Abstract
Clinical research involves direct observation or data collection on human subjects. This study was conducted to evaluate the profile of pediatric neurology clinical research over a decade. Trends in pediatric neurology clinical research were documented through a systematic comparative review of articles published in selected journals. Eleven journals (five pediatric neurology, three general neurology, three general pediatrics) were systematically reviewed for articles involving a majority of human subjects less than 18 years of age for the years 1990 and 2000. Three hundred thirty-five clinical research articles in pediatric neurology were identified in the 11 journals for 1990 and 398 for 2000, a 19% increase. A statistically significant increase in analytic design (21.8% vs 39.5%; P = .01), statistical support (6% vs 16.6%; P < .0001), and multidisciplinary team (69.9% vs 87%; P = .003) was observed. In terms of specific study design, a significant decline in case reports (34.3% vs 10.3%; P < .0001) and an increase in case-control studies (11.3% vs 22.9%; P = .02) were evident over the 10-year interval. This comparative audit revealed that there has been a discernible change in the methodology profile of clinical research in child neurology over a decade. Trends apparently suggest a more rigorous approach to study design and investigation in this field.
| Original language | English |
|---|---|
| Pages (from-to) | 882-886 |
| Number of pages | 5 |
| Journal | Journal of Child Neurology |
| Volume | 19 |
| Issue number | 11 |
| DOIs | |
| Publication status | Published - Nov 2004 |
| Externally published | Yes |
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Clinical Neurology
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In: Journal of Child Neurology, Vol. 19, No. 11, 11.2004, p. 882-886.
Research output: Contribution to journal › Review article › peer-review
}
TY - JOUR
T1 - Comparative audit of clinical research in pediatric neurology
AU - Al-Futaisi, Amna
AU - Shevell, Michael
N1 - Funding Information: Al-Futaisi Amna MD, FRCPC Departments of Neurology/Neurosurgery, McGill University, Montreal, QC Shevell Michael MD, CM, FRCPC Department of Pediatrics McGill University, Montreal, QC, michael.shevell@muhc.mcgill.ca 11 2004 19 11 882 886 Clinical research involves direct observation or data collection on human subjects. This study was conducted to evaluate the profile of pediatric neurology clinical research over a decade. Trends in pediatric neurology clinical research were documented through a systematic comparative review of articles published in selected journals. Eleven journals (five pediatric neurology, three general neurology, three general pediatrics) were systematically reviewed for articles involving a majority of human subjects less than 18 years of age for the years 1990 and 2000. Three hundred thirty-five clinical research articles in pediatric neurology were identified in the 11 journals for 1990 and 398 for 2000, a 19% increase. A statistically significant increase in analytic design (21.8% vs 39.5%; P = .01), statistical support (6% vs 16.6%; P < .0001), and multidisciplinary team (69.9% vs 87%; P = .003) was observed. In terms of specific study design, a significant decline in case reports (34.3% vs 10.3%; P < .0001) and an increase in case-control studies (11.3% vs 22.9%; P = .02) were evident over the 10-year interval. This comparative audit revealed that there has been a discernible change in the methodology profile of clinical research in child neurology over a decade. Trends apparently suggest a more rigorous approach to study design and investigation in this field. ( J Child Neurol 2004;19:882—886). sagemeta-type Other search-text 882 Original ArticleComparative Audit of Clinical Research in Pediatric Neurology SAGE Publications, Inc.2004DOI: 10.1177/08830738040190110601 AmnaAl-Futaisi MD, FRCPC Departments of Neurology/Neurosurgery, McGill University, Montreal, QC MichaelShevell MD, CM, FRCPC Department of Pediatrics McGill University, Montreal, QC, michael.shevell@muhc.mcgill.ca Received Dec 17, 2003. Received revised Jan 8, 2004. Accepted for publication Jan 13, 2004. ABSTRACT Clinical research involves direct observation or data collection on human subjects. This study was conducted to evaluate the profile of pediatric neurology clinical research over a decade. Trends in pediatric neurology clinical research were documented through a systematic comparative review of articles published in selected journals. Eleven journals (five pediatric neurology, three general neurology, three general pediatrics) were systematically reviewed for articles involving a majority of human subjects less than 18 years of age for the years 1990 and 2000. Three hundred thirty-five clinical research articles in pediatric neurology were identified in the 11 journals for 1990 and 398 for 2000, a 19% increase. A statistically significant increase in analytic design (21.8% vs 39.5%; P = .01), statistical support (6% vs 16.6%; P < .0001), and multidisciplinary team (69.9% vs 87%; P = .003) was observed. In terms of specific study design, a significant decline in case reports (34.3% vs 10.3%; P < .0001) and an increase in case-control studies (11.3% vs 22.9%; P = .02) were evident over the 10-year interval. This comparative audit revealed that there has been a discernible change in the methodology profile of clinical research in child neurology over a decade. Trends apparently suggest a more rigorous approach to study design and investigation in this field. (J Child Neurol 2004;19:882—886). No universally accepted consensus definition of clinical research exists. In a 1997 report on clinical research, a direc- tors' panel of the National Institutes of Health (NIH) defined it broadly as being of three types: (1) research conducted with human subjects for which an investigator directly interacts with the subjects, (2) epidemiologic or behavioral studies, or (3) outcomes or health services research.1 A narrow, more focused definition characterizes clinical research as the component of medical health research involving direct inter- action with the patient for data collection.2 Ideally, clinical research is a structured process for investigating facts, concepts, or theories. It provides a “sys- tematic and objective way to examine clinical conditions and outcomes, to establish relationships amongst clinical phe- nomena and to provide the impetus for improving methods of practice.”3 It can be descriptive or analytic in design and approach. Its crucial importance for future health and well- being is well recognized.4 It has been characterized as “the neck of the scientific bottle with a bi-directional flow of insight and information from bench to bedside.”3 Pediatric neurology is a relatively young specialty, emerging with distinct professional societies and board certification slightly more than 30 years ago. Many of its orig- inal pioneers remain active in the twilight of their profes- sional practice. New disciplines are particularly dynamic and creative, with a broad range of clinical issues to be addressed on a variety of fronts: nosologic (diagnostic constructs), eti- ology (mechanisms), modes of investigation (new tech- nologies), natural history (outcomes), and interventions (therapies). One would expect in such a setting for clinical research to be particularly vigorous and productive. In addition to this youthful vigor of a young specialty, the 1990s were designated as the “Decade of the Brain” by presidential proclamation in July 1990.5 Through intera- gency efforts (the Library of Congress and the National Institute of Mental Health of the National Institutes of Health) partnering with relevant professional groups and pri- vate associations, there was a considerable effort to enhance public awareness of the benefits to be derived from research on neurologic disorders.5 Presumably, this increasing aware- 883 ness and emphasis would impact on the domain of neuroscience-relevant clinical research. Our objective was to evaluate the profile of clinical research in pediatric neurology at the beginning and end of the Decade of the Brain. Through a systematic “audit” of a portion of the published literature, trends in clinical research in this discipline were documented and examined. METHODS An audit refers to a full check, verification, or formal examination.6 This concept is usually applied to financial accounts. To extend this concept to an assessment or “snapshot” of the state of clinical research in pediatric neurology in 2 calendar years (1990 and 2000) separated by the interval of the Decade of the Brain (1990s), 11 jour- nals were selected for systematic review. These journals were selected from the English literature as representing different forums (ie, different target audiences) for the publication of child neurology research. Five journals targeting primarily child neu- rologists (Brain and Development, Developmental Medicine and Child Neurology, Journal of Child Neurology, Neuropediatrics, Pediatric Neurology), three journals targeting a general neurology readership (Annals of Neurology, Archives of Neurology, Neu- rology), and three journals with a pediatric targeted audience (Archives of Diseases of Childhood, Journal of Pediatrics, Pedi- atrics) were reviewed. Each issue of the above journals for the calendar years 1990 and 2000 was systematically reviewed by a single investigator (A.A- F.). For inclusion in this study, an article had to be an original pub- lication and meet all of the following inclusionary criteria: (1) patient-oriented research, (2) direct interaction between the inves- tigator and subject (as a whole) required for data collection, and (3) majority of subjects less than 18 years of age at study entry. Identified articles were classified according to the following parameters: (1) study design, (2) study profile, (3) statistical sup- port, (4) interdisciplinary or multidisciplinary group of investiga- tors, (5) multicenter subject recruiting, (6) external funding support and source, and (7) topic addressed. Study design was stratified according to the following terms and definitions7: (1) case reports: description of 9 subjects or less with a unique or unusual occurrence; (2) case series: description of 10 subjects or more with a specific condition or occurrence; (3) cross- sectional: the sample is drawn from an affected population, and at a single point in time, a specific measurement is undertaken; (4) case control: a sample of affected individuals is compared with a sample of unaffected similar individuals for the occurrence of prior specific variables; (5) cohort: a group of similar individuals is followed over time to evaluate the occurrence of specific outcomes of interest; (6) randomized controlled trial: a cohort study involving the random dis- tribution (allocation) of an experimental condition between two groups of similar individuals who are then followed for the occur- rence of an outcome of interest. Collectively, case reports, case series, and cross-sectional studies are considered descriptive, whereas case-control, cohort, and randomized controlled trials are considered analytic. An additional aspect of study design stratified separately was whether the study featured retrospective or prospec- tive data collection. Retrospective refers to a study based on previ- ously existing data, whereas prospective was defined as a study involving the creation and collection of new data. Study profile was classified as natural history, therapeutic, out- come or prognosis, diagnostic, or other depending on the princi- pal focus of the report. Statistical support required the presence of an identified epidemiologist or biostatistician as one of the coauthors or as a specific acknowledgment. An interdisciplinary group was determined to exist if one of the authors held a primary appointment outside a university's department of neurology or pediatrics. A multicenter study was defined as one in which sub- ject recruitment occurred at more than one site for patient case man- a g e m e n t . E x t e r n a l f u n d i n g s u p p o r t r e q u i r e d specific acknowledgment in the publication, with the source of funding sup- port stratified into governmental, pharmaceutical, institutional (academic or hospital), disease society, or other (foundation). Data regarding the above parameters were collected separately for the calendar years 1990 and 2000 and compared. Chi-square analysis was undertaken comparing the years 1990 and 2000 for the variables highlighted, with a level of statistical significance taken as P < .05. RESULTS Systematic review of the 11 selected journals revealed 733 original pediatric neurology clinical research articles for the years 1990 and 2000. A statistically significant (P = .01) 19% increase in articles published in 2000 (n = 398) was noted compared with articles published in 1990 (n = 335). The dis- tribution of published articles in the selected journals for 1990 and 2000 is provided in Table 1. For the non–child neurology journals, the percentage of clinical research arti- cles in pediatric neurology as a proportion of the total num- ber of articles published that year for the particular journal is also provided. Comparing the years 1990 and 2000 in the 11 journals selected for detailed review, there was an increase in the pro- portion of clinical research articles published in the pedi- atric neurology journals (1990, 64.5%; 2000, 71.6%). There was a corresponding decline in the proportion of clinical research articles published in the six selected journals targeting prin- cipally either a general pediatric or a neurology readership. It is interesting to note that the pediatric journals had a pro- portionally greater content of pediatric neurology clinical research publications than did the neurology journals. Also, in absolute terms, the pediatric journals were a favored site for publication compared with the neurology journals. The distribution of study design for the clinical research articles published for the 2 years investigated is provided in Table 2. Chi-square analysis revealed a statistically sig- nificant decline in the proportion of case reports published (115/335 [34.3%] vs 41/398 [10.3%]; P < .0001) and a corre- sponding statistically significant increase in the proportion of published articles employing a case-control study design (38/335 [11.3%] vs 91/398 [22.9%]; P = .02) over the decade. A statistically significant shift toward an analytic study design as a proportion of all studies published was evident over the decade as well (73/335 [21.8%] vs 157/398 [39.4%]; 884 Table 1. Distribution of Pediatric Neurology Clinical Research Articles in Selected Journals (1990 and 2000) In 1990, pediatric neurology–specific journals (Brain and Development, Developmental Medicine and Child Neurology, Journal of Child Neurology, Neuropediatrics, Pediatric Neurology) published 216 clinical research articles (64.5%) of the total general pediatric journals (Archives of Diseases of Childhood, Journal of Pediatrics, Pediatrics) published 74 (22.5%) clinical research articles, and general neurology journals (Annals of Neurology, Archives of Neurology, Neurology) published 45 clinical research articles (13.4%). In 2000, pediatric neurology–specific journals published 285 clinical research articles (71.6%) of the total, general pediatric journals published 67 clinical research articles (16.8%) and general neurology journals published 46 clinical research articles (11.6%). P = .01). Although there was a trend toward a prospective design compared with a retrospective design, this did not reach actual statistical significance (204/335 [60.9%] vs 288/398 [72.4%]; P = .10). The distribution of study profiles for 1990 and 2000 is provided in Table 3. A trend for a proportional decline in nat- ural history studies over the decade was observed (66/335 [19.7%] vs 82/398 [11.1%]; P = .08). The distribution of articles featuring statistical sup- port, an interdisciplinary study team, multicenter subject intake, and external funding is provided in Table 4. A sta- tistically significant increase in acknowledged statistical participation was noted (20/335 [6%] vs 66/398 [16.6%]; P < .001), although it was a feature in only a small minority of studies overall. Similarly, a statistically significant increase in an interdisciplinary study team was observed over the decade (234/335 [70%] vs 346/398 [86%]; P = .03), with this being a feature in the overwhelming majority of study teams in both years evaluated. Multicenter studies were rare in both years, with a trend toward a statistically significant increase noted (8/335 [2.4%] vs 28/398 [7%]; P = .09). External funding sup- port increased over the decade, approaching the threshold of statistical significance (105/335 [31.3%] vs 174/398 [43.7%]; P = .06); however, this support was provided in only a minor- ity of published studies in both years evaluated. The distribution of external funding support is shown in Table 5. It should be noted that 26 articles had multiple sources of funding in 1990 and 62 articles in 2000. Comparing these 2 years, a decline in governmental funding support and a statistically significant increase in pharmaceutical support were observed. However, in both years, external funding in the majority of instances was provided by governmental sources. The distribution of topics reported in the published article for the 2 calendar years is provided in Table 6. Only epilepsy and neonatal neurology topics were covered in more than 10% of published articles in both 1990 and 2000, with cerebral palsy topics also being covered in 10% of pub- lished articles in 2000, whereas neuromuscular topics fell below this 10% threshold in 2000. Table 2. Distribution of Study Design of Pediatric Neurology Clinical Research Articles (1990 and 2000) NS = not significant. *Descriptive = case report + case series + cross-sectional. †Analytic = case control + cohort + randomized controlled trial. ‡Statistically significant. 885 Table 3. Distribution of Study Profile of Pediatric Neurology Clinical Research Articles (1990 and 2000) NS = not significant. DISCUSSION Our comparative audit contrasted two snapshots of clinical research in pediatric neurology separated by a decade's interval. The quantity and quality of relevant clinical research undertaken can be taken as a rough index of the scientific and intellectual vigor of a particular discipline. Our audit reveals a 19% increase in published articles in clinical research in pediatric neurology in a sample of representa- tive journals that serve as forums for publication from this discipline. Furthermore, the shift toward analytic studies, a decline in case reports, and an increase in case-control stud- ies suggest a trend to more rigorous study design and inves- tigation. This might reflect an ongoing professional trend toward the practice of evidence-based medicine. This is further supported by an increasing proportion of studies fea- turing prospective data collection. The profile of studies undertaken with respect to issues in management addressed continues to emphasize diag- nostic challenges, with an emerging trend away from nat- ural history studies to those addressing issues of prognosis and outcome. This likely reflects the maturation of the dis- cipline over the decade under study. Studies addressing issues of therapeutic relevance remain a distinct minority, perhaps reflecting the inherent difficulties of conducting a randomized controlled trial, which is the gold standard of therapy evaluation. Although one cannot speak in absolute terms because of our study design, it is interesting to note a declining pro- portion of studies published in the general pediatric and neu- rology literature on pediatric neurology clinical research topics. This reflects a trend toward subspecialty-specific jour- nals as the forum for the exchange of scientific information. One can observe that the general pediatric literature appears to be a more receptive vehicle for publication of clinical research articles in pediatric neurology than the general neu- rology literature. On a positive note, definite trends toward increasing sta- tistical support, interdisciplinary investigatory teams, mul- ticenter studies, and external funding support were apparent. However, the presence of statistical support and multicen- ter sites for subject recruitment remains a minority occur- rence. Increasing statistical participation in study design and implementation should be sought because this expertise is often essential in conducting rigorous clinical research. Similarly, increasing the number of studies featuring mul- ticenter sites improves subject recruitment, especially in the context of relatively rare disorders for the purposes of expediting a study's completion by enhancing possibilities for subject enrollment. Features of cost, feasibility, and accessibility can limit such initiatives. It is intriguing to note the overwhelming contribution of interdisciplinary teams of researchers in this field. This suggests a complementary and holistic approach to clinical problems in the subject population. It might also reflect the efforts of external funding agencies to promote such groupings. Although external funding has increased, most clinical research in pediatric neurology appears to get done without such assistance. This suggests the use of existing clinical services infrastructure in a way that facilitates clin- ical research conduct. This research benefit needs to be fac- tored into any analysis of the use of clinical services infrastructure. The gradual shift away from governmental external funding to pharmaceutical funding suggests that the goals of clinical research might be more “benefit specific” with respect to the controlled evaluation of pharmaceuti- cal agents. Increasing involvement of the pharmaceutical industry in funding will result in increasing goal-specific Table 4. Distribution of Statistical Support, Interdisciplinary Team, Multicenter Sites, and External Funding Support of Pediatric Neurology Clinical Research Articles (1990 and 2000) *Statistically significant. 886 Table 5. Distribution of External Funding Support for Pediatric Neurology Clinical Research Articles (1990 and 2000) NS = not significant. *For 26 articles, multiple sources (two or more) of external funding were identified and attributed to individual funding source categories (ie, total identified funding sources = 132). †For 62 articles, multiple sources (two or more) of funding were identified and attributed to individual funding source categories (ie, total identified funding sources = 241). research that is more likely to be industry driven than physi- cian initiated and directed. The issue of whether this is desirable needs to be addressed. An increasingly activist role for disease-specific societies and foundations is also appar- ent, and this can serve to shift the locus of initiative and direc- tion away from academic centers to lobby groups less concerned about overall scientific rigor and curiosity than potential self-interest. The years bracketed by this particular audit were notable for tremendous change in the academic enterprise. Budgetary restraints and the widespread introduction of managed care had a considerable impact on the financial health of academic health centers, the usual locus of clini- cal research. Similarly, ethical issues relating to increasing Table 6. Distribution of Topics in Pediatric Neurology Clinical Research Articles (1990 and 2000) sensitivity to potential investigator conflicts of interest and an increasing public awareness and concern regarding the use of human subjects (particularly vulnerable children) in clinical research challenge clinical investigators.8 All of this occurred against the backdrop of rapid technologic change in informatics, neuroimaging, and molecular medicine. Clinical research is, indeed, “the neck of the scientific bottle. ”3 Its conduct, quantity, and quality are of critical importance to improve future health and well-being. At pre- sent, there are insufficient data on the distribution and character of clinical research efforts in specific disciplines. It is hoped that this comparative audit is an initial step in filling in this knowledge gap in pediatric neurology so that a thoughtful, reflective discussion on improving and enhanc- ing clinical research opportunities can occur. Such a dis- cussion will need to focus on improving training and funding opportunities, identifying funding partners, and finding mechanisms to bring research efforts into everyday clinical practice. Acknowledgments M.S.s a chercheur boursier clinicien (clinical research scholar) of the Fonds de recherche en santé du Québec MS is also grateful for the support of the Mon- treal Children's Hospital Foundation during the writing of the manuscript Alba Rinaldi provided the necessary secretarial assistance References National Institutes of Health Directors Panel on Clinical Research Report. Available at: http://www.nih.gov/news/crp/97 report/, September 1, 2004. Council for International Organizations of Medical Sciences: International Ethical Guidelines for Biomedical Research Involving Human Subjects, 1993. Portney LG, Watkins MP: Foundations of Clinical Research: Applications to Practice. Norwalk, CT, Appleton & Lange, 1993. De Angelis C: An Introduction to Clinical Research. Oxford, UK, Oxford University Press, 1990. Available at: http://www.loc/loc/brain/, September 1, 2004. Webster's New Collegiate Dictionary. Springfield , Massachusetts, G & C. Merriam Co, 1979. Halley SB, Cummings SR (eds): Designing Clinical Research: An Epidemiologic Approach. Baltimore: Williams & Wilkins; 1988. Shevell MI: Ethics of clinical research in children. Semin Pediatr Neurol 2002;9:46—52. National Institutes of Health Directors Panel on Clinical Research Report . Available at: http://www.nih.gov/news/crp/97report/ , September 1, 2004 . Council for International Organizations of Medical Sciences : International Ethical Guidelines for Biomedical Research Involving Human Subjects , 1993 . Portney LG , Watkins MP : Foundations of Clinical Research: Applications to Practice . Norwalk, CT , Appleton & Lange , 1993 . De Angelis C : An Introduction to Clinical Research. Oxford, UK , Oxford University Press , 1990 . Available at: http://www.loc/loc/brain/ , September 1, 2004 . Webster's New Collegiate Dictionary . Springfield , Massachusetts , G & C. Merriam Co , 1979 . Halley SB , Cummings SR (eds): Designing Clinical Research: An Epidemiologic Approach . Baltimore : Williams & Wilkins ; 1988 . Shevell MI :
PY - 2004/11
Y1 - 2004/11
N2 - Clinical research involves direct observation or data collection on human subjects. This study was conducted to evaluate the profile of pediatric neurology clinical research over a decade. Trends in pediatric neurology clinical research were documented through a systematic comparative review of articles published in selected journals. Eleven journals (five pediatric neurology, three general neurology, three general pediatrics) were systematically reviewed for articles involving a majority of human subjects less than 18 years of age for the years 1990 and 2000. Three hundred thirty-five clinical research articles in pediatric neurology were identified in the 11 journals for 1990 and 398 for 2000, a 19% increase. A statistically significant increase in analytic design (21.8% vs 39.5%; P = .01), statistical support (6% vs 16.6%; P < .0001), and multidisciplinary team (69.9% vs 87%; P = .003) was observed. In terms of specific study design, a significant decline in case reports (34.3% vs 10.3%; P < .0001) and an increase in case-control studies (11.3% vs 22.9%; P = .02) were evident over the 10-year interval. This comparative audit revealed that there has been a discernible change in the methodology profile of clinical research in child neurology over a decade. Trends apparently suggest a more rigorous approach to study design and investigation in this field.
AB - Clinical research involves direct observation or data collection on human subjects. This study was conducted to evaluate the profile of pediatric neurology clinical research over a decade. Trends in pediatric neurology clinical research were documented through a systematic comparative review of articles published in selected journals. Eleven journals (five pediatric neurology, three general neurology, three general pediatrics) were systematically reviewed for articles involving a majority of human subjects less than 18 years of age for the years 1990 and 2000. Three hundred thirty-five clinical research articles in pediatric neurology were identified in the 11 journals for 1990 and 398 for 2000, a 19% increase. A statistically significant increase in analytic design (21.8% vs 39.5%; P = .01), statistical support (6% vs 16.6%; P < .0001), and multidisciplinary team (69.9% vs 87%; P = .003) was observed. In terms of specific study design, a significant decline in case reports (34.3% vs 10.3%; P < .0001) and an increase in case-control studies (11.3% vs 22.9%; P = .02) were evident over the 10-year interval. This comparative audit revealed that there has been a discernible change in the methodology profile of clinical research in child neurology over a decade. Trends apparently suggest a more rigorous approach to study design and investigation in this field.
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U2 - 10.1177/08830738040190110601
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JO - Journal of Child Neurology
JF - Journal of Child Neurology
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