Although 2012 saw a rise in new drug approvals, the proportion of promising leads that fizzle out is still depressingly high. And of those that do make it to market few, if any, will achieve blockbuster status.
The low-hanging fruit was picked long ago, but there are still plenty of orphan diseases for which there is no treatment, whose existence was marked on Rare Disease Day on 28 February. It is estimated that there are 6,000–8,000 orphan diseases, affecting 30 million people in the EU alone. The global market is said to be growing at a CAGR of almost 6% and to have a value of $100bn.
By definition rare diseases have small patient populations, so it is unlikely that sales will cover the development cost. Not only do some governments offer incentives to encourage research into orphan medicines, but the EMA’s committee for orphan medicinal products is also working with EUnetHTA, which promotes scientific cooperation among Europe-based health technology assessments, to recommend which medicines can be reimbursed by EU national healthcare systems.
According to EURORDIS, an alliance representing 561 rare disease patient organisations in 51 countries, 80% of rare diseases have identified genetic origins, providing rich potential for the gene therapies that are now starting to progress into the clinic. The EMA’s committee for advanced therapies predicts a surge in market approval applications over the next five to 10 years.
However, as with any potential new drug, there are many hurdles between early phase discovery and commercialisation, and with such novel technologies there is not yet an established business model for taking these advanced therapies through to commercialisation.
With thousands of diseases and millions of patients in need of treatment, cutting edge research in need of partners to develop it, and pharma companies in need of new market opportunities, perhaps the solution to some of these issues has been hiding in plain sight and is now ready to move centre stage.