Dr. Juan Francisco Cabello, member of the Advisory Commission on Rare Diseases of the Ministry of Health, referred to the case of the boy Tomás Ross, diagnosed with Duchenne Muscular Dystrophy, and expressed the importance of addressing these issues with a focus on seeking transversal solutions.
It should be noted that Tomás’ mother managed to collect three thousand 500 million pesos in voluntary contributions, to pay for an expensive therapy that is only available in the United States and that could be of great help to your child.
Although its clinical efficacy has not yet been confirmed, it continues to generate debates about the reality that hundreds of people and families affected by this and other rare diseases face in Chile and to what extent the public health system or the State should be involved in this type of causes.
The latter became evident this Wednesday, after the woman met with President Gabriel Boric.
During the audience granted to him at the La Moneda Palace, and in which the Minister of Health, Ximena Aguilera, also participated, The president promised not to charge the taxes associated with the acquisition of said drug.
Furthermore, to evaluate some actions aimed to improve the system of access to high-cost treatments for rare pathologiesamong them the eventual creation of an organization that favors timely attention to health technologies with high budgetary impact.
The importance of addressing rare diseases as a state issue
For the neuropediatrician Juan Francisco Cabello, professor at the School of Medicine of the University of Valparaíso, who is a member of the Advisory Commission on Rare Diseases of the Ministry of Health, In these types of situations there are no absolute points of view or answers but rather multiple perspectives.
Added to the above, because beyond the objective criteria that should always be present when analyzing this type of matter, As a human being, it is impossible to reason without ceasing to be moved by anguish and pain. that those who suffer from these pathologies experience, especially when it comes to children.
In parallel, the expert in diseases and congenital errors of metabolism, maintains that the medical duty It also requires you to address these issues always considering the available scientific evidence.precisely to honor that same principle: that of respect, empathy and concern for the patients involved.
“The option to acquire the drug Elevidys, which has been conceived as a gene therapy for children with Duchenne Muscular Dystrophy, “It has ended up conditioning everything linked to this case and other similar ones in Chile.”said Dr. Cabello.
“Since groups of people and even some politicians who learned of its existence from others or read on the Internet that this drug was approved by the Federal Food and Drug Administration (FDA) of the United States, and that it can be purchased in that country, began to advocate that Elevidys could also be marketed in our country or, at leastso that the State acquires it, with a view to supplying it through the public health system”, he added.
The real discussion
Likewise, Juan Francisco Cabello suggests that the first discussion that should really be taking place now in Chile on this issue, and in a calm manner, is that until the efficacy and safety of a medication that was approved by a foreign regulatory authority is demonstrated, Even though she enjoys prestige, This should not be marketed nationally. Even less when it comes to drugs that only have a temporary or accelerated approval.
Secondly, it suggests that the relevance of creating or improving an institutional framework capable of generating better clinical trial designs should also be discussed, with a view to lowering the costs of the final products that arise from them, as long as these studies achieve the primary objectives of the investigations, that is, improve the symptoms of the disease.
“The State must protect those who suffer from rare diseases, but also guide them appropriately when it comes to opting for these types of alternatives, which are real today”he indicates.
Faced with this dilemma, the professor at the School of Medicine of the University of Valparaíso finally proposes that instead of evaluating the possibility of acquiring a medication under studyhealth authorities or the State could decide to promote or support patients affected by rare diseases as a country.
The above, so that participate in clinical trials with supervision, where medicines are provided free of charge by the manufacturer and where the risks and potential benefits are clearly disclosed from the beginning.
