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Vaccine Apr 2017This article reviews the off-label recommendations and use of vaccines, and focuses on the differences between the labelled instructions on how to use the vaccine as... (Review)
Review
This article reviews the off-label recommendations and use of vaccines, and focuses on the differences between the labelled instructions on how to use the vaccine as approved by the regulatory authorities (or "label"), and the recommendations for use issued by public health advisory bodies at national and international levels. Differences between public health recommendations and the product label regarding the vaccine use can lead to confusion at the level of vaccinators and vaccinees and possibly result in lower compliance with national vaccination schedules. In particular, in many countries, the label may contain regulatory restrictions and warnings against vaccination of specific population groups (e.g. pregnant women) due to a lack of evidence of safety from controlled trials at the time of initial licensure of the vaccine, while public health authorities may recommend the same vaccine for that group, based on additional post-marketing data and benefit risk analyses. We provide an overview of the different responsibilities between regulatory authorities and public health advisory bodies, and the rationale for off-label use of vaccines, the challenges involved based on the impact of off-label use in real-life. We propose to reduce off-label use of vaccines by requiring the manufacturer to regularly adapt the label as much as possible to the public health needs as supported by new evidence. This would require manufacturers to collect and report post-marketing data, communicate them to all stakeholders and regulators to extrapolate existing evidence (when acceptable) to other groups or to other brands of a vaccine (class effect). Regulatory authorities have a key role to play by requesting additional post-marketing data, e.g. in specific target groups. When public health recommendations for vaccine use that are outside labelled indications are considered necessary, good communication between regulatory bodies, public health authorities, companies and health care providers or vaccinators is crucial. Recommendations as well as labels and label changes should be evidence-based. The rationale for the discrepancy and the recommended off-label use of a vaccine should be communicated to providers.
Topics: Drug Approval; Humans; Off-Label Use; Product Surveillance, Postmarketing; Vaccines
PubMed: 28341112
DOI: 10.1016/j.vaccine.2017.02.056 -
The Journal of Pharmacy Technology :... Feb 2022Professional regulatory authorities play a critical role in protecting public interest. Yet, there is a growing view that trust in regulatory authorities may be on the...
Professional regulatory authorities play a critical role in protecting public interest. Yet, there is a growing view that trust in regulatory authorities may be on the decline. Awareness has been identified as important for maintaining trust. However, research that examines public awareness and trust in pharmacy regulatory authorities (PRAs) is lacking. This research explores public awareness and trust of PRAs and presents recommendations to enhance PRA communication strategies. An online survey was conducted with the Nova Scotia (Canada) public in 2020. Adopting classifications from the Communications literature, 3 media generations were explored: newspaper, television, and the Internet. The χ test of independence and Kruskal-Wallis test were adopted to explore differences between the generations. Six hundred sixty-two usable surveys were obtained. Over 80% of those surveyed were aware of the existence of the PRA. Those who had heard of the PRA were most aware of its operational responsibilities and less aware of its governance. The Internet Generation was more aware that the PRA includes members of the public in its decision making than expected and showed increased trust toward the PRA versus the other media generations. The findings should help inform PRA communication plans and set baselines to assess whether such plans enhance awareness. Future studies should explore additional aspects of PRA awareness and trust, perform comparisons across pharmacy jurisdictions, and develop and test models of the relationship between PRA awareness and various dimensions of institutional trust.
PubMed: 35141726
DOI: 10.1177/87551225211051593 -
Neurology and Therapy Dec 2017Economic sustainability is of paramount importance in the rapidly evolving therapeutic scenario of multiple sclerosis (MS). Glatiramoids are a class of drugs whose... (Review)
Review
Economic sustainability is of paramount importance in the rapidly evolving therapeutic scenario of multiple sclerosis (MS). Glatiramoids are a class of drugs whose forefather, glatiramer acetate, has been used as a disease modifying drug (DMD) in patients with MS for over 20 years. Its patent expired in 2015; new versions of such drug are nowadays available on the market, potentially contributing to lowering prices and enhancing a better allocation of economic resources. In this review, we analyze the recommendations underlying the approval of both generic drugs and biosimilars by regulatory authorities, and we provide methodological tools to contextualize the design of studies on these new classes of drugs. We examine in more detail the preclinical and clinical data of Copemyl, a new member of the glatiramoid class, focusing on its biological and immunological properties and illustrating randomized controlled trials that led to its authorization.
PubMed: 28762192
DOI: 10.1007/s40120-017-0079-3 -
Disaster Medicine and Public Health... Jul 2022Given the unstoppable spread of coronavirus disease (COVID-19), the development of a vaccine was needed to contain the pandemic. In such a situation of global emergency,...
Given the unstoppable spread of coronavirus disease (COVID-19), the development of a vaccine was needed to contain the pandemic. In such a situation of global emergency, regulatory authorities ensured timely, safe, and equitable access to the vaccine.This article aims to outline the roles of the Tunisian regulatory authority, the Directorate of Pharmacy and Medicines (DPM) at the Ministry of Health, in registration and procurement of the COVID-19 vaccine.Requirement to grant the Exceptional Provisional Authorizations of Marketing (EPAM) for COVID-19 vaccines was 27 days versus 869 days for conventional marketing authorizations (MAs). The DPM has optimized its activity through: early dialogue with manufacturers, online submission, the use of distance communication technologies. It has demonstrated unprecedented flexibility through the continuous and rolling review approach.Regulatory authorities in Tunisia and around the world have partnered with manufacturers to speed up administrative procedures while ensuring the quality, safety, and efficacy of vaccines.
PubMed: 35899949
DOI: 10.1017/dmp.2022.200 -
Frontiers in Medicine 2022The field of regulatory affairs deals with the regulatory requirements for marketing authorization of therapeutic products. This field is facing a myriad of forces... (Review)
Review
The field of regulatory affairs deals with the regulatory requirements for marketing authorization of therapeutic products. This field is facing a myriad of forces impacting all aspects of the development, regulation and value proposition of new therapeutic products. Changes in global megatrends, such as geopolitical shifts and the rise of the green economy, have emphasized the importance of manufacturing and supply chain security, and reducing the environmental impacts of product development. Rapid changes due to advances in science, digital disruption, a renewed focus on the centrality of the patient in all stages of therapeutic product development and greater collaboration between national regulatory authorities have been accelerated by the COVID-19 pandemic. This article will discuss the various trends that are impacting the development of new therapies for alleviating disease and how these trends therefore impact on the role of the regulatory affairs professional. We discuss some of the challenges and provide insights for the regulatory professional to remain at the forefront of these trends and prepare for their impacts on their work.
PubMed: 36698838
DOI: 10.3389/fmed.2022.1082384 -
Investigative Radiology Jan 2020The purpose of this manuscript is to review the successive regulatory actions and decisions following the initial publication by Kanda and colleagues in 2014 regarding... (Review)
Review
OBJECTIVES
The purpose of this manuscript is to review the successive regulatory actions and decisions following the initial publication by Kanda and colleagues in 2014 regarding gadolinium retention in the human brain after multiple gadolinium-based contrast agents (GBCAs) administrations.
MATERIALS AND METHODS
Starting from 2014, the actions and decisions made by all regulatory authorities were collected and summarized region by region. Volumes of GBCA sales in 2018 per region and main countries are also presented as an indicator of patients' exposure to those products.
RESULTS
All regulatory authorities agreed on the absence of evidence of any harmful effect of gadolinium retention in humans. However, based on the same amount of preclinical and clinical evidence available in adults and children, regulatory authorities used different approaches resulting in different actions and decisions regarding the labeling and market authorizations of GBCAs, as well as the specific actions requested to the manufacturers.
CONCLUSIONS
The manufacturers of GBCAs had to face different situations according to the countries, due to the different positions and expectations from regulatory agencies. They have adapted their responses to the different positions of the regulatory agencies and conducted specific preclinical and clinical investigations to provide the expected evidence. It is also their responsibility to continuously monitor the benefit-risk balance of the products and to propose risk minimization measures to the regulatory agencies.
Topics: Adult; Brain; Child; Contrast Media; Drug Hypersensitivity; Female; Gadolinium; Health Policy; Humans; Internationality; Male; Patient Safety
PubMed: 31725063
DOI: 10.1097/RLI.0000000000000605 -
Frontiers in Plant Science 2019This chapter provides an in-depth discussion of the legal and regulatory frameworks surrounding cannabis in the United States, including federal law-as dictated by the... (Review)
Review
This chapter provides an in-depth discussion of the legal and regulatory frameworks surrounding cannabis in the United States, including federal law-as dictated by the Controlled Substances Act (CSA) and governed by various federal agencies like the FDA and DEA-as well as state law-as regulated by each state's laws and regulations authorizing medical and/or adult use cannabis. First, the chapter discusses the definition and classification of cannabis under the CSA, including scheduling under the CSA as well as the process for and potentiality of removing cannabis from Schedule I. Then, it describes the activities relating to industrial hemp that are permitted under the 2014 and 2018 Farm Bill. Next, the chapter addresses state-level cannabis laws. The chapter also analyzes the question of whether state cannabis laws are invalidated and superseded by federal law. Moreover, this section examines the factors underlying the extent of the Department of Justice's enforcement actions relating to state-authorized cannabis activities. The chapter then turns to CBD (cannabidiol) in particular, discussing CBD's legal status under the CSA; the FDA's role in regulating and approving CBD products for medical purposes; and the steps required to take an investigational CBD product through that approval process. The chapter concludes by contending that, while cannabis has had a long and twisting history, and although cannabis-derived products face daunting obstacles to achieving FDA approval as well as rescheduling under both federal and state law, the recent success of one product (Epidiolex) should inspire other manufacturers to develop additional cannabis-derived products through the FDA process.
PubMed: 31263468
DOI: 10.3389/fpls.2019.00697 -
Therapeutic Innovation & Regulatory... Mar 2023National Regulatory Authorities (NRAs) globally are facing the challenge of evaluating pharmaceutical products in a speedy manner, whilst simultaneously ensuring...
National Regulatory Authorities (NRAs) globally are facing the challenge of evaluating pharmaceutical products in a speedy manner, whilst simultaneously ensuring adequate efficacy, safety and quality of approved products. Additionally, common expectations include that the evaluation process is competent, flexible, commensurate with risk, efficient and rapid. In 2014, the Australian regulatory system was out of step with global regulatory developments which led to a comprehensive regulatory review and reform process. As part of the reforms, two Facilitated Regulatory Pathways (FRP) were developed for prescription medicines: Priority Review (PR) and Provisional Approval (PA). Furthermore, regulatory reliance and recognition arrangements have been expanded with the Therapeutic Goods Administration (TGA) making increased use of evaluation reports by trusted NRAs. The new pathways have been utilised by the pharmaceutical industry in Australia since 2017, with the number of medicines going through these pathways gradually increasing. Additional facilitated pathways have been developed following the review, providing alternatives to the standard pathway for registration of prescription medicines in Australia. The reform is timely, helping to position Australia well in the current global regulatory climate.
Topics: Australia; Prescription Drugs; Drug Industry; Prescriptions
PubMed: 36271207
DOI: 10.1007/s43441-022-00465-2 -
Allergologie Select 2023Allergen immunotherapy (AIT) has been performed for 112 years. In this article we summarize regulatory standards and challenges based on scientific evidence on AIT. Most... (Review)
Review
Allergen immunotherapy (AIT) has been performed for 112 years. In this article we summarize regulatory standards and challenges based on scientific evidence on AIT. Most crucial and timely aspects concerning AIT are addressed from the regulatory perspective of the authors as employees of a national competent authority in Europe: (1) product specificity; (2) clinical efficacy; (3) treatment for adults and children (needs for extrapolation); (4) allergen exposure chambers; (5) biomarkers; (6) standardization; (7) real-world evidence; (8) independent official batch release (benefit and challenges); (9) harmonization on the EU level. The Paul-Ehrlich-Institut (PEI), the Federal Institute for Vaccines and Biomedicines, in Langen near Frankfurt/Main in Germany, examines and evaluates the benefits and risks of AIT products within the course of clinical development, marketing authorization, and subsequently throughout their entire life cycle to ensure high-quality, safe, and effective AIT products.
PubMed: 38143937
DOI: 10.5414/ALX02413E -
Exploratory Research in Clinical and... Sep 2022Coronavirus disease 2019 (COVID-19) is rapidly evolving with millions of cases and death reported globally. The first-ever case of COVID-19 in Bhutan was confirmed on 5...
Coronavirus disease 2019 (COVID-19) is rapidly evolving with millions of cases and death reported globally. The first-ever case of COVID-19 in Bhutan was confirmed on 5 March 2020 in a 76-year-old American tourist. The national regulatory authorities have implemented varying degrees of regulatory flexibilities to prevent unnecessary death by ensuring timely access to medical products and technologies during the pandemic. The Drug Regulatory Authority of Bhutan, as a national medicines regulatory agency responsible for ensuring the quality of medical products has implemented several regulatory mechanisms to ensure access to medical products including COVID-19 vaccines during the pandemic. In this commentary, we discuss the regulatory flexibilities that ensured access to medical products and COVID-19 vaccines during the pandemic.
PubMed: 35859611
DOI: 10.1016/j.rcsop.2022.100156