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Navigating facilitated regulatory pathways during a disease X pandemic.

ABSTRACT: In 2018, the Bill and Melinda Gates Foundation convened over thirty subject matter experts in clinical development, manufacturing, and regulatory assessment to determine how the development and approval of medical countermeasures could be accelerated in the event of Disease X. Disease X is the result of a presently unknown pathogen with epidemic or pandemic potential. A key opportunity to accelerate the scientific assessment and regulatory approval of medical countermeasures exists within efficient navigation of facilitated regulatory pathways. It was identified that not all stakeholders will be able to skillfully navigate the facilitated pathways offered by the various regulatory agencies during a public health emergency. To democratize this knowledge, we have written an overview of the facilitated approaches which have been developed and refined by Stringent Regulatory Authorities and the World Health Organization for the primary assessment of medical products. We discuss the conditions necessary for use of these approaches, scenarios in which certain pathways may be applicable, and the pros and cons of these approaches. We also address opportunities available to developers in, or developers who wish to access, low-income countries that may have nascent regulatory frameworks.

SUBMITTER: Simpson S

PROVIDER: S-EPMC7584587 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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Navigating facilitated regulatory pathways during a disease X pandemic.

Simpson Shmona S Chakrabarti Ajoy A Robinson David D Chirgwin Keith K Lumpkin Murray M

NPJ vaccines 20201023

In 2018, the Bill and Melinda Gates Foundation convened over thirty subject matter experts in clinical development, manufacturing, and regulatory assessment to determine how the development and approval of medical countermeasures could be accelerated in the event of Disease X. Disease X is the result of a presently unknown pathogen with epidemic or pandemic potential. A key opportunity to accelerate the scientific assessment and regulatory approval of medical countermeasures exists within effici ...[more]

PMID: 33110630

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Project description:BackgroundIn Australia, facilitated regulatory pathways (FRPs) became available with the introduction of priority review (PR) in 2017 and provisional approval (PA) in 2018, which aim to facilitate expedited review and approval for novel medicines. The pathways were developed in consultation with a wide range of stakeholders and have since been utilised by pharmaceutical companies for various therapeutic products. However, the perceptions of the firsthand users of these pathways have not been evaluated in Australia.ObjectivesWe have conducted a survey of Australian regulatory professionals aiming to solicit the perceived benefits, barriers to utilisation, shortcomings and proposed modifications to utilising these pathways. We have also solicited the users' perspective on key aspects of the pathways, including overall satisfaction, regulatory burden, availability and ease of use of guidelines, regulator support, impact on company strategy and recommendations for improvement.MethodsA survey was developed and distributed to Australian regulatory professionals from the pharmaceutical industry who had submission experience of new medicine applications via either PR, PA or the standard registration pathway to the Therapeutic Goods Administration (TGA). The questionnaire consisted of 44 questions with a skip logic and the option for free text comments.ResultsWe received responses from 16/42 companies that had utilised these new pathways. Nine respondents had experience with the PR pathway and ten with the PA pathway. The respondents were generally satisfied with the effectiveness of the PR process in expediting registration approvals, but they were ambivalent towards the PA pathway in terms of overall satisfaction and timelines. Respondents expressed a desire for further improvements in the speed of approval, earlier access for patients across various pathways and introduction of new Health Technology Assessment processes for medicines approved under PA.ConclusionWhile the FRPs have been an important and positive development in the Australian regulatory landscape, there remain opportunities for further improvements, some of which have been highlighted by this study and may help inform future regulatory decisions.

Project description:Clinical development for orphan drugs presents significant difficulties and challenges. There is no unique or standard design, conduct, and outcome assessment methodology and it is sometimes impractical to fit design models of rare disease trials in any practiced and well-known framework. In the European Union (EU) these challenges encompass a broad array of subjects, including trial design, study outcomes, patient recruitment, trial conduct ethics, trial cost, and chances of success. This literature-based review study aims to provide a thorough overview of the critical aspects of rare disease trials in the EU by analyzing the current landscape of rare disease trials, highlighting key challenges, delving into regulatory and research initiatives and innovation in trial designs, and proposing multi-faceted solutions to implement effective rare disease clinical trials in the region. Traditional clinical trial designs, validation, and evaluation methodologies used for nonorphan drugs often prove unsuitable for orphan drugs, given the small patient populations, sometimes fewer than 1000 cases. There is an increasing need for accessible therapies and both regulators as well as industry are trying to develop affordable and effective drugs to address this need. Despite several steps that have been taken, the timely development of drugs remains a challenge. One of the reasons behind the long development timeline is the recruitment, retention, and conduct of rare disease trials. To optimize the development timelines of orphan drugs in the EU, it is important to ensure that the safety and efficacy of the product is not compromised. Industry and regulatory agencies must implement innovative trial designs, devise flexible policies, and incorporate real-world data for assessing clinical outcomes. Collaboration among academic institutions, pharmaceutical companies (both small and major), patient groups, and health authorities is crucial in overcoming obstacles related to clinical trials and providing assistance and creative ideas. The ultimate objective of granting rare disease patients timely and affordable access to medications with a positive balance between benefits and risks is to be met.

Project description:Aim To consolidate the evidence around the experiences of nursing undergraduates and faculty members navigating through remote and online education during the COVID-19 pandemic. Background The Coronavirus disease 2019 caused by the SARS-CoV-2 Virus (COVID-19) has placed massive pressure on healthcare, economic and education systems globally. Restrictive social distancing policies and public health measures necessitated educational institutions to switch from face-to-face to remote and online education to sustain the learning process. These changes have created an uncertain path and undue stress for healthcare learners and faculty, especially for professional roles that traditionally require more hands-on and access to clinical practice particularly pre-licensure nursing students. As such, there is an urgent need to consolidate evidence on the experiences of nursing undergraduates and faculty members as they navigate the rapid transition from face-to-face to remote and online education to ensure continuity of learning in achieving optimal learning outcomes and to support them during current and future public health crises. Design A systematic review and meta-synthesis of the qualitative literature was undertaken using Sandelowski and Barroso’s approach. Methods Six electronic databases, CINAHL, Embase, ERIC, PsycINFO, PubMed and Scopus, were searched systematically using the eligibility criteria from December 2019 to September 2022. The Critical Appraisal Skills Program checklist for qualitative studies was used to conduct the critical appraisal of the selected articles. Results Forty-seven studies were included in this review, which encapsulates the experiences of 3052 undergraduates and 241 faculty members. An overarching meta-theme ‘Remote and online education: a rollercoaster ride’, emerged along with three main meta-themes: (1) Transition to remote and online education: A turbulent road, (2) Acceptance of the untravelled road, (3) Hopes and recommendations for the road ahead. Conclusion To improve nursing undergraduates’ and faculty member’s navigation of remote and online education, more institutions should move towards establishing hybrid education as the new ‘normal’ and exercise prudence in the organisation and delivery of curriculum, teaching, well-being and clinical attachment contingencies of their healthcare courses.

Project description:BackgroundEver since 2020, travelling has become complex, and increasingly so as the COVID-19 pandemic continues. To reopen Europe safely, a consensus of travel measures has been agreed between countries to enable movement between countries with as few restrictions as possible. However, communication of these travel measures and requirements for entry are not always clear and easily available. The aim of this study was to assess the availability, accessibility and harmonization of current travel information available in Europe.MethodsWe performed a systematic documental analysis of online publicly available information and synthesized travel entry requirements for all countries in the European Union and Schengen Area (N = 31). For each country we assessed entry requirements, actions after entry, how risk was assessed, and how accessible the information was.ResultsWe found varying measures implemented across Europe for entry and a range of exemptions and restrictions, some of which were consistent between countries. Information was not always easy to find taking on average 10 clicks to locate. Twenty-one countries required pre-travel forms to be completed. Forty apps were in use, 11 serving as digital certification checkers. All countries required some form of COVID-19 certification for entry with some exemptions (e.g. children). Nineteen percent (n = 6) of countries used the ECDC risk assessment system; 80% (n = 25) defined their own. Forty-eight percent (n = 15) of countries used a traffic-light system with 2-5 risk classifications.ConclusionA comprehensive set of measures has been developed to enable continued safe travel in Europe. However further refinements and coordination is needed to align travel measures throughout the EU to minimize confusion and maximize adherence to requested measures. We recommend that, along with developing travel measures based on a common set of rules, a standard approach is taken to communicate what these measures are.

Project description:BackgroundDespite the growing application of facilitated regulatory pathways (FRPs), little attention has focused on assessing the perception of pharmaceutical companies regarding their usefulness beyond increasing timeliness.ObjectivesThe aim of this study was to characterize the perceived value of four key FRPs, based on industry experiences in using these pathways. In addition, we sought to characterize the perceived impact based on benefits and barriers as well as suggested solutions for their use and recommendations as identified by companies, to outline how these FRPs may be further evolved as tools for expediting the development and regulatory review of important medicines.MethodsA study was undertaken to characterize the perceived value and impact of US FDA (i.e., Breakthrough Therapy Designation, Fast Track), European Medicines Agency (i.e., PRIME), and Japanese Pharmaceutical and Medical Devices Agency (i.e., Sakigake) FRPs through a comprehensive analysis of strengths, weaknesses, opportunities, and threats (SWOT) as well as suggested solutions based on industry experiences with their use. The finalized survey comprised six questions and was sent to senior management in regulatory affairs departments at 22 multinational pharmaceutical companies in March 2019, with a deadline for completion by April 2019. The responses were analyzed using descriptive statistics. SWOT and free-text responses were reviewed and manually grouped into key themes according to high concordance.ResultsSurvey results were returned by 11 pharmaceutical companies. Based on their perceived value and positive impact, the evaluated FRPs seem to be generally recognized as helpful tools for ensuring timely development and review of important medicines while ensuring multistakeholder involvement. Respondents overwhelmingly felt that the Breakthrough Therapy Designation carried a positive influence, both within and outside their organizations. Following closely with a positive although varied perception was Sakigake, but respondents exhibited more ambivalence about Fast Track and PRIME. Companies felt the impact of the FRPs was generally positive for most stakeholders except for health technology assessors/payers, highlighting the need to better align FRPs with flexible access and reimbursement pathways to expedite the equitable availability of high-quality, safe, effective medicines.ConclusionsThis study highlighted common recommendations across all four FRPs (relating to resource optimization, education, alignment, and communication to improve effective use), as well as agency-specific recommendations, some of which are already being addressed by the regulators.

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Navigating facilitated regulatory pathways during a disease X pandemic.

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Navigating facilitated regulatory pathways during a disease X pandemic.

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