August 7, 2017

The global orphan drug market is expected to double between 2016 and 2022 to $209bn as Pharma rethinks its business model away from blockbusters towards niche patient populations and orphan diseases, according to a new report.

A combination of drivers, including market saturation, pricing pressures and stricter r...

June 22, 2017

FDA Commissioner Scott Gottlieb, MD, testified before a Senate Appropriations Subcommittee to discuss some of the programs he is implementing to streamline the FDA review process.  Gottlieb hopes to make orphan drugs easier to be reviewed by putting a stop to the requirement of companies to undertake new clinica...

May 10, 2017

The Senate voted 57 to 42 on Tuesday to confirm Dr. Scott Gottlieb as commissioner of the Food and Drug Administration, where he will be responsible for regulating drug companies to which he has had close ties in recent years. 


For more information, please click the following link: https://www.statnews.com/2017/...

May 1, 2017

We are so excited to support and promote Ichthyosis Awareness Month (IAM)! For more information and a list of all the amazing ways you can get involved, please review the FIRST website at http://www.firstskinfoundation.org/ichthyosis-awareness-month

April 19, 2017

A recently published article by the FDA Voice, the official blog of the FDA, details the impact small businesses are having on the pharmaceutical and biotech industry.  The article states that, "Over the past decade or so, more than half of the novel drugs (i.e., those not previously marketed in the United State...

March 29, 2017

Nature Magazine publishes article depicting the recent news of New York City's $1.15 billion biotech investment plan.  According to the article, "New York has long been a leader in basic biomedical research and clinical care, but it is now making a big push to turn the city into a life-sciences hub and create so...

February 14, 2017

February 28th, 2017 marks the 10th international Rare Disease Day.  On and around this day hundreds of patient organizations from countries and regions all over the world will hold awareness-raising activities based on the theme of research.

To learn more, please visit: rarediseaseday.org

January 26, 2017

The FDA Law Blog posted an informative article yesterday sharing insight into the status of the orphan drug landscape last year.  In 2016, both orphan drug approvals and designations decreased slightly; however, the number of orphan drug designation requests skyrocketed.  2016 was still the second best year for...

December 13, 2016

On December 13, 2016, President Obama signed into law the 21st Century Cures Act.  Robert Califf, MD, Commissioner of the FDA, stated that this law "builds on FDA’s ongoing efforts to advance medical product innovation and ensure that patients get access to treatments as quickly as possible, with continued assur...

December 5, 2016

December 2016 marks Patagonia's 3 year anniversary! We're very proud of our accomplishments over the past few years and excited to keep up the momentum as we progress towards our goal of bringing new treatments to market for patients suffering from rare dermatologic diseases.

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