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 amplifies the collective voice.
Success in Rare Disease: Patient Involvement Matters!
This past December, the FDA finalized its guidance on rare diseases with the publication Rare Diseases: Considerations for the Development of Drugs and Biological Products. This FDA guidance has been in draft form since 2019.
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 of a new drug can feel similar. But the metaphor only goes so far — launch preparation is not simply about speed, and in this case, the “horses” keep changing, and the track is anything but predictable.
A better metaphor is that of an orchestra, with many instruments contributing distinctly at specific times. All play the same piece of music but contribute based on their respective roles.
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 this topic is very much a part of my focus and professional experience.
“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, this insight has stuck with me since Justice Sotomayor said it during her confirmation process in 2009.
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. In recent years, much has changed. As drug development has become more enlightened, understanding the experience of people living with health conditions — their needs, goals, and the practicalities of daily life — has become integral to the process of developing innovative medicines.
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 and challenging in all scenarios… the difference is in how significant the focus and effort will need to be.
Rare Diseases — It’s All About the Patient
Among all the days of the calendar, February 29th is the rarest of them all. That's why, back in 2008, on the 25th anniversary of the Orphan Drug ACT, EURORDIS (The European Organization for Rare Disorders) designated the last day of February as "Rare Disease Day."