Biotech, Pharma, & VCs Offer Rare Disease Patient Groups Some Advice

Today’s issue examines the surge of interest in rare disease drugs, which in the past few years have attracted significant interest from biotech firms, big pharma, and venture capitalists alike. In addition to exploring the business and policy drivers behind increased investment in orphan drugs, the multi-part story looks at the critical role patient organizations play in drawing attention to rare diseases. As such, it seemed worth highlighting advice from various stakeholders on what patient groups can do to entice drug developers to work on their disease:

Organize yourselves. Find as many patients as possible, and establish a registry that will make it easy for a drug firm to begin a clinical trial. “Beginning to identify people, getting them into a registry, and collecting natural history data is one of the most valuable things a developer can have when they’re thinking about a program,” says Genzyme’s CEO David Meeker. “Among the most helful things that patient advocates can do is to help us to understand the natural history of disease,” agrees Kevin Lee, CSO of Pfizer’s rare disease unit. “Without that understanding of how the disease progresses, and what the endpoints can be, its almost impossible to do drug development.”

–Find a way to collaborate with one another. In even the smallest of diseases, patient groups tend to proliferate. And while its natural and understandable for advocates to want to do all they can to help their own child or family member, it can lead to duplicative efforts. The disparate groups can also make it tougher for drug developers to access. “We all need to give everybody a lot of space here to do what they think is best, but in an optimal world, there are tremendous advantages to being coordinated,” Meeker says.

Be connectors. Patient organizations have the amazing ability to bring together academics who had previous not collaborated. “What I have found over and over again is that patient advocates know the investigators in their field far better than the investigators themselves do,” says Christopher Austin, director of NIH’s National Center for Advancing Translational Science (NCATS). “They can be instrumental there.”

Get the right researchers interested. Often only a handful of academic researchers are working on a given rare disease, and drug developers say attracting new scientists into the field, while also giving careful consideration about who to fund is key. Patient groups should look for someone who can use advocacy funds to attract larger grants. “If they can get some grant support, you’ll get more done,” says Emil Kakkis, CEO of Ultragenyx. “If they can’t get any grant support, you’ll have to wonder if it was just because the disease is rare, or another reason.”

Don’t cut corners. As more patient groups directly fund and organize natural history studies and early clinical trials, they need to make sure the work they support is of the same caliber as that done by biotechs or pharma. “Every data point they generate may some day be helpful in getting a drug approved,” says Philip Reilly, venture partner at Third Rock Ventures.

Take the reins. With the passage of FDASIA last year, FDA committed to allowing patients more of a seat at the table during regulatory discussions. But the role patient groups will play—how they will be allowed to particulate and how much influence they have—is still to be determined. Ritu Baral, analyst at Canaccord Genuity, thinks there’s opportunity in that vagueness. “Give an inch, take a mile. If they’re going to define it, then we can define it as a patient group,” Baral, who also sits on the board of a disease foundation, says. “We can set the markers where we want to set them.”

Help drug developers understand your needs. Drug companies are partnering with patient organizations earlier on in the drug process than in the past, convening patient advisory boards to understand how best to design a clinical trial, says Amy Waterhouse, vice president of regulatory affairs at Biomarin. That design ins’t just about regulatory practicalities, but about what families need out of the design in order to participate—a three day visit to a hospital instead of four, for example, can make all the difference. “We learn so much from discussions [with patient groups] that we wouldn’t get from the literature,” Waterhouse says.





Author: Lisa Jarvis

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