Most health authorities promote the development of orphan drugs, by offering a series of incentives to companies developing treatments or diagnostics for rare diseases.

Orphan Drug Designation

An Orphan Drug Designation is a status assigned to medicines developed for rare condition. ODD are grounded on proven medical plausibility of the orphan condition, and potentially significant benefits of the proposed treatment.

ODD in the European Union

A medicinal product can be designated as orphan if it fulfils the following three criteria:

  • It is intended for the treatment, diagnosis or prevention of a life-threatening or chronically debilitating condition;

  • The prevalence of such indication in the European Community is below 5 per 10,000 persons at the time of the application, or the marketing of the medicinal product would unlikely generate sufficient returns to justify the investment needed for its development;

  • No satisfactory method of diagnosis, prevention or treatment of the concerned condition can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Several incentives are associated with ODDs, such as market exclusivity, fee reductions and protocol assistance – a type of scientific advice specific to designated orphan medicines.

ODD applications are evaluated by the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP), which provides an opinion on whether or not the medicinal product fulfil all the requirements to be qualified as an orphan medicine for the treatment, prevention or diagnosis of a rare disease. Orphan Drugs Designations are granted by the European Commission, based on COMP opinion.

Annual reports need to be submitted to the EMA, reporting on the status of development of the medicine. At the time of marketing authorization application, the maintenance of the orphan status is assessed in order to ensure the medicinal product is still eligible for a 10-year market exclusivity incentive.


Latest Glossary Definitions

Efficacy-Effectiveness Gap

The observed discrepancy between effects of a health intervention in routine clinical practice as compared with the effects demonstrated in randomised controlled clinical trials.

Briefing Book (or briefing materials)

Documentation submitted to facilitate an Agency meeting, for example to request scientific advice in relation to a proposed drug development pathway. It should contain company questions, justifications or company positions, and appropriate summary information.

Clinical Impact

The effect that something is likely to have on the treatment of a particular group of people, or on the results of treating that group.

Commercially Confidential Information (CCI)

In relation to Policy 70, CCI shall mean any information contained in the clinical reports submitted to EMA by the applicant/MAH which is not in the public domain or publicly available and where disclosure may undermine the legitimate economic interest of the applicant/MAH.

Clinical Evaluation Report (CER)

Required under the new MEDDEV 2.7.1 Rev. 3 guidelines. Document containing information and results generated in conducting an evaluation of the clinical safety and performance of a medical device.

CE-Marking Technical File

A CE Technical File is a comprehensive collection of documents providing medical device composition, specifications, manufacturing process, rationale for qualification and classification, non-clinical testing results and clinical evaluations, risk analysis and instructions for use/Labeling.

Comparative Effectiveness Research (CER)

The conduct and/or synthesis of research comparing different benefits and harms of alternative interventions and strategies to prevent, diagnose, treat, and monitor health conditions in routine clinical practice (i.e. the real-world setting).

Core Value Dossier

Core value dossiers are used in the frame of reimbursement negotiations and to support HTA submissions, as well as national and local payers.

Core Reimbursement Dossier

A core reimbursement dossier is submitted during the late stage of development of a product, in order to be considered for reimbursement.

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