Direct-to-Patient Research: Piloting a New Approach to Understanding Drug Safety During Pregnancy

Abstract

BackgroundLittle is known about the effects of human fetal exposure when a new drug is authorized unless it was specifically developed for use in pregnancy. Since many factors may contribute to adverse fetal effects, having comprehensive information about in utero exposures will enhance our ability to make correct determinations about causality.ObjectiveThe objective of the study was to assess the extent to which women, recruited without the intervention of health care professionals (HCPs), will provide information, suitable for research purposes, via the Internet or by phone on some potential risk factors in pregnancy.MethodsTo pilot direct-to-patient research for pharmacovigilance, we conducted a prospective, noninterventional study of medication use and lifestyle factors as part of the Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium (PROTECT) Consortium. Consenting women who self-identified as pregnant and residing in the United Kingdom (UK), Denmark (DK), The Netherlands, or Poland were recruited and could then choose to provide data every 2 or 4 weeks via the Internet or a telephonic interactive voice response system (IVRS). Self-reported drug use was compared with pharmacy register data in DK and with electronic health records in the UK.ResultsRecruited women were on average older and more highly educated than the general population. Most respondents chose a frequency of every 4 weeks (56.99%, 1177/2065). Only 29.83% (464/1555) of women with due dates occurring during the study provided information on pregnancy outcome. For those responding by Internet, over 90.00% (1915/2065) reported using >1 pregnancy-related medication, 83.34% (1721/2065) reported using >1 other medicine, and 23.53% (486/2065) reported only over-the-counter medications, not counting herbals and dietary supplements. Some respondents (7.16%, 148/2065) reported that they chose not to take a prescribed medication (mostly medicines for pain or inflammation, and for depression) and 1.30% (27/2065) reported using medicines that had been prescribed to a friend or family member (oxycodone, paracetamol, and medications for acid-related problems). Relatively few respondents reported using fish oil (4.60%, 95/2065), other dietary supplements (1.88%, 39/2065), herbal products (7.07%, 146/2065), or homeopathic products (1.16%, 24/2065). Most medications for chronic conditions that were listed in the Danish prescription registry were also self-reported (83.3%, 145/174 agreement), with larger discrepancies for medications indicated for short-term use (54.0%, 153/283 agreement) and pregnancy-related medications (66.1%, 78/118).ConclusionsSelf-reported information on medication use as well as other potential teratogenic factors can be collected via the Internet, although recruitment costs are not insubstantial and maintaining follow-up is challenging. Direct data collection from consumers adds detail, but clinical input may be needed to fully understand patients’ medical histories and capture birth outcomes.

21st Century Patient Registries, Ebook Addendum to Registries for Evaluating Patient Outcomes: A User's Guide, 3rd Edition

“…[25][26][27] Another motivation of focus group patients to participate in registries was in using registries as an opportunity to connect with other patients, and creating or joining a patient community. Focus group participants who were current members of a registry described specific features that the registry provided, including educational sessions and live feedback opportunities that allowed interaction with investigators and other patients and supported a sense of community.…”

“…Many registries and PPRNs (such as Health eHeart) have enabled the collection of data from patient mobile health trackers and other web-based platforms where patients collect and store personal health information. 27 Finally, patient partners can assist in interpreting the registry findings, describing results in a way that is meaningful to patients, suggesting additional analytic questions, and communicating results in patient-friendly language.…”

“…16 The requirement of keeping up with messaging protocols, updating new versions of hardware/software versions, the need for backward-compatible "hybrid" solutions, evaluating the content validity of questions and answers during transition into a mobile app (especially for informed consent), and how to properly present a survey question are also important considerations. 16,27 Apple's ResearchKit is one example of a platform that allows researchers to build their own data collection apps that will interact with other apps in order to record health information from a multitude of sources. The perceived benefit of these systems solutions is their ability to give researchers access to a global, existing population while providing researchers with secure and HIPAA compliant data-collection options.…”

Patient- or Participant-Generated Registries

“…12,13 Purpose and Goals of Patient-or Participant-Generated Registries Like traditional investigator/sponsor-generated registries, PGRs have many purposes and are often designed to address multiple objectives. PGRs are likely to be established when the issues, concerns, or disease itself are not a priority for traditional sponsors of registries.…”

Multinational Registries: Challenges and Opportunities

“…In a recent study of medication use during pregnancy in four EU countries, it was difficult to compare prescription drug use across countries since some medications not usually covered by health insurance are reimbursed during pregnancy; practices varied by country and time period. 23 Also, in some situations, coverage decisions may limit access of the treatment or procedure to some groups of patients in one country, whereas in other countries that treatment or procedure may be more widely available. Similar to the example with approved indications, regional differences can lead to product use for diverse conditions across countries, which must be taken into account during data analysis and interpretation.…”