PROTECT and the pregnancy study
By: Stella Blackburn, MBBS | June 08, 2016
What we learned from conducting an observational study of pregnant women throughout their pregnancies.
In 2009 whilst at the European Medicines Agency, I had the great fortune, with Xavier Kurz and Jim Slattery, to start a ground-breaking program that aimed to strengthen the monitoring of the benefit-risk of medicines in Europe and to test new tools for collecting data directly from consumers of medicines. This was PROTECT: the acronym of Pharmacoepidemiological Research on Outcomes of Therapeutics by a European Consortium — a public private partnership program of 34 partners created as part of the Innovative Medicines Initiative. As part of PROTECT, one of the research areas was a pilot study that could change the way the industry collects real world outcomes data — and we demonstrated this by gathering information from two thousand pregnant women across Europe. Outcome Sciences, which became part of Quintiles, was involved in this study as one of the partners right from the start. It was only natural that when I joined Quintiles, I continued working on this with my colleague, Nancy Dreyer, who had been the original lead at Outcome.
The PROTECT Pregnancy Study was an observational study of lifestyle factors, health and use of medicines by pregnant women throughout their pregnancies. This is particularly important to study because women still need to take medicines when they are pregnant and there is limited information about the safety of medicines and their effects on the unborn baby. Traditional electronic health records (EHR) capture limited data about medication use by pregnant women. These systems only record what medications are prescribed, but not what patients actually take. They do not capture data about use of over-the-counter medications, herbals or homeopathic medicines or medication acquired by other means — e.g., “borrowed” from friends. As a result it is difficult, using existing data sets, to get a holistic view of all the medications pregnant women actually take.
We also wondered whether what is missing from the electronic record actually matters. The only way to collect such information is to ask the concerned person directly — at least that was our theory. The Pregnancy Study was designed to see whether it was possible to collect data of good enough quality to use in medication research directly from pregnant women using data collection tools developed by PROTECT.
At this year’s European Conference on Rare Diseases & Orphan Products (ECRD 2016) I presented some of the data that emerged from this initiative, and the valuable lessons we learned.
Answer questions today for the pregnancy of tomorrow!
It took quite a while to get permission from all the different ethics and data protection bodies involved and to build the systems. In 2012, we received final approval for the study and began recruiting. Our original goal was to recruit 5600 pregnant women in the UK, Poland, Denmark and the Netherlands who would anonymously answer questions about their lifestyle, health and medication use during and immediately before pregnancy. A key component of the project was to recruit and work with women outside of the healthcare environment, and to avoid recruiting them the “traditional” way via their doctors and midwives. We recruited through leaflets in pharmacies and advertised through relevant websites, Facebook and other social media outlets. We also adhered to natural language in our communications with participants to make the experience more engaging and user-friendly. We felt that such an approach would make the opportunity to participate in the research more appealing for women, and that it would create an environment where they felt comfortable reporting honestly about their experiences.
Participants who took part in the online format could choose to complete surveys either every two or four weeks during their pregnancy, and also complete two short questionnaires after the end. Women who opted to take part via telephone, using an electronic voice automated system, only completed two questionnaires: one at the start of the study and another after the date of delivery.
Not as easy as you’d think
In the end, the study was a great success, generating valuable data both about lifestyle factors and the choices made by pregnant women regarding medication as well as the benefits of these data collection techniques in enhancing our knowledge about adverse drug reactions and real-world behavior of different patient populations. At the same time, this project provided us with valuable lessons about the challenges of recruiting and retaining participants into such real-world studies.
In an ideal world we would know whether every medication was safe or not safe for use during pregnancy, but due to the limitations of research into medication and pregnancy, such a vision isn’t possible – particularly as some unwelcome effects may be very subtle and only become apparent as the child grows up. There are some very talented and dedicated researchers looking at the safety of medicines during pregnancy, but there are so many different factors that can affect the outcome of pregnancy that obtaining complete data is very difficult. But, we could do better. The Pregnancy Study demonstrates the impact that collecting data directly from women and combining it with other observational data could have on helping us understand the safety of medicines used during pregnancy. This is terribly important so that we can improve the advice given to pregnant women who need to take medicines. That kind of reassurance can be of great value to these women and to the healthcare community at large.