Orphan drugs currently account for around 10 per cent of global pharma sales, according to consultancy Alcimed. However, the company predicts that the market for these drugs will grow seven per cent a year to reach $127bn in 2018.
So, what do doctors see as the challenges in developing drugs for rare diseases, which affect five people or fewer in 10,000, and how can pharma work with them more effectively?
We spoke to Doctors.net.uk member Dr Tony Dhillon, who is a consultant clinical oncologist and focuses on relatively rare cancers such as GIST (gastronintestinal stromal tumour) and NET (neuroendocrine tumour), where the drugs are expensive and the underlying biology is still not well understood.
He said: “I would like to see pharma companies working to improve our understanding of the biomarkers involved in these diseases so that when they organise clinical trials they can find candidates who they know will respond to treatment. Without this research, patients are wasting their time being on the trials and a lot of money is wasted.”
Dr Dhillon believes there is a role for pharma in helping to educate patients about their condition and that providing information is probably the best way to help them. He says there are a quite a few companies working in these disease areas and a couple of them fund useful information for patients, including Lilly, which provides a DVD.
Asked 'what is the single biggest thing he would like to see pharma doing to make his job easier', Dr Dhillon said: “To produce cheaper treatments. The data on a new oral drug, regorafenib (Stivarga), for example, shows it can delay the progression of advanced GIST in patients who are resistant to treatment. I would like to use it but it has not been found to be cost-effective.”
He concluded: “To be honest, apart from providing information to patients I feel uneasy about drug companies moving beyond their role of providing treatments. Their job is not to provide healthcare. I know I am not alone in feeling slightly sceptical about their motives in this area.”
By Dr Tim Ringrose, CEO of M3 Europe
For more information on M3, the global provider of technology services in healthcare, and its new European Division which includes www.doctors.net.uk, www.mdlinx.com, and www.networksinhealth.com, please call Tim Ringrose on +44 (0)1235 828400, or email Tim.firstname.lastname@example.org