60-річчя В. С. Пономаренка

60 років члену редколегії журналу «Економічний вісник Донбасу», ректору Харківського національного економічного університету, доктору економічних наук, професору, заслуженому діячу науки і техніки України, кавалеру ордена «За заслуги» III ступеня, академіку Академії економічних наук України Пономаренку Володимиру Степановичу

The outcome of psur assessments of biopharmaceuticals

Background: Recent changes introduced to European legislation amend the requirements for the submission of periodic safety update reports (PSURs). However, information on the outcome of PSUR assessment is lacking. Aim: To describe the outcomes of PSUR assessments.(table present) Methods: A cross sectional analysis was performed of all PSURs and PSUR assessment reports (AR) issued between July 1st 2008 and June 30th 2010 for all biopharmaceuticals centrally approved in the European Union. PSURs and PSUR ARs were obtained from the repository of the Dutch Medicines Evaluation Board, CBG-MEB. Results: PSURs and PSUR ARs were collected for 70 products. Most products in the sample belonged to the ATC group of antineoplastic and immunomodulating agents (n = 26, 37.1%). Of the 70 PSURs included in the sample 26 (37%) covered a period of 6 months, 24 (34%) a period of 1 year and 20 (29%) a period of more than 1 year. The most common outcome of PSUR assessment was monitoring a possible safety issue, which was requested in 55 (79%) of all ARs. Of these, 23 (42%) included new safety concerns not identified before. New safety concerns were identified in 35% of the PSURs that were issued within 5 years of the international birth date (IBD) of the product, and in 40% of the PSURs submitted after 5 years of the IBD (p = 0.795). Cumulative reviews of data relating to a possible safety issue were requested in 31 (44%) of the PSUR assessments and 27 (39%) of the assessments resulted in proposals and/or requests to change the Summary of Product Characteristics (SPC). The proportion of assessments resulting in SPC changes in various subgroups is presented in table I. Conclusions: PSUR assessments are an important tool in the dialogue between regulators and marketing authorization holders. PSUR assessments are involved in the safety management of both new and well established products. New safety concerns occur throughout the lifecycle of biopharmaceuticals and may occur more often for products in different therapeutic and mechanistic classes

The contribution of periodic safety reports (PSURs) to safety related regulatory actions of biopharmaceuticals

Introduction: A periodic safety update report (PSUR), composed by marketing authorization holders and submitted to regulatory authorities on predetermined time points, provides an update of the worldwide safety experience of a pharmaceutical. Information is lacking on how PSURs contribute to safety related regulatory actions. Aim: The objective of the study is to analyze the contribution of PSUR evaluations to the initiation of safety related regulatory actions of biopharmaceuticals. Methods: We performed a retrospective analysis of all safety related type II variations of biological products centrally approved in the European Union (since 1995) for which ≥1 safety-related Direct Health Healthcare Professional Communication (DHPC) was issued until December 2009. An evaluation of the role of PSUR assessments in the initiation of safety associated regulatory actions was performed through an analysis of European Public Assessment Reports and updates of the Summary of Product Characteristics (SPC). We compared “urgent” variations, defined as variations accompanied by the distribution of a DHPC, with “less urgent” variations, i.e. safety related SPC variations for which no DHPC was distributed. For each variation we determined if any reference was made to the contribution of PSUR evaluations. We determined the data source and nature of the safety issues included in the variations. Each variation could include ≥1 safety issue and ≥1 data source could contribute in a single variation. Results: We identified 133 safety related type II variations for 15 biological products. Reference to PSUR evaluations was made in 2/24 (8.4%) of all urgent type II variations and 48/109 (44.0%) of the less urgent variations (x2, p<0.01). Data sources that contributed to the urgent variations were: 14 (58%) spontaneous reports, 9 (28%) clinical trials and 2 (8%) an analysis of pooled data. For the non-urgent variations, these were 53 (49%), 40 (37%) and 18 (17%) respectively. Overall, most of the variations concerned events from the System Organ Classes (SOCs) Infections and Infestations (32%), General Disorders and Administration Site Conditions (26%), Neoplasms (14%), Blood and Lymphatic System Disorders (14%) and Nervous System Disorders (14%). No differences in SOCs were observed between safety-related regulatory actions that did or did not result from PSUR assessments. Conclusions: The contribution of PSUR evaluations was lower in urgent safety related regulatory actions when compared with less urgent safety issues. Despite the modest role of PSURs, spontaneous reports contributed to the majority of the urgent safety related regulatory actions

The outcome of psur assessments of biopharmaceuticals

Background: Recent changes introduced to European legislation amend the requirements for the submission of periodic safety update reports (PSURs). However, information on the outcome of PSUR assessment is lacking. Aim: To describe the outcomes of PSUR assessments.(table present) Methods: A cross sectional analysis was performed of all PSURs and PSUR assessment reports (AR) issued between July 1st 2008 and June 30th 2010 for all biopharmaceuticals centrally approved in the European Union. PSURs and PSUR ARs were obtained from the repository of the Dutch Medicines Evaluation Board, CBG-MEB. Results: PSURs and PSUR ARs were collected for 70 products. Most products in the sample belonged to the ATC group of antineoplastic and immunomodulating agents (n = 26, 37.1%). Of the 70 PSURs included in the sample 26 (37%) covered a period of 6 months, 24 (34%) a period of 1 year and 20 (29%) a period of more than 1 year. The most common outcome of PSUR assessment was monitoring a possible safety issue, which was requested in 55 (79%) of all ARs. Of these, 23 (42%) included new safety concerns not identified before. New safety concerns were identified in 35% of the PSURs that were issued within 5 years of the international birth date (IBD) of the product, and in 40% of the PSURs submitted after 5 years of the IBD (p = 0.795). Cumulative reviews of data relating to a possible safety issue were requested in 31 (44%) of the PSUR assessments and 27 (39%) of the assessments resulted in proposals and/or requests to change the Summary of Product Characteristics (SPC). The proportion of assessments resulting in SPC changes in various subgroups is presented in table I. Conclusions: PSUR assessments are an important tool in the dialogue between regulators and marketing authorization holders. PSUR assessments are involved in the safety management of both new and well established products. New safety concerns occur throughout the lifecycle of biopharmaceuticals and may occur more often for products in different therapeutic and mechanistic classes

The contribution of periodic safety reports (PSURs) to safety related regulatory actions of biopharmaceuticals

Introduction: A periodic safety update report (PSUR), composed by marketing authorization holders and submitted to regulatory authorities on predetermined time points, provides an update of the worldwide safety experience of a pharmaceutical. Information is lacking on how PSURs contribute to safety related regulatory actions. Aim: The objective of the study is to analyze the contribution of PSUR evaluations to the initiation of safety related regulatory actions of biopharmaceuticals. Methods: We performed a retrospective analysis of all safety related type II variations of biological products centrally approved in the European Union (since 1995) for which ≥1 safety-related Direct Health Healthcare Professional Communication (DHPC) was issued until December 2009. An evaluation of the role of PSUR assessments in the initiation of safety associated regulatory actions was performed through an analysis of European Public Assessment Reports and updates of the Summary of Product Characteristics (SPC). We compared “urgent” variations, defined as variations accompanied by the distribution of a DHPC, with “less urgent” variations, i.e. safety related SPC variations for which no DHPC was distributed. For each variation we determined if any reference was made to the contribution of PSUR evaluations. We determined the data source and nature of the safety issues included in the variations. Each variation could include ≥1 safety issue and ≥1 data source could contribute in a single variation. Results: We identified 133 safety related type II variations for 15 biological products. Reference to PSUR evaluations was made in 2/24 (8.4%) of all urgent type II variations and 48/109 (44.0%) of the less urgent variations (x2, p<0.01). Data sources that contributed to the urgent variations were: 14 (58%) spontaneous reports, 9 (28%) clinical trials and 2 (8%) an analysis of pooled data. For the non-urgent variations, these were 53 (49%), 40 (37%) and 18 (17%) respectively. Overall, most of the variations concerned events from the System Organ Classes (SOCs) Infections and Infestations (32%), General Disorders and Administration Site Conditions (26%), Neoplasms (14%), Blood and Lymphatic System Disorders (14%) and Nervous System Disorders (14%). No differences in SOCs were observed between safety-related regulatory actions that did or did not result from PSUR assessments. Conclusions: The contribution of PSUR evaluations was lower in urgent safety related regulatory actions when compared with less urgent safety issues. Despite the modest role of PSURs, spontaneous reports contributed to the majority of the urgent safety related regulatory actions

Biosimilars for the Management of Inflammatory Bowel Diseases: Economic Considerations

Biological drugs revolutionized the treatment of inflammatory bowel diseases (IBD) such as Crohn’s disease and ulcerative colitis. However, not all clinically eligible patients have access to biologicals due to significant costs and budget impact. Biosimilars are highly comparable to their originator product in terms of clinical efficacy and safety. Biosimilars are priced 15-75% lower than their reference product, which makes them a less costly alternative and is expected to offer better patients access to biologicals. The total projected cost savings are significant. If the achieved budget savings were used to cover more biological therapy, several additional IBD patients could be treated. Currently, the main barriers to the increasing uptake of biosimilars are the few incentives of the key stakeholders, while physicians’ and patients’ skepticism towards biosimilars seems to be changing. Over the coming years, biosimilars are expected to gain a growing importance in the treatment of IBD, contributing to a better access to treatment, improving population-level health gain and sustainability of health systems. This review summarizes the results of the literature on the economic considerations of biosimilars in IBD and the role of biosimilar infliximab in the treatment of IBD