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Orphan disease groups

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.”

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Popular Q&A

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What is the difference between an Orphan Disease and a Rare Disease? - Quora

An orphan disease is a regulatory definition of a rare disease so it gets different regulatory treatment

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