Most new drugs for treating rare diseases not approved for sale in Ireland

New generation drugs are better able to address the challenge of rare diseases, but many have yet to get approval for sale here, according to an industry group.

The Irish Pharmaceutical Healthcare Association (IPHA) published new figures that show the State lags other western European countries on speed of access to orphan medicines.

The association, which represents bigger pharma groups that research and develop new drugs, said that less than one-third of medicines for rare diseases that are licensed for patients in Europe are available in the Republic. That ranked the Republic in the bottom half of the list of European countries, it said, and behind all other western European Union countries, including Denmark, Spain, Sweden, Italy and Austria.

However, the industry group was unable to say whether the companies behind the drugs had all applied for approval to sell them in Ireland. It is understood that some have not.

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Orphan medicines

The UK and Germany performed best, according to the figures, scoring 92 per cent and 95 per cent respectively for availability of orphan medicines. As larger countries, with a bigger cohort of patients affected by rarer medical conditions, they would be expected to be early targets for access by drug companies.

In countries such as the Republic, with a smaller population, companies would likely assess the size of the market before applying. Given the small patient populations, rare-disease drugs are generally among the most expensive to come through the approvals process.

It is understood that over the three years to 2017, the European Medicines Agency licensed 38 drugs tackling rare and orphan conditions – defined as a disease or condition that affects fewer than one in 2,000 people. The figures were compiled by IQVIA for the European Federation of Pharmaceutical Industries and Associations, of which IPHA is a member.

Less expense

Rare diseases have become an increasingly attractive target for new medicines. Regulators in the United States have encouraged research in the area by providing orphan designation, which provides a faster and often easier passage to approval, which means less expense, and an extended period of market exclusivity.

The figures, which relate to the position at the end of last year, were published on Thursday to coincide with Rare Disease Day.