The Work of the Council for International Organizations of Medical Sciences (CIOMS) in Global Pharmacovigilance

Younus, Manal M.; Zweygarth, Monika; Rägo, Lembit; Harrison‐Woolrych, Mira

doi:10.1007/s40264-020-01003-5

Cited by 5 publications

(4 citation statements)

References 2 publications

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“…The accurate assessment of adverse event (AE) rates in gene therapies is greatly hindered by the rarity of typical indications. The European Commission's guideline on summary product characteristics (SmPC) classifies adverse event risk according to several frequency categories: very rare, rare, uncommon, common, and very common, with associated event rates of < 0.01%, < 0.1%, < 1%, < 10%, and ≥ 10%, respectively 20,21 . An accurate estimation of a rate that is anything but “very common” and “common” by this bucketing is infeasible for LTFU trials that may contain fewer than 100 participants for a given gene therapy product.…”

Section: Methodsmentioning

confidence: 99%

“…Substudy ad hoc analysis/reporƟng ARTICLE categories: very rare, rare, uncommon, common, and very common, with associated event rates of < 0.01%, < 0.1%, < 1%, < 10%, and ≥ 10%, respectively. 20,21 An accurate estimation of a rate that is anything but "very common" and "common" by this bucketing is infeasible for LTFU trials that may contain fewer than 100 participants for a given gene therapy product. The key to addressing this limitation lies in well-understood and powerful statistical tools that "borrow strength" from similar categories within a cluster, which is a natural fit for such sparse data scenarios, and is further discussed in the following section.…”

Section: Master Protocol Pooled Periodic Reporɵngmentioning

confidence: 99%

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Practical and Statistical Considerations for the Long Term Follow‐Up of Gene Therapy Trial Participants

Rohde,

Huh,

D'Souza

et al. 2023

Clin Pharma and Therapeutics

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Study sponsors and market authorization holders are required by the FDA and EMA regulatory agencies to enroll patients administered a gene therapy product, whether in a trial setting or post‐licensure, in a long term follow‐up safety study to continue the safety assessments of their product. These follow‐up studies range between 5 to 15 years after dosing. This unprecedented duration of engagement with patients and caregivers raises logistical challenges that will require innovation and collaboration across sponsors and regulators. In this paper we delineate some of the key considerations for designing long term follow‐up protocols in the gene therapy setting, with an eye towards platform and master protocol approaches, and offer guidance for innovative operational and statistical methods that can help assess the safety profile and durability of response for these novel therapeutics.

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Section: Methodsmentioning

confidence: 99%

Section: Master Protocol Pooled Periodic Reporɵngmentioning

confidence: 99%

Practical and Statistical Considerations for the Long Term Follow‐Up of Gene Therapy Trial Participants

Rohde,

Huh,

D'Souza

et al. 2023

Clin Pharma and Therapeutics

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“…9 The CIOMS-I form is the universally accepted document for reporting single PV cases by the MAH to the NMRA. 10 In recent years, other means of electronic transmission have overtaken the CIOMS form, with the implementation of the E2B transmission mode by NMRAs in advanced PV systems in 2013. 11 Electronic transmission, now in its third revision, is known as E2B(R3).…”

Section: Introductionmentioning

confidence: 99%

Development of a checklist for the assessment of pharmacovigilance guidelines in Southern Africa: a document review

Makhene

Steyn

Vorster

et al. 2023

Therapeutic Advances in Drug Safety

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Introduction: National regulatory systems in Southern Africa reflect various stages of maturity, and pharmacovigilance (PV) practices are not aligned. In the absence of guidance for formulating PV guidelines in Southern African Development Community (SADC) countries, this study aimed to create a checklist that may be used to assess the rigour of PV guidelines in this region and provide guidance for the National Medicines Regulatory Agency (NMRA) authors. Methods: A document analysis was performed based on harmonised international guidelines ( n = 22) that prescribed methods of PV regulation to identify themes and items to incorporate into a checklist. The contextualisation of the checklist to the African pharmaceutical environment was accomplished by referencing peer-reviewed journal articles ( n = 7). The checklist was subjected to face and content validation by non-experts and PV experts. Results: The document review yielded 5 themes, 18 sub-themes, and 73 items structured into the checklist. Themes encompassed PV systems, definitions, individual case safety reporting, aggregate reporting, and risk management. Under PV systems, aspects of the quality management system were outlined, that is, the legal basis for PV, a description of the marketing authorisation holder’s (MAH’s) PV system, archiving of data, contracting of PV tasks, and the duties of the person responsible for the MAH’s PV obligations. Definitions of the key terms and major stakeholders were identified. Reporting of individual case safety reports (ICSRs) was explicated by considering the criteria for reporting, categories of reportable information, expedited reporting requirements, reporting timelines, and ICSR reporting format. Aggregate report submission during the development and post-marketing phases was addressed. Risk management encompassed signal detection, re-evaluation of the benefit-risk ratio, the safety decision-making process, risk management planning, risk minimisation and safety communication. Conclusion: The developed checklist can contribute towards assisting SADC NMRAs to formulate national PV guidelines that reflect current international practice, with local context incorporated. Plain Language Summary Developing a checklist for the evaluation of medicine safety guidelines in Southern Africa Introduction: In Southern African Development Community (SADC) countries, the guidelines for medicine safety [pharmacovigilance (PV)] that marketing authorisation holders (MAHs) and healthcare professionals need to adhere to, are not aligned. We saw the need to develop a checklist that can be used to evaluate these guidelines. Methods: We studied international documents issued by the World Health Organization (WHO), the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), the Council for International Organizations of Medical Sciences (CIOMS) and the European Medicines Agency (EMA). On the organisational websites, we obtained 22 documents and identified 73 checklist items. All the items were arranged under 5 themes and 18 sub-themes to create the checklist. We adapted the checklist to the local context by using seven journal articles addressing PV concerns in Africa. Experts checked the content and usability of the checklist. Results: The themes were PV systems, definitions, individual case safety reporting (ICSR), combined reporting and risk management. PV systems had six sub-themes: legal structure, description of the MAH’s PV system, contractual agreements, information storage, the qualified person responsible for PV (QPPV) and where the QPPV is located. We included the definitions of keywords and role-players. The ICSR theme had five sub-themes, i.e. criteria for reporting, categories of reportable information, expedited reporting, reporting timelines, and reporting format. Submission of summary reports comprised an overview of the safety profile of a medicine once it is approved by regulators, as well as during clinical trials. Risk management included signal detection, re-evaluation of the benefit-risk ratio, safety decision-making process, risk management planning, risk minimisation, and safety communication. The checklist is applied by allocating yes/no scoring per item. Conclusion: The checklist may be used by regulators within SADC to assess their PV guidelines for alignment with international standards and suitability to the local environment.

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“…Besides internationally recognized members like the World Medical Association, International Society of Pharmacovigilance, the International Society of Pharmacoepidemiology, and the International Union of Basic and Clinical Pharmacology, the association also has national members the Academies of Medical Sciences of Belgium, Korea, and Switzerland as well as the Medical Research Councils of Bangladesh, India, and South Africa. Working groups have been established by the council to develop guidelines on a topic where knowledge and practice gaps must be addressed worldwide [12] The Council for International Organizations of Medical Sciences (CIOMS) began to focus on pharmacovigilance and adverse drug reactions in 1993. CIOMS started working on pharmacovigilance in the late 80s through its program "the Monitoring and Terminology of Adverse Drug Reactions".…”

mentioning

confidence: 99%

Pharmacovigilance practices in South Asian Association for Regional Cooperation countries: the need for collaboration

Khan

Hamid

Babar

2022

Journal of Pharmaceutical Health Services Research

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Objectives One-fifth of the world’s population lives in eight countries that constitute the South Asian Association for Regional Cooperation (SAARC). There is very little coordination among SAARC countries regarding the harmonization of pharmaceutical regulations and medicines safety. Pakistan, India and Bangladesh have experienced medicine-related tragedies where many patients have died. This study aims to examine current pharmacovigilance activity in the SAARC region to improve pharmacovigilance practices and to make recommendations for building a platform for collaboration to improve the safety monitoring of medicines in the region. The current review utilized secondary data. We reviewed the official websites of all SAARC countries’ national regulatory authorities for pharmacovigilance-related information. A data set with eleven pharmacovigilance indicators were gathered and synthesized. Key findings All eight SAARC member countries have pharmacovigilance systems with full membership in the WHO Program for International Drug Monitoring. Out of eleven pharmacovigilance indicators, India met ten; Pakistan, Bangladesh and Bhutan nine; Maldives and Afghanistan seven; Nepal and Sri Lanka five. The SAARC countries do not have a harmonized pharmacovigilance system or centralized database. Due to positioning in different WHO regions, it is proposed to create a consortium on medicine safety among SAARC countries like other regional organizations of the world to strengthen the pharmacovigilance systems and harmonize the pharmacovigilance practices among member countries. Summary To improve the quality of medicines and to strengthen regional medicine safety, the SAARC secretariat should consider forming a technical group of all member countries’ regulatory authorities.

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The Work of the Council for International Organizations of Medical Sciences (CIOMS) in Global Pharmacovigilance

Cited by 5 publications

References 2 publications

Practical and Statistical Considerations for the Long Term Follow‐Up of Gene Therapy Trial Participants

Practical and Statistical Considerations for the Long Term Follow‐Up of Gene Therapy Trial Participants

Development of a checklist for the assessment of pharmacovigilance guidelines in Southern Africa: a document review

Pharmacovigilance practices in South Asian Association for Regional Cooperation countries: the need for collaboration

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