Definition of Clinical Research Medicine
Let us begin by defining the science that is the basis of all drug research. We call this science clinical trial medicine (CTM) and define it as the science of designing, conducting, and interpreting clinical trials. Its goal is to understand and improve methods for determining whether an intervention, such as a drug, a device, or a procedure, improves clinical outcome in patients. For example, it might address a question such as:
- “ How can one determine whether or not angiotensin converting enzyme (ACE) inhibitors slow the progression of renal disease?”
- “ How can one determine whether or not patients with angina benefi t from coronary artery bypass surgery?”
CTM is a broad field that addresses issues such as types of patients to enroll in a trial, appropriate size of a trial, and ways of maximizing the amount and quality of information elicited from a trial. Put another way, a clinical trial is concerned with finding therapies not for an individual person but rather for a group of patients with a disease. This is different from clinical practice where the goal is to treat individual patients. As an illustration, a question for a practicing clinician treating a patient may be:
- “ Will administration of ibuprofen to Mr. X
who has pain in his knee improve his symptoms? ”
- In a clinical trial, the question may be, “ Will administering ibuprofen
to patients with arthritis decrease their symptoms? ”
It is not sufficient that ibuprofen improves knee pain in Mr. X; the goal of a clinical trial is whether as a group, most (or sufficient proportion) of patients with knee pain of a certain type benefit. Clinical trials can eventually lead to improved therapies for a large group of patients if the treatment is demonstrated to be effective.
A Methodological Science
CTM is primarily a methodological science, in that it is primarily concerned with how to best answer such questions, not what the specific answer is. In other words, CTM is concerned not with the answer to questions such as, “ Do ACE inhibitors slow the progression of renal disease?” Regardless of the answer, if the results are definitive, then CTM has served its purpose. Nor is it concerned with which clinical questions to study or how to apply the results to specific patients. Rather, it is concerned with determining what is the best way to design trials to answer such questions.
Goals within Clinical Research
To put it another way, the goal of CTM is not to be able to declare, “ ACE inhibitors slow the progression of renal disease. ” Its goal is to be able to say, “ A double-blind, placebo-controlled study using measured creatinine as an endpoint at 6 months will answer the question, but a single arm study using calculated creatinine at 4 weeks will not.”
As an analogy, CTM is to clinical medicine what an architect is to the house builder, or what a coach is to an athlete. In this way, CTM is more similar to other methodological fields such as statistics, education, or epistemology than to most other branches of medicine.