Gaming out the potential high stakes of orphan drug development
Imagine you are a board member of a drug company developing an orphan drug for patients with life-threatening, rare disorders. Overall, the international orphan drug market is expected to grow nearly 30% through 2014, fueled by the entrance of big pharmaceutical companies and federal legislation favoring orphan drug development and coverage. How should your product be developed and priced? What conflicts of interest exist? How should patients be assured access to your very expensive but potentially life-saving product?
At the Biotechnology Industry Organization’s 2014 convention in San Diego June 23-26, six panelists will serve as imaginary board members of that company and simulate the decision process. Tim Mackey, an assistant professor of anesthesiology and a global public policy researcher at the University of California, San Diego, said the session will explore cuttingedge issues in bioethics and drug discoveries. “A lot of perspectives will be at play, and it will be interesting to see if these disparate actors on this board can come to a resolution,” he said.
Attendees will also hear how public policy is affecting pharmaceuticals. Caroline Pearson, vice president at Avalere Health, will participate in a convention session focusing on access to orphan drugs in the Obamacare exchange plans. With higher out-of-pocket drug costs for many exchange customers, their ability to afford such expensive specialty drugs is in doubt. “Cost burden is one of the biggest issues we are worried about,” Pearson said.
Former Secretary of State Hillary Clinton will give the keynote address June 25.