Patients suffering from a rare degenerative brain disease might soon lose funding for an expensive drug that a leading neurologist says could partially alleviate some of the effects of the disease.
The Department of Health's (DOH) committee on rare diseases decided last month that there is not enough evidence showing that Miglustat, the drug marketed as Zavesca, is effective in treating Niemann-Pick disease, a rare inherited disorder with no cure. The disease is fatal and usually strikes in childhood or adolescence, occurring in roughly one out of every 150,000 individuals.
Department officials said that there will be another expanded advisory committee meeting on this matter once the drug's maker and other experts submit more evidence on the effectiveness of Zavesca.
Currently, 50 percent of the cost is subsidized by the Bureau of Health Promotion pending an expanded advisory committee meeting on the issue. The other 50 percent is currently provided by a social welfare fund from National Taiwan University.
Hu Wu-liang (胡務亮), a physician at National Taiwan University Hospital's medical genetics department and a member of the committee on rare diseases, is strongly against the committee's decision. Hu is the doctor who first prescribed Zavesca for patients with Niemann-Pick disease in Taiwan.
Speaking to the Central News Agency, Hu said that from his clinical experience, Zavesca has helped halt the pace of deterioration in Niemann-Pick patients and even helped some patients regain the ability to swallow food.
There is no other drug out there for Niemann-Pick patients, Hu said.
However, Bureau of Pharmaceutical Affairs Director Liao Chi-chou (廖繼洲) said neither Zavesca's maker, Swiss biotech firm Actelion nor the team at the hospital have provided enough evidence showing that Zavesca actually works.
"The company only provided the result of a 29 patient study." Liao said.
"The study showed that patients taking Zavesca actually did worse than the control group by many measures," Liao said.
Liao said that he respects Hu's observations, but that the committee can only make their decision based on scientific evidence.
"I hope that both the company and the hospital submit more information soon so that the expanded advisory committee can meet and properly evaluate the effectiveness of Zavesca," Liao said.
Until the expanded committee makes a decision, the BHP will continue to subsidize 50 percent of the cost for Zavesca for Niemann-Pick patients. Zavesca costs roughly NT$320,000 per patient per month.
Last month, the European Committee for Medicinal Products for Human Use issued a negative opinion on the use of Miglustat for Niemann-Pick patients. Reuters reports that Actelion intends to ask for a re-examination of that opinion.