The Food and Drug Administration this week convened a group of experts to discuss ways to develop new drugs for preventing spontaneous early births, a major health concern for which there are no good treatments.
At the two-day workshop led by the FDA and and the Duke-Margolis Center for Health Policy, experts in maternal and fetal health, as well as advocates, discussed challenges to developing a medicine for preterm births, which affect 1 in every 10 babies in the U.S. and can jeopardize their health.
“Developing effective drugs to prevent spontaneous preterm birth is essential to improving our public health and to the lives of so many women and families who are affected by this phenomenon,” said FDA commissioner Robert Califf, at the meeting.
Yet successfully making and testing one is a complex undertaking. Experts acknowledged there’s a lack of understanding as to the underlying biological causes of preterm birth. While an expectant’s mother’s lifestyle, delivery history and cervical health can play a role, the exact triggers aren’t clear.
“The very biology that drives pregnancy, health and disease remains mostly unknown, which means every other discussion that you’re hearing is predicated on not having that knowledge,” said Michal Elovitz, dean of women’s health research at the Icahn School of Medicine at Mount Sinai.
The meeting comes about nine months after the FDA’s decision to remove a drug called Makena — for years the only available option for preterm births — from market after concluding it didn’t work. Despite negative clinical trial data, the drug’s maker had argued for its continued availability in part because of the lack of other options.
There are currently only seven approved and marketed drugs for obstetrical indications. “No other specialty in medicine has such a shortlist of drug approvals with so few indications, and this is the landscape that we in the FDA are eager to change,” said Christina Chang, the director of the agency division that reviews drugs for urology, obstetrics and gynecology.
One reason why, panelists suggested, is a historical lack of investment, compared to other fields, in conditions that primarily affect women. There are some signs investment in women’s health may be rising and the Biden Administration has launched an initiative calling for new ideas.
However, Makena’s saga is suggestive of the challenges in developing drugs for conditions in which the biological causes aren’t well understood. The medicine is made from a synthetic hormone that was believed to help prevent early deliveries. But early results that served at the basis for its 2011 approval couldn’t be confirmed in follow-up testing, leading to the FDA’s withdrawal.
Data supporting other medications given off-label to women for preterm births, such as corticosteroids and magnesium sulfate, are limited as well.
“Most pregnancy-specific conditions lack FDA-approved intervention, making pregnancy an off-label condition, and reinforcing the need for evidence,” said Anne Lyerly, a professor of social medicine at the University of North Carolina at Chapel Hill.
Experts called for more resources and urged researchers to design better trials with clearer goals.
“This is not the end of the discussion on this important topic, but rather an important step in moving the field forward so that new therapies can be developed and ultimately marketed to prevent spontaneous preterm birth.” said Nancy LaPointe, an adjust associate professor in medicine, as well as a faculty fellow, at Duke-Margolis.
