marketing authorisation
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2021 ◽  
Vol 11 ◽  
Author(s):  
Jorn Mulder ◽  
Tobias van Rossum ◽  
Segundo Mariz ◽  
Armando Magrelli ◽  
Anthonius de Boer ◽  
...  

Pancreatic cancer has a dismal prognosis and only a few treatment options are available. In the European Union, pancreatic cancer classifies as a rare disease, allowing drug developers to apply for orphan medicinal product (OMP) designation. The aim of this study was to provide more detail on OMPs for pancreatic cancer. All applications for OMP designation submitted to the EMA between 2000 and 2019 were identified. For each medicinal product that received an OMP designation, the mode of drug action, use of protocol assistance, and current life cycle status was determined. Fifty-two medicinal products received an OMP designation. At the time of submission, eighteen OMPs were at the non-clinical and 34 OMPs were at the clinical stage of development. At least fourteen kinds of mode of action were explored in the condition. For eighteen out of 52 OMPs protocol assistance was sought. At the time of data analysis, one OMP received marketing authorisation and 24 OMPs were ongoing in development. Many medicinal products for pancreatic cancer received an OMP designation and the majority of these products was already in the clinical stage of development. Nonetheless, the success rate of OMPs for pancreatic cancer that reach the market is low, and increasing this rate is something to aspire. Fortunately, development is still ongoing for a part of the OMPs, and a few developers are planning to submit a marketing authorisation application in the near future. This however does not guarantee success, as pancreatic cancer remains a difficult disease to treat. Developers are advised to make optimal use of incentives such as protocol assistance, establishing (early) dialogue between regulators and drug developers and to agree on important topics such as clinical trial design.


2021 ◽  
Vol 8 ◽  
Author(s):  
Jorn Mulder ◽  
Robin Verjans ◽  
Ciska Verbaanderd ◽  
Elias Pean ◽  
Just Weemers ◽  
...  

After marketing authorisation, the development of a medicinal product often continues with studies investigating new therapeutic indications. Positive results can potentially lead to changes to the terms of the marketing authorisation, such as an extension of therapeutic indication(s). These studies can be initiated and sponsored by the marketing authorisation holder (MAH) or by others. When results from an investigator-initiated trial suggest that an authorised medicinal product is safe and effective for a new therapeutic indication, physicians may want to treat their patients with this medicinal product. In such a situation, it is desirable to extend the therapeutic indication(s) via the regulatory approval process, as this can facilitate patient access within the European Union. There may however be challenges when the MAH did not conduct the study and might not have access to the data. In this perspective, we focus on the possibilities to extend the therapeutic indication(s) of an already authorised medicinal product based on results from investigator-initiated trials. We address: (1) the advantages of an extension of indication; (2) the regulatory requirements for a variation application; (3) investigator-initiated trials as a basis for regulatory approval; (4) the role of the MAH in extending the indication. With this article, we want to emphasize the importance of a collaborative approach and dialogue between stakeholders with the aim to facilitate access to effective medicinal products.


2021 ◽  
Vol 25 (76) ◽  
pp. 1-228
Author(s):  
Peter Murphy ◽  
David Glynn ◽  
Sofia Dias ◽  
Robert Hodgson ◽  
Lindsay Claxton ◽  
...  

Background The first histology-independent marketing authorisation in Europe was granted in 2019. This was the first time that a cancer treatment was approved based on a common biomarker rather than the location in the body at which the tumour originated. This research aims to explore the implications for National Institute for Health and Care Excellence appraisals. Methods Targeted reviews were undertaken to determine the type of evidence that is likely to be available at the point of marketing authorisation and the analyses required to support National Institute for Health and Care Excellence appraisals. Several challenges were identified concerning the design and conduct of trials for histology-independent products, the greater levels of heterogeneity within the licensed population and the use of surrogate end points. We identified approaches to address these challenges by reviewing key statistical literature that focuses on the design and analysis of histology-independent trials and by undertaking a systematic review to evaluate the use of response end points as surrogate outcomes for survival end points. We developed a decision framework to help to inform approval and research policies for histology-independent products. The framework explored the uncertainties and risks associated with different approval policies, including the role of further data collection, pricing schemes and stratified decision-making. Results We found that the potential for heterogeneity in treatment effects, across tumour types or other characteristics, is likely to be a central issue for National Institute for Health and Care Excellence appraisals. Bayesian hierarchical methods may serve as a useful vehicle to assess the level of heterogeneity across tumours and to estimate the pooled treatment effects for each tumour, which can inform whether or not the assumption of homogeneity is reasonable. Our review suggests that response end points may not be reliable surrogates for survival end points. However, a surrogate-based modelling approach, which captures all relevant uncertainty, may be preferable to the use of immature survival data. Several additional sources of heterogeneity were identified as presenting potential challenges to National Institute for Health and Care Excellence appraisal, including the cost of testing, baseline risk, quality of life and routine management costs. We concluded that a range of alternative approaches will be required to address different sources of heterogeneity to support National Institute for Health and Care Excellence appraisals. An exemplar case study was developed to illustrate the nature of the assessments that may be required. Conclusions Adequately designed and analysed basket studies that assess the homogeneity of outcomes and allow borrowing of information across baskets, where appropriate, are recommended. Where there is evidence of heterogeneity in treatment effects and estimates of cost-effectiveness, consideration should be given to optimised recommendations. Routine presentation of the scale of the consequences of heterogeneity and decision uncertainty may provide an important additional approach to the assessments specified in the current National Institute for Health and Care Excellence methods guide. Further research Further exploration of Bayesian hierarchical methods could help to inform decision-makers on whether or not there is sufficient evidence of homogeneity to support pooled analyses. Further research is also required to determine the appropriate basis for apportioning genomic testing costs where there are multiple targets and to address the challenges of uncontrolled Phase II studies, including the role and use of surrogate end points. Funding This project was funded by the National Institute for Health Research (NIHR) Evidence Synthesis programme and will be published in full in Health Technology Assessment; Vol. 25, No. 76. See the NIHR Journals Library website for further project information.


2021 ◽  
Author(s):  
Jane Mashingia ◽  
S Maboko ◽  
P I Mbwiri ◽  
A Okello ◽  
S I Ahmada ◽  
...  

A review of the East African Community (EAC) joint regulatory review process was conducted, registration timelines analyzed and key milestones, challenges and opportunities documented for the period of July 2015 to January 2020. A total of 113 applications were submitted for joint scientific review. Among these, 109 applications were assessed, 57 were recommended for marketing authorisation, 52 applications had queries to applicants and four applications were under review. A total median approval time for all products ranged from 53 to 102 days. The maximum time taken by a regulator to review the dossier was 391 days and the minimum time was 44 days. For applicants, the maximum time to respond to queries was 927 days and the minimum time was nine days. The total median time for granting marketing authorisation by the National Medicines Regulatory Authorities (NMRA) decreased from 174 to 39 working days in 2015 and 2019 respectively. However, not all EAC NMRA has granted marketing authorisation to all 57 products due to non-payment of applicable fees by applicants. Long regulatory approval timelines were contributed by limited capacity for timely scientific review of dossier by some NMRA, lack of online portal to share dossiersand assessment reports, delay in responding to queries by applicants and deficiencies in dossier. The metric tool and register of medical products submitted for joint scientific review had incomplete data. Challenges were identified and actions recommended to ensure regional regulatory system optimization, efficiency, transparency, sustainability and accountability.


2021 ◽  
Vol 14 (2) ◽  
Author(s):  
Katarzyna Miaskowska-Daszkiewicz

The development and marketing authorisation of COVID-19 vaccines has given the authorities a much-anticipated instrument to fight a pandemic. At the same time, however, for the extinction of the epidemic to become real, according to epidemiologists' estimates, the threshold of herd immunity must reach the value of 50-70 percent. To ensure mass vaccination, it should be considered whether a compulsory vaccination against COVID-19 would be an acceptable solution. It is a sensitive issue in the context of the right to self-determination, guaranteed both in Article 8 European Convention on Human Rights, as well as most modern constitutions. The aim of this paper is to investigate whether the compulsory vaccination against COVID-19 could be the next step in the fight against the pandemic. In particular, whether the current approach of the ECHR and national courts to compulsory vaccination can be considered adequate in relation to COVID-19 vaccines with a conditional marketing authorisation.


2021 ◽  
Vol 9 (4) ◽  
pp. 185-190
Author(s):  
E. O. Zhuravleva ◽  
N. Yu. Velts ◽  
G. V. Kutekhova

Since 1 January 2021 the authorisation of medicines in the Russian Federation has to be performed according to the Eurasian Economic Union (EAEU) procedure. The Pharmacovigilance System Master File (PSMF) is the main document describing the pharmacovigilance system of the marketing authorisation holder (MAH) or its authorised representative.  The aim of the study was to analyse noncompliances with the EAEU requirements, which were revealed during PSMF assessment.Materials and methods: the authors analysed 687 pharmacovigilance documents included in registration dossiers that were submitted for assessment from 1 January to 30 June, 2021.  Results: the authors identified and systematised the main noncompliances with the EAEU requirements in terms of presentation, content, completeness of each PSMF section. They analysed the frequency of noncompliances in PSMFs and identified the most frequent flaws of MAHs’ pharmacovigilance systems.Conclusions: the authors give recommendations for elimination of significant noncompliances identified during PSMF assessment, which may include: timely updating, maintenance, and revision of the documents in accordance with changes in legislation and any other significant changes, regular training of pharmacovigilance staff, etc. The results of this review will be useful for MAHs as the main participants of the marketing authorisation process who are directly involved in the pharmacovigilance system management at the pre- and post-authorisation stages, and will help them prevent potential mistakes when drawing up pharmacovigilance system documents.


2021 ◽  
pp. 336-347

This chapter begins by defining intellectual property rights as the protection of the ‘creation’ of the mind and describing many different rights that are protected by both statute and common law. It divides intellectual property into two broad categories: industrial property and copyright. It also explores the various statutory and common law intellectual property regimes that have their own idiosyncratic criteria in order to qualify for the protection they offer. The chapter distinguishes relevant intellectual property rights for pharmaceutical product marketing authorisation holders from ‘traditional’ intellectual property rights to regulatory exclusivities. It explores the characteristics of regulatory exclusivities that are akin to other intellectual property rights but have their own unique criteria for qualification and enforcement.


2021 ◽  
pp. 1-26

This chapter begins with the early regulation of medicines, which was concerned with quality rather than any scientific assessment of efficacy and safety. It describes the latter half of the nineteenth century wherein the need for objective scientific assessment of the safety and efficacy of new medicines and the concept of risk–benefit of medicines was born. It also talks about the configuration of the Licensing Authority in the UK and the European Medicines Evaluation Agency (EMEA) as a key provision of both the Medicines Act 1968 and the European medicines legislation. The chapter analyses Directive 2001/83/EC, which governs all the requirements for the contents of the application dossier and grant of marketing authorisation of medicinal products. It recounts the development of statutory controls in the UK that was consistent with the evolution of the overarching European medicines legislation and the formation of the central European regulatory agency.


2021 ◽  
Vol 8 ◽  
Author(s):  
Hasumati Rahalkar ◽  
Alan Sheppard ◽  
Gustavo Mendes Lima Santos ◽  
Chitralekha Dasgupta ◽  
Sonia Mayra Perez-Tapia ◽  
...  

Background: The aim of the study was to identify, interpret, and compare the current perspectives of regulatory agencies in six member countries of BRICS-TM (Brazil, Russia, India, China, South Africa, Turkey, and Mexico) on the different criteria used for biosimilar development and marketing authorisation process.Methods: A semi-quantitative questionnaire was developed covering the organisation of agency, biosimilar development criteria and marketing authorisation process and sent to seven regulatory agencies covering the BRICS-TM countries. All data was kept anonymous and confidential. Data processing and analysis was carried out; descriptive statistics were used for quantitative data and content analysis was employed to generate themes for qualitative data.Results: Out of the seven regulatory agencies included in the study, six representatives provided the responses. The perspectives of these six regulatory agencies varied on a number of aspects relating to the review criteria for biosimilar development and licencing process. The most prevalent model for data assessment is the “full review” of a marketing authorisation application. There is lack of a standard approach across the agencies on sourcing of the reference biological product, in vivo toxicity studies and confirmatory clinical studies. Most agencies restrict interaction with biosimilar developers and any scientific advice is non-binding. The marketing authorisation approval depends on scientific assessment of the dossier, sample analysis and GMP certification. The agencies do not issue any public assessment report specifying the summary basis of biosimilar approval.Conclusion: Regulatory agencies across the six emerging economies are steadily improving the regulatory mechanism in the area of biosimilars. However, there remains scope for increasing the effectiveness and efficiency of the processes by encouraging open and transparent interaction with developers, adopting a flexible approach toward accepting advanced analytical data in lieu of clinical studies and enhancing regulatory reliance amongst agencies. This will help to simplify the new biosimilar development programmes and make them more cost-effective.


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