Louise O'Reilly (Dublin Fingal, Sinn Fein)
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871. To ask the Minister for Health the details of the orphan medicinal products regulation; if his attention has been drawn to the monopoly that pharmaceutical companies can hold for orphan medicines; and if he will make a statement on the matter. [13431/17]

Simon Harris (Wicklow, Fine Gael)
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Orphan medicinal products are those products intended for the diagnosis, prevention or treatment of life-threatening, or very serious, rare conditions that affect no more than 5 in 10,000 people in the European Union.

EC Regulation No 141/2000 on orphan medicinal products was adopted to encourage the development and authorisation of medicines for rare conditions. The main objective of the Regulation is to ensure that patients suffering from rare conditions have the same quality of treatment as any other patient in the EU, where previously research on such conditions and development of treatments was not considered to be economically viable.

The Regulation lays down a procedure for the designation of ‘orphan status’ and provides incentives for research and development of such products and placing them on the market.

The European Commission has designated 1825 products in development as orphan medicinal products. Those companies responsible for these orphan medicines can benefit from incentives such as protocol assistance- scientific advice to facilitate the development and authorisation of innovative medicines; 10 years of market exclusivity - where similar medicines for the same condition cannot be placed on the market; and fee waivers for regulatory procedures may also be available.

The number of products available to treat rare diseases remains low but has grown significantly over the years. An assessment of the impact of the European Union's orphan drug regulation 10 years after it was enacted, concluded that it had boosted research and led to the increased availability of drugs to treat rare diseases. To date, the European Commission has authorised 136 orphan medicines for the benefit of patients suffering from rare diseases, whereas before the adoption of the Regulation there were only 8 treatments marketed for rare conditions. The incentives of the orphan drug legislation are therefore essential to facilitate the development of treatments for rare diseases.

In November 2016 the Commission published new guidance on how specific provisions of the Regulation should be applied. This guidance provides procedures and legal predictability for pharmaceutical companies on a number of aspects of the Regulation, including when more than one application for a marketing authorisation for an orphan medicinal product is received for the same condition; and clarity over determinations of significant benefit over existing treatments and similar medicinal products for the same condition.