The 21st century cures act: Opportunities and challenges.

On July 10, 2015, the U.S. House of Representatives overwhelmingly voted (344-77) to approve the 21st Century Cures Act, setting the stage for the Senate to consider its own version of the bill this fall. Such strong bipartisan support, rare in an age of congressional gridlock, might logically be interpreted as a sign of the Act's promise to foster innovative drug and device development. However, careful inspection reveals that while the Act contains some positive features, it represents a poor deal for Americans on balance that could result in the entry of more dangerous and ineffective drugs and devices onto the market and erode trust in the US Food and Drug Administration (FDA).

Are risk evaluation and mitigation strategies associated with less off-label use of medications? The case of immune thrombocytopenia.

Using data from a large commercial health insurer, we studied prescribing of romiplostim (Nplate) and eltrombopag (Promacta), two drugs for primary immune thrombocytopenia (ITP) for which risk evaluation and mitigation strategies (REMS) with elements to assure safe use were initially imposed and then removed. We identified 103 and 117 new users of romiplostim and eltrombopag, respectively. Use was almost exclusively for FDA-approved indications ("on-label") while the REMS with elements to assure safe use were in place. After these elements were lifted, off-label use of eltrombopag among patients with hepatitis C virus (HCV), a subsequently approved indication, increased. The ratio of incidence rate ratios of off-label/HCV to on-label initiation of eltrombopag between the two time periods was significant (13.41; P < 0.001). Our finding of an association with reduced off-label prescribing suggests that REMS with elements to assure safe use can help promote patient safety but may also prevent promising off-label drug uses.