RESEARCH SKILLS II

CHE Level 5November 2014

Sian Moss

Research that generates numerical data or data that can be converted into numbers.

• Research using clinical trials, quantifies results to assess effectiveness

VAS for assessing pain

• National Census, which counts people and households.

Involves questions like: 'How many people visit their GP each year?‘'What proportion of children have had this vaccine?'.

Quantitative research

Quantitative research

Qualitative Research• Explores people's beliefs, experiences, attitudes, behaviour and interactions• Asks questions about how and why. Eg why people want to stop smoking, rather

than asking how many people have tried to stop. • Generates non-numerical data, such as a person's description of their pain rather

than a measure of pain • Qualitative research techniques such as focus groups and in depth interviews

may be used eg producing NICE guidance to find out more about the views and experiences of the target population or practitioners.

Focus group

• Used for some types of qualitative research. Usually, between 6 and 12 people have a group interview or discussion on a particular topic. It is a good way to find out how people feel or think about an issue, or to come with possible solutions to a problem.

In depth interview

• A face to face conversation between a researcher and a respondent with the purpose of exploring issues or topics in detail. Does not use pre-set questions, but is shaped by a defined set of topics or issues

http://www.nice.org.uk/website/glossary/glossary.jsp

Qualitative research

Qualitative research

DEDUCTIVE and INDUCTIVEresearch approaches

These illustrate process behind Quantitative and Qualitative methods

http://www.drburney.net/INDUCTIVE%20&%20DEDUCTIVE%20RESEARCH%20APPROACH%2006032008.pdf

Deductive research approach

• Reasoning works from• General to specific• Top-down approach• Conclusion follows

Theory

Hypothesis Observation

Confirmation

Inductive research approach• Specific observation to broader

generalisations• Bottom up• Conclusion likely based on the

premise (an assertion or proposition which forms the basis for a work or theory)

• Involves a degree of uncertainty

Theory

Tentative Hypothesis

Pattern

Observation

TRIANGULATIONMixed method research

Combines several methods to study one thing

Triangulation is a way of assuring the validity of research results through the use of avariety of research methods and approaches . It is a means of overcoming the weaknesses and biases which can arise from the use of only one method

For example, a researcher might choose to begin their research with an unstructured interview.

This will allow them to identify key issues and appropriate terms which they can then use as a basis for more formal interviews and structured questionnaires.

Terms and Definitions

Case studiesDefinition • An article that describes and interprets an individual case• Case reports often describe: • Unique cases that cannot be explained by known diseases or syndromes • Cases that show an important variation of a disease or condition • Cases that show unexpected events that may yield new or useful information • Cases in which one patient has two or more unexpected diseases or disorders • Case reports are considered the lowest level of evidence, but they are also the first line of

evidence, because they are where new issues and ideas emerge. This is why they form the base of EBM pyramid.

• If multiple case reports show something similar, the next step might be a case-control study to determine if there is a relationship between the relevant variables.

Advantages • Can help in the identification of new trends or diseases • Educational – a way of sharing lessons learned • Identifies rare manifestations of a disease

Case studiesDisadvantages • Cases may not be generalizable • Not based on systematic studies • Causes or associations may have other explanations • Can be seen as emphasizing the bizarre or focusing on misleading elements

Design pitfalls to look out for • The patient should be described in detail, allowing others to identify patients with

similar characteristics. • Does the case report provide information about the patient's age, sex, ethnicity, race,

employment status, social situation, medical history, diagnosis, prognosis, previous treatments, past and current diagnostic test results, medications, psychological tests, clinical and functional assessments, and current intervention?

• Case reports should include carefully recorded, unbiased observations. • Does the case report include measurements and/or recorded observations of the case?

Does it show a bias? • Case reports should explore and infer, not confirm, deduce, or prove. They cannot

demonstrate causality or argue for the adoption of a new treatment approach. • Does the case report present a hypothesis that can be confirmed by another type of

study?

Cohort studiesDefinition • A cohort study is a type of observational study that follows a group of people over time. Cohort

studies can either be prospective or retrospective. • In a prospective cohort study, the cohort is formed and then followed over time.• In a retrospective cohort study, data is gathered for a cohort that was formed sometime in the

past - usually in a group where good records have been kept and all exposures and outcomes have already occurred. In both retrospective and prospective cohort studies, the goal is to break down the cohort into a group of exposed and a group of non-exposed individuals and follow their outcomes over time.

• In a large enough cohort study, many exposures and outcomes may be evaluated at once.

Advantages • Standardization of criteria/outcome is possible • Easier and cheaper than a randomized controlled trial (RCT)

Disadvantages • No randomization, which means that imbalances in patient characteristics could exist • Blinding/masking is difficult • Outcome of interest could take time to occur

US National Library of Medicine 2008 definitions for the Publication

Controlled Clinical Trial

Work consisting of a clinical trial involving • one or more test treatments, at least one control treatment, specified outcome measures for

evaluating the studied intervention

• a bias-free method for assigning patients to the test treatment. The treatment may be drugs, devices, or procedures studied for diagnostic, therapeutic, or prophylactic effectiveness

• Control measures include placebos, active medicine, no-treatment, dosage forms and regimens, historical comparisons, etc

• Randomization uses mathematical techniques are employed to assign patients to test or control treatments

Randomised Controlled Trial

Randomized Controlled Trial Definition A study design that randomly assigns participants into an experimental group or a control group. As the study is conducted, the only expected difference between the control and experimental groups in a randomized controlled trial (RCT) is the outcome variable being studied.

Advantages • Good randomization will help prevent any population bias • Easier to blind/mask than observational studies • Results can be analyzed with well known statistical tools • Populations of participating individuals are clearly identified

Disadvantages • Expensive in terms of time and money • Volunteer biases: the population that participates may not be representative of the whole • Does not reveal causation • Loss to follow-up attributed to treatment

Design pitfalls to look out for An RCT should be a study of one population only. Was the randomization actually “random” The variables being studied should be the only variables between the experimental groups

Are there any confounding variables between the groups? See Appendix 1

Effectiveness v Efficacy“efficacy” and “effectiveness” mean different things!!!!

EfficacyIs a narrower definition that means how well something works in an ideal or controlled setting, such as a clinical trial.

EffectivenessIs the extent to which a drug achieves its intended effect in the usual clinical setting real world!

Effectiveness, for example, takes into consideration how easy a drug is to use, and potential side effects, whereas efficacy measures only how well it produces the desired result.

Clinical trialsPhase I• aim to test the safety of a new medicine.• A small number of healthy volunteers, are given the medicine and researchers test for side

effects and calculate what the right dose might be to use in treatment • Usually the first time medicine tried on humans, so there is an unavoidable element of risk.

To minimise the risk, start with small doses and only increase the dose if the volunteers do not experience any side effects, or if they only experience minor side effects.

Phase II• Test the new medicine on a larger group of people who are ill, to get a better idea of whether

it works and how well it works in the short-term.Phase III• Only for medicines that have already passed phases I and II. Test medicines in larger groups of

people who are ill, and compare the new medicine v existing treatment or a placebo to see if it works better in practice and if it has important side effects.

• Often last a year or more and involve several thousand patients.Phase IV• The safety, side effects and effectiveness of the medicine continue to be studied while it is

being used in practice. Phase four trials are not required for every medicine.

Research in ‘Homeopathy’A distinction can made between the four different possible meanings of the term ‘homeopathy’

‘Homeopathy’ can be used to mean any of the following:• Treatment by a homeopath• Homeopathic remedy• The principles/ laws of homeopathy (law of similars, law

of minimum dose)• The system of medicine

It can be argued that if no distinction is made then claims about the research for or against ‘homeopathy’ are vague and of little use Discussions/ arguments then fail to shed any light upon the many different types of questions there are about ‘homeopathy’ research and thedebate continues on in the dark……

This is also true of other systems of medicine

Experimental v ObservationalObservational evidence • Evidence from routine practice of treatment by homeopaths and/or taking

homeopathic medicines, case reports, audits, cohort studies, case series etc• Provides evidence as to the strength of association between A and B

Eg Witt, 2009 Homeopathic treatment for patients with dysmenorrhea Arch Gynecol Obstet 280:603–611

Concluded Patients with dysmenorrhea improved under homeopathic treatment. Controlled studies should investigate efficacy and effectiveness

Experimental evidence• Evidence derived from RCTs / systematic reviews and meta analyses of RCTs• Provides evidence as to causation between A and B

Eg Bell, 2004 Improved clinical status in fibromyalgia patients treated with individualized homeopathic remedies versus placebo Rheumatology 2004;43:577–582 Concluded Individualized homeopathy is significantly better than placebo in lessening tender point pain and improving the quality of life and global health of persons with fibromyalgia

How to read the studieswhat to look for

M.A.A.R.I.E.http://www.studyingastudy.com/jc_maarie_main.html

The M.A.A.R.I.E. framework outlined below is the basis for a step-by-step approach to reading the clinical research literature.

Useful paper to readCorcoran M, 2006. Using the MAARIE Framework To Read the Research Literature. The American Journal of Occupational Therapy Volume 60, Number 4 p367-368

Examples of actual trials to read to gain understanding of methodologies

Homeopathic treatment of patients with dysmenorrhea:a prospective observational study with 2 years follow-upClaudia M. Witt · Rainer Lüdtke · Stefan N. Willich

Available athttp://www.writerscafe.org/uploads/stories/6811ade799993e3741071e1cfb2d2318.pdf

Randomised controlled trial of homoeopathy versusplacebo in perennial allergic rhinitis with overview offour trial seriesMorag A Taylor, David Reilly, Robert H Llewellyn Jones, Charles McSharry, Tom C Aitchison

Available athttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC27460/pdf/471.pdf

For discussion it provokedhttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC1119423/

Appendix 1 Confounding variablesConfounding Variable: Is an extraneous variable whose presence affects the variables being studied so that the results you get do not reflect the actual relationship between the variables under investigation.

When conducting an experiment, the basic question that any experimenter is asking is: "How does A affect B?" where A is the probable cause, and B is the effect.

Eg. you want to study whether bottle-feeding (Cause) is related to an increased risk of diarrhea in infants (Effect) It would seem logical that bottle-fed infants are more prone to diarrhea since water and the bottle could get contaminated, milk could go bad, etc. But when conducting this study, you would learn that bottle-fed infants are less likely to develop diarrhoea than breast-fed infants. It seems that bottle-feeding actually protected against the illness. But in truth, you would have missed a very important confounding variable - mother's education.

On taking the mother's education into account, you would learn that better-educated mothers were more likely to bottle-feed their infants, who are also less likely to develop diarrhoea due to better hygienic practices of the mothers.

In other words, mother's education is related to both the Cause and the Effect. Not only did the Confounding Variable suppress the effect of bottle-feeding, it even appeared to reverse it – confounding the results!

Appendix 2aHomeopathic arnica therapy in patients receiving knee surgery: Results of three randomised double-blind trialsB. Brinkhaus et al 2006

Abstract

Objectives: We investigated the effectiveness of homeopathic Arnica montana onpostoperative swelling and pain after arthroscopy (ART), artificial knee joint implantation(AKJ), and cruciate ligament reconstruction (CLR)

Design: Three randomised, placebo-controlled, double-blind, sequential clinicalTrials

Setting: Single primary care unit specialised in arthroscopic knee surgery.Participants: Patients suffering from a knee disease that necessitated arthroscopicSurgery

Interventions: Prior to surgery, patients were given 1×5 globules of the homeopathicdilution 30× (a homeopathic dilution of 1:1030) of arnica or placebo. Followingsurgery, 3×5 globules were administered daily

Primary outcome measures: The primary outcome parameter was difference in kneecircumference, defined as the ratio of circumference on day 1 (ART) or day 2 (CLRand AKJ) after surgery to baseline circumference

Results: A total of 227 patients were enrolled in the ART (33% female, meanage 43.2 years;), 35 in the AKJ (71% female, 67.0 years), and 57 in theCLR trial (26% female; 33.4 years) The percentage of change in knee circumference was similar between the treatment groups for ART (group difference =−0.25%, 95% CI: −0.85 to 0.41, p = 0.204) and AKJ (=−1.68%, −4.24 to 0.77, p = 0.184) and showed homeopathic arnica to have a beneficial effect compared to placebo in CLR (=−1.80%, −3.30 to −0.30, p = 0.019).

Conclusions: In all three trials, patients receiving homeopathic arnica showed a trendtowards less postoperative swelling compared to patients receiving placebo. However,a significant difference in favour of homeopathic arnica was only found in the CLR trial.

Appendix 2bHomeopathic arnica therapy in patients receiving knee surgery: Results of three randomiseddouble-blind trialsB. Brinkhaus et al 2006