Source: PricewaterhouseCoopers LLP
Consumers taking charge: Kids V Cancer
The drug development process can stretch for over a decade, though many important decisions occur before any clinical development or company investments occur. Here at the start is when consumers are exerting greater influence.
“The question isn’t how to get a drug approved. The question is how you get a private company to make the drug in the first place,” says Nancy Goodman, founder and executive director of Kids V Cancer, a patient organization that promotes pediatric cancer research.
The group was instrumental in advocating for the 2012 Creating Hope Act, which encourages manufacturers to develop new drugs for children with rare diseases. Under the law, a company that develops a drug for a pediatric rare disease receives a voucher when the drug is approved by the FDA. The voucher can be used to obtain priority review by the FDA for any new drug application, which can shave months off of a product’s review time. The voucher can be sold or transferred from one holder to another without limitation.
In July 2014, the first priority review voucher was sold by BioMarin Pharmaceutical Inc. for $67.5 million. The company received the voucher for a drug it developed to treat Morquio A Syndrome, a rare inherited disease that occurs in 1 out of every 200,000 births.60 BioMarin sold the voucher to Sanofi and Regeneron, which used it to obtain priority review from the FDA for a new cardiovascular drug—a market which is fiercely competitive. BioMarin CEO Jean-Jacques Bienaimé said the company will use the proceeds from the sale to reinvest in developing products to treat rare diseases.
According to Goodman, the program is correcting market failures that keep pharmaceutical companies from developing certain drugs. “Large pharmaceutical companies have a responsibility to their shareholders,” she told HRI. “Part of their mission is to maximize financial wealth. There’s nothing wrong with that. It’s the system we’ve created. But the way you maximize profits is by having the biggest hit. That’s never going to be [a drug for] kids with cancer the same way it’s with a drug like Lipitor.”
It may be too soon to tell how successful the program will be in generating more research, treatments and cures. But the outlook appears good. A second voucher was sold to Gilead Sciences for $125 million by Knight Therapeutics in November 2014.62 And some in Congress are looking to expand the use of vouchers for other diseases and conditions. Lawmakers recently added Ebola to the list of tropical diseases that could qualify a manufacturer for a priority review voucher if they develop a vaccine or treatment.