Advancing Public Health Using Regulatory Science to Enhance Development and Regulation of Medical Products: Food and Drug Administration Research at the Center for Biologics Evaluation and Research

Kusinitz, Marc; Braunstein, Emily; Wilson, Carolyn A.

doi:10.3389/fmed.2017.00071

Cited by 5 publications

(6 citation statements)

References 39 publications

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“…Countries need fully functional regulatory systems in order to respond to public health needs as well as to enhance access to safe and effective medicines ( Kusinitz M et al, 2017 ). One of the determinants of access to essential medicines is regulatory filing and registration ( Sillo et al, 2020 ).…”

Section: Introductionmentioning

confidence: 99%

Evaluation of the effectiveness and efficiency of the East African community joint assessment procedure by pharmaceutical companies: Opportunities for improvement

Ngum

Mashingia²,

Ndomondo-Sigonda³

et al. 2022

Front. Pharmacol.

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Background: A 2021 study to determine the viewpoints among the seven member countries regarding the effectiveness (i.e., achieving the intended outcomes) and efficiency (i.e., achieving the intended outcomes in timely manner with the resources available) of the East African Community Medicine Regulatory Harmonisation (EAC-MRH) Joint Assessment Procedure recommended the conduct of a similar study among pharmaceutical company applicants. The aim of this study then was to evaluate the effectiveness and efficiency of the current EAC-MRH operating model from the applicants’ perspective, including the challenges and opportunities for improvement.Methods: Using the Process Effectiveness and Efficiency Rating for Industry questionnaire developed by the authors, data were collected from company representatives responsible for EAC joint procedure submissions.Results: Responses from 14 study participants underlined the support of pharmaceutical companies for the EAC-MRH initiative, which has facilitated the harmonisation of registration requirements across the EAC region leading to one registration for all countries and a reduction of the workload for both applicants and assessors. In addition, it is expected that shorter timelines for approval will lead to improved access to quality-assured essential medicines in the region. Access to various markets at the same time was also noted as an important benefit to pharmaceutical companies. Noted challenges include a lack of process information, a lack of centralised submission and tracking process and a lack of mandated central registration. A key strategy proposed by participants is the establishment of a regional administrative body to centrally receive and track EAC applications and the eventual establishment of a Regional EAC Medicines Authority.Conclusion: This is the first study evaluating the performance of the EAC work-sharing initiative from the point of view of the applicants. In general, the applicants believe that the system performs efficiently and fulfils its promise. However, some participants indicated that in some countries an EAC positive recommendation does not directly result in an individual country approvals. Following the recommendations listed in this report may mitigate identified areas for improvement and facilitate the overall goal of the EAC-MRH initiative to expedite the availability of needed quality-assured medicines to patients in the region.

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Section: Introductionmentioning

confidence: 99%

Evaluation of the effectiveness and efficiency of the East African community joint assessment procedure by pharmaceutical companies: Opportunities for improvement

Ngum

Mashingia²,

Ndomondo-Sigonda³

et al. 2022

Front. Pharmacol.

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“…This poses increased regulatory challenge of development of a manufacturing process and “functionally closed” disposable that can ensure aseptic processing and the absence of unwanted infectious agents. Blood products, like cells, have biologically active components that cannot be subjected to terminal sterilization . Manufacture of dried plasma using glass containers and rubber stoppers was successfully implemented in WWII.…”

Section: Regulatory Issuesmentioning

confidence: 99%

“…Pooling provides uniformity of product and makes generating specifications and testing for product release more straightforward. Manufacturers of dried plasma made from single‐donor units have to take unique approaches to testing for safety and potency, as most testing is destructive and it would be cost prohibitive to test every unit . Additionally, current plasma products such as FFP and PF24 are not tested for sterility or coagulation factor levels.…”

Section: Product Issuesmentioning

confidence: 99%

“…There are multiple cell types to be investigated for their therapeutic potential. Products’ mechanism of action will need to be sufficiently understood to identify appropriate biomarkers of clinical effect and/or to develop appropriate potency assays . Development of a commercially viable cell therapy product will require defining a cell type for a discrete therapeutic indication and target for action in a traumatic injury that can demonstrate the benefit of cell therapy in clinical settings.…”

Section: Lessons Learned and How They May Apply To Cell And Other Novmentioning

confidence: 99%

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Challenges to producing novel therapies – dried plasma for use in trauma and critical care

Buckley

Gonzales²

2019

Transfusion

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C hallenges to modern dried plasma development exist at multiple points along the product development cycle and include the regulatory pathway, funding, logistics, implementation, and commercial viability. The regulatory path for these products requires a clinical development pathway similar to a drug that entails longer timelines and substantial funding. US efforts to develop a dried plasma product have been military driven in response to a demand arising from the Operation Iraqi Freedom and Operation Enduring Freedom conflicts. A memorandum of understanding has existed between the Department of Defense (DoD) and the Food and Drug Administration (FDA) since 1974 for the purpose of expediting review of special DoD requirements to meet national defense considerations and set the requirement for clinical testing through submission of an investigational new drug (IND) or investigational device exemption (IDE) application. 1 Modern development of a US-based dried plasma product has been under way since the early 2000s, challenging expectations for an expeditious process. A new initiative between the DoD and FDA was announced in January 2018, with the goal of streamlining the product development path. This initiative, coupled with continued clinical investigation, will result in US dried plasma products being available on the market soon, and may demonstrate that these efforts have the potential to hasten future product development pathways. BRIEF HISTORY OF DRIED PLASMADemand for freeze-dried plasma (FDP) production arose from military need for a resuscitation fluid to treat hemorrhagic shock due to blood loss resulting from battlefield injuries. 2 Widespread use of dried plasma began in the 1940s by US and British military forces during World War II for the primary indication of management of shock. 2 Due to the large quantities of plasma needed, freeze-dried (lyophilized) plasma was produced by several biologic manufacturers by pooling thousands of units that were dispensed into glass bottles, freeze dried, stoppered upon completion of drying, and then packaged in tin cans to resist breakage. Location of the manufacturers was selected based on proximity to plasma collection centers to streamline production. 2 The French similarly began manufacture of FDP to support military use during the Indochine War. 3 US production efforts were eventually discontinued due to contamination of the large plasma pools with hepatitis B virus (HBV), with reported rates of transmission of hepatitis B virus during the Korean War of up to 21%. 4 Pooled lyophilized plasma was formally withdrawn from use by the FDA in 1968 because of known risk of pathogen transmission. 5 Concerns regarding transmission of human immunodeficiency virus suspended further development efforts in the 1980s.Production was reinitiated by the French in the early 1990s during the Gulf War to support military operations. 3 The German Red Cross Blood Service West also began manufacturing in the 1990s a lyophilized pooled product that used the solvent/detergent metho...

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“…Summarizing this topic, regenerative medicine aims to treat human diseases provoked by deficits in particular cells, restoring injured tissue, and emerging as innovative therapeutic strategies to assist a wide range of health disorders. To date, Regenerative Medicine Advanced Therapy is the newest program considered by the FDA, responsible to design drug development and review processes for promising pipeline products . In this context, preliminary in vitro approaches and preclinical strategies are needed .…”

Section: Introductionmentioning

confidence: 99%

Layered Double Hydroxides Are Promising Nanomaterials for Tissue Bioengineering Application

et al. 2019

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Layered double hydroxides (LDHs) have emerged as promising nanomaterials for human health and although it has achieved some progress on this matter, their application within bioengineering is not fully addressed. This prompted to subject fibroblasts to two compositions of LDHs (Mg2Al‐Cl and Zn2Al‐Cl), considering an acute response. First, LDH particles are addressed by scanning electron microscopy, and no significant effect of the cell culture medium on the shape of LDHs particles is reported although it seems to adsorb some soluble proteins as proposed by energy‐dispersive X‐ray analysis. These LDHs release magnesium, zinc, and aluminum, but there is no cytotoxic or biocompatibility effects. The data show interference to fibroblast adhesion by driving the reorganization of actin‐based cytoskeleton, preliminarily to cell cycle progression. Additionally, these molecular findings are validated by performing a functional wound‐healing assay, which is accompanied by a dynamic extracellular matrix remodeling in response to the LDHs. Altogether, the results show that LDHs nanomaterials modulate cell adhesion, proliferation, and migration, delineating new advances on the biomaterial field applied in the context of soft tissue bioengineering, which must be explored in health disorders, such as wound healing in burn injuries.

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Advancing Public Health Using Regulatory Science to Enhance Development and Regulation of Medical Products: Food and Drug Administration Research at the Center for Biologics Evaluation and Research

Cited by 5 publications

References 39 publications

Evaluation of the effectiveness and efficiency of the East African community joint assessment procedure by pharmaceutical companies: Opportunities for improvement

Evaluation of the effectiveness and efficiency of the East African community joint assessment procedure by pharmaceutical companies: Opportunities for improvement

Challenges to producing novel therapies – dried plasma for use in trauma and critical care

Layered Double Hydroxides Are Promising Nanomaterials for Tissue Bioengineering Application

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