scholarly journals Evaluation of institutional readiness at sites within the UK NHS using a novel advanced therapy medicinal product assessment tool

2021 ◽  
Author(s):  
Lisa Wotherspoon ◽  
Ruaridh Buchan ◽  
Ewan Morrison ◽  
Greg Amatt
Keyword(s):  
Assessment Tool ◽  
Self Assessment ◽  
Advanced Therapy Medicinal Product ◽  
Advanced Therapy ◽  
Independent Expert ◽  
Treatment Centre ◽  
Product Assessment ◽  
Uk National Health Service ◽  
The Uk ◽  
Therapy Treatment

Aim: This paper reports on a study to assess institutional readiness (IR) of UK National Health Service sites that form part of the Northern Alliance Advanced Therapy Treatment Centre to deliver advanced therapy medicinal products (ATMPs). The paper discusses the development of an assessment tool to support self-assessment of IR in healthcare institutions. Methods: The tool utilized criteria developed by clinicians to self-assess IR to deliver four classes of ATMP over a series of time points. Each assessment was independently analyzed and validated by independent expert groups. Results & conclusion: The collated results indicated an overall trend toward IR for all classes of ATMP. The study highlighted areas where IR is evidenced, areas where work is ongoing and areas where further work is required to achieve IR. The study also facilitated validation of the IR assessment tool.

Re-engineering the innovator-clinic interface for adoption of advanced therapies

2021 ◽  
Author(s):  
Nicholas Medcalf
Keyword(s):  
Systems Engineering ◽  
Value Chain ◽  
Lifetime Cost ◽  
Innovation Process ◽  
Adoption Process ◽  
Advanced Therapy ◽  
Advanced Therapies ◽  
The Uk ◽  
The Relationship ◽  
Therapy Treatment

The relationship between regenerative medicine innovators and the clinics that will use their inventions continues to evolve. In the UK, the Advanced Therapy Treatment Centres exemplify this. The agents in the value chain are becoming collaborators in a shared innovation process. This paper proposes a larger role for systems engineering in this change and a cost-based representation of institutional readiness in designing suitable operational models for clinical adoption. The proposed approach places this value in a whole-lifetime cost framework. The current value for the adoption process can then be estimated for comparison with the sum of the adoption costs, the costs of operating at steady state and, if need be, replacement costs at end of life of the innovation.

scholarly journals Processing and Ex Vivo Expansion of Adipose Tissue-Derived Mesenchymal Stem/Stromal Cells for the Development of an Advanced Therapy Medicinal Product for Use in Humans

Cells ◽  
2021 ◽  
Vol 10 (8) ◽  
pp. 1908
Author(s):  
Anna Labedz-Maslowska ◽  
Agnieszka Szkaradek ◽  
Tomasz Mierzwinski ◽  
Zbigniew Madeja ◽  
Ewa Zuba-Surma
Keyword(s):  
Adipose Tissue ◽  
Medicinal Product ◽  
Stromal Cells ◽  
Manufacturing Process ◽  
Cellular Therapy ◽  
Ex Vivo ◽  
Ex Vivo Expansion ◽  
Advanced Therapy Medicinal Product ◽  
Differentiation Potential ◽  
Advanced Therapy

Adipose tissue (AT) represents a commonly used source of mesenchymal stem/stromal cells (MSCs) whose proregenerative potential has been widely investigated in multiple clinical trials worldwide. However, the standardization of the manufacturing process of MSC-based cell therapy medicinal products in compliance with the requirements of the local authorities is obligatory and will allow us to obtain the necessary permits for product administration according to its intended use. Within the research phase (RD), we optimized the protocols used for the processing and ex vivo expansion of AT-derived MSCs (AT-MSCs) for the development of an Advanced Therapy Medicinal Product (ATMP) for use in humans. Critical process parameters (including, e.g., the concentration of enzyme used for AT digestion, cell culture conditions) were identified and examined to ensure the high quality of the final product containing AT-MSCs. We confirmed the identity of isolated AT-MSCs as MSCs and their trilineage differentiation potential according to the International Society for Cellular Therapy (ISCT) recommendations. Based on the conducted experiments, in-process quality control (QC) parameters and acceptance criteria were defined for the manufacturing of hospital exemption ATMP (HE-ATMP). Finally, we conducted a validation of the manufacturing process in a GMP facility. In the current study, we presented a process approach leading to the optimization of processing and the ex vivo expansion of AT-MSCs for the development of ATMP for use in humans.

scholarly journals Temporomandibular Joint Osteoarthritis: Regenerative Treatment by a Stem Cell Containing Advanced Therapy Medicinal Product (ATMP)—An In Vivo Animal Trial

2021 ◽  
Vol 22 (1) ◽  
pp. 443
Author(s):  
Robert Köhnke ◽  
Marcus Oliver Ahlers ◽  
Moritz Alexander Birkelbach ◽  
Florian Ewald ◽  
Michael Krueger ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hyaluronic Acid ◽  
Medicinal Product ◽  
Temporomandibular Joint ◽  
Stromal Cells ◽  
Stromal Cell ◽  
Advanced Therapy Medicinal Product ◽  
Animal Trial ◽  
Advanced Therapy ◽  
Temporomandibular Joint Osteoarthritis

Temporomandibular joint osteoarthritis (TMJ-OA) is a chronic degenerative disease that is often characterized by progressive impairment of the temporomandibular functional unit. The aim of this randomized controlled animal trial was a comparative analysis regarding the chondroregenerative potency of intra-articular stem/stromal cell therapy. Four weeks after combined mechanical and biochemical osteoarthritis induction in 28 rabbits, therapy was initiated by a single intra-articular injection, randomized into the following groups: Group 1: AB Serum (ABS); Group 2: Hyaluronic acid (HA); Group 3: Mesenchymal stromal cells (STx.); Group 4: Mesenchymal stromal cells in hyaluronic acid (HA + STx.). After another 4 weeks, the animals were euthanized, followed by histological examination of the removed joints. The histological analysis showed a significant increase in cartilage thickness in the stromal cell treated groups (HA + STx. vs. ABS, p = 0.028; HA + ST.x vs. HA, p = 0.042; STx. vs. ABS, p = 0.036). Scanning electron microscopy detected a similar heterogeneity of mineralization and tissue porosity in the subchondral zone in all groups. The single intra-articular injection of a stem cell containing, GMP-compliant advanced therapy medicinal product for the treatment of iatrogen induced osteoarthritis of the temporomandibular joint shows a chondroregenerative effect.

Efficacy and safety data for the Ab interno XEN45 gel stent implant at 3 Years: A retrospective analysis

2021 ◽  
pp. 112067212110143
Author(s):  
Itay E Gabbay ◽  
Mordechai Goldberg ◽  
Felicity Allen ◽  
Zhiheng Lin ◽  
Christine Morley ◽  
...  
Keyword(s):  
Intraocular Pressure ◽  
Failure Rate ◽  
Open Angle Glaucoma ◽  
Safety Data ◽  
Angle Closure ◽  
Efficacy And Safety ◽  
Antihypertensive Medications ◽  
Uk National Health Service ◽  
The Uk ◽  
Comparative Audit

Purpose: To report efficacy and safety measures of the XEN45 gel stent at 36 months in the UK National Health Service setting. Methods: Retrospective non-comparative audit of the records of patients who underwent XEN45 implantation between June 2015 and May 2017 was performed. Main outcome measures were intraocular pressure and number of antihypertensive medications used. Failure was defined as need for further surgery or stent removal. Success was defined as a 20% decrease in intraocular pressure without the need for additional glaucoma medications or a reduction in antihypertensive medications without an increase in baseline intraocular pressure. Needling rates and preoperative factors effect were assessed. Results: The cohort included 205 patients (205 eyes) with primary open angle glaucoma (84.4%), angle closure glaucoma (7.8%), or refractory glaucoma (7.8%), 62.9% had a stand-alone procedure and 37.1%, a combined phaco-XEN45 procedure. Mean intraocular pressure was 22.6 ± 7.0 mmHg at baseline compared to 14.7 ± 3.8 mmHg at 24 months and 14.0 ± 2.9 mmHg at 36 months ( p < 0.001 for both). Mean number of medications used was 2.6 ± 1.1 at baseline compared to 0.5 ± 0.9 and 0.6 ± 1.0, at 24- and 36-months, respectively ( p < 0.001 for both). The failure rate at 36 months was 25%. Needling was required in 36.6%. Evaluation of background factors yielded an increased failure rate in non-Caucasians compared to Caucasians (74% vs 21%, p < 0.001). Conclusion: XEN45 implantation is effective and safe at 36 months’ follow-up. Patients should be advised regarding the risk of failure and possible need for bleb revisions. Careful patient selection may be required.

scholarly journals Generation and Evaluation of Novel Biomaterials Based on Decellularized Sturgeon Cartilage for Use in Tissue Engineering

Biomedicines ◽  
2021 ◽  
Vol 9 (7) ◽  
pp. 775
Author(s):  
Olimpia Ortiz-Arrabal ◽  
Ramón Carmona ◽  
Óscar-Darío García-García ◽  
Jesús Chato-Astrain ◽  
David Sánchez-Porras ◽  
...  
Keyword(s):  
Tissue Engineering ◽  
Ex Vivo ◽  
Cartilage Damage ◽  
Human Mesenchymal Stem Cells ◽  
In Silico Analysis ◽  
Cartilage Tissue ◽  
Advanced Therapy Medicinal Product ◽  
Advanced Therapy ◽  
Novel Scaffold

Because cartilage has limited regenerative capability, a fully efficient advanced therapy medicinal product is needed to treat severe cartilage damage. We evaluated a novel biomaterial obtained by decellularizing sturgeon chondral endoskeleton tissue for use in cartilage tissue engineering. In silico analysis suggested high homology between human and sturgeon collagen proteins, and ultra-performance liquid chromatography confirmed that both types of cartilage consisted mainly of the same amino acids. Decellularized sturgeon cartilage was recellularized with human chondrocytes and four types of human mesenchymal stem cells (MSC) and their suitability for generating a cartilage substitute was assessed ex vivo and in vivo. The results supported the biocompatibility of the novel scaffold, as well as its ability to sustain cell adhesion, proliferation and differentiation. In vivo assays showed that the MSC cells in grafted cartilage disks were biosynthetically active and able to remodel the extracellular matrix of cartilage substitutes, with the production of type II collagen and other relevant components, especially when adipose tissue MSC were used. In addition, these cartilage substitutes triggered a pro-regenerative reaction mediated by CD206-positive M2 macrophages. These preliminary results warrant further research to characterize in greater detail the potential clinical translation of these novel cartilage substitutes.

scholarly journals Testing a self-assessment tool for HP competencies with participants of training courses in Austria

2020 ◽  
Vol 30 (Supplement_5) ◽  
Author(s):  
G Lang
Keyword(s):  
Assessment Tool ◽  
Core Competencies ◽  
Empirical Studies ◽  
Reliability And Validity ◽  
International Standards ◽  
Online Questionnaire ◽  
Self Assessment ◽  
High Quality ◽  
Training Courses ◽  
Training Programmes

Abstract Background High quality health promotion (HP) depends on a competent workforce for which professional development programmes for practitioners are essential. The “CompHP Core Competencies Framework in HP” defines crucial competency domains but a recent review concluded that the implementation and use of the framework is lacking. The aim was to develop and validate a self-assessment tool for HP competencies, which should help evaluate training courses. Methods A brief self-assessment tool was employed in 2018 in Austria. 584 participants of 77 training courses submitted their post-course assessment (paper-pencil, RR = 78.1%). In addition, longitudinal data are available for 148 participants who filled in a pre-course online questionnaire. Measurement reliability and validity was tested by single factor, bifactor, multigroup, and multilevel CFA. A SEM proved for predictive and concurrent validity, controlling gender and age. Results A bifactor model (X2/df=3.69, RMSEA=.07, CFI=.95, sRMR=.07) showed superior results with a strong general CompHP factor (FL&gt;.65, wH=.90, ECV=.85), configurally invariant for two training programmes. On course level, there was only minimal variance between trainings (ICC&lt;.08). Structurally, there was a significant increase in HP competencies when comparing pre- and post-course measurements (b=.33, p&lt;.01). Participants showed different levels of competencies due to prior knowledge (b=.38, p&lt;.001) and course format (b=.16, p&lt;.06). The total scale had good properties (m = 49.8, sd = 10.3, 95%-CI: 49.0-50.7) and discriminated between groups (eg by training length). Conclusions The results justify the creation of an overall scale to assess core HP competencies. It is recommended to use the scale for evaluating training courses. The work compensates for the lack of empirical studies on the CompHP concept and facilitates a broader empirical application of a uniform competency framework for HP in accordance with international standards in HP and public health. Key messages The self-assessment tool provides a good and compact foundation for assessing HP competencies. It provides a basis for holistic, high quality and sustainable capacity building or development in HP.

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