In the context of the Interdisciplinary Thematic Networks of the University of Lisbon (ULisboa) and with the objective of defining the University’s strategy for research in the area of Health Sciences and Technologies, the redeSAÚDE (Health Network) aims to promote the development of research of excellence, taking into account the challenges posed by national and international programmes supporting research in the field of Health. It focuses on the joint development of complementary research strategies involving the different Schools and Research Units of ULisboa, seeking to reinforce their activities and potential. It is particularly relevant to promote interdisciplinarity, bridging the gap between complementary areas of knowledge for the innovation process in Health. In addition, it seeks to establish platforms through which projects with critical mass can bring together the public, private and social sectors, in order to Increase their impact and contribute significantly to society.

The network promotes ULisboa’s capacity in areas of excellence related to Health and seeks to identify where to leverage ULisboa’s potential in order to promote a healthy living system and to respond to the scientific challenges related to innovation in products and services, including the health care sector. It also contributes to the development of an interface between Science and Public Policies that stimulates collaborative action to meet national and international objectives, activating a strategic network of partnerships in the area of Health.

In January 2017, a new Cycle of Conferences of the Interdisciplinary Thematic Networks of Ulisboa began. This cycle takes place in the various Schools of Ulisboa, aiming to involve all lecturers and researchers of the university, thus contributing to the development of research of excellence in the strategic areas of intervention of the University.

On 6 February, another conference of the Interdisciplinary Thematic Network was held in the Egas Moniz Building of the Faculty of Medicine of the University of Lisbon (FMUL) on the topic “Rare Diseases and Orphan Medicinal Products: Challenges and Opportunities“.

This theme brought together representatives of the various schools of ULIsboa: Medicine, Pharmacy, and Sciences, among others. The introduction of the topic was the responsibility of Professor Bruno Sepodes of the Faculty of Pharmacy and President of the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), who moderated the entire debate on Orphan Medicinal Products.

The European Commission (EC) has adopted Regulation No 141/2000, which has created conditions for the development of orphan medicinal products, and, as a result, since 2000 the Committee for Orphan Medicinal Products has evaluated about 2,500 applications for designation as a medicinal product orphan. Orphan medicinal products are intended for the diagnosis, prevention or treatment of a life-threatening disease that is chronically debilitating and affects up to five out of every 10,000 citizens in the European Union or, alternatively, a life-threatening disease that is serious and chronic, and which, without adequate incentives, the marketing of this medicinal product will hardly to be able to generate revenues that will provide the return on the investment needed for its development.

Professor Maria José Diógenes, holder of a PhD in Neurosciences from the FMUL and Assistant Professor at the same faculty, presented the topic “Research on Rett Syndrome in Ulisboa“.

The study conducted by the Professor at the Institute of Pharmacology and Neurosciences in partnership with the Institute of Molecular Medicine and with other European experts, is based on the evaluation of changes in the control of synaptic activity in pathologies such as Rett Syndrome.

This was followed by the presentation of the topic “New approaches to the treatment of metabolism hereditary errors: from enzyme replacement therapy to pharmacological chaperones“, by Professor Paula Leandro, Assistant Professor of the Faculty of Pharmacy of Ulisboa.

Dr Nuno Oliveira Lages, President of the Portuguese Association of Bio-Industry Companies (P-BIO), presented the topic “The Portuguese industry and support to the development of orphan medicinal products“, in which he spoke about the creation and growth of national start-up companies in promoting their national and international business development. This national investment is the key element for the development and support of Biotechnology in Portugal. The Association he represents is fundamental in the interconnection between business and relevant government partners, investors, regulatory agencies and other institutions linked to the industry.

Most of the innovation we have witnessed regarding orphan medicinal products results from significant advances in biotechnology, for which reason investment in this area will continue to represent a unique opportunity for the national competitive potential in the development of new drugs. The focus on research and its link to industry is an undeniable way of attracting investment and creating therapeutic innovation with a positive impact on the national economy.

Raquel Moreira

Editorial Team

news@medicina.ulisboa.pt