Expanding patient access for Orphan Drugs in Asia Pacific - vast differences in patient access to Orphan Drugs exist around the world and unfortunately Asia Pacific stands out for all the wrong reasons – there is a clear lack of access to these life changing medicines. For companies with commercialized orphans the patient opportunity is vast but the barriers they encounter can appear overwhelming.
Many are aware that over 370 Orphan Drugs are marketed in the US and a further 64 are marketed in Europe (as of May 2011). When looking at these lists it can be seen that there is little overlap between the two lists and therefore more than 400 marketed Orphan Drugs are potentially available to patients worldwide. Unfortunately only a small fraction of these orphans reach Asia Pacific patients.
Could the reason be that there is no patient opportunity for orphans in Asia Pacific? By definition orphan indications affect a small percentage of the population – but the reality is that vast populations live in the Asia Pacific region and this means many patients are not getting treatment. The figure shows that in the selected Asia Pacific countries below 2 billion people currently live (Vs less than 310 million in the US and less than 510 million in Europe). Even if we ignore China just three countries (Indonesia, the Philippines and Vietnam) account for more than 400 million people – many of whom are living with rare diseases.

If we extrapolate for one orphan indication e.g. we look at the prevalence of idiopathic pulmonary fibrosis (estimated from the literature at between 14-42 per 100,000 of population) we can see that somewhere between 275,000 patients and 826,000 patients are currently suffering from idiopathic pulmonary fibrosis across the region – this represents a huge potential patient pool with no readily available medicine.

Even the smallest countries such as Singapore and New Zealand each have between 600 and 1800 patients who could benefit from treatment if therapy was available and funding achievable. It is therefore clear that many patients could benefit from Orphan Drugs and that lack of patient opportunity is not the major reason for why we find very low penetration of orphans in Asia Pacific.
This blog posting is part of a series of articles that focus on the opportunity and the challenges faced when bringing Orphan Drugs to patients in Asia Pacific. If you would like more information about the series please contact giles@aftpharm.com.





