Like many medical institutions around the world this week The Lancet (Haematology) ,an offshoot of the well known medical journal, featured articles about rare diseases.
The main article covered the usual issues facing those 300 million around the world living with one or more of 6000 or so rare diseases. Issues such as difficulty in getting a diagnosis and the lack of treatments. Only one in 20 of those diseases has a treatment.
Frequently progress is only made through patient advocacy for research and awareness. Resulting in legislation to deal with this specific social injustice by incentivising pharmaceutical developments.
Then facing the economic reality that breakthrough therapies are prohibitively expensive. Ravulizumab for Paroxysmal Nocturnal Haemolgobinuria PNH is cited as an example in the article because of its $450k cost per year.
The Lancet article goes on to discuss the issues in low to medium income countries, including China.
It is hard to imagine given its current priorities of handling the coronavirus crisis but China has begun to act for its rare disease population. Although it does not have a rare disease health policy like many other countries the Lancet reports that some progress is evident.
In 2018 China issued its first list of 121 rare diseases, known as The Chinese Rare Disease List. And yes atypical haemolytic Uraemic syndrome is one of those 121 diseases, as is PNH , more information can be read here.)
A year later China’s drug administration agency acted to speed up the approval of orphan drugs by changing the law so that evidence from drug trials conducted outside of China could be used in its drug evaluation and approval process.
Also of interest and found elsewhere it has been reported that in January 2020 the China National Medicine Product Administration (NMPA) has approved a Phase 3 trial of Samsung Bioepis’ SB12, which is bio similar of eculizumab. The trial will just be for the treatment of PNH but it is a hopeful sign for Chinese aHUS patients.
Samsung Bioepis and Affamed Pharmaceutics have an agreement that the latter will take responsibility for getting Chinese Government approval and then will have exclusive marketing rights to the drug , which is called AMT 904 in China. Singapore may also benefit from it.
No one knows how many aHUS patients there are in China , possibly between 2000 and 3000. Just some of the 16 million plus rare disease patients in China. 16 million is lot of people but only a small number compared to a population of around 1.4 billion. Many are rare in China but there is a glimmer of hope for aHUS.