Category Archives: Orphan Drugs

FDA: From Risk Aversion to Approval Activism

By Ben Comer | Published: May 7, 2013
During the Rutgers Business School’s annual healthcare symposium, an FDA official encouraged industry to put its drugs on the reviewing table and be prepared for good news. On a panel titled “Activist FDA: Transformation Agent,” Prevision Policy founder and former Pink Sheet editorial head Cole Werble relayed the tale of Acadia Pharmaceuticals, a San Diego-based [...]
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Drug Pipelines in Canada: Is There a Buyer for Future Innovation?

By Clark Herman | Published: April 15, 2013
Last week, Canada’s National Prescription Drug Utilization Information System (NPDUIS), a federal-provincial fact-finding panel that works closely with the Patented Medicines Prices Review Board (PMPRB), issued its fourth New Drug Pipeline Monitor (NDPM) looking at drugs currently under development that may have an impact on future drug expenditures. The report is another example of how [...]
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Reflections on Rare Disease Day

By Clark Herman | Published: March 1, 2013
The rare disease advocacy community convened yesterday for its annual Rare Disease Day celebration, marking another year of advances in the fight against ailments once almost entirely ignored by big Pharma. The fact that hundreds of rare diseases are finally attracting real attention from major researchers, not to mention big dollars, is attributable not [...]
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It's Blind Leading the Blind on Companion Diagnostics

By Clark Herman | Published: January 25, 2013
A new report from the Tufts Center for Study of Drug Development (CSDD), confirms that companion diagnostics are an important factor for pharma companies to consider when seeking reimbursement of their drugs. But companies still have a ways to go in allaying the concerns of investors, regulators and payors alike, and fixing this will require [...]
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The Orphan Drug Act at 30 Years: What's Next?

By Clark Herman | Published: January 8, 2013
30 years ago last week, the US Orphan Drug Act came into being, and with it, a door of possibilities opened up for patients with literally thousands of untreated diseases. The anniversary marks the beginning of a journey where medicines for rare diseases have gained a foothold in the portfolios of drug companies large and [...]
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