Did you know that orphan drugs are those that are designed to treat so-called rare diseases? Well, over the past 20 years, research has made a leap forward with regard to new drugs. In any case, much remains to be done, since only 5% of these diseases are treated.
The World Health Organization estimates that today there are about 7,000 different rare diseases that affect patients in terms of their physical and mental abilities, as well as their sensory and behavioral qualities. And according to data compiled by the Spanish Federation of Rare Diseases, FEDER is estimated that 3.5 to 5.9% of the world’s population, more or less, will be affected by any of these pathologies, which is equivalent in Spain. more than 3 million citizens.
Over the past two decades, the area of pharmaceutical research has made great strides in the development of orphan drugs. This is possible thanks to the fact that at the beginning of 2000, the Official Journal of the European Union published the Orphan Medicines Regulation to provide solutions for the needs of 30 million European patients who live with one of the so-called rare diseases or infrequently.
And the fact is that before the approval and implementation of this Regulation, therapeutic research in this area was limited, mainly due to the complexity of research on these drugs and the fact that these pathologies affect very few people. people, thereby increasing the economic risk of their development.
Remarkably only eight drugs for rare or rare diseases were authorized European Medicines Agency (EMA) until 2000. Thus, stimulating research in this area was at that time a vital task: the development of regulations on orphan drugs provided an encouraging response to those diseases that did not help them. … His impulse was a very significant stimulus until it reached 184 drugs for the treatment of almost a hundred different pathologies, already in 2019, along with more than 2,100 orphan designations, that is, new indications for rare diseases authorized by the EMA for existing drugs.
So while in 2019, out of 30 drugs with new active ingredients approved by the EMA, seven were intended for treatment considered neglected, which means that one in four new drugs was for patients with rare diseases. But of course in this sense 2018 was the most fruitful year, when 22 new treatments were introduced, which was an important milestone in this area.…
IN Regulation of Orphan Medicines also benefited the rest of Europe by helping better understanding of rare diseasesneeded to develop new treatments, has helped to establish 24 reference networks, and 23 Member States have developed national plans to control this type of disease.
In addition, 220 companies were created, accounting for 51% of the orphan drugs approved in Europe, and patient work and associative movement are advancingwhich in turn improves communication and knowledge of these pathologies among healthcare professionals, researchers, pharmaceutical companies and health authorities.
To move all of this work forward, the Regulation includes incentives such as expediting the approval process, creating a dedicated EMA assistance protocol and guidelines, and providing financial assistance for clinical trials, and guarantees a period of market exclusivity after a new drug is approved in 10 or 12 years years if the drug is approved for use in pediatrics.
The rule was clearly reflected in the Spanish clinical trial scenario. According to data compiled by the Spanish Registry of Clinical Trials (REEC), coordinated by the Spanish Agency for Medicines and Health Products (Aemps), of over 4,000 ongoing trials during 2019, 837 of them are devoted to rare diseases, more than 4200 patients took part in them.…
But promoting R&D funding is key, especially when you consider that only three out of ten new drugs pay off the R&D investments made by pharmaceutical companies, while the average cost of a trial for a new drug is around 2,500 million euros.… BUT NONETHELESS, This ongoing investment in research has benefits for health, economy and society.…
Over the past two decades, the Regulation has worked to create tools that provide the necessary incentives for the research, development and commercialization of treatments for rare diseases such as atrophic lateral sclerosis (ALS), cystic fibrosis or disease. by Fabry.
It is thanks to this support that the orphan drug industry is one of the areas in which the pharmaceutical industry is paying more attention. Farmaindustria, which represents the pharmaceutical industry in Spain, and Efpia, which does it in Europe, highlight the benefits provided by policymakers, such as a system of government incentives for small companies in the sector that research these drugs for rare diseases.
For the Goal 2027 platform, promoted by the Spanish Federation for Rare Diseases (Feder) and the International Consortium for Research on Rare Diseases, health systems must work to ensure that “all rare disease patients receive an accurate diagnosis within a year, approve a thousand new treatments and develop methods, allowing to assess the impact of diagnoses and applied treatment methods on patients ”.