Regulatory and policy issues for T1DM immunotherapy

Fleming, Alexander

doi:10.4161/hv.7.1.14527

Cited by 4 publications

(3 citation statements)

References 14 publications

(12 reference statements)

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“…However, for clinical and biostatistical reasons, measurement of a reliable change in group mean C-peptide levels in a small group of participants in less than 6 months is extremely difficult even if the underlying efficacy is high (27). Additionally, if all other factors were equal, efficacy would be easier to measure in children because, due to generally more intense autoimmunity, their C-peptide decline is more rapid than those of adults (25).…”

Section: Regulatory Considerationsmentioning

confidence: 99%

Alternate Approaches for Pediatric Type 1 Diabetes Drug Development and Potential Regulatory Approval: A Perspective

Turner

Fleming³

et al. 2015

Diabetes Care

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The incidence and prevalence of pediatric type 1 diabetes are increasing globally, including in the U.S. While the increasing number of cases of pediatric diabetes makes expeditious availability of new medical products and therapies for diabetes care essential, there have been many barriers encountered in bringing some drugs and devices to pediatric patients who may benefit. Newer insulins have been studied and approved for use in children. However, hurdles exist in the inclusion of children in studies of therapies aimed at preventing β-cell loss in those with new-onset diabetes and those at risk for type 1 diabetes. This Perspective focuses on potential solutions to the challenges experienced in bringing new drugs for pediatric type 1 diabetes to marketing approval. Given their central importance as the users of medical products, patient perspectives are included along with scientific and regulatory considerations.

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Section: Regulatory Considerationsmentioning

confidence: 99%

Alternate Approaches for Pediatric Type 1 Diabetes Drug Development and Potential Regulatory Approval: A Perspective

Turner

Fleming³

et al. 2015

Diabetes Care

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“…In contrast to trials that evaluate glucose-lowering therapies and use HbA1c as the primary endpoint, trials aimed at T1D autoimmunity evaluate the treatment’s effect on the decline in endogenous insulin secretion as reflected by stimulated C-peptide levels [ 6 ]. Because of the high variability in stimulated baseline C-peptide levels, these trials must be longer and larger than typical trials with HbA1c as the endpoint [ 7 , 8 ].…”

Section: Introductionmentioning

confidence: 99%

“…Despite huge unmet clinical need for therapies specifically approved for T1D and defined regulatory pathways for T1D indications [ 8 , 16 , 17 ], to date (and for a variety of reasons) no drug remains in Phase 3 development. Four drugs reached that stage, GAD65 (Diamyd), DiaPep277 (Andromeda), otelixizumab (GlaxoSmithKline), and teplizumab (Lilly/MacroGenics), but all have gone back to Phase 2 or have been discontinued.…”

Section: Introductionmentioning

confidence: 99%

Addressing Unmet Medical Needs in Type 1 Diabetes: A Review of Drugs Under Development

Mittermayer¹,

Caveney

Oliveira

et al. 2017

CDR

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Introduction: The incidence of type 1 diabetes (T1D) is increasing worldwide and there is a very large need for effective therapies. Essentially no therapies other than insulin are currently approved for the treatment of T1D. Drugs already in use for type 2 diabetes and many new drugs are under clinical development for T1D, including compounds with both established and new mechanisms of action.Content of the Review: Most of the new compounds in clinical development are currently in Phase 1 and 2. Drug classes discussed in this review include new insulins, SGLT inhibitors, GLP-1 agonists, immunomodulatory drugs including autoantigens and anti-cytokines, agents that regenerate β-cells and others.Regulatory Considerations: In addition, considerations are provided with regard to the regulatory environment for the clinical development of drugs for T1D, with a focus on the United States Food and Drug Administration and the European Medicines Agency. Future opportunities, such as combination treatments of immunomodulatory and beta-cell regenerating therapies, are also discussed.

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Regulatory Considerations for Early Clinical Development of Drugs for Diabetes, Obesity, and Cardiometabolic Disorders

Fleming¹

2014

Translational Research Methods for Diabetes, Obesity and Cardiometabolic Drug Development

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Regulatory and policy issues for T1DM immunotherapy

Abstract: To cite this article: Alexander Fleming (2011) Regulatory and policy issues for T1DM immunotherapy, Human Vaccines, 7:1, 50-55,

Cited by 4 publications

References 14 publications

Alternate Approaches for Pediatric Type 1 Diabetes Drug Development and Potential Regulatory Approval: A Perspective

Alternate Approaches for Pediatric Type 1 Diabetes Drug Development and Potential Regulatory Approval: A Perspective

Addressing Unmet Medical Needs in Type 1 Diabetes: A Review of Drugs Under Development

Regulatory Considerations for Early Clinical Development of Drugs for Diabetes, Obesity, and Cardiometabolic Disorders

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