Category Archive: Rare Diseases

Apr 24 2019

New FDA Guidance on Natural History Studies for Rare Diseases

In a recent Ask Cato blog post, I summarized a revised FDA guidance issued in January 2019, Rare Diseases: Common Issues in Drug Development.1 The guidance covers a range of topics, including the use of surrogate biomarkers, nonclinical flexibility, and natural history studies, to assist sponsors in conducting more efficient drug development programs for rare …

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Feb 28 2019

Revised FDA Guidance on Developing Rare Disease Therapies

by Kristen Biernat, Ph.D., Scientist at Cato Research   Rare diseases, defined as conditions affecting fewer than 200,000 people in the United States, pose a significant health care concern.1 There are approximately 7,000 rare diseases that cumulatively affect more than 25 million Americans, half of which are children.2 In addition, most rare diseases are serious …

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Oct 26 2016

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 2016

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 2015

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 2014

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 2013

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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