Ethical and legal framework and regulation for off-label use: European perspective

Lenk, Christian; Duttge, Gunnar

doi:10.2147/tcrm.s40232

Cited by 73 publications

(62 citation statements)

References 16 publications

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“…Many older drugs are off patent, so there is no financial incentive for a manufacturer to seek an FDA indication for their use in migraine, since the expense of the trials and FDA application could not be recouped. This is an area where government or nonprofit funding could be useful …”

Section: How Should Clinicians Judge the Evidence?mentioning

confidence: 99%

Pharmacologic Prevention of Migraine: A Narrative Review of the State of the Art in 2018

Loder

Rizzoli

2018

Headache

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This paper describes current non-antibody pharmacologic approaches to the prevention of migraine in adults. Preventive therapy should be considered for patients with migraine who routinely have more than 6 headache days per month or in other special circumstances. Choices for preventive therapy are based on patient preferences about side effects and evidence of efficacy. The evidence level and commonly used doses for selected categories of migraine preventive medication are reviewed, including antiepileptic drugs, antihypertensive drugs, and antidepressants. Propranolol, timolol, topiramate, and divalproex sodium are approved for migraine prevention by the US FDA. OnabotulinumtoxinA is approved for prevention of chronic migraine. Several off-label drugs, especially lisinopril, candesartan, and amitriptyline also have good evidence of benefit. The spectrum of response to preventive therapy varies; in general, complete cessation of headaches is uncommon, although there are "super-responders" to every therapy, as illustrated by patient reports of dramatic responses to treatment. Preventive treatment should be started at a low dose and doses increased slowly until therapeutic benefit is achieved or side effects preclude continued use.

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Section: How Should Clinicians Judge the Evidence?mentioning

confidence: 99%

Pharmacologic Prevention of Migraine: A Narrative Review of the State of the Art in 2018

Loder

Rizzoli

2018

Headache

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“…In Europe, the regulation of off-label drug prescribing is summarized by the European Medicines Agency's (EMA). Off-label prescribing provisions include strong scientific evidence or guaranteed safety of use by the consensus of professional organizations [6]. In Indonesia, there is no clear regulation regarding the off-label drug use.…”

Section: Discussionmentioning

confidence: 99%

A Retrospective Study: The Off-Label Use of Anticonvulsants at a Private Hospital in Indonesia

Rahajeng

Ikawati

Andayani

et al. 2018

Int J Pharm Pharm Sci

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Objective: Anticonvulsant is one class of drugs often used off-label. This study was conducted to investigate the prevalence and the indication of the off-label use of anticonvulsants in a private hospital in Java, Indonesia. Methods:This was an observational study with a retrospective data collection in a private hospital in Java. Data were obtained on the prescription of anticonvulsants. Indications of the use of anticonvulsants were obtained from the medical records of patients who were prescribed anticonvulsants. The off-label use of anticonvulsants was defined a prescribing of medication outside the indication approved by The National Agency of Drug and Food Control Indonesia (NA-DFC). The use off-label of anticonvulsants was calculated by descriptive analysis and presented as a percentage.Results: It showed that in one year there were 5,310 for 1,316 patients: of this 462 patients (35.11%) were for an off-label use. The anticonvulsants used off-label were oxcarbazepine 67.27% (37/55), carbamazepine 46.15% (54/117), pregabalin45.45% (60/132), phenytoin37.62% (225/598), valproate 25.34% (37/109), and gabapentin 18.28% (49/219). The highest off-label use of anticonvulsants was found in neurological and psychiatric disorders 67.32% (n=311), and on 97.19% of them were not supported by strong clinical evidence. Conclusion:The off-label use of anticonvulsants occurred in one-third of patients receiving prescriptions of anticonvulsants, even though for most of them there was a lack of evidence. More attention must be paid to the efficacy and risk of side effects of the drug used.

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“…This misconception among advanced cancer patients could possibly lead to the demand of off‐label drugs use beyond approved usage . As a recommendation, patients should be properly educated about every off‐label drug use, supporting evidence, the risk of toxicities and financial impact on treatment costs . Obtaining informed consent was highly recommended by participants to safeguard patient autonomy leading to better healthcare decision with off‐label use and to prevent any future legal liabilities .…”

Section: Discussionmentioning

confidence: 99%

Perception of oncology practitioners towards off-label use of anticancer medicines in Singapore

Saiyed

Ong

Chew

2017

J Pharm Health Serv Res

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Objectives Prescribing drugs outside regulatory recommendation is known as off‐label use. Studies have reported widespread off‐label drug use in oncology practice. But the clinical, economic and humanistic outcomes for such practice are often uncertain. Hence, it is important to gather oncology practitioners’ perspective for the creation of clinical practice framework and policies. The aim of the study was to evaluate perception of oncology practitioners on off‐label use in cancer therapy. Methods This study employed a cross‐sectional design implemented in the form of the self‐administered questionnaire at National Cancer Centre Singapore where more than 50% cancer patients of the country are treated. Key findings Eighty‐one practitioners involving nurses (38%), medical oncologist (37%) and pharmacists (25%) were surveyed. The majority of practitioners (57%) agreed off‐label use as indispensable practice in cancer therapy. Main reasons cited include advanced cancers where standard lines of treatment are exhausted (58%), lack of alternative approved drug (43%) and rare tumours (47%). Compared with other practitioners, medical oncologists viewed Phase 2 (73% versus 31%, P = 0.001) and conference abstracts (56% versus 23%, P = 0.003) as reasonable evidence for off‐label use. Major concerns included lack of efficacy (58%), patient understanding (47%), uncertain safety (43%) and out‐of‐pocket cost (41%) associated with off‐label use. Most viewed need for obtaining informed consent (86%) and institutional guidance (75%) as important elements in practice framework. Conclusion The study provides insights about off‐label drug use practice and establishes the need for robust clinical guidance and educational strategies for oncology practitioners.

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Ethical and legal framework and regulation for off-label use: European perspective

Cited by 73 publications

References 16 publications

Pharmacologic Prevention of Migraine: A Narrative Review of the State of the Art in 2018

Pharmacologic Prevention of Migraine: A Narrative Review of the State of the Art in 2018

A Retrospective Study: The Off-Label Use of Anticonvulsants at a Private Hospital in Indonesia

Perception of oncology practitioners towards off-label use of anticancer medicines in Singapore

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