Clinical Research by Developmental and Behavioral Pediatrics Specialists: Which Models Work Best?
In light of the calls for involvement of more DBP subspecialists in research and development of efficacious and scalable research practices, we should praise the authors for systematically assessing this lean approach, which, in spite of its virtues, proved lean in too many ways. Among the meanings given for the adjective “lean” at m-w.com, we find not only positive senses, such as “characterized by economy,” but also negative ones: “lacking productiveness,” and “deficient in an essential or important quality or ingredient.”1 In harmony with these latter definitions, this lean model was insufficiently productive, likely due to a shortage of important ingredients. The lack of productivity was shown by the fact that only 7% of potential participants (11% of those approved for recruitment by their physician) ultimately were recruited. Thus, leanness did not translate into economic efficiency. From a methods standpoint, such low participation rates preclude any generalization of study results.
In regard to deficient ingredients, what was missing from this effort? In clinical research, subject recruitment is the foundation on which all is built. If there are too few subjects, there is no study. We can only speculate, but it seems that resources devoted to and experience in subject recruitment were both lacking. Over half of potential subjects were lost at the lead site because caregivers were not reached after 3 phone calls. Loss of potential subjects at this critical juncture was even more pronounced at the 4 other sites. The authors do not reveal the extent to which they piloted their recruitment protocol, but I suspect that insufficient resources may have limited piloting across sites and precluded closer monitoring of study recruitment. A brief period of pilot testing should have quickly revealed a problem and prompted an earlier shift in study protocol and resources. In the 21st century, multimodal recruitment strategies are needed. Three phone calls is a fraction of the number typically required to get through to families in an era when contact mainly occurs in the evenings, after parents return home from work. Also, parents are highly suspicious of phone calls from unknown numbers, so letters and postcards (and even texts) warning families in advance of upcoming recruitment phone calls often must be used. Other resources may have been missing as well, but the voltage drop between the number of potential subjects and those actually reached was the key. In addition, study-specific blood draws dissuaded many of the parents who were reached. A different protocol might have capitalized on blood needed for clinical purposes.