Of approved drugs and unapproved uses

New Straits Times

a day ago

NOT many Malaysians know what off-label drug prescriptions are, but Dr Kamini Shanmugaiah, a senior lecturer at the University Wollongong Australia KDU Penang University College writes in her op-ed in this newspaper that they may account for up to 50 per cent of all medication use, particularly in paediatric and geriatric patients.
One won't be wrong in calling such practice a common use, controversial though it may be. If before we were worried about side effects of modern medicine, now we have to add off-label drug prescriptions to the list.
But what exactly is off-label drug prescription? It is the unapproved use of approved drugs. The use of the cancer drug, methotrexate, for psoriasis is an example of unapproved use. Because of the lack of clinical trial data, the patients prescribed such medicines may be exposed to severe side effects, Kamini warns.
If so, why resort to off-label prescriptions? The simple answer is there are no other drugs available or these drugs have not been tested on certain patients such as children and geriatric patients.
Corrosive commerce plays a part, too. Pharmaceutical companies choose not to invest in clinical trials on such patients because they are not profit generators. No one expected capitalism to be compassionate, anyway.
For the physicians who have to treat their patients even if there aren't any approved drugs for their ailments, it is one tightrope to walk. But so long as they follow guidelines issued by professional bodies such as the Malaysian Medical Council and act responsibly, they can keep medical negligence cases as far away as possible.
Doctors serving in public hospitals would, however, need the approval of a few committees before they can prescribe off-label drugs. The idea is to ensure that such prescriptions are medically justified. Informed consent of patients — meaning physicians must disclose to patients any material risks posed by the drugs — is critical to avoid any legal liability.
Surprisingly, for an old practice of using approved drugs for unapproved uses, there appears to be no Malaysian case law directly on point, as far as we can tell, which means a litigant must rely on general principles of medical negligence and malpractice.
The court will obviously consider the standard of care appropriate for off-label prescriptions, which means the need for the physician to answer several questions. One, was the unapproved use based on guidelines issued by a medical body?
Two, did the physician obtain the informed consent of the patient? Three, was the use in the best interest of the patient?
Vulnerable patients such as children and the elderly have long been ignored by clinical studies conducted by pharmaceutical companies. Malaysia may not be able to do much about getting Big Pharma to be inclusive, but it can get local ones to do so.
The European Union and the United States are using the legal regime to mandate paediatric trials. Malaysia must follow their lead and include geriatric trials as well.
The EU and the US are right in moving up from guidelines to regulatory oversight to ensure patient safety. Malaysia must move up, too.