NPC Patients Ask for Medication Reimbursement

Niemann-Pick Patients Ask for Medication Reimbursement   

Patient association for Niemann-Pick Disease had its inauguration ceremony on Sep. 07, 2006 and at the same time, the medical team from medical  genetics department of National Taiwan University Hospital also brought us a good news that Miglustat, which was originally used to treat Gaucher Disease Type I, can also be used to treat Niemann-Pick Disease Type C, as the clinical study result showed. Two patients participating in the clinical study have shown great improvement in mobility, feeding, swallowing ability, and intelligence.  

Dr. Wuh-Liang Hwu, chairman of Medical Genetics Department, NTUH and Dr. Yin-Hsiu Chien showed a film about the patients' improvement at the opening ceremony of Niemann-Pick Patient Association.  After receiving Miglustat for some time, A-Shen is able to slowly walk with assistance and able to eat food, instead of being fed through a stomach tube.

Even though this is great news for Niemann-Pick patients, there are still some issues regarding their medical rights.  Currently, Miglustat is still under clinical trials and hasn't received market approval from the U.S. Food and Drug Administration for Niemann-Pick Disease. Following in its footsteps, Taiwan National Health Insurance also hasn't included Miglustat as one its orphan drugs. Therefore, there's no official reimbursement from NIH.  Furthermore, the Miglustat used in the clinical study was donated by a pharmaceutical company and will be running out at end of September.  Without NIH reimbursement, it will cost one patient NT$300,000 (more than US$9,000) per month to receive treatment.  

It is estimated that the U.S. FDA will approve Miglustat for Niemann-Pick Type C in 1.5 year.  Patients can't wait that long.  We can't help worrying that patients may soon lose the life-saving medication and will be forced to give up treatment due to the high cost of the drug before the government approves reimbursement for Miglustat. 

The Niemann-Pick Patient Association and TFRD hope that the government can see the patients' needs and that in the foreseeable future, Miglustat can be approved to treat both Gacher Diease and Niemann-Pick Type C disease.  Because of our requests, the Bureau of Health Promotion has promised that for cases like this, doctors can apply for their patients 
about the medical cost before approval and they will provide necessary assistance.