The FDA’s Humanitarian Device Exemption Program

Eydelman, Malvina B.; Chen, Eric A.

doi:10.1377/hlthaff.2011.0550

Cited by 6 publications

(4 citation statements)

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“…32 However, the device has been shown to produce clinically meaningful improvement, and it remains on the market as an adjunctive therapy for patients with severe, chronic, treatment resistant, obsessive compulsive disorder who have not responded to at least three selective serotonin reuptake inhibitors. 33 Safety issues with some humanitarian devices have emerged after approval. A randomised controlled trial of an intracranial neurovascular stent 34 led the FDA to restrict the label indication to a substantially smaller patient population after approval.…”

Section: Performance Of Humanitarian Devicesmentioning

confidence: 99%

Assessment of US pathway for approving medical devices for rare conditions

Hwang

Carpenter

Kesselheim

2014

BMJ

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Section: Performance Of Humanitarian Devicesmentioning

confidence: 99%

Assessment of US pathway for approving medical devices for rare conditions

Hwang

Carpenter

Kesselheim

2014

BMJ

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“…The Humanitarian Device exemption regulatory path incentivizes device development for orphan populations by requiring that such a test 1) addresses an unmet need and has no predicate for the intended use, 2) does not pose an unreasonable or significant risk of injury, and 3) demonstrates probable benefit which outweighs the risk of injury or illness related to its intended use. 10 Impending regulation of in-vitro diagnostics is likely to foster interest in this mechanism for rare and high-risk diseases. 11,12 …”

Section: Introductionmentioning

confidence: 99%

Predicting Cellular Rejection With a Cell-Based Assay

et al. 2017

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Background Allospecific CD154+T-cytotoxic memory cells (CD154+TcM) predict acute cellular rejection (ACR) after liver or intestine transplantation (LTx, ITx) in small cohorts of children and can enhance immunosuppression management, but await validation and clinical implementation. Methods To establish safety and probable benefit, CD154+TcM were measured in cryopreserved samples from 214 children <21 years (NCT#1163578). Training set samples, n=158, were tested with research-grade reagents and 122 independent validation set samples were tested with cGMP-manufactured reagents after assay standardization and reproducibility testing. Recipient CD154+TcM induced by stimulation with donor cells were expressed as a fraction of those induced by HLA-non-identical cells in parallel cultures. The resulting immunoreactivity index (IR) if > 1 implies increased rejection-risk. Results Training and validation set subjects were demographically similar. Mean coefficient of test variation was <10% under several conditions. Logistic regression incorporating several confounding variables identified separate pre-transplant and post-transplant IR thresholds for prediction of rejection in respective training set samples. An IR ≥ 1.1 in post-transplant training samples, and IR ≥1.23 in pre-transplant training samples predicted LTx or ITx rejection in corresponding validation set samples in the 60-day post-sampling period with sensitivity, specificity, positive and negative predictive values of 84%, 80%, 64%, and 92%, respectively (AUC 0.792), and 57%, 89%, 78%, and 74%, respectively (AUC 0.848). No adverse events were encountered due to phlebotomy. Conclusions Allospecific CD154+T-cytotoxic memory cells predict acute cellular rejection after liver or intestine transplantation in children. Adjunctive use can enhance clinical outcomes.

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“…The pre-marketing evaluation of this test system under the Humanitarian Device Exemption route requires that Pleximmune™ 1) addresses an unmet need and has no predicate for the intended use, 2) does not pose an unreasonable or significant risk of injury, and 3) demonstrates probable benefit which outweighs the risk of injury or illness related to its intended use [27]. …”

Section: Introductionmentioning

confidence: 99%

Profile of the Pleximmune blood test for transplant rejection risk prediction

Sindhi

Ashokkumar

Higgs

et al. 2016

Expert Review of Molecular Diagnostics

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Summary The Pleximmune™ test (Plexision Inc., Pittsburgh, PA, USA) is the first cell-based test approved by the US FDA, which predicts acute cellular rejection in children with liver- or intestine transplantation. The test addresses an unmet need to improve management of immunosuppression, which incurs greater risks of opportunistic infections and Epstein–Barr virus-induced malignancy during childhood. High-dose immunosuppression and recurrent rejection after intestine transplantation also result in a 5-year graft loss rate of up to 50%. Such outcomes seem increasingly unacceptable because children can experience rejection-free survival with reduced immunosuppression. Pleximmune test sensitivity and specificity for predicting acute cellular rejection is 84% and 81% respectively in training set–validation set testing of 214 children. Among existing gold standards, the biopsy detects but cannot predict rejection. Anti-donor antibodies, which presage antibody-mediated injury, reflect late-stage allosensitization as a downstream effect of engagement between recipient and donor cells. Therefore, durable graft and patient outcomes also require an accurate management of cellular immune responses in clinical practice.

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The FDA’s Humanitarian Device Exemption Program

Cited by 6 publications

References 0 publications

Assessment of US pathway for approving medical devices for rare conditions

Assessment of US pathway for approving medical devices for rare conditions

Predicting Cellular Rejection With a Cell-Based Assay

Profile of the Pleximmune blood test for transplant rejection risk prediction

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