Bin Sawad Aseel, Seoane-Vazquez Enrique, Rodriguez-Monguio Rosa, Turkistani Fatema
a International Center for Pharmaceutical Economics and Policy, MCPHS University , Boston , MA , USA.
b Department of Clinical Pharmacy , Umm Al-Qura University , Makkah , Kingdom of Saudi Arabia.
Curr Med Res Opin. 2016 Nov;32(11):1783-1788. doi: 10.1080/03007995.2016.1208644. Epub 2016 Aug 2.
This study assessed trends in the average wholesale price (AWP) at the market entry of disease-modifying therapies (DMTs) approved by Food and Drug Administration (FDA) in the period 1987-2014.
DMT regulatory information was derived from the FDA website. The AWPs per unit at market entry data were derived from the Red Book (Truven Health Analytics Inc.). The AWP history for each DMT was collected from its date of approval to 31 December 2014. The FDA approved label defined daily dose (DDD) for adult patients was obtained from FDA approved labels. The AWP per DDD and the AWP/DDD per year of therapy were computed. Descriptive statistics, Wilcoxon tests, t-test, and multiple linear regression were performed. The statistical significance level was set at 0.05.
The FDA approved 12 multiple sclerosis (MS) DMTs, including five new drug applications (NDAs) and seven biologic license applications (BLAs) as of 31 December 2014. The FDA granted orphan designation to five DMTs. There was one DMT approved by the FDA in the 1980s, three in the 1990s, three in 2000s, and five in the period 2010-2014. The market entry inflation-adjusted AWP per DDD was $10.23 for the first DMT (mitoxantrone hydrochloride) that was approved in the 1980s. The median market entry inflation-adjusted AWP per DDD was $12.41 (interquartile range [IQR] = 4.51) for DMTs approved in the 1990s, $71.26 (IQR = 58.35) in the 2000s, and $172.56 (IQR = 84.97) in the period 2010-2014. The median AWP per DDD was statistically significantly different (p = 0.011) for orphan (median = $41.82, IQR = 56.077) compared to non-orphan drugs (median = $171.32, IQR = 199.29). Year of market entry was positively associated with DMT prices at US market entry (p = 0.01).
The AWP per DDD for DMTs at market entry increased substantially over time. The increase in DMTs prices exceeded the general consumer price index.
本研究评估了1987 - 2014年期间美国食品药品监督管理局（FDA）批准的疾病修饰疗法（DMTs）进入市场时的平均批发价格（AWP）趋势。
DMT监管信息来源于FDA网站。进入市场时每单位的AWP数据来源于《红皮书》（Truven Health Analytics公司）。收集每种DMT从批准日期到2014年12月31日的AWP历史数据。从FDA批准的标签中获取成年患者的FDA批准标签定义的每日剂量（DDD）。计算每DDD的AWP以及每年治疗的AWP/DDD。进行描述性统计、Wilcoxon检验、t检验和多元线性回归。设定统计显著性水平为0.05。
截至2014年12月31日，FDA批准了12种用于治疗多发性硬化症（MS）的DMTs，包括5份新药申请（NDAs）和7份生物制品许可申请（BLAs）。FDA授予5种DMTs孤儿药认定。20世纪80年代FDA批准了1种DMT，90年代批准了3种，21世纪批准了3种，2010 - 2014年期间批准了5种。20世纪80年代批准的首个DMT（盐酸米托蒽醌）进入市场时经通胀调整后的每DDD的AWP为10.23美元。90年代批准的DMTs进入市场时经通胀调整后的每DDD的AWP中位数为12.41美元（四分位间距[IQR]=4.51），21世纪为71.26美元（IQR = 58.35），2010 - 2014年期间为172.56美元（IQR = 84.97）。与非孤儿药（中位数 = 171.32美元，IQR = 199.29）相比，孤儿药（中位数 = 41.82美元，IQR =
