I look forward to the NORD Rare Summit each year. It is a time to reflect on the work all of us do in the rare disease community−whatever area we work in, be it drug development, clinical research, regulatory compliance or patient advocacy. It’s a wonderful opportunity to collaborate as a community, truly learn from one another, and most importantly return to our work with a renewed focus and passion for advancing treatments for rare disease patients, knowing that the patient is central to all our work.
If you haven’t noticed UBC in the news lately, you may not be looking hard enough. Our experts have been busy talking to media in the industry, sharing insights you don’t want to miss. Here are articles published this month with contributions from four of UBC’s thought-provoking experts.
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.
Disease natural history data is critical to rare disease medical product development where information may be severely limited. In fact, this type of data is often required or encouraged by regulators as it can help with interpretation of both clinical trials and real-world experience post-approval.
On February 28, rare-disease communities around the world come together to advocate for an increase in research focused on rare diseases. When it comes to clinical research for rare and orphan diseases, the relationship with the patient and advocacy groups is critical to success.
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 UBC examined the FDA’s draft guidance related to the development of these drugs and presented the results in a poster at ISPOR.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.
Rare-disease patients need special attention, not only upon therapy initiation, but throughout their entire therapy journey. UBC has the capabilities to start at the very beginning of this journey — transitioning patients from a trial drug to a commercialized product — and still provide high-touch care — coordinating pharmacy triage and beyond.
Today, rare-disease communities around the world are coming together to raise awareness for the roughly 7,000 different types of rare diseases and disorders. Shazia Ahmad, our Patient and Physician services rare-disease expert, will be attending Rare Disease Day at NIH to celebrate this year’s theme, “Patient Voice.”
For rare-disease patients, a successful therapeutic journey is as unique as they are. Here is a look at some of the most pressing questions rare-disease patients face during their therapeutic journey.