Event high­lights plight of rare dis­ease suf­fer­ers

China Daily (USA) - - CHINA - By ZHANG YI zhang_yi@chi­nadaily.com.cn

The ninth in­ter­na­tional Rare Dis­ease Day, ini­ti­ated by the Euro­pean Or­ga­ni­za­tion for Rare Dis­eases, was marked on Feb 29 this year.

The date was cho­sen be­cause such “leap days” are rare, only hap­pen­ing once ev­ery four years.

On and around this day, med­i­cal and re­search in­sti­tu­tions as well as other or­ga­ni­za­tions all over the world hold ac­tiv­i­ties to raise aware­ness of rare dis­eases.

One in ev­ery 2,000 new­borns in China has a hered­i­tary dis­ease, such as amy­otrophic lat­eral scle­ro­sis or Pompe dis­ease.

But ac­cord­ing to Huang Ru­fang, di­rec­tor of the Chi­nese Or­ga­ni­za­tion for Rare Dis­or­ders, there is no sys­tem of na­tional med­i­cal care for suf­fer­ers, es­pe­cially as there is no clear def­i­ni­tion of what con­sti­tutes a rare dis­ease in China at present.

“Leg­is­la­tion for rare dis­eases faces mul­ti­ple hin­drances, there are barely any med­i­cal re­search funds in the field and the ad­mis­sion mech­a­nism for or­phan drugs (phar­ma­ceu­ti­cal agents de­vel­oped specif­i­cally to treat rare med­i­cal con­di­tions) is not in place,” Huang said.

Sun Zhaoqi, a deputy to the Na­tional Peo­ple’s Congress, has brought for­ward a pro­posal re­gard­ing the preven­tion and treat­ment of

Leg­is­la­tion for rare dis­eases faces mul­ti­ple hin­drances ... Huang Ru­fang, di­rec­tor of the Chi­nese Or­ga­ni­za­tion for Rare Dis­or­ders

rare dis­eases for 10 con­sec­u­tive years, but this has never been adopted, as there is no govern­ment pol­icy to re­fer to and there­fore no med­i­cal in­sur­ance for pa­tient re­im­burse­ment.

Ac­cord­ing to an in­ves­ti­ga­tion into China’s small- and medium-sized phar­ma­ceu­ti­cal en­ter­prises car­ried out by Yangcheng Evening News in Fe­bru­ary, most are keen to re­search and de­velop widely used medicines, but are less in­clined to work on drugs for rare dis­eases due to the rel­a­tively small size of the mar­ket and slim profit mar­gins on of­fer.

In ad­di­tion, for­eign drugs for rare dis­eases are not of­ten in­tro­duced to China ei­ther, ac­cord­ing to the in­ves­ti­ga­tion.

Even if the drugs are im­ported, they are rarely used in clin­i­cal treat­ment be­cause pa­tients can­not be re­im­bursed un­der ex­ist­ing med­i­cal in­sur­ance poli­cies.

FAN CHANGGUO / XIN­HUA

Pa­tients with brit­tle bone dis­ease per­form at a shop­ping mall in Jinan, Shan­dong prov­ince, to raise aware­ness of rare dis­eases.

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