Small patient populations are big business. In 2000, worldwide sales of orphan drugs for rare diseases accounted for just 6% of global prescription drug sales. Last year they accounted for US$114 billion in revenue and nearly 16% of total prescription drug sales, and they are on track to account for 21% of sales by 2022. For Birgitte Volck, head of rare disease R&D at GlaxoSmithKline, the explosion of this space has tracked with the arrival of a new era in rare disease drug discovery and development. She told Asher Mullard about how new drug modalities, the voice of the patient and genomics are reshaping the field.

中文翻译：

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比尔吉特·沃尔克（Birgitte Volck）

病人人数少是大生意。2000年，罕见病孤儿药的全球销量仅占全球处方药销量的6％。去年，他们的收入达到了1,140亿美元，占处方药总销售额的近16％，并有望在2022年占到总销售额的21％。对于葛兰素史克（GlaxoSmithKline）罕见病研发部门负责人比尔吉特·沃尔克（Birgitte Volck）而言，随着稀有疾病药物发现和开发新时代的到来，这一领域也得到了追踪。她告诉阿舍·穆拉德（Asher Mullard）有关新药形式，患者声音和基因组学如何重塑这一领域的信息。