Promoting the Off–label Use of Medicines: Where to Draw the Line?

2016 ◽  
Vol 7 (2) ◽  
pp. 426-433
Author(s):  
Genevra Forwood ◽  
James Killick
Keyword(s):  
Public Health ◽  
Clinical Trials ◽  
Patient Safety ◽  
Marketing Authorisation ◽  
Regulatory System ◽  
Eu Law ◽  
Off Label Use ◽  
Compassionate Use ◽  
Off Label ◽  
Label Use

In Europe, medicines can only be marketed once they have passed through a strict regulatory process, designed primarily to protect patient safety. It is only after in–depth testing on the targeted disease population, including three phases of clinical assessment and clinical trials, that a medicine will obtain a ‘marketing authorisation’. Given its primary goal of ensuring patient safety, EU law only allows a few narrow exceptions to the requirement of amarketing authorisation. Adrug can only be used “off–label”, meaning outside the limits of its marketing authorization, in authorised clinical trials or under one of the strictly defined exceptions, such as severe public health risk, compassionate use for groups of patients or for individual patients on a named patient basis.However, in recent years, a trend has emerged Among Member States to push the boundaries of the existing regulatory system, and actively promote the off–label use of medicines on the ground that they are cheaper than the alternative, authorised medicine. It is questionable whether this trend is in line with EU law.

Safety and Evidence of Off-Label Use of Approved Drugs at the National Cancer Center Hospital in Japan

2020 ◽  
pp. OP.20.00131
Author(s):  
Seiko Bun ◽  
Kan Yonemori ◽  
Hiroko Sunadoi ◽  
Rena Nishigaki ◽  
Emi Noguchi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Pharmaceutical Products ◽  
National Cancer Center Hospital ◽  
Phase Iii ◽  
Cancer Center ◽  
Off Label Use ◽  
Pharmaceutical Development ◽  
Off Label ◽  
Center Hospital ◽  
Label Use

PURPOSE: In Japan, for pharmaceutical products to be covered by public medical insurance, their efficacy and safety must first be confirmed in clinical trials. To our knowledge, this study is the first investigation into the off-label use of pharmaceutical products at a high-volume cancer treatment center in Japan. The objective of this study is to explore the framework necessary for future pharmaceutical development and regulatory approval in the field of oncology by surveying the frequency of and indications for off-label use of pharmaceutical products at the National Cancer Center Hospital in Tokyo, Japan. MATERIALS AND METHODS: The pharmaceutical products used off-label in daily practice from 2003 to 2015 at the National Cancer Center Hospital were retrospectively examined based on applications that had been submitted to an internal review committee requesting off-label use. RESULTS: A total of 1,390 applications were submitted during the study period. The most frequently used supporting documents were the results of phase II trials, followed by case series and phase III trials. The cancer most frequently treated with off-label drugs was sarcoma (15.1%), followed by urologic cancer (9.2%) and GI cancer (7.6%). CONCLUSION: As reported in previous studies, pharmaceutical products were generally used off-label for the treatment of rare cancers, for which large-scale clinical trials are difficult to conduct. Continued discussion of the types of frameworks that are needed to guide pharmaceutical development is necessary.

The off-label use of antipsychotics for insomnia disorder: a major public health concern

2019 ◽  
Vol 64 ◽  
pp. S103
Author(s):  
M. El Gewely ◽  
C. Dusablon ◽  
J.Y. Montplaisir ◽  
A. Desautels ◽  
S.C. Warby
Keyword(s):  
Public Health ◽  
Health Concern ◽  
Off Label Use ◽  
Public Health Concern ◽  
Off Label ◽  
Major Public Health Concern ◽  
Insomnia Disorder ◽  
Label Use

Chapter 6 - Clinical trials and off-label use of drugs

2016 ◽  
Vol 17 (3-4) ◽  
pp. 409-430
Author(s):  
Giovanni Maria Cavo
Keyword(s):  
Clinical Trials ◽  
Off Label Use ◽  
Off Label ◽  
Label Use

Off-label drugs in palliative care: a Group Delphi treatment recommendation process

2020 ◽  
pp. bmjspcare-2019-002165
Author(s):  
Constanze Remi ◽  
Kathrin Weingärtner ◽  
Vera Hagemann ◽  
Claudia Bausewein ◽  
Farina Hodiamont
Keyword(s):  
Palliative Care ◽  
Patient Safety ◽  
Online Survey ◽  
Delphi Process ◽  
Treatment Quality ◽  
Direct Patient Care ◽  
Treatment Recommendation ◽  
Off Label Use ◽  
Off Label ◽  
Label Use

ObjectivesThe use of drugs beyond their marketing authorisation, that is, off-label use, is common practice in palliative care with over 70% of off-label use having little or no scientific support. The lack of evidence makes recommendations for off-label use essential, in order to increase the safety of drug therapy and thus patient safety. The aim of this study was to develop a guide for preparing and consenting drug-specific recommendations for off-label use in palliative care.MethodsGroup Delphi Study with three rounds and a prior online survey to identify topics of dissent. Participants represented professional groups working in palliative care involved in direct patient care and/or drug management and various care settings. Furthermore, representatives of relevant professional associations, experts with academic, non-clinical background and experts with international expertise were invited.Results18/20 invited professionals participated in the prior online-survey. 15 experts participated in the Group Delphi process. Six domains, including identification of drugs, drug uses, assessment of evidence, formulation, consensus and updating of recommendations were generated and respective statements were included in the Group Delphi process. The consensus process resulted in 28 statements forming the guide for recommendations.ConclusionsThe resultant systematic approach for preparing and consenting drug-specific recommendations for off-label use will allow the development of recommendations with transparent and reproducible monographs. This will help to increase treatment quality and patient safety as well as security of decision-making in palliative care. The developed guide is part of a larger project aiming to provide therapy recommendations for areas that have little or no scientific evidence.

Research on patient safety for off-label use of recombinant factor VIIa

2015 ◽  
Vol 72 (8) ◽  
pp. 600-602 ◽  
Author(s):  
Agnes E. Aysola ◽  
Monika M. Wahi ◽  
Russell P. McKelvey ◽  
Cynthia Russell Gerdik
Keyword(s):  
Patient Safety ◽  
Recombinant Factor Viia ◽  
Factor Viia ◽  
Off Label Use ◽  
Off Label ◽  
Label Use

scholarly journals Evaluation of the decision-making process underlying the initial off-label use of vaccines: a scoping review protocol

BMJ Open ◽  
2021 ◽  
Vol 11 (3) ◽  
pp. e042748
Author(s):  
Dieynaba Diallo ◽  
Caroline Quach
Keyword(s):  
Public Health ◽  
Decision Making ◽  
Scoping Review ◽  
Grey Literature ◽  
Decision Making Process ◽  
Off Label Use ◽  
Advisory Committees ◽  
Off Label ◽  
Vaccination Recommendations ◽  
Label Use

IntroductionVaccination has become a central part of public health prevention. Vaccines are introduced after licensure by national regulatory authorities, whereas recommendations for use of licensed vaccines are made by national or international advisory committees and may include off-label use. The methodological and decision-making processes that are used to assess novel initial off-label vaccine use are unclear. This review aims to examine the off-label assessment processes to map evidence and concepts used in the decision-making process and present a common approach between all recommendations and specifics of each decision.Methods and analysisThe methodological framework described at the Joanna Briggs Institute will be applied to this scoping review. A search strategy was developed, in collaboration with an experienced senior health research librarian, by combining Mesgarpour’s highly sensitive search strategies. Peer-reviewed and grey literature will be systematically identified using PubMed, Medline and EMBASE; governmental agency and pharmaceutical websites; and search engines, such as Google Scholar. Reports and studies on off-label vaccine use in public health will be included. Screening will be independently undertaken by two reviewers. Data will be extracted using a standard form. Results will be narratively summarised to highlight relevant findings and guide the development of an analytical framework for off-label vaccination recommendations.Ethics and disseminationThis research does not require ethical approval. This scoping review will provide decision-making elements and a synthesis of knowledge on vaccines off-label use. Findings will be relevant to decision-makers/advisory committees and public health. These will be disseminated through peer-reviewed articles and conferences.

scholarly journals Guide for recommendations on specific drug-related off-label treatment in palliative care: A Group Delphi process

2019 ◽  
Author(s):  
Constanze Remi ◽  
Kathrin Weingärtner ◽  
Vera Hagemann ◽  
Claudia Bausewein ◽  
Farina Hodiamont
Keyword(s):  
Palliative Care ◽  
Patient Safety ◽  
Delphi Study ◽  
Online Survey ◽  
Delphi Process ◽  
Treatment Quality ◽  
Direct Patient Care ◽  
Off Label Use ◽  
Off Label ◽  
Label Use

Abstract Background The use of drugs beyond their authorisation label, i.e. off-label-use, is common practice in palliative care with over 70% of off-label-use having little or no scientific support. Recommendations for off-label-use are essential to increase the safety of drug therapy and thus patient safety. The aim was to develop a guide for preparing and consenting drug-specific recommendations for off-label-use in palliative medicine.Methods Group Delphi Study with three rounds and a prior online survey. Participants represented professional groups working in palliative care involved in direct patient care and/or drug management (doctors, pharmacists, nurses) and various care settings (inpatient/community, university/non-university). Furthermore, representatives of relevant professional associations, experts with academic, non-clinical background and experts with international expertise were invited.Results For the preliminary online-survey 18/20 invited participants returned 18 questionnaires. Six domains, including identification of drugs, drug uses, assessment of evidence, formulation, consensus and updating of recommendations were generated and eventually 22 statements were included in the Group Delphi process. 15 experts participated in this consensus process. In combination with the survey results, consensus was achieved over a 28 statements after 3 Delphi rounds.Conclusions The resulted systematic approach for preparing and consenting drug-specific recommendations for off-label-use will allow to develop such recommendations with transparent and reproducible monographs. This will help to increase treatment quality and patient safety as well as security of decision-making in palliative care. The developed guide is part of a larger project aiming to provide therapy recommendations for areas that have little or no scientific evidence to date.

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