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Frontiers in Immunology 2021Despite the availability of a lot of effective disease-modifying drugs, multiple sclerosis (MS) (in particular the progressive forms) still represents an important unmet... (Review)
Review
Despite the availability of a lot of effective disease-modifying drugs, multiple sclerosis (MS) (in particular the progressive forms) still represents an important unmet medical need, because of issues in terms of effectiveness, duration of response, safety, and patient compliance. An increasing body of evidence from randomized clinical trials and real-world data suggest that rituximab is a highly effective alternative in both relapsing and progressive MS, with a low discontinuation rate, related to a good benefit/risk profile, and a good compliance. To date, the use of rituximab in patients with multiple sclerosis is not in accordance with the authorized product information (off-label use). However, the use of this medicine is widespread in several countries, and in some cases, it is the most commonly used disease-modifying drug for MS subtypes. This use could be officially recognized by national regulatory authorities, according to specific procedures, to ensure equal access for patients to a safe and effective option.
Topics: Drug Approval; Humans; Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive; Multiple Sclerosis, Relapsing-Remitting; Randomized Controlled Trials as Topic; Recurrence; Rituximab
PubMed: 34295328
DOI: 10.3389/fimmu.2021.661882 -
EFSA Journal. European Food Safety... Oct 2017The conclusions of EFSA following the peer review of the initial risk assessments carried out by the competent authorities of the rapporteur Member State, Greece, and... (Review)
Review
The conclusions of EFSA following the peer review of the initial risk assessments carried out by the competent authorities of the rapporteur Member State, Greece, and the co-rapporteur Member State, the United Kingdom, for the pesticide active substance etoxazole and the assessment of applications for maximum residue levels (MRLs) are reported. The context of the peer review was that required by Commission Implementing Regulation (EU) No 844/2012. The conclusions were reached on the basis of the evaluation of the representative uses of etoxazole as an acaricide on pome fruits, plums, peaches, nectarines, apricots, cherries (sweet), citrus, grapes, strawberries, tomatoes/eggplants, cucurbits inedible peel, cotton seeds and ornamental plants. MRLs were assessed in strawberries, cucurbits inedible peel, plums, tomatoes and aubergines/eggplants. The reliable end points, appropriate for use in regulatory risk assessment and the proposed MRLs, are presented. Missing information identified as being required by the regulatory framework is listed. Concerns are identified.
PubMed: 32625285
DOI: 10.2903/j.efsa.2017.4988 -
Virusdisease Jun 2021COVID-19 outburst initiated from the city of Wuhan in China in December 2019 and has been declared as a public health emergency of international concern. This pandemic... (Review)
Review
UNLABELLED
COVID-19 outburst initiated from the city of Wuhan in China in December 2019 and has been declared as a public health emergency of international concern. This pandemic portrays a spectrum of clinical complications, primarily affecting the respiratory system reported to be caused by a pathogen SARS-CoV-2 belonging to the family of beta coronavirus. Currently, the main objective of the government authorities of all affected countries and research organizations is to find a potential solution in the form of a pharmacological intervention or a vaccination to eradicate the disease and to have a long-term solution to deal with the pandemic. A multitude of anti-viral regimens is proposed based on the repurposing of currently available drugs for other issues or the compassionate use of drugs to immediately control and optimize the healthcare facilities. Meanwhile, a number of agencies are proposing new drug candidates to recreate the possibility of treating the disease. Similarly, a lot of research work is going on worldwide for the development of vaccination. Currently, a good number of candidates has finally reached the borders of Clinical Trials and are expected to be launched as soon as possible. However, the regulatory framework and authorization of these candidates is the most significant aspect of the whole scenario, which needs a specific set of time for validation purposes. The present work is widely focused on the general aspects of COVID-19 and the regulatory landscape for the emergency authorization of repurposed drug candidates, compassionate use of drugs, investigational entities, and vaccine development worldwide.
SUPPLEMENTARY INFORMATION
The online version contains supplementary material available at 10.1007/s13337-021-00684-5.
PubMed: 33969152
DOI: 10.1007/s13337-021-00684-5 -
EFSA Journal. European Food Safety... Aug 2022The conclusions of EFSA following the peer review of the initial risk assessments carried out by the competent authorities of the rapporteur Member State Austria and... (Review)
Review
The conclusions of EFSA following the peer review of the initial risk assessments carried out by the competent authorities of the rapporteur Member State Austria and co-rapporteur Member State Malta for the pesticide active substance abamectin are reported. The context of the peer review was that required by Commission Implementing Regulation (EU) No 844/2012, as amended by Commission Implementing Regulation (EU) No 2018/1659. The conclusions were reached on the basis of the evaluation of the representative uses of abamectin as an insecticide and acaricide on tomato and strawberry, and updated following the request from Commission to review the exposure and risk assessments as regards birds and mammals, aquatic organisms and soil macroorganisms. The risk assessment to bees and non-target arthropods was also updated. The reliable end points, appropriate for use in regulatory risk assessment are presented. Missing information identified as being required by the regulatory framework is listed. Concerns are identified.
PubMed: 36052014
DOI: 10.2903/j.efsa.2022.7544 -
Frontiers in Medicine 2023The World Health Organization (WHO) advocates the use of reliance practices to enable national regulatory authorities (NRAs) to improve patients' access to medicines....
Evaluation of the impact of reliance on the regulatory performance in the South African Health Products Regulatory Authority: implications for African regulatory authorities.
INTRODUCTION
The World Health Organization (WHO) advocates the use of reliance practices to enable national regulatory authorities (NRAs) to improve patients' access to medicines. This study considered whether reliance review translates into swifter medicine authorization.
METHODS
Abridged review outcomes were examined for New Chemical Entity (NCE) and generic applications to the South African Health Products Regulatory Authority (SAHPRA) in Chemistry, Manufacturing and Controls (CMC) and clinical/bioequivalence (BE), as well as overall NCE authorization times.
RESULTS
SAHPRA NCE CMC review time was 91 days (abridged) vs. 179 days (full), applicant response time was 34 vs. 105 days, respectively, and there was a >2-fold time reduction for abridged vs. full CMC review (125 vs. 284 days). There was a 99-day decrease in clinical approval time through an abridged review (230 vs. 329 days) and a decrease in marketing authorization time for NCE abridged assessment (446 vs. 619 days). SAHPRA review time for generic applications was 97 days (abridged) vs. 191 days (full); applicant response time was 26 days (abridged) vs. 81 days (full) and there was a >2-fold time reduction for CMC and BE abridged vs. full review (122 vs. 272 days).
CONCLUSION
These results clearly support World Health Organization recommendations for the use of reliance-based regulatory review to expedite the worldwide availability of safe, effective and needed medications.
PubMed: 37937144
DOI: 10.3389/fmed.2023.1265058 -
Journal of Pharmaceutical Policy and... 2024The pharmaceutical sector in Pakistan has grown over a period, however, there are several barriers in the framework governing the growth of the country's...
BACKGROUND
The pharmaceutical sector in Pakistan has grown over a period, however, there are several barriers in the framework governing the growth of the country's pharmaceuticals. The lack of academia-industry linkage (AIL) is among the critical barriers; hence the focus of the study is to find out the reasons for the lack in the above collaboration. Understanding barriers may help their redressal.
METHOD
This qualitative phenomenology-based study has been conducted in the most prominent pharmacy institutes, located in Lahore, Islamabad, Peshawar, Sargodha, and Quetta. Academic participants, with a minimum experience of 10 years and designation of assistant professor or above were recruited with a two-stage selection process, purposive sampling and snowball sampling. The data were collected using semi-structured interviews with academic experts. Thematic content analysis was employed to conclude the data.
RESULTS
Analysis of data yielded 8 themes with 18 codes. The main reasons for neglected AIL were explained by a partial or complete lack of industrial research and development activities. Other key factors for the scarcity of AILs were the lack of positive attitude from both industry and academia, applied research in academics, and the research and development of the new molecules in the pharmaceutical industry. Support by the government and the drug regulatory authority of Pakistan in terms of regulatory and academic policies was also perceived to be absent. New horizons in research and development could be opened by providing applied research to industry, including but not limited to new molecule development.
CONCLUSION
Academia-industry linkage could be boosted with government-backed funded projects and policies. Academia should focus on the industrial-demanded applied research.
PubMed: 38590643
DOI: 10.1080/20523211.2024.2332872 -
Clinical and Translational Science May 2020Developing a novel drug, including discovery, nonclinical toxicology studies, initial clinical trials, and thorough pivotal studies, may take many years. Once an...
Developing a novel drug, including discovery, nonclinical toxicology studies, initial clinical trials, and thorough pivotal studies, may take many years. Once an applicant has generated this comprehensive body of data, the final step prior to regulatory approval is Health Authority review of the marketing authorization application. Review by regulatory authorities to evaluate whether the data support a positive benefit/risk profile takes many months, adding additional time before patients may access therapy. In this paper, we discuss the various opportunities the US Food and Drug Administration and the European Medicines Agency offer to expedite the drug development and regulatory approval timelines for drugs intended to treat serious diseases and unmet medical needs.
Topics: Drug Approval; European Union; United States; United States Food and Drug Administration
PubMed: 31909876
DOI: 10.1111/cts.12745 -
Nursing Ethics Feb 2022A small minority of nurses are investigated when they fail to meet the required professional standards. Unprofessional conduct does not just affect the nurse but also...
BACKGROUND
A small minority of nurses are investigated when they fail to meet the required professional standards. Unprofessional conduct does not just affect the nurse but also patients, colleagues and managers. However, it has not been clearly defined.
OBJECTIVE
The objective was to identify unprofessional conduct by registered nurses by examining disciplinary decisions by a national regulator.
DESIGN
A retrospective document analysis.
DATA AND RESEARCH CONTEXT
Disciplinary decisions delivered to 204 registered nurses by the Finnish national regulatory authority from 2007 to 2016. The data were analysed with quantitative statistics.
ETHICAL CONSIDERATION
The study received permission from the Finnish National Supervisory Authority for Welfare and Health and used confidential documents that were supplied on the basis of complete anonymity and confidentiality.
FINDINGS
The mean age of the registered nurses who were disciplined was 44 years and 81% were female. Two-thirds had worked for their employer for 5 years or less, 53% had two or more employers and 18% had a criminal history. All the decisions included a primary reason for why the nurses were investigated, but there were also 479 coexisting reasons. In most cases, unprofessional conduct was connected to substance abuse (96%). In addition, stealing of medicine, a decreased ability to work and neglect of nursing guidelines were reported.
DISCUSSION
We found that the nurses were investigated for unprofessional conduct for complex combinations of primary and coexisting reasons. Our study highlighted that more attention needs to be paid to the key markers for unprofessional conduct.
CONCLUSION
Unprofessional conduct is a complex phenomenon that is connected to nurses' individual and working backgrounds and has an impact on their work performance. More research is needed to identify how nursing communities can detect, manage and limit the serious effects and consequences of unprofessional conduct.
Topics: Adult; Female; Humans; Nurses; Retrospective Studies
PubMed: 34583555
DOI: 10.1177/09697330211015289 -
Disaster Medicine and Public Health... Jun 2022In view of the possible disruptions in the manufacturing and supply of health products following the coronavirus disease 2019 (COVID-19) pandemic, the Tunisian medicines...
In view of the possible disruptions in the manufacturing and supply of health products following the coronavirus disease 2019 (COVID-19) pandemic, the Tunisian medicines regulatory authority was mobilized to guarantee patient safety. Teleworking has become the ultimate way of service continuity. The planning was revised according to health priorities. Work procedures were set online. A minimum list of medicines known as "medicines of health and strategic interest" was established. The Directorate of Pharmacy and Medicines (DPM) has been working on updating medicines stock data. A provisional suspension of authorizations for medicines export for 1 mo was decided. A fast-track procedure allowing the validation of alternative sources of raw materials has been put in place. An appeal for a fast track manufacture of hydroalcoholic gel/solutions was launched. A Monitored Emergency Use of Unregistered and Investigational Interventions (MEURI) procedure has been adopted in order to dispense off-label prescriptions of hydroxychloroquine and azithromycin combination. Focus groups were organized in order to set up therapeutic trials exploring possible strategies of COVID-19 treatment, such as serotherapy and BCG vaccine. This proactive and anticipatory policy has made it possible to meet the health challenges dictated by this crisis.
Topics: Humans; COVID-19; Tunisia; Pandemics; COVID-19 Drug Treatment; COVID-19 Serotherapy
PubMed: 33143805
DOI: 10.1017/dmp.2020.432 -
Human Gene Therapy. Clinical Development Sep 2019Cell and gene therapy products belong to a diverse class of biopharmaceuticals known as advanced therapy medicinal products. Cell and gene therapy products are used for... (Review)
Review
Cell and gene therapy products belong to a diverse class of biopharmaceuticals known as advanced therapy medicinal products. Cell and gene therapy products are used for the treatment and prevention of diseases that until recently were only managed chronically. The objective of this study was to examine the characteristics of market authorizations, discontinuations, and prices of cellular and gene therapy products worldwide. We conducted an electronic search of authorized cell, tissue-engineered, and gene therapy products from the databases of the main drug regulatory agencies. The analysis excluded hematopoietic progenitor cell cord blood products authorized by the U.S. Food and Drug Administration. Price information was derived from the Red Book (Truven Health Analytics) for the United States, health technology assessment agencies for Europe, and other public sector sources and company news for other countries. We also searched the scientific literature for authorizations, discontinuations, and price information using MEDLINE/PubMed, Cochrane Library, Google Scholar, and EMBASE databases. All cost data were converted to U.S. dollars. Descriptive analysis was conducted in this study. There were 52 different cell, tissue engineering and gene therapy products with 69 market authorizations in the world as of December 31, 2018. The products included 18 (34%) cell therapies, 23 (43.4%) tissue engineered products, and 12 (22.6%) gene therapies. There were 21 (30.4% of all authorizations) cell therapy, 26 (37.7%) tissue-engineered, and 22 (31.9%) gene therapy market authorizations. The EMA withdrew the authorization for two tissue engineering products, one cell therapy and one gene therapy, and New Zealand lapsed approval of one cell therapy. Most products were first authorized after 2010, including 10 (83.3%) gene therapies, 13 (72.2%) cell therapies, and 13 (56.5%) tissue-engineered products. The treatment price for four allogenic cell therapies varied from $2,150 in India to $200,000 in Canada. The treatment price for three autologous cell therapies ranged from $61,500 in the United Kingdom to a listed price of $169,206 in the United States. Tissue-engineered treatment prices varied from $400 in South Korea to $123,154 in Japan. Gene therapy treatment prices ranged from $5,501 for tonogenchoncel-L in South Korea to $1,398,321 for alipogene tiparvovec in Germany. A significant number of new cell, tissue, and gene therapies have been approved in the past decade. Most products were conditionally authorized and targeted rare cancers, genetic diseases, and other debilitating diseases. However, there are also products approved for cosmetic reasons. Cell, tissue, and gene therapies are among the most expensive therapies available. Healthcare systems are not prepared to assume the cost of future therapies for a myriad of rare diseases and common diseases of epidemic proportions.
Topics: Cell- and Tissue-Based Therapy; Costs and Cost Analysis; Genetic Therapy; Government Agencies; Government Regulation; Humans; Tissue Engineering
PubMed: 30968714
DOI: 10.1089/humc.2018.201