Research in children in the emergency department

29.1 Research in children in the emergency department



Franz E. Babl, Meredith Borland, Andrew J. Davidson




Essentials



1 Research is essential to establish an evidence base in paediatric emergency medicine and optimise individual treatment.

2 At the core of any research project is the development of a viable research question which drives the study design.

3 A biostatistician should be involved early in the study design.

4 Agreement on key data elements and outcomes, including definitions, feasibility of collection and validity of observations must happen early in the study design.

5 Apart from a lack of subjects, the major problem in clinical research projects is poor design.

6 Good clinical research practice is important in all studies.

7 Consent and risk are key ethical issues in all studies involving children

8 Multicentre research can address the difficulty of recruiting adequate numbers of paediatric patients.


Introduction


Research is an important part of emergency medicine as it provides the scientific basis for optimal patient care. Key elements for conducting high quality, ethical research are the development of a good research question, use of an appropriate study design, adherence to good clinical research practice and an understanding of the ethical basis for research. Study design, the quality of the conduct of the study and its ethics are all intricately linked.


Increasing numbers of emergency physicians, nurses and allied health personnel are involved in research, and trainees are now required to complete research projects during training. Yet few emergency clinicians have formal research training. In addition, over the past years both national and local regulatory requirements have become more complex. Research funding is often difficult to obtain in both the emergency and paediatric setting. Hospitals and departments often have to focus on clinical care with limited resources for dedicated staff, time for research, research assistants or infrastructure for studies. Hence, there is a critical and overdue requirement for the development of paediatric emergency medicine within university academic settings, where research is highly valued and an intrinsic part of daily business.


In addition, serious outcomes and adverse events are rare in children and data collected at paediatric tertiary institutions may not be applicable to other settings. Informed consent is difficult to obtain in the emergency setting and the ethics of research in children, as a particularly vulnerable group, creates an additional degree of complexity.


However, despite these difficulties, a number of strategies can be used to overcome the perceived barriers and achieve quality research in children in the emergency department.



Research science


All research should have a sound scientific basis. Getting the science right is essential before starting any project.



Research question


The most crucial component of every project is the research question. The question defines all the other elements of the research design, including the hypothesis and objective of the project.


A good study question should be:


• original or add significantly to what is known;

• relevant and important;

• feasible, based on resources, skills, time, subject availability;

• ethical;

• plausible, that is there should be a scientific basis for the question;

• clearly defined.

Determining these factors requires clinical perspective and a detailed literature search. In clinical research the most relevant or important questions are those that actually change clinical practice.


A question can be defined in terms of the acronym PICOT:


• population (who should be in the study?);

• indicator (what is the intervention or exposure of interest?);

• comparator (what is the gold standard? comparison vs baseline vs control group?);

• outcome (what is the outcome of interest?);

• timeframe (over what time period?).

The outcome measures are often the most difficult to determine and must be clearly defined, usually with a single primary outcome to answer the study question and further secondary outcomes, which may be multiple, to expand on this question. These should be determined in advance, not after study results are in.



Literature review and level of evidence


The literature review should provide the background to the study question. Literature databases like Pubmed, Medline, Google scholar internet search engines, EMBASE and CINAHL (Cumulative Index to Nursing and Allied Health Literature) are useful but may initially be overwhelming. Helpful starting points can be standard textbooks, the Cochrane library, a search of BestBets (www.bestbets.org), BMJ Clinical Evidence (http://clinicalevidence.bmj.com) and the assistance of a medical librarian.


It is important to grade the importance of medical research evidence. There are many grading systems in use internationally. A commonly used example from National Health and Medical Research Council of Australia (NHMRC) is shown in Table 29.1.1.1 Systematic reviews often use such a grading system as the basis for clinical management recommendations. The current standard of research evidence is the randomised clinical trial (RCT), and the highest level of evidence is meta-analysis of RCTs.

























Table 29.1.1 Levels of evidence according to type of research question
Level Study design
I Evidence obtained from a systematic review of all relevant randomised controlled trials
II Evidence obtained from at least one properly designed randomised controlled trial
III-1 Evidence obtained from well-designed pseudo-randomised controlled trials (alternate allocation or some other method)
III-2 Evidence obtained from comparative studies (including systematic reviews of such studies) with concurrent controls and allocation not randomised, cohort studies, case-control studies, or interrupted time series with a control group
III-3 Evidence obtained from comparative studies with historical control, two or more single arm studies, or interrupted time series without a parallel control group
IV Evidence obtained from case series, either post-test or pretest/post-test

Adapted from NHMRC 1999.



Types of studies


Studies can be grouped in a number of ways. Studies can be descriptive, analytic or interventional. Intervention studies can either assess the efficacy of the intervention (does it work in a study?) or the effectiveness (does it work in the everyday ED situation?) (Table 29.1.2).







Table 29.1.2 Types of studies
image


The ethics of medical research


Following atrocities during the Second World War, written codes of medical ethics have been developed such as the Nuremberg Code2, the Declaration of Helsinki3 and the Belmont Report.4 The key principles developed in these documents still underpin most ethical guidelines.



Key principles


The key principles of research ethics include respect for persons, beneficence and justice as well as ‘research merit and integrity’.


Respect for persons recognises the value of autonomy to an individual. Participants must have the power to make their own decisions, if possible. Having respect for persons requires due regard for beliefs, customs and cultural heritage of individuals as well as respecting privacy and confidentiality. Consent is the key element of respect. The consent to participate in a research project must be free and informed. Free consent implies a voluntary choice not influenced by external coercion, pressure or inducement. Informed consent implies that the participant has sufficient information and adequate understanding of the proposed research, and, particularly in the paediatric context, is sufficiently mature to understand the consequences of the decision to take part in the study.


The principle of beneficence includes the concept of maximising possible benefits and minimising possible harm while avoiding unnecessary burdens. Non-therapeutic research or research where the risk of harm is possibly greater than the risk of benefit is not beneficent.


The principle of justice implies there should be no inequality in sharing the burden or risks and the benefits of research. The most obvious examples of injustice are where research benefits or risks are unevenly distributed amongst the wealthy and poor, or conducting research exclusively in minority populations to benefit non-minority populations.



Ethics of research involving children


Due to the exploitation of children prior to the Nuremberg Code there is added complexity relating to research in children. This relates to the key component of informed consent. The age at which a child develops the maturity to understand, give informed consent or accept risk for altruistic reasons is not predefined by chronological age. There is variability in this, based on complexity of the proposed intervention. It is important to note that ethical standards and criteria for study participation evolve over time, and what is acceptable now may be controversial or unacceptable in the future, just as previous standards may now be considered questionable. This is particularly so in relation to children.


Assent by the child is a requirement used in some countries (e.g. USA) from the age of approximately 7 years for studies involving children, although formal consent is still required from the legal guardians.


There are in general four recruitment scenarios for children presenting to the ED who may be enrolled in research studies:5


Infants/toddlers without the capacity to understand or take part in discussions regarding a research project and whose parents/guardians are approached for consent.

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Sep 7, 2016 | Posted by in EMERGENCY MEDICINE | Comments Off on Research in children in the emergency department

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