Kontoghiorghes George J
Postgraduate Research Institute of Science, Technology, Environment and Medicine, Limassol 3021, Cyprus.
Medicines (Basel). 2021 Jul 7;8(7):36. doi: 10.3390/medicines8070036.
Regulatory policies on drugs have a major impact on patient safety and survival. Some pharmaceutical companies employ all possible methods to achieve maximum sales in relation to the monopoly of their patented drugs, leading sometimes to irregularities and illegal activities. Misinformation on the orphan drug deferasirox has reached the stage of criminal investigations and fines exceeding USD 100 million. Additional lawsuits of USD 3.5 billion for damages and civil fines were also filed by the FBI of the USA involving deferasirox and mycophenolic acid, which were later settled with an additional fine of USD 390 million. Furthermore, a USD 345 million fine was also settled for bribes and other illegal overseas operations including an EU country. However, no similar fines for illegal practises or regulatory control violations have been issued in the EU. Misconceptions and a lack of clear guidelines for the use of deferasirox in comparison to deferiprone and deferoxamine appear to reduce the effective treatment prospects and to increase the toxicity risks for thalassaemia and other iron loaded patients. Similar issues have been raised for the activities of other pharmaceutical companies promoting the use of new patented versus generic drugs. Treatments for different categories of patients using new patented drugs are mostly market driven with no clear safeguards or guidelines for risk/benefit assessment indications or for individualised effective and safe optimum therapies. There is a need for the establishment of an international organisation, which can monitor and assess the risk/benefit assessment and marketing of drugs in the EU and globally for the benefit of patients. The pivotal role of the regulatory drug authorities and the prescribing physicians for identifying individualised optimum therapies is essential for improving the survival and safety of millions of patients worldwide.
药品监管政策对患者安全和生存有着重大影响。一些制药公司采取一切可能的方法，以实现其专利药品垄断下的最大销售额，有时会导致违规和非法活动。关于孤儿药地拉罗司的错误信息已达到刑事调查阶段，罚款超过1亿美元。美国联邦调查局还就地拉罗司和霉酚酸提起了35亿美元的损害赔偿和民事罚款诉讼，后来又追加了3.9亿美元罚款才达成和解。此外，还因贿赂和包括一个欧盟国家在内的其他非法海外业务支付了3.45亿美元罚款。然而，欧盟尚未对非法行为或违反监管规定的行为开出类似罚单。与去铁酮和去铁胺相比，地拉罗司使用方面存在误解且缺乏明确指南，这似乎降低了有效治疗前景，并增加了地中海贫血和其他铁过载患者的毒性风险。对于其他推广使用新专利药而非仿制药的制药公司的活动，也提出了类似问题。使用新专利药治疗不同类型患者的情况大多由市场驱动，对于风险/效益评估指标或个体化有效且安全的最佳疗法，没有明确的保障措施或指南。有必要建立一个国际组织，该组织能够为了患者的利益，在欧盟和全球范围内监测和评估药品的风险/效益评估及营销情况。监管药品当局和开处方医生在确定个体化最佳疗法方面的关键作用，对于提高全球数百万患者的生存率和安全性至关重要。
