Homeopathy has been practiced for the last 221 years, and for most of that time curing the sick was all that mattered. But over the last few decades formal documentation and analysis of treatment has become a necessity due to the increasing focus on evidence-based medicine.
The moment we hear about evidence-based medicine, we start thinking about randomized controlled trials (RCT). But these are only one of the many research tools and methodologies available and they are not suitable for answering many research questions. One of the other popular research methodologies in clinical research is ‘Outcome Research’.
An outcome study is a generic term that refers to investigations of the results of therapeutic interventions regardless of the type of investigation used.[i] Outcomes research is a body of literature that identifies, measures, and evaluates the results of health care services in general and includes clinical effects, economic data, and quality of life information.
In this paper, I’ll first outline the difference between Randomized Controlled Trials and Outcome Studies, give more details about Outcome Research and then explain how homeopaths can build effective outcome studies even in a very small clinic setup, with an example. Since this paper is meant for clinicians, I’ll purposefully avoid some technical research and statistics jargon.
Difference between RCT and Outcome Studies
Outcome studies are believed (by some) to be less scientific because they do not follow the standard format of a randomized controlled trial (RCT). However, outcome studies can be just as rigorous as RCTs, but the scope of the studies may vary. The primary types of research questions that RCTs and outcome studies are designed to answer differ considerably. RCTs are suited to answer questions about safety and efficacy. RCTs take place in highly controlled settings designed to maximize patient compliance and limit extraneous confounding factors. They are typically short in duration and focus on laboratory or biomedical endpoints.
Outcome studies are designed to answer “real-world” questions pertaining to how drugs are used in the broad population.[ii] This information can be used to answer policy or clinical management questions. Outcome studies focus on cost impact, clinical outcome, and quality-of-life issues. These studies are most useful when real- world variables (e.g., cost, quality of life impact, long-term morbidity) are allowed to have an effect on the data, because outcomes do not depend solely on the activity of the drug but also on the patients and their environment. RCTs are often referred to as efficacy studies, whereas outcome studies may be referred to as effectiveness studies. The differences between RCT and outcomes studies are summarized in the table below[iii].
Outcome studies are gaining importance in all areas of medical practice due to managed care’s growing emphasis on evidence-based medicine. Most outcome studies are designed to evaluate a program’s success rate as well as identify areas of treatment that may need improvement. Moreover, outcome studies serve as a valuable motivational tool for therapists and clinicians because these studies substantiate the positive impact of the professionals’ work.
Benefits of Outcome Studies
- A first important advantage is that – unlike funded research studies – clinical outcome investigations can continue without preset funding cut-off dates, enabling outcome data registries to grow ever larger (and thus more valuable) over time.
- A second important advantage is that resulting outcome studies document changes following interventions in real world settings, thus addressing the issue of actual clinical effectiveness.
- Finally, outcome research may reveal promising – but previously unsuspected – trends that may fruitfully guide the formulation of questions to be investigated in subsequent scientifically controlled research.
PROPERTIES of OUTCOME STUDIES
- Properly conducted, outcome studies can accomplish much that is done also by scientifically controlled experimental studies. The main inherent limitation is that – in contradistinction to scientifically controlled experimental study designs – they cannot establish causality; in its stead, they can document correlations.
- In common with scientifically controlled experimental designs, outcomes studies can document the existence of clinical changes following specified interventions, the magnitudes of the changes, their directions, statistical significance, along with analyses of statistical power.
- Outcome studies can (and often should) use the very same assessment instruments and protocols as scientifically controlled experimental research, making them comparable in terms of psychometric properties such as study validity, reliability, and sensitivity.
- Outcome studies can also probe intervention effects longitudinally to study maintenance effects; and they can also probe generalization of treated behaviors with proper design.
Process of Outcome Research
- Identifying the patient outcome
- building the team
- selecting the instrument
- measuring the patient outcome
- analyzing the data
- summarizing the findings
- applying the findings to practice
- planning future patient outcomes projects
Designing an Outcome Study
In the section below, I’ll describe a simple yet effective approach to designing and implementing an outcome study that will deliver meaningful results in a small clinical setup. The main goal is to evaluate and improve the hospital’s/clinic’s treatment programs. Such small clinical setup trials (also called office-based clinical trials) are not something new. They are frequently used in conventional medicine in Phase IV of new drug trials, where physicians test the drugs in their practice. But in such cases the overall trial design is usually an RCT, and except for the administration of intervention, all aspects of the design and implementation are usually controlled by the pharmaceutical companies funding the research.
But here we are talking about planning and implementing an outcome study in an independent clinical setup.
We will divide the whole process into three parts:
- Planning Stage
- Implementation Stage
- Analysis & Presentation Stage
The four basic questions for the planning stage are:
- What are the objectives of the outcome study?
- What data needs to be collected and what instruments* will be used?
- When to collect the data?
- Who is involved?
(* The word ‘instrument’ here does not refer just to a mechanical tool but to all tools like questionnaires, laboratory reports, clinical assessment and tests done with mechanical instruments, which are used to collect data and monitor the effect of the treatment.)
Providing detailed answers to these questions not only helps design the outcome study but also ensures that the information gathered is reliable and provides appropriate feedback for improvements.
What are the objectives of the outcome study?
The most frequent goal of any clinical outcome study is to find and monitor the success rate of a clinical program, approach or intervention.
Measuring a treatment’s success rate is the very first challenge of setting up an outcome study because success in any program can be defined using different variables in different ways. Is success related to reduction of symptom severity, or reduction of some pathological markers, or adherence of the patient, or the financial benefits of the treatment?
It is important to define from the very beginning how much change is expected in a specific patient population over time. Do we want to see any difference that is statistically significant over time, such as a decrease in symptom severity from first consultation to the last? Or do we want to reach a specific target value that we know reflects a clinically meaningful change, such as a complete change in a pathological marker?
A clear understanding of the meaning of success is a key first step. These definitions become essential not only to identify the goals of the outcome study but also to select the variables to measure that success.
What data needs to be collected?
Once success has been defined, the quantifiable variables are selected to measure that success. Usually, quantifiable variables are extracted from well-established instruments/guides that measure symptom severity and disease evolution for each patient. The selection of these instruments should meet four criteria:
* Accurate and reliable.
* Widely available.
* Easy to use and interpret.
In essence, the chosen instruments should be recognized as accurate and reliable tools in their respective fields. For e.g. if a study seeks to ascertain the severity of asthma in children in the city, it can use as instrument, a symptom-based questionnaire used in epidemiological research like the ISAAC questionnaire or another instrument developed with an internationally accepted clinical classification of asthma severity (GINA) as its basis. For most disease conditions, internationally accepted instruments are available to plan your research around.
Based on the selection of instruments, it is easy to see how the information obtained from the outcome study can be used to provide valuable insight into which treatment components are effective and which may need to be revised to improve the success rate.
When to collect the data
Once the objectives have been set and the instruments have been chosen, the next step is to decide when to collect the data to obtain meaningful results.
There are two main types of outcome studies; retrospective and prospective. Retrospective studies are faster and easier because they consist of either analyzing data that has been collected in the past or contacting former program participants to ask about past treatment.
Prospective studies are the more common choice; they consist of collecting data at consistent time intervals, preferably starting with the registration or first consultation.