"A disease is regarded as rare when it affects a small proportion of the population, more specifically, no more than five people out of every 10,000 people.
The term "Orphan drugs," which first appeared in Regulation EC No. 141/2000 to identify drugs used to treat an "orphan" disease, often genetic pathologies, is considered of low impact for manufacturing companies, which are known to evaluate research and large-scale investments.
Today, many rare diseases have received proper attention from research and pharmaceutical companies thanks to the renewed commitment of both the US and European governments, but many other pathologies that have a low impact on the population are still "waiting" for effective treatments, due to a lack of experimentation and research results. Access to the market is frequently hampered by both bureaucratic issues and safety concerns.
Advancement in science is slow, particularly when it comes to evaluating side effects. All the results obtained so far are due to "facilitations" provided to industries producing orphan drugs by health policies mindful of resource redistribution to benefit those affected by rare diseases who, as citizens and taxpayers have every right to access "specific and personalized” care aimed at improving the quality of life and survival.
For these reasons, spending on orphan drugs accounts for only about 5% of all pharmaceutical spending in Italy. This low spending puts patients at risk of access to care and of excessive expenses as more people seek treatment for given rare diseases.
To encourage the growth of the "orphan drug sector," there is a need for a serious economic and political commitment to patients affected by these diseases. It is a problem of social fairness to balance the demands of industry and current legislation with this ethical and then political dilemma. In fact, all patients have the right to adequate care, regardless of whether they are dealing with illnesses that affect a small percentage of the general population.
This is an interview of Prof Antonio Giordano for Sud Reporter, click here to read the original interview in Italian.