Orphan medicinal products are medicinal products that have been designated as orphan due to the rarity of the diseases they treat. Measures were first put in place to create incentives for developing orphan medicinal products in America in 1983. Incentives are required, as there are more than 5000 identified diseases that affect less than 0.05% of the population for which there are currently no satisfactory treatments. This US initiative has proved so successful in stimulating research that Japan, Singapore and Australia have introduced similar regimes as has the EU in the form of Regulation 141/2000/EC on orphan medicinal products (Orphan Regulation).

The Orphan Regulation establishes a community procedure for the designation of orphan medicinal product status and provides incentives to encourage the research, development and marketing of designated orphan medicinal products. The Orphan Regulation also looks to encourage Member States to adopt similar or complementary incentive measures at national level.

Since the introduction of the regime, in the EC, over 155 applications have been submitted for designation of orphan status and to date 78 of these have been approved, and only 2 applications have been rejected.

For a drug to be granted orphan medicinal product status the sponsor must apply to the EMEA prior to submitting an application for a marketing authorisation.

The application should be formatted subject to the guidelines produced by the Committee for Orphan Medicinal Products (COMP), the EMEA committee established by the Orphan Regulation is to advise and administrate all issues regarding orphan medicinal products.

The criteria for orphan designation are set out in Article 3 of the Orphan Regulation, which states that:

• the product must be intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that either:

  affects less than 0.05% of the Community population; or
  without incentives it is unlikely that the medicinal product would generate sufficient return to justify the expenditure;

• a satisfactory method of diagnosis or treatment must not already exist.

The success of the regime is in the main dependant on the allure of the incentives offered. They must be such that enough assistance is offered to ensure that the profits from marketing an orphan product balances the outlay of research, development and other related costs.

The Orphan Regulation introduced a number of community wide incentives including:

• Market exclusivity

• Fee waivers

• Protocol assistance

• Direct access to centralised marketing authorisation application procedure

To complement the incentives introduced by the Orphan Regulation, many Member States are beginning to adopt national measures.

At present there are not a large number of additional incentives. However the number is increasing. Many Member States have introduced fee waivers in respect of applications for Marketing Authorisations, the UK included. A number of Member States are also looking to introduce further measures. For example France has initiated an extensive research programme, the “Genome”, which includes research into genetic diseases, and has drafted legislation which will provide tax exemptions for companies developing orphan medicinal products. Also the Netherlands have introduced and have drafted proposals for a vast number of measures, from developing a structure for co-ordinating research and bringing together patient organisations, to extending a subsidy programme which will look for opportunities to invest in orphan medicinal products.

Presently in the UK there are no additional incentives other than that of fee waivers for applications to the MCA.

The introduction of the Orphan Regulation nearly 2 years ago has stimulated research into orphan medicinal products immensely, and the introduction of additional measures at national levels can only serve to increase this.

This article first appeared in IPeye, the intellectual property newsletter published by Eversheds Solicitors. For more information about, or to receive copies of IPeye, please contact Janet Knowles on 0161 831 8207 or 0113 243 0391 or at janetknowles@eversheds.com.

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