Science can often be described as a search for answers. The scientist who finds The Answer might even get to name it. Think of Henry Judah Heimlich, who established the Heimlich Maneuver in the 1970s, or of Alois Alzheimer, the psychiatrist forever associated with dementia after being the first to diagnose it.
But an answer is only as good - or as useful - as the question that sparked the research. That's why the real key to solving medical challenges like pressure injuries is the process of asking the right questions. Achieving real world outcomes that make a practical difference in people’s lives means discovering what matters to the patients and directing the research there.
Without questions that point the research in the right direction, the results will produce a series of metrics that may be of great interest to an academic studying the phenomenon, but will be unlikely to produce an improvement in the condition of the person suffering from painful pressure injuries.
Designing Experiments and Gathering Evidence
According to Prof. Esther Middelkoop of the VU University Medical Center in the Netherlands, the primary endpoint of confirmatory phase-3 trials should provide something of value to the patient.
To do so, she offers a three-step process for designing clinical trials and collecting and analyzing the data that comes out of it.
1. Define the primary endpoint consistent with the question that the trial seeks to answer.
Forming the right question is the essential first step of the process. Without it, there is no way to know if the research will wind up helping a patient directly.
Clinical trials are effective in finding crucial information. Health workers could discover so much about the recovery time for a particular injury or which creams offer the quickest and more pain-free solutions to particular wounds. But these real world conclusions can only be drawn when researchers take the time to discover what patients, their caregivers and their health staff really need to know. Starting with the end in mind helps focus the trials and ensures a result that adds something of value to the patient.
But a good question is only part of the process. To meet clinical standards, the outcome must also have a number of other characteristics. Dr. Middelkoop lists four of them:
- Easy to diagnose - an evaluator does not need to rely on personal judgment to identify the result
- Free of measurement error - repeated measures yield the same result each time
- Internal validity - outcome is linked to the point of interest
- External validity - result can be generalized to a wider population
When it comes to wound trials, the main areas for research are safety, wound healing, rate of wound closure, quality of wound healing, and cost effectiveness.
2. Use validated tools to measure characteristics of the primary end point.
Once the goal is clearly defined and the standards for an effective process have been met, the next step is to find the most effective tools for measuring the outcomes. The only way to improve a process is to measure its effectiveness and then use the clinical trials to test new ways of improving it.
Every process has its own tools of measurement. What's important is that the tools are recognized by the medical establishment. For example, a study that looked for ways to reduce scarring in large burns used POSAS (Patient and Observer Scar Assessment Scale) to measure vascularity, pigmentation, thickness, relief, pliability and surface area of the scar.
The measure of validity in scientific research is how accurately the results answer the question at the heart of the study. Validity, therefore, represents the credibility of the study.
It is also crucial, from the beginning, to be clear not only on the objective of the study but also the stage of the trial. If the study is a stage 1 safety trial, it will have a different protocol from a stage 3 confirmatory trial. Matching the objectives to the validation tools will yield usable results.
3. Even with validated tools, multicenter and multinational trials require specific training and evaluation of the outcomes.
Multinational trials produce far-reaching results that could increase validity and scope. But they also require more intensive training to avoid drawing the wrong conclusions. The methods used to analyze the data require specialized training.
In many cases, that means having a specialist on board at the time of the trial. Alternatively, it may be possible to turn the data over to a trained expert to help draw the proper conclusions.
The Importance of Getting it Right
The steps Dr. Middelkoop outlines go a long way in focusing research so that it brings the best, and more importantly, most practical results. They ensure that the research is focused on the patient, not the particular interests of the researcher. If there is no specific benefit for the patient, it would be revealed in the first stage.
The last two stages deal primarily with the credibility of the results. Health workers and people in decision-making positions need to know that the results are applicable to the problems they are trying to solve. They also need to know that the analysis has been performed by trained specialists.
Clinical trials are costly endeavors but they have the potential to unlock so many secrets. When they are carried out with the explicit aim of helping patients, there is no limit to the benefits they can bring.