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.

中文翻译：

---

美国药品定价的危机：神经肌肉疾病患者，医生和社会的后果，第1部分。

在美国，药品价格已达到惊人的高度，对患者和社会产生了负面影响。处方药支出中的绝大部分都花在了品牌药上，而FDA排他性和知识产权专利保护了品牌药免受典型的市场压力。在所有药物中，治疗与神经肌肉临床医生特别相关的“孤儿”疾病的药物是最昂贵的一些。《孤儿药法案》的初衷是激励人们创造毒品，否则这些毒品几乎不会带来经济收益。尽管它为我们的患者带来了令人难以置信的改变生命的药物，但它也已成为滥用的来源。根据孤儿药法案批准的许多昂贵药物以前都可以以同情心的价格使用，也可以以更低的价格用于其他适应症。