Drug companies, politicians, hospitals, doctors, and patients all agree that there isn’t nearly enough information about whether a given medicine is safe for pregnant and breastfeeding women to use or whether it will work.

A sweeping new effort in Europe aims to change that by bringing all of those groups together to address a public health issue they say demands urgent attention.

The project, dubbed ConcePTION, is a $32 million effort from the Innovative Medicines Initiative, a public-private partnership between the drug industry and the European Commission, the executive branch of the European Union. There are more than 200 researchers and 88 organizations involved in ConcePTION, which is being led by Novartis (NVS) and the University Medical Center Utrecht. The European Medicines Agency, the FDA’s counterpart in Europe, has also joined the consortium.

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“We’re really trying to combine our efforts to change the situation,” said Dr. Miriam Sturkenboom, a pharmacoepidemiology professor at UMC Utrecht and the scientific coordinator of ConcePTION.

Many women take a prescription medication at some point during their pregnancy. But only 5% of medications have adequate safety information for pregnant and breastfeeding women, according to the project’s leaders. That’s left patients and their providers guessing about whether many medicines are safe to take, are being delivered at the right dose, or will impact milk supply. ConcePTION aims to address that by creating an ecosystem that culls several different types of data — including case reports, electronic health records, and studies on drug transfer into breast milk — and distills it into evidence-based information that women and their health care providers can use.

The project’s launch comes as U.S. health officials bolster their own efforts to tackle the information gap. In the past year, the FDA has released several draft guidances on how drug makers can study medication safety and efficacy in pregnant and lactating women.

STAT spoke with Sturkenboom about the new effort. This conversation has been lightly edited and condensed.

Whose responsibility is it to address the lack of information around medication safety and efficacy for these groups?

It’s a societal issue. It’s our duty to solve it. … We do not have enough information, especially for pregnant women. We need to really do a better job using the information that is generated in practice or routine care to make information available to women that are pregnant and their health care providers. There’s just not good enough evidence.

You have a broad range of groups involved. How will you bring them together to address this?

The most important thing is that we all share this vision that this needs to change. So I think already, the fact that we’re all together and wanting to change this builds some momentum that is really necessary to change the current state.

There will be differences in perspective or in interests that people may have. We’re trying to find consensus and solutions together. Because [each company and organization] is part of the building process, it’s [more likely] that each will find the solutions acceptable and workable and continue this work after the project.

How will you approach that, and what data will you look at?

The project is actually going to use four different sources of information to generate evidence that can be useful for women. One is case reports that are being reported to regulators or company and women that report pregnancy data themselves.

Another source of data will be electronic health records, which can be used to look at pregnancy outcomes and drug exposure…. In the U.S., you have the Sentinel system. It’s similar — it’s using existing data to monitor safety. We will also use population-based cohorts or registries that exist already. There’s some pregnancy registries, and they will be part of it.

We are also going to work on breastfeeding and information about the passage of drugs into milk. We will start there in a more experimental manner, namely by developing new models that can predict when drugs will transfer. So that will be in vitro and in vivo work in animals, and then validating that in human lactation studies. We will also be setting up a biobank with milk from breastfeeding women.

What’s important is that we streamline and harmonize all of these so that at the end, based on all these types of different information, we can actually assemble all the knowledge and make it available. That will give concrete advice for women and physicians [in] an outward-facing knowledge bank that is easy to comprehend and summarizes all the evidence.

You were just awarded this five-year grant. What are you hoping to have accomplished five years from now?

We hope that we have a system that can generate information in a rapid and robust manner, and that can be trusted by regulators and by women.

How might this benefit women outside Europe?

We are collaborating with [people in the] U.S., partners in Africa, and also with Asia. So we do think this may spin off to other parts of the world. … The more people that join, the better it will be. … We hope that women across the world will benefit from this effort.

What are the challenges to addressing this information gap? 

It’s a nice thing that [the stakeholders] all have done, but one of the things we have to do is to come to a common understanding and build consensus toward how to do this. The second will be to coordinate all of these different organizations. In total, we have 88 organizations, so it’s not easy. But we have the same goals.

All of the rest is just to get the pieces together. So many of the pieces of what we will be doing have been done somehow, but we need to build them into a system that works and produces the right evidence.

For the companies, one challenge will be whether the evidence that is generated will be able to fulfill regulatory requirements. Currently, the FDA likes to see pregnancy registries, [which collect information from women who take medicines while pregnant]. But these registries cost a lot of money and they only accrue a few women. What companies want, and which I think is sensible, is for regulators to see that there are sources of evidence other than pregnancy registries that are larger and an improvement on quality.

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  • As early as the mid 1980s, Burroughs Wellcome, now part of GlaxoSmithKline, began the use of pregnancy registries for its antiviral products for herpes and HIV and extended them later to epilepsy and other areas. I believed this helped support that treatment of HIV-infected women could reasonably safely reduce the risk of unborn infants being born with HIV. Although this was voluntary, the data was regularly made available to FDA.
    I believe that Hugh Tilson, MD, MPh, the head of safety, deserves at least some credit for such innovations as well as helping implement responsible monitoring of one of the largest set of early access programs such as Treatment INDs.
    Yes, pharma has made some mistakes, but the ethical leadership of some companies should not be forgotten or ignored.
    Glad to see that a new approach may advance the great start that BW made and proud to have been part of ethical pharma.

  • Of course : a European initiative with collaborative partners that collectively enable succes in this long-overdue research. Good for Europe !!! And … where is the mighty self-proclaimed powerhouse, the USA? Still stuck in endless useless regulation dilemmas and discussions ……. materializing nothing. The US machines are too slow, too big, and they succumb to in-fighting. They can not match up to creative bolder and bigger thinking and organizing. For shame.

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