Ireland an outlier without access to new cell and gene therapy, report says

Irish patients will not be able to access expensive cutting-edge medicines without a rethink of the reimbursement model used to pay for new treatments, according to a report published on Thursday.

Ireland is currently almost alone among western European countries in not granting patients access to modern cell and gene therapy treatments, it says.

Money is a major drawback, the Pathfinder Study on the Adoption of Cell and Gene Therapies in Ireland acknowledges. Finding new ways to pay for drugs will also require new ways of measuring their value to patients and to the health system, it argues.

Cell and gene therapies are usually one-time treatments that can add months, sometimes years, to a patient's life, replacing a lifetime of treatment, the report commissioned by the Irish Pharmaceutical Healthcare Association (Ipha), which represents the major research-driven drug companies, states.

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It accepts that the upfront cost of such treatment is very high but it “could reduce the long-term direct and indirect costs of chronic treatment for certain illnesses, improving patient outcomes”.

Genetic diseases

Cell therapy replaces diseased, faulty or missing cells with healthy versions. Gene therapy replaces faulty DNA to cure genetic diseases. These breakthrough treatments are being used to treat – and potentially in some cases cure – genetic and acquired diseases.

The European Medicines Agency, which regulates the drugs across Europe, has approved five such therapies in cancer, ophthalmology, spinal muscular atrophy and Crohn's disease, but none are available in Ireland as yet. The only other countries in western Europe in a similar position are Sweden and Luxembourg.

Funding such treatments will require a move to new models like stage payments or rebates linked to longer-term patient outcomes, and moving away from the current model of single-year health budgets, the report says. It might also require a different way of assessing value for money under health technology assessments carried out on new drugs.

New “shared risk” payment models will also require investment in digitisation by the health service to adequately track patient outcomes, the report says.

Public policy need

PwC's Shane Gannon, the report's lead author, said he hoped it would lead to healthcare policy change in the near term.

“The new therapies bring clear clinical benefits but they can be expensive. How the health system pays for them, and how their value is measured in the community, are areas for further exploration,” he said.

“We hope this study can prompt a debate among all stakeholders for how Ireland can realise the potential of new therapeutic interventions in a way that makes sense for the healthcare system.”

Ipha chief executive Oliver O'Connor said: "We must be open to adopting new innovations affordably and at pace in our health services. This study is an industry contribution to an urgent public policy need. It recognises that current reimbursement models will not work for cell and gene therapies. We need to explore how we can deliver both clinical benefit and value for money from cell and gene therapy adoption,"

"Cell and gene therapies are revolutionising medicine," said Prof David Keegan, a consultant at the Mater hospital in Dublin, and a specialist in ophthalmology. "They often offer better treatment options for life-threatening illnesses. In some cases, they have the potential to cure diseases altogether."