Skip to main content
. 2015 Nov 6;80(6):1269–1280. doi: 10.1111/bcp.12750

Table 1.

An overview of the studies

Title (Author, year, reference) Country Study aim Study design (duration) Method Target group Survey instrument/tool characteristics Outcome domains Response rate (%) Results
Awareness about and views of parents on the off‐label drug use in children (Bang et al., 2014) 14 India To explore awareness among parents regarding both off‐label drug use in children Cross‐sectional study (1 year) Face‐to‐face interview using structured questionnaire Parents (n = 400) Validated, structured questionnaire Parental views on safety and labelled use of drugs in children 98.8 (400/405) Participants felt that drugs used in children in hospital (89.5%) and community setting (80.3%) were either safe or extremely safe
To explore willingness to allow their child to participate in clinical research
Comprised of 18 questions Awareness regarding off‐label use in children 30% parents were aware of off‐label drug use in children
Face validity done for translated version of questionnaire (English to Hindi and Marathi languages) Communication from healthcare worker about off‐label drug use in children 93% wanted to be informed whenever drug prescribed in off‐label manner
Parental views on off‐label drug use in children 73% felt the off‐label drug use is illegal and 57% would ask for a change to labelled drug
Willingness of allowing child participation in clinical trial Participants not keen to allow their healthy children to participate in clinical trials participate
Results from the 2012–2013 paediatric national survey on off‐label drug use in children in Spain (OL‐PED study) (Pérez et al., 2014) 15 Spain To estimate the current state of knowledge on off‐label use of drugs among Spanish paediatricians Multicentre, descriptive, cross‐sectional study (8 months) Online questionnaire Paediatricians (n = 673) Comprised of 10 questions Knowledge and views on off‐label prescribing 7.5 (673/9027) 75.1% of participants knew the meaning of off‐label use
To assess the need to adopt measures to improve current practice Distributed via email Informing parents/guardians and documentation 47% knew the importance of noting the off‐label use in medical records. However only 22% wrote it in the medical records
To estimate how well informed they are about legal liabilities arising from off‐label use of medications and to ascertain the sources paediatricians use to obtain information on drugs View on factors of support from the point of view of medical liability
Sources of information used to obtain information on drugs
A questionnaire‐based study in Calabria on the knowledge of off‐label drugs in paediatrics (Saullo et al., 2013) 20 Italy To evaluate the knowledge of off‐label drugs in paediatricians of Calabria region in Italy 2‐phase cross‐sectional study (8 months) Online anonymous questionnaire Paediatricians (n = 85) Comprised of 10 questions; all question had tick box answers Drugs and their off‐label use 47.3 (85/180) 40% used off‐label drugs ‘sometimes’
General guidelines on risk and limitation of off‐label drugs in clinical practice 74% did not have good knowledge about this practice
Children's view on unlicensed/off‐label paediatric prescribing and paediatric clinical trials (Mukattash et al., 2012) 22 Northern Ireland To explore the views and perspectives of children on the unlicensed/off‐label use of medicines in children and on the participation of children in clinical trials Cross‐sectional study (not reported) Focus group discussions School children (n = 123) Sessions moderated by trained researcher Views on the unlicensed use of medicines in children 63.4 (123/194) Pupils viewed unlicensed/off‐label use of medicine in children as unsafe and unethical
Facilitator was familiarized with focus group sessions by conducting 2 simulation sessions Informing parents/guardians and children Pupils felt it is necessary to test medicines in children to improve availability of licensed products
Study guide was used to ensure uniformity of focus group discussions Clinical trials and willingness to participate They felt that older children and parents should be informed when drug used in off‐label manner
Illness and participation in clinical trials
Healthcare professional experiences and attitudes on unlicensed/off‐label paediatric prescribing and paediatric clinical trials (Mukattash et al., 2011) 16 Northern Ireland To investigate the knowledge and views of a range of healthcare professionals regarding the use of unlicensed/off‐label medicines in children and the participation of children in clinical trials Cross‐sectional study (not reported) Prospective questionnaire survey Consultant paediatricians, general practitioners, community pharmacists, paediatric nurses (n = 563) Survey instrument designed after an extensive review of the literature Experiences and views of healthcare professionals regarding the use of unlicensed and off‐label medicines in children 46.5 (563/1212) More familiarity with the term unlicensed compared to off‐label prescribing
Comprised of 39 questions Parental involvement in decision making regarding the medicine being prescribed for their child 30.7% reported informing parents/guardians on the use of unlicensed/off‐label medicines in children
Questionnaire was piloted using a small number of healthcare professionals (n = 20; pharmacists and doctors) and respondents indicated that the questionnaire was clear and easy to understand Information sources that healthcare professionals use when prescribing, dispensing and administering unlicensed/off‐label medicines to children Willingness to be actively involved in clinical trials is highest among consultant paediatricians
Distributed via postal services and email Paediatric clinical trials
4‐week window to complete the questionnaire Dose‐related issues when prescribing unlicensed and off‐label medicines in children
Perceptions and attitudes of Jordanian paediatricians towards off‐label paediatric prescribing(Mukattash et al., 2011) 17 Jordan To investigate the knowledge and views of Jordanian paediatricians regarding off‐label prescribing in children Cross‐sectional study (not reported) Prospective questionnaire survey Hospital based paediatricians (n = 250) Survey instrument designed after an extensive review of the literature Experiences and views of paediatricians regarding off‐label prescribing to children 83 (250/300) 69% of participants were familiar with the term off‐label medicines but only 28% knowingly prescribed off‐label medicine to children
Comprised of 39 questions Parental involvement in decision making regarding the medicine being prescribed for their child Majority did not obtain informed consent or tell parents when they prescribe off‐label medicine
Face and content validity done by panel of experts
Survey instrument was piloted prior to use Dose – related issues with off‐label prescribing
Survey instrument was personally delivered
Collected after 4 weeks
Off‐label, off‐limits? Parental awareness and attitudes towards off‐label use in paediatrics (Lenk et al., 2009) 13 Germany To explore knowledge and view regarding off‐label prescribing and participation in clinical trials Cross‐sectional study (4 months) Questionnaire survey Parents (n = 94) Comprised of three sections Knowledge and view of off‐label use in paediatrics Parents of children with renal disease: 54 (43/80) Parents of healthy children 64 (51/80) Knowledge about off‐label drug use was poor in both groups
Participation in clinical trials Refusal to off‐label drug use was low
Parents with poor knowledge about off‐label drug use tend to refuse to volunteer their child for participation in clinical trials
Public awareness and views on unlicensed use of medicines in children (Mukattash et al., 2008) 12 Northern Ireland To explore awareness and views of the general public on unlicensed use of medicines in children Multicentre, cross‐sectional study (4 months) Face‐to‐face interview using structured questionnaire Parents (n = 1000) Draft survey was examined for fitness for purpose and face validity in focus group Views on safety and labelled use of drugs in children Not reported Majority had no previous knowledge about unlicensed/off‐label drug use
To explore participation of children in clinical trials Final version was piloted in a sample of 20 members of public Awareness regarding off‐label use in children Most parents felt that they should be told when drug prescribed unlicensed/off‐label manner
Interview conducted by trained interviewer Communication from healthcare worker about off‐label drug use in children Views on clinical participation varied according health status of children
Views on off‐label drug use in children
Willingness of allowing child participation in clinical trial
Attitudes and experiences of community pharmacists towards paediatric off‐label prescribing: a prospective study (Stewart et al., 2007) 21 United Kingdom To identify community pharmacist experiences of, and attitudes towards paediatric off‐label prescribing Cross‐sectional study (not reported) Prospective questionnaire survey Community pharmacists (n = 482) Comprised of 21 questions with combination of tick‐box responses and written comments Knowledge of and reasons for paediatric off‐label prescribing 32.1 (482/1500) Familiarity with concept of off‐label prescribing (73%), mainly gained through dispensing experience (64%)
Piloted prior to use Classes of drugs dispensed off‐label 40% knowingly dispensed off‐label prescription to children
Most common reasons for concerns when dispensing off‐label Most common reason of off‐label prescription was age (84.6%)
Sources of information for dispensing to children Major concern regarding off‐label drug use is lack of dosage data (60%)
Transfer of information to parents and prescribers Majority feels that pharmacist should inform parents and prescribers if drug is used in off‐label manner (66% and 78%)
A prospective questionnaire assessment of attitudes and experiences of off‐label prescribing among hospital based paediatricians (McLay et al., 2006) 18 Scotland To assess current attitudes of hospital based paediatricians to off‐label prescribing, and the performance of clinical trials in children Cross‐sectional study (1 year) Prospective questionnaire survey Hospital based consultants and specialist registrars (n = 151) Comprised of 24 questions with combination of tick‐box responses and written comments Perceived reasons for off‐label prescribing 59 (151/257) Familiarity with concept of off‐label prescribing (92.8%)
Concerns about off‐label prescribing 90% knowingly prescribed off‐label prescription to children
Parental and GP involvement Most common reason of off‐label prescription was age
Paediatric clinical trials Major concern regarding off‐label drug use is lack of efficacy data (63.8%)
Off‐label prescribing to children: attitudes and experience of general practitioners (Ekins‐Daukes et al., 2005) 19 Scotland To identify experience with and attitudes towards paediatric off‐label prescribing in primary care Cross‐sectional study (not reported) Prospective questionnaire survey General practitioners (n = 202) Questionnaire was piloted prior to use Prior knowledge of off‐label prescribing and licensing recommendations 58 (202/346) Majority familiar with off‐label concept (705) but 40% admitted knowingly prescribing off‐label dugs
Attempt to increase response rate was made Acknowledgement of and perceived problems with off‐label prescribing Prescribing information sources used in practice Ranked development of paediatric formulations and clearer dosage information highly as a means to reducing off‐label prescribing
Comprised of 13 questions Importance of proposed methods or reducing off‐label prescribing
Collected after 4 weeks