scholarly journals Regulatory updates are needed to prevent commercialization of islet transplantation in the US

2021 ◽  
Author(s):  
Piotr Witkowski ◽  
Rolf N. Barth ◽  
Anthony Japour ◽  
Gail Javitt ◽  
Jordan S. Pyda ◽  
...  
Keyword(s):  
Islet Transplantation ◽  
The Us

scholarly journals Arguments against the Requirement of a Biological License Application for Human Pancreatic Islets: The Position Statement of the Islets for US Collaborative Presented during the FDA Advisory Committee Meeting

2021 ◽  
Vol 10 (13) ◽  
pp. 2878
Author(s):  
Piotr Witkowski ◽  
Jon Odorico ◽  
Jordan Pyda ◽  
Roi Anteby ◽  
Robert Stratta ◽  
...  
Keyword(s):  
Advisory Committee ◽  
Islet Transplantation ◽  
Current System ◽  
Alternative Pathway ◽  
Position Statement ◽  
Cellular Tissue ◽  
Human Islets ◽  
Negative Consequences ◽  
License Application ◽  
The Us

The Food and Drug Administration (FDA) has been regulating human islets for allotransplantation as a biologic drug in the US. Consequently, the requirement of a biological license application (BLA) approval before clinical use of islet transplantation as a standard of care procedure has stalled the development of the field for the last 20 years. Herein, we provide our commentary to the multiple FDA’s position papers and guidance for industry arguing that BLA requirement has been inappropriately applied to allogeneic islets, which was delivered to the FDA Cellular, Tissue and Gene Therapies Advisory Committee on 15 April 2021. We provided evidence that BLA requirement and drug related regulations are inadequate in reassuring islet product quality and potency as well as patient safety and clinical outcomes. As leaders in the field of transplantation and endocrinology under the “Islets for US Collaborative” designation, we examined the current regulatory status of islet transplantation in the US and identified several anticipated negative consequences of the BLA approval. In our commentary we also offer an alternative pathway for islet transplantation under the regulatory framework for organ transplantation, which would address deficiencies of in current system.

scholarly journals Islets Transplantation at a Crossroads - Need for Urgent Regulatory Update in the United States: Perspective Presented During the Scientific Sessions 2021 at the American Diabetes Association Congress

2022 ◽  
Vol 12 ◽  
Author(s):  
Piotr Witkowski ◽  
Louis H. Philipson ◽  
John B. Buse ◽  
R. Paul Robertson ◽  
Rodolfo Alejandro ◽  
...  
Keyword(s):  
United States ◽  
Islet Transplantation ◽  
Organ Procurement ◽  
The United States ◽  
Regulatory Framework ◽  
Human Organs ◽  
Cell Tissue ◽  
License Application ◽  
The Us ◽  
Drug Manufacturing

Clinical islet allotransplantation has been successfully regulated as tissue/organ for transplantation in number of countries and is recognized as a safe and efficacious therapy for selected patients with type 1 diabetes mellitus. However, in the United States, the FDA considers pancreatic islets as a biologic drug, and islet transplantation has not yet shifted from the experimental to the clinical arena for last 20 years. In order to transplant islets, the FDA requires a valid Biological License Application (BLA) in place. The BLA process is costly and lengthy. However, despite the application of drug manufacturing technology and regulations, the final islet product sterility and potency cannot be confirmed, even when islets meet all the predetermined release criteria. Therefore, further regulation of islets as drugs is obsolete and will continue to hinder clinical application of islet transplantation in the US. The Organ Procurement and Transplantation Network together with the United Network for Organ Sharing have developed separately from the FDA and BLA regulatory framework for human organs under the Human Resources & Services Administration to assure safety and efficacy of transplantation. Based on similar biologic characteristics of islets and human organs, we propose inclusion of islets into the existing regulatory framework for organs for transplantation, along with continued FDA oversight for islet processing, as it is for other cell/tissue products exempt from BLA. This approach would reassure islet quality, efficacy and access for Americans with diabetes to this effective procedure.

scholarly journals Pancreas Transplantation

2008 ◽  
Vol 90 (5) ◽  
pp. 368-370 ◽  
Author(s):  
SA White ◽  
DW Manas
Keyword(s):  
Islet Transplantation ◽  
Pancreas Transplantation ◽  
Insulin Independence ◽  
Secondary Complications ◽  
Complications Of Diabetes ◽  
Beneficial Effects ◽  
The Us ◽  
The Uk

Over the last 5 years, there has been a resumed interest in treating diabetes by transplantation, particularly islet transplantation. However, despite advances being reported in Canada and the US, replication in the UK has been much more difficult. At present there is still only one treatment that can consistently reverse insulin independence in the long-term and that is whole pancreas transplantation. Long-term normoglycaemia has beneficial effects on preventing and ameliorating the secondary complications of diabetes and will be discussed.

The Eleventh Circuit Holds that Agreements in which Pharmaceutical Companies Pay Generic Companies Not to Compete May be Valid

2004 ◽  
Vol 32 (1) ◽  
pp. 181-184
Author(s):  
Amy Garrigues
Keyword(s):  
Pharmaceutical Companies ◽  
Court Decisions ◽  
Patent Holder ◽  
Trial Court ◽  
Court Of Appeals ◽  
The Us ◽  
Antitrust Laws ◽  
Per Se ◽  
Appeals Court ◽  
Two Sides

On September 15, 2003, the US. Court of Appeals for the Eleventh Circuit held that agreements between pharmaceutical and generic companies not to compete are not per se unlawful if these agreements do not expand the existing exclusionary right of a patent. The Valley DrugCo.v.Geneva Pharmaceuticals decision emphasizes that the nature of a patent gives the patent holder exclusive rights, and if an agreement merely confirms that exclusivity, then it is not per se unlawful. With this holding, the appeals court reversed the decision of the trial court, which held that agreements under which competitors are paid to stay out of the market are per se violations of the antitrust laws. An examination of the Valley Drugtrial and appeals court decisions sheds light on the two sides of an emerging legal debate concerning the validity of pay-not-to-compete agreements, and more broadly, on the appropriate balance between the seemingly competing interests of patent and antitrust laws.

Relation of Statistically Significant, Abnormal, and Typical WAIS-R VIQ-PIQ Discrepancies to Full Scale IQs

2000 ◽  
Vol 16 (2) ◽  
pp. 107-114 ◽  
Author(s):  
Louis M. Hsu ◽  
Judy Hayman ◽  
Judith Koch ◽  
Debbie Mandell
Keyword(s):  
Graphical Method ◽  
The United States ◽  
Full Scale ◽  
True Score ◽  
Absolute Value ◽  
Normative Population ◽  
The Us ◽  
The Mean ◽  
And Performance ◽  
Quadratic Models

Summary: In the United States' normative population for the WAIS-R, differences (Ds) between persons' verbal and performance IQs (VIQs and PIQs) tend to increase with an increase in full scale IQs (FSIQs). This suggests that norm-referenced interpretations of Ds should take FSIQs into account. Two new graphs are presented to facilitate this type of interpretation. One of these graphs estimates the mean of absolute values of D (called typical D) at each FSIQ level of the US normative population. The other graph estimates the absolute value of D that is exceeded only 5% of the time (called abnormal D) at each FSIQ level of this population. A graph for the identification of conventional “statistically significant Ds” (also called “reliable Ds”) is also presented. A reliable D is defined in the context of classical true score theory as an absolute D that is unlikely (p < .05) to be exceeded by a person whose true VIQ and PIQ are equal. As conventionally defined reliable Ds do not depend on the FSIQ. The graphs of typical and abnormal Ds are based on quadratic models of the relation of sizes of Ds to FSIQs. These models are generalizations of models described in Hsu (1996) . The new graphical method of identifying Abnormal Ds is compared to the conventional Payne-Jones method of identifying these Ds. Implications of the three juxtaposed graphs for the interpretation of VIQ-PIQ differences are discussed.

Factorial Structure of the Therapist Adherence Measure-Revised (TAM-R) Within Multisystemic Therapy

2020 ◽  
Vol 36 (2) ◽  
pp. 427-431
Author(s):  
Aurelie M. C. Lange ◽  
Marc J. M. H. Delsing ◽  
Ron H. J. Scholte ◽  
Rachel E. A. van der Rijken
Keyword(s):  
Factor Analysis ◽  
Factor Structure ◽  
Factor Model ◽  
Multisystemic Therapy ◽  
Model Fit ◽  
Validity And Reliability ◽  
Adherence Measure ◽  
Therapist Adherence ◽  
Confirmatory Factor ◽  
The Us

Abstract. The Therapist Adherence Measure (TAM-R) is a central assessment within the quality-assurance system of Multisystemic Therapy (MST). Studies into the validity and reliability of the TAM in the US have found varying numbers of latent factors. The current study aimed to reexamine its factor structure using two independent samples of families participating in MST in the Netherlands. The factor structure was explored using an Exploratory Factor Analysis (EFA) in Sample 1 ( N = 580). This resulted in a two-factor solution. The factors were labeled “therapist adherence” and “client–therapist alliance.” Four cross-loading items were dropped. Reliability of the resulting factors was good. This two-factor model showed good model fit in a subsequent Confirmatory Factor Analysis (CFA) in Sample 2 ( N = 723). The current finding of an alliance component corroborates previous studies and fits with the focus of the MST treatment model on creating engagement.

Measurement Invariance of a Universal Behavioral Screener Across Samples From the USA and Germany

2018 ◽  
Vol 34 (2) ◽  
pp. 87-100 ◽  
Author(s):  
Gino Casale ◽  
Robert J. Volpe ◽  
Brian Daniels ◽  
Thomas Hennemann ◽  
Amy M. Briesch ◽  
...  
Keyword(s):  
Measurement Invariance ◽  
Student Behavior ◽  
Cross Cultural ◽  
Cross Cultural Comparison ◽  
School Based ◽  
Cultural Research ◽  
Universal Screener ◽  
Confirmatory Factor ◽  
The Us ◽  
The Usa

Abstract. The current study examines the item and scalar equivalence of an abbreviated school-based universal screener that was cross-culturally translated and adapted from English into German. The instrument was designed to assess student behavior problems that impact classroom learning. Participants were 1,346 K-6 grade students from the US (n = 390, Mage = 9.23, 38.5% female) and Germany (n = 956, Mage = 8.04, 40.1% female). Measurement invariance was tested by multigroup confirmatory factor analysis (CFA) across students from the US and Germany. Results support full scalar invariance between students from the US and Germany (df = 266, χ2 = 790.141, Δχ2 = 6.9, p < .001, CFI = 0.976, ΔCFI = 0.000, RMSEA = 0.052, ΔRMSEA = −0.003) indicating that the factor structure, the factor loadings, and the item thresholds are comparable across samples. This finding implies that a full cross-cultural comparison including latent factor means and structural coefficients between the US and the German version of the abbreviated screener is possible. Therefore, the tool can be used in German schools as well as for cross-cultural research purposes between the US and Germany.

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