Center for Innovation in Regulatory Science

London, United Kingdom

Center for Innovation in Regulatory Science

London, United Kingdom
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Lipska I.,University Utrecht | McAuslane N.,Center for Innovation in Regulatory Science | Leufkens H.,University Utrecht | Hovels A.,University Utrecht
International Journal of Technology Assessment in Health Care | Year: 2017

Objectives: The objective of this study is to illustrate and provide a better understanding of the role of health technology assessment (HTA) processes in decision making for drug reimbursement in Poland and how this approach could be considered by other countries of limited resources. Methods: We analyzed the evolution of the HTA system and processes in Poland over the past decade and current developments based on publicly available information. Results: The role of HTA in drug-reimbursement process in Poland has increased substantially over the recent decade, starting in 2005 with the formation the Agency for Health Technology Assessment and Tariff System (AOTMiT). The key success factors in this development were effective capacity building based on the use of international expertise, the implementation of transparent criteria into the drug reimbursement processes, and the selective approach to the adoption of innovative medicines based on the cost-effectiveness threshold among other criteria. Conclusions: While Poland is regarded as a leader in Central and Eastern Europe, there is room for improvement, especially with regard to the quality of HTA processes and the consistency of HTA guidelines with reimbursement law. In the “pragmatic” HTA model use by AOTMiT, the pharmaceutical company is responsible for the preparation of a reimbursement dossier of good quality in line with HTA guidelines while the assessment team in AOTMiT is responsible for critical review of that dossier. Adoption of this model may be considered by other countries with limited resources to balance differing priorities and ensure transparent and objective access to medicines for patients who need them. Copyright © Cambridge University Press 2017


Leong J.,National University of Singapore | Walker S.,Center for Innovation in Regulatory Science | Salek S.,University of Hertfordshire | Salek S.,Institute for Medicines Development
Frontiers in Pharmacology | Year: 2015

Purpose: The importance of a framework for a systematic structured assessment of the benefits and risks has been established, but in addition, it is necessary that the benefit-risk decisions and the processes to derive those decisions are documented and communicated to various stakeholders for accountability. Hence there is now a need to find appropriate tools to enhance communication between regulators and other stakeholders, in a manner that would uphold transparency, consistency and standards. Methods: A retrospective, non-comparative study was conducted to determine the applicability and practicality of a summary template in documenting benefit-risk assessment and communicating benefit-risk balance and conclusions for reviewers to other stakeholders. The benefit-risk (BR) Summary Template and its User Manual was evaluated by 12 reviewers within a regulatory agency in Singapore, the Health Sciences Authority (HSA). Results: The BR Summary Template was found to be adequate in documenting benefits, risks, relevant summaries and conclusions, while the User Manual was useful in guiding the reviewer in completing the template. The BR Summary Template was also considered a useful tool for communicating benefit-risk decisions to a variety of stakeholders. Conclusions: The use of a template may be of value for the communicating benefit-risk assessment of medicines to stakeholders. © 2015 Leong, Walker and Salek.


Donelan R.,Quintiles | Walker S.,Center for Innovation in Regulatory Science | Salek S.,University of Hertfordshire
Pharmacoepidemiology and Drug Safety | Year: 2015

Currently, there is no qualified understanding of the influences, behaviours and other factors that impact the decision-making of individuals and organisations involved in the development of new medicines. The aim of this qualitative study was to investigate and identify the important issues that influence quality decision-making. Methods: Semi-structured interviews were carried out with 29 senior decision-makers from the pharmaceutical industry and regulatory authorities. The study participants were invited to discuss and review their perception of decision-making within their organisation, its role in drug development and the regulatory review and their awareness and use of decision-making techniques and the impact and monitoring of decisions. Results: The analyses (using NVivo 8 software) resulted in the identification of 32 major and 97 sub-themes that were consolidated into 19 overarching themes. These included items such as quality and validity of data, time considerations, organisational and cultural influences, analytical and logical approach, qualification and experience, subjective and personal considerations, political influences, precedents for similar previous decisions, understanding of the decision in question, impact analyses, audit trail, education and awareness, individual versus corporate decision-making and frameworks. Relationships between themes were identified. The 19 overarching decision-making themes were integrated into a framework for quality decision-making. Conclusion: This study has achieved its aim of exploring decision-making from the perspective of the individual and the organisation working in drug development and the regulatory review and has identified issues and considerations relating to making good quality decisions and allowed for the generation of a framework to aid quality decision-making. © 2015 John Wiley & Sons, Ltd.


Donelan R.,Quintiles | Walker S.,Center for Innovation in Regulatory Science | Salek S.,University of Hertfordshire | Salek S.,Institute for Medicines Development
Frontiers in Pharmacology | Year: 2016

Introduction: The impact of decision-making during the development and the regulatory review of medicines greatly influences the delivery of new medicinal products. Currently, there is no generic instrument that can be used to assess the quality of decision-making. This study describes the development of the Quality of Decision-Making Orientation Scheme QoDoS© instrument for appraising the quality of decision-making. Methods: Semi-structured interviews about decision-making were carried out with 29 senior decision makers from the pharmaceutical industry (10), regulatory authorities (9) and contract research organizations (10). The interviews offered a qualified understanding of the subjective decision-making approach, influences, behaviors and other factors that impact such processes for individuals and organizations involved in the delivery of new medicines. Thematic analysis of the transcribed interviews was carried out using NVivo8® software. Content validity was carried out using qualitative and quantitative data by an expert panel, which led to the developmental version of the QoDoS. Further psychometric evaluations were performed, including factor analysis, item reduction, reliability testing and construct validation. Results: The thematic analysis of the interviews yielded a 94-item initial version of the QoDoS© with a 5-point Likert scale. The instrument was tested for content validity using a panel of experts for language clarity, completeness, relevance and scaling, resulting in a favorable agreement by panel members with an intra-class correlation coefficient value of 0.89 (95% confidence interval = 0.56, 0.99). A 76-item QoDoS© (version 2) emerged from content validation. Factor analysis produced a 47-item measure with four domains. The 47-item QoDoS© (version 3) showed high internal consistency (n = 120, Cronbach's alpha = 0.89), high reproducibility (n = 20, intra-class correlation = 0.77) and a mean completion time of 10 min. Reliability testing and construct validation was successfully performed. Conclusion: The QoDoS© is both reliable and valid for use. It has the potential for extensive use in medicines development by both the pharmaceutical industry and regulatory authorities. The QoDoS© can be used to assess the quality of decision-making and to inform decision makers of the factors that influence decision-making. © 2016 Donelan, Walker and Salek.


Fronsdal K.,Norwegian Knowledge Center for Health Services | Pichler F.,Center for Innovation in Regulatory Science | Mardhani-Bayne L.,Institute of Health Economics | Henshall C.,University of York | And 3 more authors.
International Journal of Technology Assessment in Health Care | Year: 2012

There has been an increased focus on the relationship between health technology assessment (HTA) and regulatory assessments and how regulatory, HTA and coverage bodies, and industry can work better together to improve efficiency and alignment of processes. There is increasingly agreement across sectors that improved communication and coordination could contribute to facilitating timely patient access to effective, affordable treatments that offer value to the health system. Discussions on aspects of this relationship are being held in different forums and various forms of coordination and collaboration are being developed or piloted within several jurisdictions. It is therefore both timely and of value to stakeholders to describe and reflect on current initiatives intended to improve interactions between regulatory, HTA and coverage bodies, and industry. Drawing on 2011 meetings of the HTAi Policy Forum and the Center for Innovation in Regulatory Science (CIRS), this study aims to describe and compare initiatives, and point to success factors and challenges that are likely to inform future work and collaboration. © Copyright 2012 Cambridge University Press.


Levitan B.,Janssen Research & Development LLC | Phillips L.D.,Management Science Group | Walker S.,Center for Innovation in Regulatory Science
Therapeutic Innovation and Regulatory Science | Year: 2014

Assessing the utility of structured approaches to benefit-risk assessment of medicinal products is challenging, in part due to the lack of a gold standard for results and the uncertainty inherent in the data. In place of conducting formal testing, obtaining feedback from users of structured approaches provides insight into their value and limitations. The authors conducted a simulated single-session benefit-risk decision in which 3 groups applied the PhRMA BRAT(Pharmaceutical Research and Manufacturers of America Benefit-Risk Action Team) framework or the multicriteria decision analysis approach. The groups were provided with background and data for a hypothetical triptan for acute migraine in a population with cardiovascular risk factors and were asked to determine and defend an approval decision. Three insights emerged consistently from the groups: (1) the value of a structured approach to benefit-risk assessment, (2) the clarity provided by real-time visualization tools, and, most critically, (3) the importance of bringing the patient into the discussion early. © The Author(s) 2014.


Leong J.,University of Cardiff | Mcauslane N.,Center for Innovation in Regulatory Science | Walker S.,University of Cardiff | Walker S.,Center for Innovation in Regulatory Science | Salek S.,University of Cardiff
Pharmacoepidemiology and Drug Safety | Year: 2013

Purpose: To explore the current status and need for a universal benefit-risk framework for medicines in regulatory agencies and pharmaceutical companies. Methods: A questionnaire was developed and sent to 14 mature regulatory agencies and 24 major companies. The data were analysed using descriptive statistics, for a minority of questions preceded by manual grouping of the responses. Results: Overall response rate was 82%, and study participants included key decision makers from agencies and companies. None used a fully quantitative system, most companies preferring a qualitative method. The major reasons for this group not using semi-quantitative or quantitative systems were lack of a universal and scientifically validated framework. The main advantages of a benefit-risk framework were that it provided a systematic standardised approach to decision-making and that it acted as a tool to enhance quality of communication. It was also reported that a framework should be of value to both agencies and companies throughout the life cycle of a product. They believed that it is possible to develop an overarching benefit-risk framework that should involve relevant stakeholders in the development, validation and application of a universal framework. The entire cohort indicated common barriers to implementing a framework were resource limitations, a lack of knowledge and a scientifically validated and acceptable framework. Conclusions: Stakeholders prefer a semi-quantitative, overarching framework that incorporates a toolbox of different methodologies. A coordinating committee of relevant stakeholders should be formed to guide its development and implementation. Through engaging the stakeholders, these outcomes confirm sentiments and need for developing a universal benefit-risk assessment framework.© 2013 John Wiley & Sons, Ltd..


Alsager S.,North Ring Road Al Nafal Unit | Hashan H.,North Ring Road Al Nafal Unit | Walker S.,Center for Innovation in Regulatory Science | Walker S.,University of Cardiff
Pharmaceutical Medicine | Year: 2015

Objective: This study sought to assess the current regulatory review process in Saudi Arabia, identify the key milestones, evaluate the measures used for Good Review Practices (GRevP) and to suggest opportunities for an enhanced regulatory review of medicines.Methods: A questionnaire completed by the Saudi Food and Drug Authority (SFDA) was divided into three parts: Organisation of the Agency, Key Milestones and Timelines and GRevP.Results: Currently the SFDA carries out a full assessment for the review of all major applications, although they currently lack the expertise to evaluate the preclinical portion of the product file. A Certificate of Pharmaceutical Product (CPP) is required at the time of registration and a pricing agreement internally must be developed before authorisation. Applications may have to wait 2–6 months before review, although priority products are taken out of the queuing system. The median review times for new active substances from submission to approval were 340 working days (2011) and 372 working days (2013); however, the target time was 290 working days. Standard operating procedures (SOPs), review templates and an electronic submission tracking system are in place, but the GRevP framework is still evolving.Conclusion: Based on the available resources and capabilities, the SFDA is unable currently to meet its overall target timelines, partly due to the sponsor’s time in responding to agency questions. Therefore, it either needs to increase its resources or to implement a risk stratification system based on the Singapore model, which takes into account reviews by reference agencies. The SFDA is encouraged to develop GRevP guidelines to ensure the quality of the review. © 2015, Springer International Publishing Switzerland.


Al-Essa R.,University of Cardiff | Salek S.,University of Cardiff | Walker S.,Center for Innovation in Regulatory Science
Drug Information Journal | Year: 2012

The aim of this study was to evaluate how each Gulf Cooperation Council (GCC) regulatory authority is building quality into the assessment and registration process and to establish opportunities for the exchange of best practice between the GCC states. A questionnaire was completed by the 7 Gulf States (Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, United Arab Emirates [UAE], and Yemen), which provided details of the quality attributes that characterize the extent of the scientific assessment in the region. The results showed that each authority has its unique practices that characterize their 7 milestones in comparison with the other GCC authorities. Bahrain uses good review practice (GRP) guidelines and has placement arrangements in competent authorities, while Kuwait has separate pricing and registration departments. Oman engages external audit programs from accredited bodies, while Saudi Arabia and UAE conduct formal training for their assessors. © Drug Information Association 2012.


Woolley K.L.,University of Queensland | Woolley K.L.,University of The Sunshine Coast | Gertel A.,Beardsworth Consulting Group Inc | Gertel A.,Center for Innovation in Regulatory Science | And 3 more authors.
Annals of Pharmacotherapy | Year: 2013

In this commentary, we present evidence that unethical authorship (eg, guest and ghost authoring) and other publication practices are not restricted to the pharmaceutical industry; they also occur in academia. Such practices are not an industry problem-they are a research problem. To enhance trust in industrysponsored research, companies have made rapid and far-reaching changes to their publication guidelines, policies, and procedures. Professional medical writers have adopted, and continue to implement, these changes. Although evidence indicates that industry practices are improving, there is certainly more to do, both in industry and academia. We invite readers to join ongoing efforts to promote ethical publication practices. © 1967-2013 Harvey Whitney Books Co. All rights reserved.

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