The pharmaceutical industry reaffirms its commitment to R&D in rare diseases
The number of new treatments available against rare pathologies has multiplied by 20 in Europe since 2000
The industry insists on collaboration with governments and health systems to facilitate patients’ access to medicines
Pharmaindustry joins the new phase of the European initiative #WeWontRest, focused on rare diseases
Source: www.farmaindustria.es
“We will not rest until treatments for rare diseases are not uncommon.” This is one of the main messages of the new phase of the European information initiative #WeWontRest, which this time has as main objective to raise awareness in society about the importance of continuing to work in the research and development of new therapies for patients suffering infrequent pathologies and explain the commitment that the pharmaceutical industry has for this purpose.
As in the previous stages of #WeWontRest, which started two and a half years ago in order to reach society about the pharmaceutical industry’s commitment to research and people’s health, Farmaindustria has joined this phase of the campaign through their social media profiles. In this case, it should be noted that, although very significant progress has been made since 2000, the challenge ahead is still very demanding.
The general director of the European Federation of the Pharmaceutical Industry (Efpia), Nathalie Moll, has underlined the commitment of pharmaceutical companies in Europe to the investigation of new remedies against these pathologies, as evidenced by the fact that only between 2000 and 2016 The number of clinical trials against these ailments has grown by 88%.
In the case of Spain, “the commitment of the pharmaceutical industry for the research and development of treatments for infrequent diseases is indisputable and, as the data shows, growing”, explains the deputy director general of Farmaindustria, Javier Urzay, who emphasizes “the international reference position that our country plays in clinical research ”. Thus, one in six clinical trials promoted in Spain by pharmaceutical companies already corresponds to drugs designated as orphans. These are figures obtained from the database of the BEST Project for the promotion of biomedical R&D promoted by Farmaindustria, which monitors the research activity of 50 pharmaceutical companies, four cooperative research groups and 54 hospitals in the country.
As a result of these efforts of the innovative pharmaceutical sector, from the year 2000 to 2018, 164 new treatments have been approved in Europe against almost a hundred rare diseases, while before 2000 there were only eight treatments available against these pathologies, which in their together they affect 30 million people in Europe (one in 17), half of them children. A key factor in this progress was the approval, in 2000, of the European Orphan Medicines Regulation, which established incentives for its research, development and commercialization.
Despite these advances, it is estimated that only 5% of rare diseases have an approved treatment, which shows that there is still much work to be done, given, on the one hand, the low prevalence of these diseases, and, on the other, the fact that its approach is very complex from the scientific point of view. For all these reasons, Moll defends the need for “a stable and predictable regulatory framework that allows us to continue supporting and inspiring new research”.
Promote access
But it is of little use to research and develop new medications against infrequent diseases if patients then have difficulty accessing new treatments. “Once a new drug is approved in Europe, industry, healthcare systems and governments have a shared responsibility to ensure that patients will have access to it,” says Moll, who understands that all parties have to work in a way joint to make such access as quick as possible, for example, through “flexible financing formulas”, as is already being considered in different European countries.
In the specific case of the new gene or cell-based therapies that have curative capacity and that come to replace traditional treatments that were extended throughout the patient’s life, Efpia insists that governments, health systems and The industry itself must continue to cooperate to find new ways to reconcile patients’ access to new treatments with the sustainability of the health system.
In Distefar we add to the importance of focusing efforts in the study of the treatments of infrequent diseases that affect so many people.
