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Radiopharmaceuticals for Radionuclide Therapy
Published in Michael Ljungberg, Handbook of Nuclear Medicine and Molecular Imaging for Physicists, 2022
Meltem Ocak, Emre Demirci, Jessie R. Nedrow, Rebecca Krimins
Yttrium-90 (T1/2=64.2 h) is a therapeutic radionuclide that emits a β-particle with a mean energy of 0.94 MeV and an average tissue penetration range of 2.5 mm. The selective targeting of Yttrium-90-TARE was first reported by Dr. Irving Ariel. In his study, patients with primary pancreatic and liver cancers were treated with 90Y-labeled ceramic microspheres injected intra-arterial at either the celiac axis or hepatic artery. The study concluded that the complications were minimal, and a significant number of patients experienced palliative effects as well as a few patients appearing to have increased survival [19]. Furthermore, the results were supportive of the continuation of exploring the use of Yttrium-90 for TARE. Yttrium-90 TARE agents are today still utilizing microspheres, which are either glass-based (Thrashers) or resin-based (SIR-Spheres) [20, 21]. Both TARE agents are FDA approved as devices under the humanitarian device exemption. Clinical trials mainly throughout the 1990s demonstrated that these agents were safe and effective in HCC (Thrashers) and metastatic colorectal cancer (SIR-Spheres) leading to their FDA approval in the late 1990s and early 2000s.
Electrical Brain Stimulation to Treat Neurological Disorders
Published in Bahman Zohuri, Patrick J. McDaniel, Electrical Brain Stimulation for the Treatment of Neurological Disorders, 2019
Bahman Zohuri, Patrick J. McDaniel
DBS has been studied as a treatment for depression or obsessive compulsive disorder (OCD). Currently, there is a Humanitarian Device Exemption for the use of DBS to treat OCD, but its use in depression remains only on an experimental basis. A review of all 22 published studies testing DBS for depression found that only three of them were of high quality because they had not only a treatment group but also a control group which did not receive DBS. The review found that across the studies, 40–50% of people showed receiving DBS greater than 50% improvement.
Perspectives on the Translational Aspects of Articular Cartilage Biology
Published in Kyriacos A. Athanasiou, Eric M. Darling, Grayson D. DuRaine, Jerry C. Hu, A. Hari Reddi, Articular Cartilage, 2017
Kyriacos A. Athanasiou, Eric M. Darling, Grayson D. DuRaine, Jerry C. Hu, A. Hari Reddi
Additional pathways to market include the humanitarian device exemption (HDE) and the PDP for Class III devices. HDE devices cannot have a profit margin, as this pathway is intended for the development of devices to treat rare (<4,000 patients per year) conditions. To speed the time to approval, regulatory burdens on the manufacturer are lessened for HDE devices; these devices do not have to demonstrate efficacy in large sample sizes since patients may be rare. HDE devices need to be approved by the IRB where they are used, and surgeons must be aware of the dearth of efficacy data for these devices. A PDP is an alternative to the PMA. To pursue this pathway, a company would work with the FDA in designing preclinical and clinical studies, protocols, assessment methods, and acceptance criteria. Few products have been approved through this pathway, though it may be speedier than a PMA, since the FDA would be involved from the initial development of the product all the way to market.
Rarely mentioned: how we arrived at the quantitative definition of a rare disease
Published in Baylor University Medical Center Proceedings, 2022
On a side note, Meyers related another story with a numerical population medical twist. The Safe Medical Devices Act of 1990 was amended with the Humanitarian Device Exemption so “medical devices for conditions affecting fewer than 4,000 people could get on the market with minimal evidence of safety and effectiveness.” Insurance companies began refusing to cover the use of these devices since they were experimental, although the FDA had approved them. The cutoff at 4,000 had to do with one particular surgical instrument that would have been used on fewer than 4,000 patients per year. Meyers questioned the wisdom of this conclusion remarking, “Surely it is not good public policy to design a law based on the needs of one product and one company.”4 Based on data from the FDA Office of Orphan Drugs Development, “Since 1990, the FDA has approved 76 medical devices for orphan indications under the Agency’s Humanitarian Device exemption.”12
Trends in safety and cost of deep brain stimulation for treatment of movement disorders in the United States: 2002–2014
Published in British Journal of Neurosurgery, 2021
Hansen Deng, John K. Yue, Doris D. Wang
The United States (U.S.) Food and Drug Administration (FDA) approved DBS to treat ET in 1997, followed by PD in 2002.7 Dystonia was approved in 2003 as a humanitarian device exemption (HDE).8,9 In addition to FDA approval, there has been mounting evidence from randomized controlled trials (RCTs) that neurostimulation of the subthalamic nucleus (STN) or globus pallidus internus (GPi) is superior to the best medical therapy available in treating PD.10–12 Recent results from a novel N-of-1 trial by Hyam et al. provided Class I evidence on the efficacy of DBS in tremor reduction for patients with ET.13 Other RCTs on Vim DBS showed significant functional improvement in both PD and ET patients with resistant tremors, even 6-years post-implantation.14,15 Results from two sham-controlled RCTs, Kupsch et al. and Volkmann et al., demonstrated that pallidal neurostimulation reduced symptoms in patients with dystonia.16,17 Long-term follow-up showed that symptom reduction persisted at 12 months.18,19
Biosynthetic alternatives for corneal transplant surgery
Published in Expert Review of Ophthalmology, 2020
May Griffith, Bijay Kumar Poudel, Kamal Malhotra, Naoufal Akla, Miguel González-Andrades, David Courtman, Victor Hu, Emilio I. Alarcon
In the USA, trials are conducted under Investigational Device Exemptions (IDE), allowing the use of unapproved devices in defined and regulated clinical trials. There are two main study types. The Early Feasibility Studies (EFS) program [128] was set up to stimulate ‘device innovation.’ The EFS program essentially allows innovators to work together with their sponsors, clinicians, and FDA review teams. 510 K and humanitarian device exemption (HDE) devices can also be evaluated clinically through this program. The aim of the EFS program is to allow limited clinical testing in <15 subjects, prior to finalization of the device design. It is usually intended to provide proof of principle and initial clinical safety data for the device or for use for a specific indication. The second IDE study type is the First in Human (FIH) study for a medical device, which is when that specific indication is evaluated for the first time in human subjects. A FIH can be an EFS, but not all FIH studies would be deemed EFSs. A program also exists for combination product called the Breakthrough Devices Program [129]. This is a ‘voluntary program for certain medical devices and device-led combination products that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or condition’. It replaces the Expedited Access Pathway and Priority Review for medical devices.