REPOSITORY ITEMS

08-06-2021
Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Estimating the budget impact of orphan medicines in Europe: 2010 - 2020
"Annotated by Carlos Campillo-Artero Servei de Salut de les Illes Balears. Centre de Recerca en Economia i Salut. Universitat Pompeu Fabra and Dr. Julio Martínez Clinical Coordinator. Head of the Pharmacy Service. Vall d'Hebron University Hospital. Barcelona".

"To avoid difficulties in accessing these medicines, a European framework of consensus on pricing and financing appropriate to the special characteristics of the population and orphan drugs must be designed.

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

08-06-2021
Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research.
Albert Figueras Professor, Department of Pharmacology, Therapeutics and Toxicology. Autonomous University of Barcelona

When knowing how to say "no" enhances the value of "yes".

Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research.

08-06-2021
Sustainable public health systems for rare diseases
Marta Trapero-Bertran. Lecturer and researcher at the Universitat Internacional de Catalunya (UIC).

"Sustainability of the system without taking into account economic evaluation?"

Sustainable public health systems for rare diseases

08-06-2021
Marketing authorisation of orphan medicines in Europe from 2000 to 2013
AELMHU

The causes are related to adherence to European Medicines Agency guidelines and the size of the laboratories developing these drugs. This study retrospectively compares marketing authorisation applications for orphan medicinal products versus applications for products without this designation that have been submitted by the European Medicines Agency (EMA), through its centralised procedure, between 2000 and 2013, i.e. during the first 14 years of the existence of the orphan drug regulatory framework in the European Union. All authors are directly linked to the EMA, except two of them, who belong to CeMSIIS (Centre for Intelligent Systems, Medical Informatics and Statistics) at the Medical University of Vienna.

Marketing authorisation of orphan medicines in Europe from 2000 to 2013

30-11-2020
Schooling of children and young people with rare diseases
Yolanda Ahedo Infante and Montserrat Cabrejas del Campo

Children with RD have difficulties in the school environment and educational needs of varying type and degree. Knowing them, understanding them and attending to them appropriately helps to reduce school failure and dropout, and ensures the maximum development of these pupils' abilities.

Schooling of children and young people with rare diseases

31-08-2020
Rare diseases and COVID-19: clinical and care implications
María Luz Couce Pico

Patients with rare or minority diseases constitute a population at risk for COVID-19. This implies that they must strictly follow the measures recommended by the health authorities to prevent infection, and at the same time there must be a very careful follow-up with specific protocols by the healthcare professionals who care for them, including the measures to be taken in each case whether they are infected by COVID-19 or by their underlying pathology.

Rare diseases and COVID-19: clinical and care implications

29-06-2020
Policies and actions to tackle rare diseases at European level. Ann Ist Super Sanita
AELMHU

No single country has the knowledge and capacity to treat all types of rare diseases, but thanks to international cooperation, best practices are available to all patients in the European Union.

Policies and actions to tackle rare diseases at European level. Ann Ist Super Sanita

21-05-2020
Epidemiology of rare diseases: a cartographic approach to the study of their mortality in Spain
Verónica Alonso Ferreira, Germán Sánchez Díaz, Manuel Posada de la Paz and Francisco J. Escobar Martínez

The medical care of people affected by rare diseases (RD) represents a significant burden and an additional challenge for health systems, compared to some common diseases. The specific characteristics of RD, such as the low number of patients, their diagnostic complexity, high morbimortality and lack of specialised knowledge, among many others, together with the methodological difficulties derived from them, make these conditions a distinct group of scientific interest and high impact. The more traditional epidemiological research aimed at identifying and distributing the determinants of RD continues to be a pending issue, which limits scientific knowledge and, above all, the planning of resources and their cost, making this group of diseases a public health problem.

Epidemiology of rare diseases: a cartographic approach to the study of their mortality in Spain

13-02-2020
Developing interactions with industry in rare diseases: lessons learned and continuing challenges.
AELMHU

"This article describes the experience of collaboration between the biopharmaceutical industry and the Urea Cycle Disorders Consortium (UCDC); and how that collaboration has favoured the development of new treatments and diagnostic methods useful in the management of this group of rare diseases. "

Developing interactions with industry in rare diseases: lessons learned and continuing challenges.

20-01-2020
Alternative access schemes for pharmaceuticals in Europe: towards an emerging typology
AELMHU

"Alternative access methods are a phenomenon of increasing frequency and importance in the European Union. This paper systematically classifies and analyses them and develops a conceptual framework to advance their study".

Alternative access schemes for pharmaceuticals in Europe: towards an emerging typology