Among the number of diseases affecting people, there are some that affect few individuals. Consequently, there is no interest from pharmaceutical companies to study and develop drugs and medicines, since it prevails the equation: few patients, few earnings.
But this was a temporally referable reality at the beginning of the millennium: rare diseases were called “orphans” because they were orphans of research, treatment and investment. But now everything has changed and we are witnessing, for some years now, a real revolution.
As reported in the Italian newspaper La Repubblica of February 26, 2019 “the supports of EU and USA for these forgotten diseases have turned the problem into an opportunity. The rare disease market, with therapies that cost an average of $ 140,000 a year per patient, have become the new goldmine of the drug giants “.
In this regard, always La Repubblica, reports the brilliant experience of the Swiss firm Roche that has bought for 4.8 billion Spark Therapeutics: The American company “has only 250 employees and 68 million of revenues in 2018 (more or less like a SME). However, it has one of the most promising products in the world of orphan drugs: Luxturna, a product able to avoid blindness in some serious eye conditions. The patients affected by this disease are not many, but the cost of therapy – about $ 450 thousand – added to the public subsidies, have made possible to discover, develop and get approved a medicine at cost of about 700 million. Thus, the small Spark turned into a jewel of multibillion dollar value”.
The rare diseases become a new business for pharmaceutical giants.