Rare Disease Day … The Power of Many Voices
Rare Disease Day unites this wide range of conditions under one shared umbrella, fostering a global community that…
Success in Rare Disease: Patient Involvement Matters!
This past December, the FDA finalized its guidance on rare diseases with the publication Rare Diseases: Considerations…
Medical Affairs: An Orchestra, Not a Horse Race
Every horse race has a starting bell, a push to move as fast as possible, and a finish line. In many ways, the launch…
Rare Diseases: Shaping Biopharma’s Future from the Past
We just completed Rare Disease month. With that in mind, Sue has asked me to step in for today’s e-conversation, as…
“Rare Disease” Is More Than Just a Day
You might think it odd for me to begin this month’s e-conversation with a quote from a Supreme Court justice. However,…
Patient Advocacy – Delivering on Patient Centricity in Drug Development
Historically, biopharma companies developed drugs for patients with an illness — but the illness was the primary focus.…
Gaining Participation in Clinical Trials
When it comes to patient recruitment and retention for clinical trials, the bottom line is simply this: It is important…
Rare Disease Patient Advocacy and Medical Affairs: Why They Come First
Among all the days of the calendar, February 29th is the rarest of them all. That's why, back in 2008, on the 25th…