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

Monitoring Plan

Describes the monitoring methods, responsibilities, and requirements for the trial. It includes a brief description of the study, its objectives, and the critical data and study procedures.

The plan describes the reference policies and procedures to follow.

Project Management Plan

The purpose of this document is to define the scope of activities, outline responsibilities (define task and roles) and provide guidance on a specific project.

EUDAMED

Is the European Databank on Medical Devices. It’s a secure, web-based portal that acts as a central hive for the exchange of information between national competent authorities and the European Commission.

ISO14155

International standard addressing good clinical practice for the design, conduct, recording and reporting of clinical investigations carried out in human subjects to assess the safety or performance of medical devices for regulatory purposes.

Redaction

In relation to Policy 70, the process of permanently obscuring CCI or PPD in the clinical documentation submitted to EMA by the applicant/MAH before the documents are published. Tthe redaction should appear as a light blue box with a black 'PPD' label or as a black box with a red 'CCI' label.

Protection of personal data (PPD)

In relation to Policy 70, “Personal data” is any information relating to an identified or identifiable natural person; an identifiable person is one who can be identified, directly or indirectly, in particular by reference to an identification number or to one or more factors specific to his

Informed Consent Form (ICF)

Patients, and/or in specific circumstances their legally designated representative(s), are requested to give written consent before participating in a clinical trial.

Lay person summary (of a clinical trial)

The EU Clinical Trials Regulation 536/2014 (Article 37) requires sponsors to provide summary results of clinical trials in a format understandable to laypersons. These lay person summaries will be made available in the EU Portal and Database.

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