Photography: Eyewire Artwork: Paul Belci
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It is axiomatic, because we work in clinical research, that we think we know what clinical research is. It's too fundamental
an issue, too basic a concept, to give it much thought. However, there are compounds administered to patients (not subjects),
as well as procedures performed on patients that are unapproved, untested, and could in no way be considered the generally
accepted standard of care. These therapies, albeit well intentioned, are largely unreviewed for scientific merit. For example,
a physician hears from a colleague that off-label use of a drug might be helpful in treating a patient's clinical condition
and so prescribes the drug for this off-label indication. There are no statutory prohibitions on this approach, and the physician
feels that he or she is in the best position to make this determination. Regulatory agencies are loath to interfere with these
kinds of clinical approaches, lest innovative care be stifled.
Innovation and experimentation are part of the daily life of some physicians and dentists. They may inadvertently cross the
line from innovative care to clinical research. Both medical and dental activities are regulated by state law regarding their
practice, but clinical research has additional GCP requirements, including FDA and IRB oversight. Individual patients may
benefit from innovative care, but society also benefits from innovative clinical research; individual patients must be protected
from substandard care and research subjects must additionally be protected from substandard research. So there are significant
differences in processes and procedures applied to specific clinical situations depending upon a regulatory agency, sponsor,
or IRB determination of whether it is innovative care or clinical research.
This short paper explores current thinking/best practices regarding distinctions between routine care, innovative care, and
clinical research including research that may represent innovative care. It begins with three scenarios that approach, and
possibly cross, an unmarked, "fuzzy" border into clinical research, describes how regulatory agencies and IRBs look at these
scenarios, and finally adds dimension and perspective to the discussion.
Approaching the borderScenario #1: A pulmonologist decides he would like to experiment with a compound that has been previously studied 30 years ago in the
treatment of asthma. He obtains IRB approval to try this compound on a few subjects, but like the surgeon below, he "innovates"
and makes changes as his series of patients expands; he varies the dosing conditions and he changes the premedication treatment.
Scenario #2: For a certain kind of benign tumor that's usually found in the femur, the standard treatment is to remove the tumor, curette
the area, then fill the hole in the bone with cement, or cement plus some hardware, to strengthen the bone and hopefully avert
a later fracture.
An orthopedic surgeon thinks this last step might be accomplished better with cement and a slight modification of the hardware
that is usually applied, or by adding hardware specially designed by the surgeon. He has a patient he thinks might benefit
from this innovative approach and he will decide which hardware to install once his patient's bone is exposed. He expects
he'll probably use this surgical approach for more than just this one patient, and if he does, he'll probably do a retrospective
chart review and publish his results. However, his description of this activity is that he's just going to do what he thinks
is best for each individual patient, and he is being an innovator in his surgical practice.
Scenario #3: An infectious disease consultant has just read a paper describing the use of an old generic antibiotic for staphylococcal
skin abscesses. Coincidently, she is called to the ER that weekend to see such an abscess. With the results of the study fresh
in her mind, she uses the old and cheaper, but non-standard of care—antibiotic—on this patient.
None of these physicians seeks FDA approval of their activities, and neither consents patients with a consent that describes
the treatment as experimental. Are these approaches research that fall under GCP rules and regulations or are they simply
innovative care? Can treatment of a single patient constitute "research?"
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