Rising costs may not be a result of orphan drugs

Published Online: Sep 12,2016 Laurie Toich, Assistant Editor From Daraprim’s price rising 5556% by Turing Pharmaceuticals under the leadership of Martin Shkreli, to the most recent pricing scandal with Mylan’s EpiPen, Americans have been faced with high out-of-pocket costs for many prescription drugs. Government institutions, such as the US Centers for Medicare and Medicaid, have begun crafting novel ways to reduce …

Rare diseases

Sharing the mission to conquer rare diseases

Closer collaboration between pharmaceutical companies and patient organizations will accelerate drug development At an ever-quickening pace, biopharmaceutical companies and patient advocacy groups are banding together to hasten development of new drugs for rare diseases. Advocacy groups rely on their partners for financial and scientific support while providing access to repositories of patient data – the key to understanding rare diseases …

FDA Commissioner announcement

Keynote announced – FDA Commissioner Dr. Robert Califf to tackle precision medicine focus

NEW YORK – Dr. Robert Califf, the recently appointed Commissioner of the U.S. Food and Drug Administration (FDA), will be presenting the opening address on the second day of the inaugural World Precision Medicine Congress USA 2016. His talk will be at 9:00 AM on November 15th, 2016. Download the preview agenda here – http://goo.gl/pMGfsn > The FDA recently developed a platform …

genomeweb whitepaper

Alternative Proposals for Lab Test Regulation

Are There Opportunities for Consensus? A GenomeWeb Report February 2016 Amid multiple proposals on LDT regulation, are there opportunities for consensus? By Turna Ray The lab industry entered 2016 in a staring contest with the US Food and Drug Administration that began two years ago when the agency released its draft guidance on regulating laboratory-developed tests (LDTs). During the course …

US-Market-Size-Infographic

Infographic: What is the size of the Orphan Drugs Market in the USA?

Here at World Orphan Drug Congress USA, we understand that sometimes it is really useful to have the stats! So here they are – in a handy infographic! We thought this breakdown and overview might be helpful to some in the orphan drug sector. We took this from the FDA’s list of approved and designated orphan drugs and is one …

“Characterization of cell-based regenerative medicine products–current challenges and possible solutions”

Sema Rosinbum of FDA  joined us at Stem Cells & Regenerative Medicine Congress USA 2014 to present about ‘ Characterization of cell-based regenerative medicine products–current challenges and possible solutions’  Download the presentation now to learn more about: Cell therapy challenges Islet cell transplantation in diabetic mice Graded vs. Binary Responses …and much more! Get your copy here! To find out more about current challenges and …

Private Cord Blood Banks vs Public Health: Is it Time to Bury the Hatchet?

Can private cord blood banks ever work in synergy with public and national health services? Cord blood is the blood that remains in the placenta and umbilical cord after a baby is born. It is rich in stem cells, which can help to cure many life threatening diseases. The stem cells found in cord blood restore the function of the …

Download Ebook: 5 breakthrough therapies to watch

The US FDA’s Breakthrough Therapy demands sufficient preliminary clinical evidence to prove a compelling proof-of-concept; for instance the drug must show substantial improvement on a significant clinical endpoint when compared with current therapies. If these criteria are met, the US FDA is able to expedite the development and review of drug candidates that are able to treat serious of life …

investment in cell therapies

Promising scale and market success for cell therapies after large financial injection

In a report released this week, by BCC Research, it was forecasted that the global market for stem cells (including cell therapies) is to reach US $10.6 billion in 2018. However with currently no medicines, based on stem cells, having yet received marketing authorisation from the EMA*, or the FDA*, how are we going to get there? In 2013, the stem cell …