背景:Elexacaftor/tezacaftor/ivacaftor(ETI)彻底改变了囊性纤维化(CF)治疗。然而,先前的研究表明,在诊断和治疗获得方面存在巨大的全球差异。如果未解决,这些威胁扩大现有的健康不平等。因此,在本分析中,我们旨在重新评估高收入国家(HIC)与中低收入国家(LMICs)在诊断和治疗公平性方面的差距,并评估进展.
方法:对158个国家/地区的CF人口进行了估计,系统的文献检索,以及对14名CF专家的国际调查。使用文献检索和注册覆盖率数据中确定的流行病学研究对未诊断CF的全球负担进行了估计。使用公开的收入数据和对23个国家药品定价数据库的调查估计了接受ETI的人的比例。
结果:估计在96个国家/地区有188,336(163,421-209,204)人患有CF。其中,诊断为112,955(60%),接受ETI的51,322(27%)。未确诊的病人负担估计为75381人,82%的低收入国家。ETI在35个HIC中得到报销,但只有一个LMIC.批准四年后,有14,911人被诊断患有CF,他们生活在一个ETI无法进入的国家。当包括估计的未诊断人群时,这一数字增加到76,199。
结论:公平获得CFTR调节剂必须成为国际CF界的首要任务。ETI每年的成本高达322,000美元,但可以以5000美元的价格制造,以允许在自愿许可下访问。鉴于差距的程度,还应考虑其他规避制造商的改善准入的机制。
BACKGROUND: Elexacaftor/tezacaftor/ivacaftor (ETI) has revolutionized cystic fibrosis (CF) treatment. However, previous research has demonstrated profound global disparities in diagnosis and treatment access. If unaddressed, these threaten to widen existing health inequities. Therefore, in this analysis we aimed to reappraise gaps and evaluate progress in diagnosis and treatment equity in high-income (HIC) versus low- and middle-income countries (LMICs).
METHODS: Estimates of the global CF population were made in 158 countries using patient registries, systematic literature searches, and an international survey of 14 CF experts. Estimates of the global burden of undiagnosed CF were made using epidemiological studies identified in literature searches and registry coverage data. The proportion of people receiving ETI was estimated using publicly available revenue data and a survey of 23 national drug pricing databases.
RESULTS: 188,336 (163,421-209,204) people are estimated to have CF in 96 countries. Of these, 111,767 (59%) were diagnosed and 51,322 (27%) received ETI. The undiagnosed patient burden is estimated to be 76,569 people, with 82% in LMICs. ETI is reimbursed in 35 HICs, but only one LMIC. Four years after approval, there are 13,723 people diagnosed with CF who live in a country where ETI is inaccessible. This increases to 76,199 when including the estimated undiagnosed population.
CONCLUSIONS: Equitable access to CFTR modulators must become a top priority for the international CF community. ETI costs up to $322,000 per year but could be manufactured for $5000 to allow access under a voluntary license. Given the extent of disparities, other mechanisms to improve access that circumvent the manufacturer should also be considered.