Dataset Information

Expanding Patient Access to Investigational Drugs: Single Patient Investigational New Drug and the "Right to Try".

ABSTRACT: With drug approval times taking an average of 8 years from entry into clinical trials to full U.S. Food and Drug Administration (FDA) approval, patients with life-threatening and severely debilitating disease and no reasonable therapeutic options are advocating for expanded access (EA) to investigational drugs prior to approval. Special investigational new drug (IND) application categories allow patients who meet specific criteria to receive treatment with non-approved drugs. The FDA approves over 99% of all single-patient INDs, providing emergency approval within hours, and non-emergency approval within an average of 4 days. "Right-to-try" laws passed in 38 states would allow patients to bypass FDA processes altogether, but contain controversial provisions that some claim risk more harm than benefit to desperate and vulnerable patients. This review focuses on FDA EA to non-approved drugs through a special category of IND-the single-patient IND-and "right-to-try" (R2T) access outside of the FDA.

SUBMITTER: Van Norman GA

PROVIDER: S-EPMC6059004 | biostudies-literature | 2018 Apr

REPOSITORIES: biostudies-literature

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Publications

Expanding Patient Access to Investigational Drugs: Single Patient Investigational New Drug and the "Right to Try".

Van Norman Gail A GA

JACC. Basic to translational science 20180401 2

With drug approval times taking an average of 8 years from entry into clinical trials to full U.S. Food and Drug Administration (FDA) approval, patients with life-threatening and severely debilitating disease and no reasonable therapeutic options are advocating for expanded access (EA) to investigational drugs prior to approval. Special investigational new drug (IND) application categories allow patients who meet specific criteria to receive treatment with non-approved drugs. The FDA approves ov ...[more]

PMID: 30062214

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Project description:BackgroundHepatic and biliary diseases are prevalent worldwide, but the majority of people lack access to diagnostic medical imaging for their assessment. The liver and gallbladder are readily amenable to sonographic examination, and ultrasound is a portable, cost-effective imaging modality suitable for use in rural and underserved areas. However, the deployment of ultrasound in these settings is limited by the lack of experienced sonographers to perform the exam. In this study, we tested an asynchronous telediagnostic system for right upper quadrant abdominal ultrasound examination operated by individuals without prior ultrasound experience to facilitate deployment of ultrasound to rural and underserved areas.MethodsThe teleultrasound system utilized in this study employs volume sweep imaging and a telemedicine app installed on a tablet which connects to an ultrasound machine. Volume sweep imaging is an ultrasound technique in which an individual scans the target region utilizing preset ultrasound sweeps demarcated by easily recognized external body landmarks. The sweeps are saved as video clips for later interpretation by an experienced radiologist. Teleultrasound scans from a Peruvian clinic obtained by individuals without prior ultrasound experience were sent to the United States for remote interpretation and quality assessment. Standard of care comparison was made to a same-day ultrasound examination performed by a radiologist.ResultsIndividuals without prior ultrasound experience scanned 144 subjects. Image quality was rated "poor" on 36.8% of exams, "acceptable" on 38.9% of exams, and "excellent" on 24.3% of exams. Among telemedicine exams of "acceptable" or "excellent" image quality (n = 91), greater than 80% of the liver and gallbladder were visualized in the majority of cases. In this group, there was 95% agreement between standard of care and teleultrasound on whether an exam was normal or abnormal, with a Cohen's kappa of 0.84 (95% CI 0.7-0.98, p <0.0001). Finally, among these teleultrasound exams of "acceptable" or "excellent" image quality, the sensitivity for cholelithiasis was 93% (95% CI 68.1%-99.8%), and the specificity was 97% (95% CI 89.5%-99.6%).ConclusionThis asynchronous telediagnostic system allows individuals without prior ultrasound experience to effectively scan the liver, gallbladder, and right kidney with a high degree of agreement with standard of care ultrasound. This system can be deployed to improve access to diagnostic imaging in low-resource areas.

Dataset Information

Expanding Patient Access to Investigational Drugs: Single Patient Investigational New Drug and the "Right to Try".

Publications

Expanding Patient Access to Investigational Drugs: Single Patient Investigational New Drug and the "Right to Try".

Similar Datasets

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