Zampoli M, Morrow B M, Paul G
Department of Paediatrics and Child Health and Red Cross War Memorial Children's Hospital, University of Cape Town, Cape Town, South Africa.
Division of Pulmonary Medicine, Nationwide Children's Hospital, Columbus, OH, United States.
Front Pharmacol. 2023 Mar 27;14:1163391. doi: 10.3389/fphar.2023.1163391. eCollection 2023.
The third Sustainable Development Goal (SDG), to ensure healthy lives and promote well-being for all at all ages, has particular relevance and implementation challenges amongst people living with rare diseases such as cystic fibrosis (CF). Although the treatment and projected outcome of CF has significantly improved with the advent of CF transmembrane conductance regulator protein modulator (CFTRm) therapy, there remains significant global inequality with regards to access to these life-saving and life-altering drugs. Elexacaftor, tezacaftor, and ivacaftor (ETI) triple combination therapy, first licensed in the United States in 2019, has rapidly become the standard of care for children aged 6 years and older in most high-income countries for individuals with CFTR variants responsive to ETI. Negotiated agreements for access to ETI are currently in place in North America,Europe, Israel ,Australia and New Zealand. However, less priority has been given to negotiate agreements for access to CFTRm in low-middle income countries(LMIC) with significant CF populations such as Central and South America, India, the Middle East, and Southern Africa. These countries and individuals living with CF are therefore effectively being left behind, in direct conflict with the stated principle of the 2030 SDGs. In this review, we highlight the current global inequity in access to CFTRm drugs and its impact on widening disparities between high-income countries and LMIC in CF outcomes and survival. We further discuss the reasons for this inequity and explore the ethical- and human rights-based principles and dilemmas that clinicians, families, governments, and healthcare funders must consider when prioritizing fair and affordable access to expensive CFTRm drugs. Lastly, we propose possible solutions to overcoming the barriers to accessing affordable CFTRm drugs in LMIC and illustrate with examples how access to drug therapies for other conditions have been successfully negotiated in LMIC through innovative partnerships between governments and pharmaceutical industries.
第三个可持续发展目标是确保所有人在所有年龄段都能过上健康的生活并促进福祉，这对于患有囊性纤维化（CF）等罕见疾病的人群具有特殊的相关性和实施挑战。尽管随着囊性纤维化跨膜传导调节蛋白调节剂（CFTRm）疗法的出现，CF的治疗和预期结果有了显著改善，但在获取这些挽救生命和改变生活的药物方面，全球仍存在巨大的不平等。依列卡托、替扎卡托和依伐卡托（ETI）三联联合疗法于2019年在美国首次获批，迅速成为大多数高收入国家中6岁及以上对ETI有反应的CFTR变异个体的标准治疗方案。目前在北美、欧洲、以色列、澳大利亚和新西兰已达成获取ETI的协商协议。然而，在中低收入国家（LMIC），如中美洲、南美洲、印度、中东和南部非洲等有大量CF患者的国家，在协商获取CFTRm的协议方面得到的重视较少。因此，这些国家和患有CF的个体实际上被抛在了后面，这与2030年可持续发展目标所阐述的原则直接冲突。在本综述中，我们强调了目前在获取CFTRm药物方面的全球不平等及其对扩大高收入国家和中低收入国家在CF治疗结果和生存率方面差距的影响。我们进一步讨论了这种不平等的原因，并探讨了临床医生、家庭、政府和医疗保健资助者在优先考虑公平且可负担地获取昂贵的CFTRm药物时必须考虑的基于伦理和人权的原则及困境。最后，我们提出了克服中低收入国家获取可负担CFTRm药物障碍的可能解决方案，并举例说明通过政府与制药行业之间的创新伙伴关系，中低收入国家如何成功协商获取其他疾病的药物治疗。
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