The Good and Bad: FDA’s Fast-Track Approval for Preterm Birth Prevention

by Jermane Bond, Ph.D.

In February, the U.S. Food and Drug Administration (FDA), through its accelerated approval regulations, approved and granted exclusivity to KV Pharmaceuticals to market Makena (17Alpha Hydroxyprogesterone Caproate).  The drug is also known as 17P, a synthetic hormone delivered by weekly intramuscular injection to slow the progression of pre-term birth (PTB) before 37 weeks of pregnancy, among pregnant women with at least one previous spontaneous PTB.  Although the drug is not for use for women with other risk factors for PTB or women pregnant with twins, it has been sold for years without approval.  Today, the drug just comes with a large price tag.

PTB is one of the leading causes of neonatal mortality in the United States.  Major risk factors for PTB include behavioral and demographic characteristics such as race/ethnicity, maternal age,  history of preterm delivery, stress, income, education, employment, housing, prenatal care utilization, smoking, alcohol consumption and marital status.  Already, the preterm birth rate is roughly 13 percent of live births, accounting for 500,000 infant deaths annually.

On March 14, 2011 the new 17P, Makena was officially brought to market, marking the first FDA approval of a drug for the prevention of PTB.  Subsequently, the prescription cost increased from $20 to $1500 per dose.  Recent studies support the use of progesterone supplementation for patients at risk for PTB.   The American College of Obstetricians and Gynecologists (ACOG) has promoted the use of 17P injections since 2003, following two successful randomized placebo-controlled trials.  Research has shown that approximately one-third of pregnant women receiving weekly 17P injections have had successful outcomes resulting in the prevention of PTB.  And in 2008, ACOG issued a Committee Opinion indicating that progesterone for the prevention of PTB should be offered to women with a history of spontaneous PTB.

Some obstetricians, maternal health and public health practitioners, including family planning advocates, are outraged at the dosage price increase for 17P and have spoken out and written letters to KV.  As a result, the St. Louis drug company has been criticized for increasing the price of Makena.  In April, the company announced that it would cut the price by more than half (in fact KV reduced the dosage price to $690 two days after the FDA publicly invited competition by announcing that it would continue to allow compounding pharmacies to make and sell a generic version of the drug).  Now, instead of a pregnancy costing $30,000, a much improved price of $13,800 will make the drug more accessible.

Jermane Bond, Ph.D. is Research Associate in the Health Policy Institute at the Joint Center for Political and Economic Studies. More information on Dr. Bond and his work can be found on the Joint Center website.