Current status and prospects of usefulness of research in
preterm birth
The multidimensional assessment of usefulness can map the strengths and
weaknesses of a large field. The remarkable absence of reference to
patient centeredness in our assessment could be the first ‘low hanging
fruit’ to improve usefulness in future trials. Luckily, an extensive
patient/parents and clinicians research priority list of 15 research
questions related to PTB has already been developed through the James
Lind Alliance.(24) Participants have also been involved in a ‘core
outcome set’ for PTB prevention studies.(25) The essential step is
implementing these two tools in future PTB trials.
Different studies may require very different levels of financial
investment and may differ substantially in how much we can learn from
them.(5) An assessment of value of information through formal modelling
(13) can help reduce the conduct of studies that do not convincingly
change practice. Hopefully, this will help decrease the use of surrogate
and composite outcomes and the conduct of underpowered studies.(26)
Proper power calculations require clinicians’ and women’s input to
define the minimal clinically important difference. For PTB research,
this is a remarkable knowledge gap. We found only one study
investigating the minimal clinically important difference for preventive
PTB strategies (cerclage, progesterone).(27)
The very low number of pragmatic trials may also be a missed
opportunity. Traditional trials recruit highly selected participants,
seen in specialist environments and meet rigorous inclusion/exclusion
criteria such as being free of comorbidities, which might influence
generalizability of the results. In contrast, pragmatic trials support
the generation of evidence relevant to real-world decision making.(28)