Here's a new phrase you will want to add to your vocabulary: Patient Opinion Leaders, or POLs. And here is what it means: You can either involve patients in drug development, or you can watch as they work around your company to get to their goals.
This lesson rises up from the orphan drug space where famously the Cystic Fibrosis Foundation has demonstrated the power of patients in funding through venture philanthropy, and more recently, patient power is credited with a reversal in an FDA decision on eteplirsen injection, which it approved as the first drug approved to treat patients with Duchenne muscular dystrophy.
POLs are real, said Wendy White, the chair at Global Genes, a leading rare disease patient advocacy organization.
Wendy White, Chair at Global Genes"They are out there and they are talking to each other thanks to the access the internet has given them," she said during a panel session at BIO-Europe Spring® in Barcelona.
Eighty percent of rare diseases are genetic, and 50 percent affect children, she explained, "so when we talk about active patients, we are really talking about parents. And what will they not do for their child? They will do anything, giving time, talent and treasure. Before people might have gone to the library to learn about a disease," she said. "Today they do it faster. With access to the internet and access to other people through social media they connect, and these are the people they are going to trust."
Where these home-grown activists may once have used the public library for information, she said what is happening in the rare disease space today is that they find each other and organize. And then they begin to do nontraditional, unexpected things.
"If a scientific conference does not exist, they will start their own. They may ask each other if they should start their own biotech company. They all see the Cystic Fibrosis model that is out there," she said.
In September, 2017 Global Genes will host the first partnering event focused exclusively on rare diseases in the framework of its increasingly successful Patient Summit held in Southern California.
According to Michael Pistone at the Cincinnati Children's Hospital, "When we speak about partnerships today, it is not just us and an industry partner, it is just as often also with a foundation or patient advocacy group.
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Michael Pistone, Cincinnati Children's Hospital
"There is still a disconnect here that we are working to bridge with these incredibly strong patient advocacy groups. It is not so much about funding and philanthropy but about outcomes and bringing the patients in from the front lines. This becomes really powerful. For us more and more partnership is a three-legged stool," he said.
Offering a different angle on the emerging power of patients was Timothy Coté who leads what he calls a boutique full-service regulatory group with a laser focus on orphan drugs.
"Bring the patients into the regulatory process," he suggested. "Patients are the real, true experts in endpoints, which is the hardest part of developing a drug. Yet we do not involve them in the process for the most part. We need to bring them in because without the patients, our work becomes largely irrelevant."
Up until a few years ago, Coté led what he called the orphanage at the FDA, signing off on 1,400 orphan drugs designation applications and seeing 150 products go to full marketing approval.
"Orphan drugs are wicked hot. Everyone wants to be an orphan," he said. "Rare diseases are actually making their way into the US President's speeches now. Wall Street loves the Orphan Drug Designation. Even if you do not receive the designation, you receive a long letter from the FDA that tells you the agency's thinking. So you win if you get it, and you win if you do not.
"This is what is working for us. This kind of power and energy makes it the most exciting thing in our industry," said Coté.
Big pharma arrived very tardy to this party, very slow, he said. "The real rocket fuel in the industry are the micro companies that today can start with mothers who don't want their kids to die, a scientist who has a really cool idea about how to prevent these children from dying, and then some guy with a bag of money who sees a business in this. They all have some skin in the game."
Currently there are 600 approved orphan drugs in the US but with overlap only 200 of the 7,000 identified rare diseases are being treated.
"Do the math and there are 6,800 diseases that have nothing, which is terrible," he said. "We now have the technology to really change that. To make a dent in that number by treating at least a 1,000 of these by 2050. I think that's reachable and we should aim for that."
According to White at Global Genes, "You cannot count on having an organized patient advocacy group to walk into the FDA with you. Eighty-five percent of rare diseases do not yet have a patient advocacy group. You need to think about how you are going to make that work. The more you can involve patients at the beginning, figure out their role and treat them like a real partner, the more successful you will be, and the more efficient you will be."