Current practice and awareness of pediatric off-label drug use in Shanghai, China a questionnaire-based study Full Text

Currently the most comprehensive information about off-label drug usage is from the National Disease and Therapeutic Index , which relies on periodic surveys of office-based physicians. We believe that off-label use can be learned systematically, eco sober house ma in a data-driven manner directly from electronic medical records. Our work represents the first effort to detect novel off-label usage from clinical free text over the entire range of drugs and indications observed in the medical record.

We manually examined these to filter out known usages that were missed in Medi-Span and the NDF-RT, leaving us with 403 well-supported novel off-label usages. Psychiatry is more and more becoming a field that is conceptualized with neuroanatomical-based circuits and intricate neurotransmitter signaling at its core. It is also a field that is in desperate need of new medications that leverage the rapidly advancing neurobiological research to provide more effective treatment options for psychiatric disorders.

JAMA Pediatrics

Because of these limitations in the evidence base, off-label prescription of psychotropic medications may be more the rule than the exception. There are relatively few medications approved for use in adolescents, and even fewer in children. The same applies in geriatric psychiatry, where medications are often prescribed, using data from nongeriatric populations, which places the elderly at high risk for negative outcomes. The potential for considerable benefit to patient care underscores the need for opportunities where off-label medication use is presented in a systematic and peer-reviewed manner. This potential benefit must be balanced by discussion of legal risks, particularly in off-label promotion of these medications. Health care providers should understand these risks and benefits, especially when promoting off-label medication use.

We created a gold standard of positive and negative examples of known drug usage. We created negative examples by randomly selecting positive examples and then randomly choosing a drug and indication with roughly the same frequency of mentions in STRIDE as the real usage. These were then checked against Medi-Span to filter out inadvertently generated known usages. The gold standard dataset contained 4 negative examples for each positive case. For each drug-indication pair in the gold standard, we calculated features summarizing the pattern of mentions of the drugs and indications in 9.5 million clinical notes from STRIDE.

For instance, we predict that alendronic acid is used to treat osteopenia, the clinical precursor to osteoporosis. However, Medi-Span and the NDF-RT list the indication as osteoporosis instead of osteopenia — i.e., they encode the used-to-prevent relationship. As a result, although we can detect used-to-treat relationships quite well, recognizing whether or not uses are already known is difficult. Since you may not always know if you are taking a medication in a way that has been approved by the FDA, you can ask your provider to make sure that you are fully informed about the risks and what to expect from treatment. But it is sometimes used off-label to treat impulse-control disorders like gambling.

Medications for psychiatric disorders are also frequently used for unapproved indications.12,32 Patients with psychiatric disorders are often excluded from clinical trials, and these disorders are inherently difficult to study. Moreover, there is often crossover in symptoms from disease state to disease state, which has lead physicians to use psychiatric medications approved for one psychiatric condition for additional unapproved indications. Alexander et al32 estimated that the cost of off-label antipsychotic drug use in 2008 was $6.0 billion. Off-label drug uses can become widely entrenched in clinical practice and become predominant treatments for a given clinical condition. For example, tricyclic antidepressants do not have FDA approval as a treatment for neuropathic pain, yet this class of drugs is considered a first-line treatment option.15 The use of aspirin provides another interesting example of OLDU.

The method has characteristics that allow it to generalize to sites beyond Stanford. First, the system does not require training text labeled with mentions of drugs and indications, and the relationships between them. Second, our method is very flexible with respect to the target drug and indication vocabulary. Third, the system is very fast — annotation of 9.5 million clinical notes takes only two hours on a single machine; constructing features, training a classifier and making predictions takes an additional few hours. It is thus conceivable to process clinical text from a large number of sites, providing a picture of off-label usage across a wide spectrum of institutions. Off-label usage of drugs is an important enough aspect of drug safety to warrant a full issue of Nature Clinical Therapeutics and Pharmacology devoted to the topic .

Off-label drug use was defined as use of a specific drug in a patient younger than the Food and Drug Administration–approved age range for any indication of that drug. Administrative database containing inpatient resource utilization data from January 1 to December 31, 2004, from 31 tertiary care pediatric hospitals in the United States. Some may require evidence of effectiveness or failure with conventional treatments, especially if the drug is expensive.

What does the FDA say about off-label use?

One of the side effects is that it can lower your appetite, so it has gained popularity for off-label use in helping people lose weight. However, if your doctor prescribes a drug for an off-label use to treat your cancer, they are basing the decision on knowledge of and experience with the drug, as well as on research that shows it might be helpful for your stage and type of cancer. Drugs can be legally sold in the United States only after the Food and Drug Administration has approved them.

Whether you or someone you love has cancer, knowing what to expect can help you cope. Here you’ll find in-depth information on specific cancer types – including risk factors, early detection, diagnosis, and treatment options. For example, a person may not have access to a particular FDA-approved drug, or they may have exhausted all other FDA-approved treatment options for their condition.

Current practice and awareness of pediatric off-label drug use in Shanghai, China -a questionnaire-based study

Physicians are known to miss medication-related symptoms, and patients may not inform their physicians about all of their symptoms.44 Second, ADEs among patients with comorbidities may not be as easily attributed to the drug and may not be identified. Third, patients with severe ADEs may visit hospitals and other physicians, and the ADEs might not be recorded in their EHR. All these limitations https://sober-house.org/ might have resulted in an underestimation of ADE incidence. LIbertarians have long chafed at the extreme expense and delay caused by the process of FDA approval of a drug for a new indication. This landmark study demonstrates that on-label (FDA-approved) prescribing is no safer than off-label (FDA-unapproved) prescribing, provided the latter is backed by strong evidence.

These combinations might include one or more drugs not approved for the type of cancer they are being used to treat. Another example is lorazepam (Ativan®), an anti-anxiety drug that is often used as an anti-nausea drug in cancer treatment. In oncology, lorazepam is most commonly given under the tongue , which is also not listed on the drug label. In this case, it’s being given for an off-label use and by an off-label route. One of the biggest problems related to widespread off-label use is the lack of information about how to best use the drug other than for what it was approved. The drug label is one of the most reliable and easy-to-find sources of information available to health professionals, caregivers, and patients.

U.S. Food and Drug Administration

You can learn more about how we ensure our content is accurate and current by reading our editorial policy. With the increase in direct-to-consumer marketing by pharmaceutical manufacturers, in 2010 the FDA introduced the Truthful Prescription Drug Advertising and Promotion Program. This program provides a mechanism by which health care professionals and patients can report illicit OLDU promotion to the FDA. It is important to know that before a drug can be approved, a company must submit clinical data and other information to FDA for review. The company must show that the drug is safe and effective for its intended uses.

For example, a well designed and conducted observational study with a large sample size may provide stronger evidence than a poorly designed and conducted randomized controlled trial with a small sample size. The American Hospital Association (the „AHA”) has not reviewed, and is not responsible for, the completeness or accuracy of any information contained in this material, nor was the AHA or any of its affiliates, involved in the preparation of this material, or the analysis of information provided in the material. The views and/or positions presented in the material do not necessarily represent the views of the AHA.

Although the existence of off-label drug use has its rationality, it may cause several problems. First, off-label prescribing can jeopardize patient safety in certain clinical scenarios where a positive benefit-risk ratio is not fully established. This is mainly due to the fact that off-label drug use is not systematically appraised by regulators, guideline formulators or even healthcare policymakers. Second, off-label use raises issues of liability in the case of adverse events which makes physicians vulnerable to potential legal sanctions. Moreover, drugs used in an off-label manner are usually not reimbursed and would ultimately increase costs to patients and society . Due to its high prevalence and regulatory challenge in pediatric medical practice, off-label drug use has become a worldwide problem .

Calculation of risk and cost indices

Hanbin Wu from the Tongji University School of Medicine proposed grading management to address innovative off-label medication use in China, which provided some references for medical institutions . Furthermore, healthcare professionals’ inadequate training in off-label use was speculated to be another barrier to guideline use since almost all respondents expressed the desire for more education and guidance on off-label drug use. Overall, these results not only identified some barriers to the current expert consensus implementation but also emphasized the need for training on this topic.

• Food and Drug Administration.”38,39 By way of legal precedent and similar FDA regulatory processes, the same standard would apply to OLDU.
• Thus, in this case both the used-to-treat and causal relationships may be true.
• Physicians are known to miss medication-related symptoms, and patients may not inform their physicians about all of their symptoms.44 Second, ADEs among patients with comorbidities may not be as easily attributed to the drug and may not be identified.
• Since the Food and Drug Administration does not regulate the practice of medicine, OLDU has become common.

FAERS case reports explicitly link indications and the drugs used to treat them . These reports are created by patients, health care providers and drug manufacturers, and directly reflect clinical practice. In contrast, MEDLINE provides curated annotations of the biomedical literature with terms from the National Library of Medicine’s Medical Subject Headings vocabulary. We found that 766 novel off-label usages are supported by at least 10 records in FAERS, and 537 of those are also supported by at least two articles co-annotated with the drug and indication in MEDLINE . We then filtered out usages that appeared to be bona fide drug adverse events listed in SIDER 2 in order to eliminate drug-disease pairs that are actually drug-adverse event relationships, leaving us with 466 candidate novel off-label usages.

What is off-label drug use?

Racial differences in the receipt of drugs off-label were noted in the endocrine and respiratory tract drug categories. Male patients were more likely than female patients to receive a drug off-label in the cardiac, endocrine, and respiratory tract categories. Most patients, regardless of whether they died or survived to hospital discharge, received at least 1 drug off-label .

Patient discharge disposition was also included, that is, discharged to home, died, or other . Resource use data included length of hospital stay and case mix index , a widely used severity of illness and risk of mortality adjustment. The LOS weight for each category in the All-Patient Refined–Diagnosis-Related Group Classification System, version 20, and severity level combination is computed as the ratio of the average LOS for patients in this group to the average LOS for all other patients. Drug use, the dependent variable, was categorized as either appropriate for age or off-label. The term “off-label drug use” indicated the use of a specific drug in a patient who at the time of hospital discharge was younger than the FDA-approved age range for any indication of that drug. Drug use was considered appropriate for age if the patient met the age criteria approved by the FDA regardless of indication.