Organisations and
DOCUMENTS
of interest
Where can you get +INFORMATION on
rare diseases and orphan drugs?
CIBERER
The Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), dependent on the Instituto de Salud Carlos III, coordinates and promotes research on rare diseases in Spain. It is made up of 57 research groups linked to 29 consortium institutions.
BELIEVE
The State Reference Center for the Care of People with Rare Diseases and their Families (Creer), located in Burgos, has the strategic objective of achieving better care for people affected by these minority pathologies.
ERDERA
ERDERA (European Rare Disease Research Alliance) is a European association that brings together more than 170 public and private organizations from 37 countries with a single goal: to turn cutting-edge science into tangible benefits for the 30 million Europeans living with a rare disease.
EURORDIS
It is a non-profit alliance of more than 1,000 rare disease patient organizations from 74 countries, working together to improve the lives of more than 30 million people living with a rare disease in Europe.
ERDF
The Spanish Federation of Rare Diseases (FEDER), which brings together more than 370 Spanish entities, promotes the rights of those living with a rare disease and in search of diagnosis, generating strategies that contribute to improving their quality of life.
OBSER
This is the Observatory on Rare Diseases, a FEDER project that was created with the aim of sharing and generating knowledge about the needs of patients with rare diseases.
Orphanet
Created and coordinated by INSERM (French National Institute of Health and Medical Research), it is a portal where you can find information on types of rare diseases, a list of approved orphan drugs, a directory of associations of patients with rare diseases, reports, clinical trials, etc.
CSIC Rare Diseases Network
This network of research groups from centers of the Spanish National Research Council (CSIC) aims to lead and coordinate research efforts in the field of rare diseases in Spain.
RELEVANT DOCUMENTS
Strategy sponsored by the Ministry of Health, Equality and Social Services, which includes seven lines of action, such as prevention and early detection of rare diseases, health and social and health care, promotion of research, and training and information for professionals, affected persons and their families.
Royal Decree 1015/2009, of 19 June, which regulates the availability of medicines in special situations. The aim of this regulation is to facilitate access to investigational medicinal products for patients who do not have a satisfactory treatment and suffer from a seriously debilitating or life-threatening disease.
seriously debilitating or life-threatening illness.
Link where you can find Laws, Royal Decrees, Orders and derogations on clinical trials in Spain.
Document explaining the functioning of the European medicines regulatory system.
Guidance to help plan for and better understand the challenges that researchers will encounter as they move forward in the development of such new orphan therapeutics.
Unit to develop epidemiological research on rare diseases in order to broaden knowledge and improve the quality of information on these pathologies.
Methodological guide to economic evaluation applied to orphan drugs published by the Max Weber Institute in 2015, to assist in decision-making on funding, access and rational use of treatments for rare or low prevalence diseases.
The book, published in 2016, seeks to address the ethical issues inherent to any experimental study conducted on human subjects and the ethical issues specific to rare diseases.
AELMHU is not responsible for the content of these links.
