Orphan Drugs: a lifeline for rare diseases

The World Orphan Drug Congress slated for May 23 to 25 in Washington DC, brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors, and solution providers to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies. 

This year’s focus is on promoting collaboration to help rare disease patients. We recently wrote about collaborations and their importance to this community. On a grand scale, collaborative work leads to simple but powerful outcomes like more resources for people that need them. Other outcomes include common goals that result in better, more effective, and on-target work. For example, more people have access to essential and helpful information, more people are connected and exchange information, more good science-based educational and informational tools become available, and critically, the chances that the problems that continually affect patients can be solved quickly and efficiently increases exponentially. With collaborative approaches, problems can be better understood, defined, and ranked in terms of their importance and ability to affect meaningful change. It’s like a symphony orchestra with talented musicians and fine-tuned instruments, all doing their part and playing from the same sheet of music. That’s the only way beautiful music happens!

Join us as we cover and amplify the critical work being done with orphan drugs.  

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