Eamon Scanlon (Sligo-Leitrim, Fianna Fail) | Oireachtas source

I commend Deputy Brassil on introducing the Bill to reform the HSE's reimbursement process by establishing a unique process for assessing orphan drug for reimbursement in Ireland. There are around 7,000 rare or orphan diseases for which no authorised or satisfactory method of treatment exists. Orphan drugs treat, diagnose or prevent diseases that affect fewer than five in 10,000 people. Many are life-threatening or debilitating, and they particularly affect the very young, who often do not survive beyond adolescence. Without treatment, the quality of life of these patients is seriously affected and their lives may be shortened. This represents the grim reality they and their families are facing.

There have been great advances in developing orphan drugs but at present they are assessed under the same guidelines and procedures as standard drugs despite the clear differences between the two.

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Other EU countries have adapted their processes for this very reason. It is time we did so.

The purpose of the Bill is to put in place a structure for the HSE to appropriately assess orphan drugs when making a relevant decision regarding adding an item to the reimbursement list. The Joint Committee on Health recommended considerable change to the evaluation process of orphan drugs. It agrees that the current assessment is not effective and that this process neglects to ensure the best outcome for patients. Reforming the process for assessing the impact and benefit of orphan and rare drugs would be a fairer way of ensuring people get the treatment they need and deserve. Much-needed orphan drugs would reach and benefit patients with rare fatal genetic diseases sooner. It is not acceptable that citizens suffering from severe illnesses are forced to take to the streets or go public in the media to have their voices heard.

We read daily about or meet patients who suffer from severe, often life-threatening, rare diseases who cannot secure access to medicine. Some notable examples of orphan drugs, on which we have all made representations previously, include Orkambi for cystic fibrosis, CF, Vimizim, which overcomes the deficiency causing mucopolysaccharidosis type IV, or Morquio syndrome, and Respreeza, the life-changing drug for emphysema caused by Alpha-1. Orkambi is a game-changer because it is the first drug to treat the underlying cause of CF in over half the CF population in Ireland. The successful funding of Orkambi will result in more research and development into rare disease drugs, not just for those with CF but also for those with other rare diseases.

In short, the funding of Orkambi will save more lives and not only within the CF community. Vimizim is by no means a cure for the rare Morquio disease but it has increasingly enhanced the quality of lives and ensured people have more energy and improved health. The disease causes lifelong problems including heart disease, skeletal abnormalities, vision and hearing loss and respiratory issues. Vimizim is the only medicine available to treat the condition. It replaces the enzyme that the body is missing. Finally, Respreeza is the only therapy clearly shown to slow the progression of emphysema caused by Alpha-1, resulting in a reduction in the frequency and severity of chest infections and associated hospital admissions, and, therefore, increased life expectancy. This Bill brings a long overdue and credible solution to the ongoing crisis surrounding access to medicine for patients with rare diseases in Ireland. I again commend Deputy Brassil on his work in respect of this Bill.

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