Tag Archive: pediatric

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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Jun 11

U.S. Pediatric Product Development

In the United States, there are two regulations in place regarding pediatric trials and related incentives: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA).  The PREA requires pediatric assessments for applications submitted for changes in active ingredient, indication, dosage form, dosing regimen, or route of administration unless a waiver …

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Mar 06

FDA’s New Guidances for February 2013

Several new guidance documents were released by FDA in February 2013, covering a wide range of subjects. S10 Photosafety Evaluation of Pharmaceuticals Alzheimer’s Disease: Developing Drugs for the Treatment of Early Stage Disease Class II Special Controls Guideline: Temporary Mandibular Condyle Reconstruction Plate Immunogenicity Assessment for Therapeutic Protein Products Clinical Study Designs for Surgical Ablation …

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Mar 06

FDA's New Guidances for February 2013

Several new guidance documents were released by FDA in February 2013, covering a wide range of subjects. S10 Photosafety Evaluation of Pharmaceuticals Alzheimer’s Disease: Developing Drugs for the Treatment of Early Stage Disease Class II Special Controls Guideline: Temporary Mandibular Condyle Reconstruction Plate Immunogenicity Assessment for Therapeutic Protein Products Clinical Study Designs for Surgical Ablation …

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Feb 15

Everything You Need to Know About Priority Review Vouchers: Part 2 of 2

The Rare Pediatric Disease PRV, the Difference in the PRVs, and the Future of the Program The Rare Pediatric Disease Priority Review Voucher (If you haven’t read Part 1 yet, start there first!) A new Priority Review Voucher (PRV) has been introduced that could be a game changer with respect to the program.  Nancy Goodman …

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Feb 12

Everything You Need to Know About Priority Review Vouchers: Part 1 of 2

The Basics, History, and Record of the Priority Review Voucher Program The Basics If your company’s goals include funding pipeline development or increasing speed to market you should, at least, be familiar with the concept of the Priority Review Voucher (PRV). A PRV would be aptly described as an obscure or esoteric regulatory curiosity by …

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Oct 24

Considerations for the Assessment of Pain in Non-Verbal and Pediatric Subjects

Studies of drugs, biologics or devices that are intended for use in pediatric populations fall under far more stringent guidelines than those intended for use in adults.  The issues with pediatric studies range from informed consent concerns to stricter risk-to-benefit analyses.  Another problem that can arise in these studies, is that very young children cannot …

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Aug 07

FDASIA, Part 3: Generating Antibiotic Incentives Now (GAIN) and Drug Approval and Patient Access Changes

On 09 July 2012, the Food and Drug Administration Safety and Innovation Act (FDASIA, otherwise known as PDUFA V) was signed into law.  In a 4-part weekly series, we are covering the major changes in FDASIA as follows: Part 1: The “UFAs”: PDUFA, MDUFA, GDUFA, and BsUFA Part 2: Pediatric Drugs and Devices and Medical …

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Jul 27

FDASIA, Part 2: Pediatric Drugs and Devices and Medical Device Regulatory Improvements

On 09 July 2012, the Food and Drug Administration Safety and Innovation Act (FDASIA, otherwise known as PDUFA V) was signed into law.  In a 4-part weekly series, we are covering the major changes in FDASIA as follows: Part 1: The “UFAs”: PDUFA, MDUFA, GDUFA, and BsUFA Part 2: Pediatric Drugs and Devices and Medical …

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May 25

PDUFA V: A Funny Sounding Name for a Very Important Bill (Updated!)

US Capitol

Can you say PDUFA (pŭ-dōō-fŭ)?  The United States Senate can.  A very partisan Senate showed uncharacteristically bipartisan support for the Prescription Drug and User Fee Act (PDUFA) V, voting on 24 May 2012 to reauthorize PDUFA by a 96 to 1 vote.  This reauthorization allows for a 6% increase in fees paid by drug and …

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