Department of Neurology, University of Virginia, Charlottesville, Virginia, USA.
Beth Israel Deaconess Medical Center, Jerome H. Grossman M.D. Graduate Fellow, Harvard Kennedy School, Boston, Massachusetts, USA.
Muscle Nerve. 2020 Nov;62(5):567-572. doi: 10.1002/mus.27015. Epub 2020 Jul 28.
Drug prices in the United States have reached astounding heights, negatively impacting patients and society. The vast majority of prescription drug spending is on brand name drugs, which are protected from typical market pressures by FDA exclusivity and intellectual property patents. Drugs to treat "orphan" diseases, of particular relevance to neuromuscular clinicians, are some of the most expensive in all of medicine. The Orphan Drug Act's original intent was to incentivize the creation of drugs that would otherwise provide little economic payoff. While it has facilitated incredible, life-changing drugs for our patients, it has also become a source of abuse. Many expensive drugs approved under the Orphan Drug Act were previously available for compassionate use or for another indication at much lower prices. As patients increasingly face high drug prices, it is important for clinicians to understand a drug's risk for inducing financial toxicity, as the financial and emotional consequences of an overpriced low-value drug may outweigh its intended benefit.
美国的药品价格已经达到了令人震惊的高度，对患者和社会都产生了负面影响。绝大多数处方药的支出都用于名牌药品，这些药品受到 FDA 专有权和知识产权专利的保护，免受典型的市场压力影响。治疗“孤儿病”的药物是所有药物中最昂贵的药物之一，对神经肌肉科医生来说尤为相关。《孤儿药法案》的初衷是鼓励开发那些否则经济回报微薄的药物。虽然它为我们的患者带来了令人难以置信的、改变生活的药物，但它也成为了滥用的根源。许多根据《孤儿药法案》批准的昂贵药物之前可用于同情用药或其他适应证，价格要低得多。随着患者越来越多地面临高昂的药品价格，临床医生了解药物引发财务毒性的风险非常重要，因为高价低价值药物的财务和情感后果可能超过其预期的益处。
