GLOSSARY
OF ORPHAN DRUGS AND RARE DISEASES
AELMHU has compiled in this glossary the most frequently used terms related to the orphan drugsThe aim is to help in the understanding and knowledge of this type of treatment, its research, development, approval and financing.
Authorisation to market a medicinal product. In Spain, the body that grants authorisation to market a medicinal product is the AEMPS.
A code or registration number that identifies each presentation of a medicinal product. It is an element of unequivocal identification and traceability. The code consists of seven digits and is displayed on the two main faces of the outer packaging.
A public law corporation that represents, coordinates and cooperates with the Pharmaceutical Profession, both at national and international level.
It is the first step in the development of any orphan medicinal product but involves approval of the use of the medicinal product for the designated condition. This designation is granted by the Committee for Orphan Medicinal Products and Medicinal Products (COMP) of the European Medicines Agency (EMA).
Documentation on a product that the company submits to the DGCSF in order to initiate the negotiation of financing by SNS.
Clinical research that generates high quality knowledge to develop therapeutic tools that contribute to the prevention, alleviation and cure of diseases and to the improvement of the quality of life of the population.
Clinical study other than a clinical trial.
It is a private health establishment of public interest, expressly defined by state and regional laws, from which the community pharmacist ensures the population's access to medicines and health products, helping patients in the correct process of safe, effective, efficient and responsible use, getting involved in the achievement of health results.
Pharmaceutical speciality concerned with serving the population's pharmaceutical needs, through the selection, preparation, acquisition, control, dispensing, information on medicines and other activities aimed at achieving appropriate, safe and cost-effective use of medicines and medical devices, for the benefit of the patients treated in the hospital and its sphere of influence.
The science and activities relating to the detection, assessment, understanding and prevention of adverse effects of medicinal products or any other drug-related problems.
Monitoring the side effects and efficacy of medicines, once marketed.
Acronyms and
abbreviations on orphan drugs and rare diseases:
Marketing Authorisation.
Cost-Benefit Analysis
Cost-Effectiveness Analysis
Cost-utility analysis
Spanish Agency for Medicines and Health Products.
Primary Care.
Quality Adjusted Life Years
Researcher's Charter and Code of Conduct for the Recruitment of Researchers.
Catalan Health Service.
Autonomous Communities.
External Scientific Committee.
Internal Scientific Committee.
Research Ethics Committee.
Clinical Research Ethics Committee.
Committee on the Ethics of Research on Medicinal Products.
Committee for Medicinal Products for Human Use .
Centre for Biomedical Research Network on Rare Diseases.
AEMPS online medicines information centre.
Interministerial Commission on Medicines Prices.
National Code.
Committee for Orphan Medicinal Products and Medicinal Products.
Committee for Orphan Medicinal Products
Contract research organisation.
These are reference centres, services or units.
Directorate General for the Common Portfolio of Services of the National Health System and Pharmacy.
Directorate General for Public Health.
Hospital Diagnosis.
Clinical trial.
Special Medical Control.
Clinical trials.
Rare diseases.
Rare diseases.
European Medicines Agency.
Rare disease
European reference networks.
US Food and Drug Administration.
Spanish Federation for Rare Diseases
Clinical Practice Guideline.
Institute for Rare Disease Research.
Institute for Health Research.
Principal Investigator.
Therapeutic positioning reports.
International Rare Diseases Research Consortium.
Carlos III Health Institute.
Orphan Drug
Orphan drugs.
National Health Service
National Institute for Health and Care Excellence
Open Access.
Orphan Designation.
Data Management Plans.
Royal Decree.
Spanish Society of Hospital Pharmacy.
Galician Health Service.
National Health System.
Child Protection System.
Reference Price System.
Marketing Authorisation Holder.
European Union.
Hospital use.
Sources used:
Spanish Agency for Medicines and Health Products
Portal on rare diseases and orphan drugs (Orphanet)
Centre for Biomedical Research on Rare Diseases Network (Ciberer)
General Council of Pharmaceutical Associations
Spanish Federation for Rare Diseases
SEFH Working Group on Rare Diseases and Orphan Drugs
AELMHU has compiled in this glossary the
terms most commonly used around the
orphan drugs, with the objective of
to help in the understanding and knowledge of
this type of treatment, its research,
development, approval and financing.
