Target product profiles for novel medicines to prevent and treat preeclampsia: An expert consensus

Abstract

Background: Preeclampsia and eclampsia are a leading cause of global maternal and newborn mortality. Currently, there are few effective medicines that can prevent or treat preeclampsia. Target Product Profiles (TPPs) are important tools for driving new product development by specifying upfront the characteristics that new products should take. Considering the lack of investment and innovation around new medicines for obstetric conditions, we aimed to develop two new TPPs for medicines to prevent and treat preeclampsia.

Methods and findings: We used a multi-methods approach comprised of a literature review, stakeholder interviews, online survey, and public consultation. Following an initial literature review, diverse stakeholders (clinical practice, research, academia, international organizations, funders, consumer representatives) were invited for in-depth interviews and an online international survey, as well as public consultation on draft TPPs. The level of stakeholder agreement with TPPs was assessed, and findings from interviews were synthesised to inform the final TPPs. We performed 23 stakeholder interviews and received 46 survey responses. A high level of agreement was observed in survey results, with 89% of TPP variables reaching consensus (75% agree or strongly agree). Points of discussion were raised around the target population for preeclampsia prevention and treatment, as well as the acceptability of cold-chain storage and routes of administration.

Conclusion: There is consensus within the maternal health research community on the parameters that new medicines for preeclampsia prevention and treatment must achieve to meet real-world health needs. These TPPs provide necessary guidance to spur interest, innovation and investment in the development of new medicines to prevent and treat preeclampsia.

Fig 1. Stakeholders’ distribution by WHO global regions.

The proportion of stakeholders who participated in the (A) interviews (n = 23) and (B) survey (n = 46), in each of the WHO global regions. AFR = African region (yellow), AMR = Region of the Americas (blue), SEAR = South-East Asian region (light green), EUR = European region (red), EMR = Eastern Mediterranean region (dark green), WPR = Western Pacific region (black).

Fig 2. Survey responses.

Results from international stakeholder survey (n = 46). Percentage of respondents that strongly agreed (dark green), agreed (light green), were neutral (grey), disagreed (orange) or strongly disagreed (red) in response to the minimum and preferred variable in the TPPs for new medicines to prevent (A = minimum criteria, B = preferred criteria) and treat (C = minimum criteria, D = preferred criteria) preeclampsia. Consensus was considered agreement greater than 75% (black dotted line).