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Life Science Innovation 2021

We have a chance to realise the promise of cell and gene therapies

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Bernard Mallee

Director of Communications and Advocacy, Irish Pharmaceutical Healthcare Association

In recent years, there has been a steady acceleration in the number of cell and gene therapies in development and authorised for use by patients. They should be available to patients in Ireland and it should be an investment location for manufacturing and supplying these breakthrough treatments, too.

Cell and gene therapies are a revolution in science. Cell therapy replaces diseased, broken or missing cells with healthy versions. Gene therapy replaces faulty DNA to cure genetic diseases. These therapies treat, prevent and potentially cure diseases, whether genetic or acquired. With data so far showing transformative results, they are game-changers for patients.

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. In the US, the Food and Drug Administration says it expects to approve between 10 and 20 new cell and gene therapies over the next four years.

Replacing a lifetime of treatment

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.

Cell and gene therapies can add months, sometimes years, to a patient’s life, replacing a lifetime of treatment.

Ireland at the forefront of cell and gene therapy

Ireland has a chance now to be an adopter of cell and gene therapies, aligning with most other countries in Western Europe. We should look to the investment opportunity in manufacturing, production and supply chains for these treatments. Both the traditional commercial and manufacturing models for medicines need a re-think when it comes to cell and gene therapies. How we make them, supply them and pay for them are major challenges that demand collective action.

The industry recently commissioned PwC to examine ways to integrate the treatments into the care pathway. Related work is under way, in conjunction with IDA Ireland and NIBRT and our industry through IPHA and BPCI, to understand how we can create centres of manufacturing excellence. 

Bringing all this work together requires an initiative led by central Government. Unlocking the potential of cell and gene therapy landscape requires cross-stakeholder dialogue, especially between the State, the industry, doctors and patients. This is a matter of urgency.

The pace at which Ireland can translate scientific breakthroughs in cell and gene therapies into better healthcare outcomes and new jobs will depend on how quickly stakeholders can come together in common purpose.

Let us work towards realising the clinical and economic promise of one of the most radical developments in modern science.

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