ZAVESCA for Niemann-Pick C Is Finally Reimbursed

Taiwan's regulations and subsidization for drugs considering rare diseases is 
based on the discussion of the "Rare Disease and Orphan Drug Committee". 
If the Committee believes that the drug has enough effect, it would be designated 
as orphan drug and will be reimbursed by National Health Insurance. As for the 
definition of "effective", the committee considers the decisions of the FDA and 
EMEA or the clinical trial conducted in different countries. However, creating a 
new kind of drug is difficult, and the amount of patients that actually gets the 
help is limited. 

A few years ago, the clinical trial in US and Europe showed that the drug that was 
originally for Gaucher's disease, ZAVESCA, may has positive effect on Niemann 
Pick Type C disease. In Taiwan, the Division of Genetics in National Taiwan 
University Hospital's Genetics division has been conducting similar trials since 
2005 in hopes of giving good treatments to patients in Taiwan. During the process, 
the two children shoed great improvement on swallowing and eye ball rolling. But as 
the parents grew excited, the support from drug companies were about to end. 
Though the medical team attempted to fight for it, but as the experiment was not 
completed, whether the medicine is effective remains a controversial question. 
Most of the committee members could not agree to the subsidy as the data is not 
supportive enough with only two patients that the medical team has been working 
with and the lack of data from other countries. Most of the committee members have 
medical backgrounds and their perspectives are fixed on medical terms. 

However, having always been on the patients' perspective, our foundation believes 
that regardless of the weak data support, the patients should receive the best care 
possible, as it is suppose to be the whole purpose of the laws regarding rare disorders' 
protection and medical treatment. Therefore, as our representative, vice president 
Dr. Min-Chieh Tseng, continued to fight for the subsidy, the committee ultimately 
decided to let the National Taiwan University Hospital conduct a project with 5 patients 
that lasts a year. At the same time, the committee continues to keep an eye on the p
rogress of other countries such as the United States. The finance of this experiment is 
funded by the National Health Department, our foundation, and the hospital itself. 

As we fight for the patients' welfare, we realize the importance of conducting more 
clinical trials, yet the help we could receive from the government is limited. So our 
foundation established a fund especially for clinical trials in hopes of expediting the 
process of finding new treatments. Other similar foundations also donated to the fund, 
we raised 5 million NT dollars to start with. Our foundation grants the clinical trial for 
the Niemann Pick Disease 1.5 million NT dollars, all the medical expense and testing 
was supported by this fund. 

After a year of treatment, 5 victims of the Niemann Pick Type C disease are starting to 
improve on the swallowing problems; lung problems also decreased. Their lives were 
generally improved. Moreover, It became easier for the patients to control their body 
and communicate with others. The families saw hope, so did the drug use of rare 
disorders. In December, 2008, EMEA finally announced that ZAVESCA can indeed be 
effective on the Niemann Pick Type C patients. The National Health Insurance also has 
started the reimbursement on ZAVESCA for these patients in August 2009. 

Looking back to the entire process, we found that it would have been impossible to have 
this plan go through if it was not for the support of the Bureau of Health Promotion, the 
foundation, the National Taiwan University Hospital, and everyone from each corner of 
the society. As the foundation complete its 10th year of journey, we believe that we not 
only need to pay attention to the laws and regulations regarding the patients' welfare, 
but we also have to work on the present ones to make them better and benefit more people. We believe that more and more success for other diseases will occur following the efforts we put in and the results we received.