Each year since 2009, the National Institutes of Health (NIH) Clinical Center and National Center for Advancing Translational Sciences (NCATS) has hosted Rare Disease Day at NIH to participate in the global observance. This year, we attended the event and heard several inspiring presentations from a range of rare disease stakeholders.
Members from both UBC and Accredo recently attended NORD’s Rare Diseases and Orphan Products Breakthrough Summit in Arlington, VA. This was a great opportunity for leaders from both organizations to collaborate with key industry stakeholders to discuss the progress as well as challenges in rare disease diagnosis, clinical trials, patient engagement and access to orphan products.
Moving pharmaceutical products from development into commercialization is no small feat. Doing so for a rare disease and improving speed to therapy is even more challenging — but, because of UBC’s unique solutions and strategic partnerships, it’s a challenge that we can take on.
Pharmaceutical and biotech sponsors still face hurdles when seeking FDA approval of new medications for rare diseases. UBC examined the FDA’s draft guidance related to the development of these drugs and presented the results in a poster at ISPOR.
Shazia Ahmad, Director, Patient and Physician Services, participated in Rare Disease Day 2016 while attending the National Institutes of Health (NIH) earlier this month. In our latest blog post, she reviews three key insights from the event.
On February 28, patient communities around the world will celebrate Rare Disease Day to raise awareness about rare diseases and their impact on patients’ lives.
Rare diseases are a hot topic in healthcare today. Google “rare disease” and you’ll find hundreds of news stories about the research efforts of many companies and advocacy groups. Interestingly, 40% of novel drug approvals by the FDA in 2014 were indicated for rare conditions.
Pediatricians are known for saying that children aren’t just small adults; and their healthcare needs shouldn’t be treated that way. Giving children less of the same medicine or ordering a smaller MRI just doesn’t work.