Target Health Blog

Rare and Orphan Diseases

April 2, 2018

What's New

This past week, BioNJ (New Jersey) sponsored a meeting entitled: "Developing Rare Disease Regulatory Strategy Under Current Global Regulatory Statutes." Our friend and colleague Dr. Andrew Mulberg chaired the meeting and attendees were from patient advocacy groups, industry and FDA. There was the theme that clinical programs and regulatory strategies for rare and orphan diseases must be different from traditional development program, and that the "Totality of the Evidence" must be part of the strategy. While there are no black and white formulas to address the "Totality of the Evidence", it was clear that Quality of Life must be a key factor, especially when there are soft and non-validated clinical endpoints. Another striking idea was for companies collaborate with each other and FDA when working on the same indication. This way all can use the same pre-competitive natural history and available patient data, and then agree on how to study effectiveness. This would allow for the harmonization of drug development and avoid sponsors going in different directions when studying the same patient population. One exciting breakout session was on the roles of Registries both as sources of natural history data, as well a backbone for registry embedded clinical trials. 

Target Health is very active in the orphan and rare disease spaces and we have come up with creative strategies to accelerate time to market.

Spring is Here, a Little Late this Year: 2018 The Big Apple 

Last week, the 4th nor'easter storm hit New York City. It snowed all day and accumulated over 1 foot of snow in the NY metropolitan area.

Crocuses and others, struggle up through the snow in NYC   ©Target Health Inc.

For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 165). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel. The Target Health software tools are designed to partner with both CROs and Sponsors.

Joyce Hays, Founder and Editor in Chief of On Target

Jules Mitchel, Editor

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