Common Drugs with Effective Off-Label Uses

The term off-label drug use (OLDU) is utilized broadly in the medical literature. It is a polarizing term since it may be related to incredible advantage or harm to patients. OLDU is defined as drug uses that not included in the indications or dosage regimens listed in the drug labeling. The main issue with off-label use is that there is insufficient information supportsthe use ofthe drug so this review aims to give brief information about some common drugs with effective and useful off-label uses based on scientific study and to encourage the researcher toprovidesufficientinformation for the physician and health care providersaboutoff-label uses to decrease the risk of harm to the patients. There are many drugs with various off-label uses that play an important role clinically such as using atorvastatin in chronic heart failure (CHF) due to its pleiotropic action also it can show a significant reduction in the frequency of hospitalization due to CHF exacerbation. However, that make off-label uses an important is several diseases do not have approved drugs, partly because the diseases are rare or conducting clinical trials and marketing the drug for such diseases may not be gainful.

Consumption pattern of gabapentinoids in a tertiary health-care system: a five year study from 2012-2017

Background: Treating chronic pain has always been a great challenge for the treating physician. As the available pharmacotherapy do not always yield desired results, wider armamentarium is needed in tackling the chronic pain. Hence gabapentinoids are often considered, but it has led to widespread off label use of these groups of medications. Hence a study of consumption pattern was carried out to assess the usage of gabapentinoids.Methods: The consumption pattern of gabapentinoids (gabapentin/ pregabalin/ any combination) was recorded from the medical stores and dispensary of tertiary care teaching hospital.Results: There is an upward trend in the usage of all gabapentinoids between 2012 to 2017 and pregabalin was seen to be the commonly used gabapentinoid. In the recent years apart from individual gabapentinoids there is a trend favoring use of FDCs of gabapentinoids with nortryptyline and methylcobalamine.Conclusions: The increase in the usage pattern should alarm us to look into the off label uses of gabapentinoids. As there is a possibility of abuse and misuse of gabapentinoids clear guidelines regarding the same is the need of the hour.

Survey on off-label uses of drugs in 24 hospitals / 中国药学杂志

OBJECTIVE: To investigate the off-label uses of drugs in domestic hospitals, so as to provide baseline data for policy-making and strategy for risk management. METHODS: The off-label uses of drugs in 24 hospitals were collected and analyzed according to the categories of drugs and off-label uses and the strength of evidence for the off-label use of drugs. RESULTS: One thousand six hundred and fifty-two cases of off-label uses involving 998 drugs were included in this study. The top three hospitals with the largest numbers of off-label uses were Beijing Hospital (297 cases, 18.0%), Peking University Third Hospital (282 cases, 17.1%) and Yantai Yuhuangding Hospital (256 cases, 15.5%). There were 998 kinds of drugs which had off-label uses in these 24 hospitals. The top three categories of drugs with the highest frequncies of off-label uses were antineoplastics (388 cases, 23.5%), immunomodu-latingagents (315 cases, 19.1%), and cardiovascular system agents (177 cases, 10.7%). The off-label use of drugs concentrated in indication (1493 cases, 90.4%), dosage (349 cases, 21.1%), route and administration (154 cases, 9.3%), and population (65 cases, 3.9%) in this study. One thousand one hundred and fifty-five (73.3%) off-labels were supported by foreign package inserts, international guidelines, domestic guidelines, expert consensus, monographs and literatures. CONCLUSION: Off-label uses of drugs existed widely in these 24 hospitals, which pose potential risk on medication safety and efficacy, law responsibility and ethical issues. The government should accelerate the development and promulgation of correspondent law to promote rational use of drugs. And the relevant associations should develop guidelines for off-label uses and provide reliable evidence for safe medication.

Technological process with cochrane library of reasonability assessment of off-label uses of drugs found in comments on prescription / 中国药学杂志

OBJECTIVE: Off-label uses of drugs incur much debate, which is often encountered when doing prescription comment. The reasonability assessment of off-label uses is the key problem and requires solution urgently. This paper attempts to set up a normative evaluation process of off-label uses in routine prescription comment. METHODS: The off-label uses gathered in 2014 prescription comment were used for example. The off-label uses were searched in the Cochrane Library, and the results such as CDSR (Cochrane Database of Systematic Review), DARE (Database of Abstracts of Reviews of Effectiveness), RCT (Random Clinical Controlled Trial) and CCT (Clinical Controlled Trial) were screened according the criterion and analyzed. RESULTS: Eight Off-label uses were found with prescription comment. The edaravone for head injury, L-ornithine-L-aspartate for rise of hepatic transaminase, progesterone for urinary calculi and levocarnitine for myocardium protection were no evidence found in Cochrane Library, and were treated as irrationality. According to results of CDSR and DARE the edaravone for brain hemorrhage, and levocarnitine for cancer fatigue were not supported, but thymalfasin for cancer were supported. After assessing risk of bias with Cochrane tool, only under special circumstances can alprostadil for pulmonary hypertension (PH) or pulmonary arterial hypertension (PAH) be evaluated as conformation with evidence-based medicine. CONCLUSION: Assessment of reasonability for Off-label uses according to evaluation process using Cochrane Library can improve medication reasonability and safety, and increase the knowledge and competence of drug administration.

Review of the legal issues of off-label drug usage / 中华医院管理杂志

This paper identified misunderstandings of the measures taken in China to overcome the problems incurred by off-label uses (namely reliance on drug makers to modify their medicine specifications,on medical institutions to enhance their regulation and management of off-label uses,and on informed consent to avoid risks).Based on such findings,the paper named defects found with such measures,and puts forward feasible ideas and methods to make such off-label uses legitimate and reasonable.These include clarification of the legal status of the medicine specifications,encouraging authoritative guidance for off-label uses,and determining the subject of evaluation to approve the off-label uses as reasonable.Such efforts aim at helping off-label uses out of the legal difficulties.

Clinical use and control of the dispensing of thalidomide in Brasilia-Federal District, Brazil, from 2001 to 2012 / Uso clínico e controle sobre a dispensação de talidomida em Brasília-DF, Brasil, de 2001 a 2012

The use of thalidomide was never discontinued in Brazil where it is prescribed for leprosy type 2 reaction. Babies with birth defects compatible with the thalidomide embryopathy phenotype were born after 1965, an indication that control on drug dispensing and use failed in the country. The article reports data on thalidomide dispensing and clinical uses in the Federal District in 2011/12, when new rules were put into effect, and data on drug dispensing and use obtained ten years earlier. It was found that the number of patients making use of thalidomide declined from 819 in 2001 to 369 in 2011/12. Leprosy accounted for over 70% of prescriptions in both time periods analyzed in this study. In the same time interval, however, use for lupus erythematosus decreased from 13.7 to 4.9%, while that for multiple myeloma increased from 2.9 to 20.3% of all prescriptions. Thalidomide prescription for the remaining approved indications was far less frequent, and so was the use for off label indications that accounted for <1% of prescriptions in 2001 and 2011/12. Registration of prescribing doctors, patients and dispensing units at the state department of health, apparently rendered this control more effective and reliable.

O uso da talidomida nunca foi interrompido no Brasil, sendo prescrita para tratar a reação tipo 2 da hanseníase. Crianças com defeitos congênitos compatíveis com o fenótipo da embriopatia causada pela talidomida nasceram após 1965, evidenciando que o controle do uso e da dispensação do medicamento falhou no país. O artigo relata dados sobre a dispensação e usos clínicos da talidomida no Distrito Federal em 2011/12, quando a nova regulamentação passou a vigorar, e dados sobre a dispensação e uso do medicamento 10 anos antes. Os resultados mostraram que o número de pacientes que usaram talidomida decresceu de 819 em 2001 para 369 em 2011/12. A hanseníase foi a indicação clínica para mais de 70% das prescrições nos períodos analisados no estudo. No mesmo período, entretanto, o uso para lupus eritematoso reduziu de 13,7 para 4,9%, enquanto o uso para mieloma múltiplo cresceu de 2.9 para 20,3% de todas as prescrições. A prescrição de talidomida para as outras indicações aprovadas foi muito menor, enquanto para indicações não aprovadas correspondeu a < 1% das prescrições em 2001 e 2011/12. O cadastro dos prescritores, pacientes e unidades dispensadoras na secretaria estadual de saúde, aparentemente tornou esse controle mais eficiente e confiável.

A Drug Utilization Study on Off-Label Uses / 薬剤疫学

Objective : Percutaneous ethanol injection (PEIT) for hepatocellular carcinoma is basic treatment of hepatocellular carcinoma. However, it is off-label uses to use ethanol for this treatment in the current state. Then, we executed the drug utilization study of Absolute ethanol injections at this time. <BR>Methods : We executed the drug utilization study of Absolute ethanol injections at this time. <BR>Results : The recovery of the questionnaire was high with 84.4% (151/179 medical institutions). The ethanol injections was made by 85% (129/151 facilities). Facilities where 100 or more ethanol injections was made annually were 76%. The raw material for making the ethanol injections was the reagent which was not the medicine in 44% of facilities. Information on safety and effectiveness concerning the ethanol injections was hardly offered from pharmacy. Finally, 96% (124/129 facilities) demanded marketing the ethanol injections. <BR>Conclusion : We investigated the research report number by using “ICHUSHI” CD-ROM version (1988-1998) which was the medical literature data base. We were able to collect reports concerning PEIT of hepatocellular carcinoma of 636 reports. We were able to collect reports which used PEIT as a treatment method of hepatocellular carcinoma by as many as 636 reports. It has been understood that PEIT is enforced in Japan. Next, we reviewed the document and collected and evaluated information on effectiveness and safety concerning the ethanol injections used for PEIT. The treatment object, the usage, and the dosage have been decided at the relating academic meeting. Moreover, there was a report concerning a lot of effectiveness and safety in the PEIT treatment method of hepatocellular carcinoma. The ethanol injections used for PEIT is one of the medicines where information and scientific evidence concerning effectiveness and safety are accumulated. Moreover, a large amount of ethanol injections are nationally made in the pharmacy in the hospital. However, it has been proved that there are a lot of problems about the making. The ethanol injections is the medicine which should be supplied and made by the pharmaceutical company.