BLOG: Pharma Finally Goes Rare! Well Done?
by Pharmacy Life - Published on 23 September 2015
Last week, Professor Stephen Hawking opened the inaugural Cambridge Rare Disease Summit and this week it has been announced that the ninth international Rare Disease Day will take place on February 29 next year.
But what exactly constitutes a ‘rare disease’ and why is big pharma increasingly interested in something that is so ‘rare’?
By definition, a rare disease is defined by the European Union as one that affects fewer than five in 10,000 of the general population. One in 17 people will be affected by such a disease at some point in their lives, equating to approximately 3.5 million people in the UK and 30 million across Europe.
In the UK, a single rare disease may affect up to about 30,000 people, although the vast majority of rare diseases will affect far fewer people. Some will, in fact, only affect a handful - or even a single person - in the whole of the UK.
At present, it is believed that there are between six and eight thousand known rare diseases, of which 80 per cent will have a genetic component. Many are chronic and life threatening. More than seventy per cent affect children, and 30 per cent of rare disease patients will die before their fifth birthday.
In the Seventies, only a handful of rare disease drugs were approved, but the 1983 Orphan Drug Act helped create interest by providing drug reviews, and benefits for drug companies. Since then, approximately 220 medicines have been approved – 93 of them in the last decade alone - and there are currently more than 450 in development.
Why the sudden upsurge? Well, for decades, pharma was reluctant to invest in rare disease treatments, preferring to focus on the mass-market drugs for common problems such as heart trouble. But, more than a decade ago, as patents on some of the most lucrative drugs began to expire, and cheaper generic drugs started wiping out billions in revenue, attention began to turn to rarer diseases – a move, which will not only put a smile on the faces of those in pharma, but on those of those affected by these diseases.
For a start, the mapping of the human genome - combined with sophisticated and affordable genetic tests - means that scientists are better placed than ever before to diagnose, treat and potentially cure rare genetic disorders. Also, where drugs for common conditions often require thousands of patients and need reviews that can take a decade and cost more than $1 billion, drugs for rare diseases are often only tested on a few dozen people, so testing is much quicker and approval is nearly assured.
Needless to say, pharma is already seeing a return on its investment. Pfizer, for example, made $3.53 billion last year selling rare-disease treatments and, according to GlobalData, it’s estimated that, by 2019, 34 rare disease drugs could have annual sales exceeding $1 billion!