Doctors, Off-Label Prescriptions, and A New Legal Precedent
Jun 16, 2009 in Drugs and Devices
The ability to prescribe medications for off-label uses (those not explicitly approved by the FDA) provides physicians with the freedom to innovate — a freedom that has enabled the discovery of many new uses for old drugs. For example, physicians prescribed aspirin for the prevention of heart attacks long before the FDA approved its use in that context. Although it lacked FDA approval, the drug was obviously clinically effective.
However, the Washington State Supreme Court recently affirmed a ruling by the Medical Quality Assurance Commission (MQAC) that expands what is considered medical malpractice in Washington State. As a result of loose wording in the conclusion, the ruling sets a new precedent for malpractice and threatens doctors who promote off-label use of drugs and medical devices. (Links to original documents: Supreme Court Ruling, Amicus Brief, and MQAC Ruling).
In 2004, the MQAC found that Dr. Ames committed malpractice (defined by RCW 18.130.180 (4) and 18.130.180 (16)) for using the Life Information System Tens (LISTEN), a diagnostic tool that measures electrical resistance in the skin, for the treatment of allergies. On June 4, the Washington State Supreme Court affirmed the MQAC ruling and concluded that “…no additional expert testimony regarding LISTEN, or Ames’s use of it, was necessary. The evidence presented demonstrated by clear and convincing evidence to a layperson that he was using a machine for purposes for which it was not designed and for which he was not specifically trained. Substantial evidence exists that he led patients to believe that LISTEN could diagnose and treat allergies, when in fact it could do neither, placing these patients at risk of harm” on the grounds that “LISTEN has not been approved by the Food and Drug Administration (FDA) for use in detecting and/or treating allergies, although biofeedback machines have been approved for other uses.”
One-fifth of all prescriptions in the United States are for off-label uses. Of those, 73% had little scientific evidence supporting their efficacy. Also, due to the limited number of studies done on children, up to 62% prescriptions given to children are considered off-label. Cardiac, psychiatric, and allergy drugs have the highest rates of off-label usage, but the practice of prescribing drugs for non-FDA approved uses is also important in the treatment of rare conditions for which no standard treatment or drug therapy exists. If FDA approval required in proving efficacy prior to prescribing medications, then doctors would be unable to pursue the aforementioned methods of treatment.
In the interest of protecting the public from ineffective treatments, the MQAC and Supreme Court ruled fairly in the case of Dr. Ames. However, the Supreme Court went too far in presuming that a lack of FDA approval suggests clinical ineffectiveness. The Supreme Court, in effect, has redefined what comprises malpractice. The legal precedent set by this ruling means the MQAC could potentially charge doctors with malpractice merely for promoting off-label treatments regardless of their actual effectiveness. As a result, the Supreme Court’s decision could greatly discourage medical innovation by Washington’s doctors and limit the number of treatment options available to patients with rare conditions.
June 16th, 2009 on 4:55 pm
“The legal precedent set by this ruling means the MQAC could potentially charge doctors with malpractice merely for promoting off-label treatments regardless of their actual effectiveness.”
I think you overlook a vital clause of the court ruling and in so doing exaggerate the extent to which malpractice lawsuits will increase. The justices did not write that the usage of the device in question represented malpractice “simply” because it was not FDA approved, but additionally that Dr. Ames:
“Led patients to believe LISTEN could treat and diagnose allergies, when in fact it could do neither, placing these patients at risk of harm.”
This statement is crucial, because it implies that if LISTEN would have held some type of legitimacy or effectiveness towards treating allergies–whether FDA approved or not–then the burden of harm may not have been reached. The efficacy of many off-label drugs, treatments and devices while not possessing the FDA stamp of approval, has been noted in reputable medical journals, demonstrated through lab studies, or largely accepted within a specific field of medicine–i.e. amongst a majority of doctors and practitioners.
If an off-label treatment held wide spread support amongst a large portion of the medical community–for instance the use of aspirin you noted before it was sanctioned–then any harm the treatment may happen to cause would have to be weighed against its observed benefits. As a matter of public policy, this is true even if the drug in question was not designed for that specific purpose.
In future cases which argue malpractice turns merely on the presence of FDA approval, the judges will surely also look to the evidence concerning the drugs’ merits.
June 16th, 2009 on 10:34 pm
My comment is not necessarily a response to your article. I would like to see you write about the drug resveratrol . Is Resveratrol just another “snake oil?”
June 16th, 2009 on 11:25 pm
“Led patients to believe LISTEN could treat and diagnose allergies, when in fact it could do neither, placing these patients at risk of harm.”
This argument can be used in the case of just about any off-label pioneering. I recall a recent medical case in which physicians experimentally injected Botox into the larynx of a young girl with Tourette syndrome. Their theory was that it would calm her vocal cords and lessen her ticks. The treatment didn’t work. Also, there was no scientific evidence to back up this usage (other than the theory post by the physician). On top of that, the treatment had real potential to harm the patient (and much more than that presented by a diagnostic tool measuring electrical resistance at that).
Although I do see your argument, and if a doctor actually made it past the first one or two cases and was able to justify some clinical effectiveness, then I can see where a judge might rule in favor of the physician. However, the true barrier to innovation lies in the impedance inherent in taking the risky step of pioneering a new method of treatment on that first patient. In such a case, where neither FDA approval nor anecdotal evidence of efficacy would be present, a physician would be hard pressed to provide evidence that his treatment was both effective and did not present a potential for harming the patient.
However, this all still presumes that the physician (or his attorney) could argue past the other clauses stating “that medical review boards are able to rely on their own expertise in evaluating medical practices” and that no other evidence was necessary to convince “a layperson that he was using a machine for purposes for which it was not designed and for which he was not specifically trained.” The evidence consisted of a lack of FDA approval and one contradictory expert opinion.
Furthermore, the ruling states that “Generally, Washington law does not require that expert testimony be provided to MQAC” and that the MQAC can “draw inferences from the evidence presented to them.” Furthermore, “it is not inappropriate for MQAC to draw its own conclusions as to the acceptable standard of care.” So, the expert testimony regarding efficacy would not have been necessary for MQAC to make their decision anyway.
I would hope that this case isn’t used as precedent for claims of malpractice. However, the wording of the affirmation still appears to loosen the reigns on what type of behavior actually constitutes malpractice. In my opinion, the Supreme Court would have been better off leaving out “the lack of FDA approval” as proof of ineffectiveness. In the case of Dr. Ames, it would have been simple enough (and less consequential) to affirm the ruling on the basis of supplemental expert testimony affirming the lack of clinical efficacy of LISTEN in treating allergies.