Regulatory Focus™ > News Articles > New Changes Meant to Simplify, Incentivize Orphan Drug Applications in the EU

New Changes Meant to Simplify, Incentivize Orphan Drug Applications in the EU

Posted 09 May 2013 | By Alexander Gaffney, RAC

The European Medicines Agency (EMA) has announced that it will, starting immediately, be instituting new changes meant to simplify the process by which fees for orphan medicine applications are paid to the agency.

So-called "orphan drugs" are those meant to treat a debilitating or life-threatening disease or condition affecting fewer than 6 in 10,000 people in the EU on average, or similar conditions for which there is no "satisfactory method" of treatment, prevention or diagnosis.

One of the primary incentives given to sponsors and manufacturers of orphan drugs is a reduction in the fees ordinarily required of applicants. Such fees can be prohibitive, and the reduction is meant to keep companies-and particularly smaller ones-better capitalized and more profitable, and therefore more likely to want to invest in bringing an orphan drug product to market.

"Medicines that have been granted orphan designation by the European Commission on the recommendation of the Agency's Committee for Orphan Medicinal Products (COMP) are eligible for fee reductions for a range of regulatory activities," EMA explained. "These include pre-authorization activities such as protocol assistance, the application for marketing authorization and inspections."


But is the eligibility process itself holding back companies as well? EMA certainly seems to think so.

In a statement released on 8 May 2013, EMA said that it had "cancelled the [fee reduction] procedure" presently used to request a reduction from the agency, and voided the related document, Procedural Advice on Fee Reductions for Designated Orphan Medicinal Products. A similar standard operating procedure (SOP/H/3048) is similarly "obsolete," EMA said.

Regulators said that going forward, they will no longer require any additional information from the sponsor in between the grant of an orphan designation and the submission of a fee reduction application.

The changes come into effect immediately.

EMA's statement may be found here.

Tags: EU

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