Articles

Medical and Regulatory Affairs

Life Science Leader – Orphan Drug Incentives & Innovations On The Rise

The year 2015 was a productive one for introduction of drugs that target rare diseases. U.S. regulators approved 21 new orphan drugs, a 40 percent increase from the previous year. European regulators approved a record 18 orphan compounds, a small increase over 2014. Any progress is a good thing, but these advances pale when weighed...

Functional Service Provider (FSP)

Scrip – The CRO View: Sharing Opportunity Vs Risk In Drug Development

We hear a lot about risk-sharing in drug development these days, and it’s a healthy trend in an industry often defined by high costs and the pressure of project deadlines. Drug development thrives on cost and schedule adherence, and the inextricable link between the two leads increasingly to risk-reward arrangements between drug makers and their...

Functional Service Provider (FSP)

Drug Development & Discovery – The Opportunity in Sharing Risk: A Productive Trend in Clinical Trials

Patient enrollment may be the single most important factor in ensuring the timely execution of clinical drug research. So when we negotiated risk-reward incentives for a trial evaluating a treatment for major depressive disorder, signing up participants accounted for most of the performance incentive structure: a significant double digit percent bonus (or penalty) tied to...

PharmaLive – Incentives, Opportunities Abound as Orphan Drug Demand Grows

If ever a drug development effort called out for priority review and approval, this was it. A U.S. pharmaceutical company was developing a treatment for hypophosphatasia, a progressive, ultra-rare metabolic disease for which only supportive therapy was available. Asfotase alfa, an innovative enzyme replacement drug, was the first real hope for those diagnosed with HPP,...

Medical Director Authors Novel Look at Kleefstra Syndrome

DURHAM, N.C., April 1, 2016 — A paper authored by a Premier Research medical director takes an unprecedented look at motor function and behavior in adults and children with Kleefstra syndrome. Susanne Schmidt, M.D., Ph.D., and four Norwegian colleagues published their findings in the European Journal of Medical Genetics. The study of four male and...