Drugs no more orphans

by Massimiliano Fanni Canelles

My involvement in the serious issues that affect patients with a rare disease is linked to personal, family, professional, and cultural background to my criticism of a society increasingly stretched for maximum profit. Necessarily associative action, as well as social and political life that has developed in the Myelin Project and the Italian Committee in @uxilia, a charity created to support and provide visibility and aid to the weakest. The diversified world of rare disease affects approximately 5 people per 10,000. It means that 3% of the population suffers from one of the 8,000 rare diseases. In the European Union, an estimated 27 million to 36 million people are involved. The drama of these patients is the lack of access to diagnosis, prevention and care dedicated. The lack of commitment by the scientific community, and then by the doctors, the lack of reference centers capable of handling such a diverse series, but, more importantly, the lack of interest of pharmaceutical companies, resulting from insufficient numbers to cover the costs of research and production of drugs. Current technology would already be able to develop an effective therapy, but the rules of the market, that pharmaceutical companies are subject, prevent new drugs reach the manufacturing and marketing. These drugs are called “orphans”, as potentially useful to treat a rare disease, but without a sufficient market to support their sales with the cost of research and development. That of ‘”health industry” is still a problem with broad spectrum. There is no question now on the ethical unavailability of pharmaceutical patent and to enter the market at low prices, drugs that can save millions of lives in developing countries. Today, 75% of the world’s population is concentrated in low-income nations can only use 15% of total drug products. Of the countless molecules synthesized in recent decades, concern only a few dozen tropical diseases. In the South, 2 billion people lack access to medical treatment of any kind. In this depressing scenario, but something is changing. From the success of Italian scientific research has established an international alliance aimed at defeating seven serious genetic diseases and to make new treatments available for all patients in the world. The agreement between the Telethon Foundation, the San Raffaele Scientific Institute (HSR-TIGET) and the multinational pharmaceutical company GlaxoSmithKline (GSK) is a milestone in the difficult challenge to genetic diseases. These are mostly rare diseases, which, until now, had benefited from a limited interest by multinational drug companies. The agreement also confirms the Italian leadership in the field of gene therapy. The Convention provides that the HSR-TIGET by GSK receives an initial investment of 10 million Euros and can then qualify for additional funding due to the completion of various milestones. The multinational pharmaceutical company obtains an exclusive license for the development and commercialization of gene therapy protocols. The results assume a historic structure is offered the gift of hope to patients suffering from rare diseases and genetic and this was made possible by the Telethon Foundation. Its transparent management of funds, and the funding mechanism that rewards merit, have supported the development of the Italian research, becoming an example throughout the world. A Foundation, the Telethon, spurred in its action by patients, their families and associations in which these people come together. The partner associations are allowing Telethon cultural change necessary to bring out a research to provide care to the sick and not given to mechanisms of pure profit. People who come together for the good of your family are an example of how Italy should present itself in the health, academic research, corporate social responsibility.

Translated by Martina Delser

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