Category Archive: Rare Diseases

Oct 26

NORD Summit 2016 Highlights

By Kimberley Cummings, Ph.D., RAC (US), Vice President, Scientific & Regulatory Strategy and Operations Cato Research   The National Organization for Rare Disorders (NORD) held their annual Rare Diseases and Orphan Products Breakthrough Summit in Arlington, VA on October 17th and 18th.  If you have never been to this meeting before, it is the most …

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Sep 21

The Current Implementation of the Drug Supply Chain Security Act (DSCSA) Implementation Plan and Rare Disease Drugs

What is the Drug Supply Chain Security Act (DSCSA)? In November 2013 the Drug Quality and Security Act (DQSA) was signed into law with the intent to decrease incidence of counterfeit, falsified, or substandard prescription medication.  Title II of this act was the Drug Supply Chain Security Act (DSCSA), which lays the ten-year framework for …

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Oct 13

One and Done: Are single pivotal studies the new norm in cancer therapeutics?

By Leanna Gentry, Ph.D., Fellow at Cato Research Clinical trials are a lengthy and costly component of drug development, with the vast majority of drugs failing to ever get to patients. Often, the efficacy seen in preclinical studies does not translate in clinical studies, and the drug fails to receive approval because of lack of …

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Apr 11

AMP’d Up Search for Biomarkers

By Kathy A. Grako, Ph.D., PMP, CCRP, Senior Clinical Strategy Scientist In February the United States government announced a unique triad brought together to “accelerate the development of life saving drugs and to help identify new treatments and cures for diseases” under the Accelerated Medicine Partnership (AMP).  FDA and NIH have teamed up with 10 …

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Oct 28

Recap: U.S. Conference on Rare Diseases and Orphan Products

The 2013 US Conference on Rare Diseases and Orphan Products, sponsored by the Drug Information Association (DIA) and the National Organization for Rare Disorders (NORD), was held on 07 – 09 October in North Bethesda, MD.  This conference sought to assemble patients, regulators, industry, academicians, physicians, and capital venture investors to discuss four major themes …

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