Governments worldwide have performed huge efforts to improve patient coverage for their health systems
These programs include easy access to most of the medicines used for some treatments focused on high-prevalence diseases (i.e. the number of individuals affected by a disease in a given period of time), which are commonly known and studied. This leads to the creation of programs that aim to prevent these diseases, as they are considered a public concern that could affect most of the population.
There are, however, diseases that cannot be found in the previous category, diseases whose low-prevalence characteristics are not considered when developing prevention or inclusion plans as they are not deemed a “public concern” because of the low numbers of people affected. This makes it difficult for patients in many countries to obtain the therapies they require.
This is a public problem also related to the research and development process of various laboratories in the private industry, where few pharmaceutical companies deem feasible the costly development phases for these kinds of products. There are even cases where the product is developed but is never marketed in some countries because of the small size of the target market, which would not justify the investment in marketing and commercial authorizations. Even worse, some medicines obtain the necessary authorizations for use in a particular country, but because of their low sales levels they are withdrawn from the market, creating shortages in the process and leaving patients untreated. The above summarizes why nowadays we speak of orphan diseases: they affect such a small part of the population that no pharmaceutical laboratory wants to invest in research and development for their treatment.
This situation means that worldwide, even while having a diagnosis for their diseases, millions of patients are unable to obtain access to the medicines which will allow them to improve their quality of life.
In recent years, health systems worldwide have used different ways to grant their patients legal access to these medicines. Some of these methods are gaining importance today, as we hear more and more terms such as “compassionate use” or “named patient”. These methods are used to provide patients with the means to obtain products that do not have marketing authorization or registration in their countries, or experience some kind of shortage, by allowing them to bring them from different countries that have authorized their use.
If you are currently in the latter category, we want you to know that you are not alone, and that we will do our best to put in your hands the therapy you require.
This is our reason for being.