. Last March, the first Irish person with haemophilia B was treated with gene therapy as part of a clinical trial. We are moving closer to making gene therapy a possible functional cure for the disease. Similarly, gene therapy for ocular diseases is a significant area of research, especially since many rare, blinding retinal diseases do not have treatments now. CAR-T is showing very promising results in the treatment of cancer.
Last week, we published a pathfinder study, undertaken for the industry by the consulting firm, PwC. Pfizer, Janssen, Novartis, Bayer, Takeda, Gilead and Astellas – all biopharmaceutical companies with significant operations in Ireland – partnered with us on the study. The aim is to prompt debate, followed swiftly by action, for how best to introduce cell and gene therapies in the health services. The study shows that cell and gene therapies have achieved ground-breaking results in a range of diseases. It urges improvements in health information infrastructure, making it easier to gather and analyse patient outcomes data. We need to continue to scale the creation of centres of excellence for cell and gene therapy at certain hospital sites, with allied investment in training and engagement for doctors and patients.
There is an urgency about making cell and gene therapies available to patients in Ireland. That will require a policy that deals with the various adoption strands – how cell and gene therapies can be assessed for clinical effectiveness and value for money, and how they can be paid for through novel reimbursement models. That work is complex, requiring dialogue among stakeholders, including policymakers, doctors, patients, the regulator and the industry. But now is the time to begin. Ultimately, we will need a pathway everyone can support. The Department of Health will be a key enabler and an agent of change.
Our thinking is aligned with Sláintecare, the health plan with cross-party political support that aims for world-class standards of care. We should be open to adopting new innovations affordably and at pace in the health services. This is a vital component of Ireland’s future as a European, even global, centre for life sciences. Our enterprise mix, the concentration of biopharmaceutical, medical technology and technology players, in close proximity to universities and research centres, means we have a unique opportunity for industry-government collaboration on tomorrow’s science.
It should not go unnoticed that, in her speech to parliament earlier this month, the Queen set a goal to make the UK “a global science superpower”. Though on a much smaller scale here, we should be no less ambitious. Cell and gene therapies are at the heart of a modal shift in medicines innovation. They are a revolution, often offering better treatment options for life-threatening illnesses and, in some cases, potentially curing them altogether. As well as making Ireland a place where cell and gene therapies are available, we should be a location for investment too, with production and manufacturing centres of excellence. The industry, through ourselves and BPCI, part of IBEC, is working with IDA Ireland, the foreign direct investment agency, NIBRT, the research and training organisation, and others, on this agenda, too.
Through the PwC study, we have laid a foundation for meaningful dialogue about how best to make cell and gene therapies available locally. Now, it is time to get on with moving Ireland a step closer to realising the promise of new science.
Bernard Mallee is Director of Communications and Advocacy at the Irish Pharmaceutical Healthcare Association.
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