Nearly a year ago, we released our first guide on rare disease clinical research. In it, we were looking to address some important questions facing the people who are committed to improving the lives of patients with rare disorders. While much progress has been made, it is critical that those of us in the health care and life sciences industry remain keenly aware of all that’s being done to further research and improve outcomes. In our latest eBook, “Focus 5: Rare Disease Research. What’s Next?” we reached out to industry experts for their insights on this evolving clinical research landscape.
For this week’s Guest Blog Spotlight and in recognition of Clinical Trials Awareness Week, we’re sharing insights on improving patient engagement from Rare Genomics Institute and Rareshare.org, and contributors to our recent Rare Disease eBook. We asked Rare Genomics CEO Paul Schindler, Director of Strategic Alliances Nolin Huddleston, and Rareshare.org’s Head of Operations and Development Alice Cheng, “How can sponsors take a more active role in building and supporting rare disease communities?”
“We know today that we can define the root cause of genetic diseases. We have a growing toolkit with which to we can aggressively tackle and modify the underlying defect, which ultimately I believe will lead to benefits for the patients, improving their quality of life, and potentially for some, it may lead to cures.”
“When so many rare disease patients commit themselves to becoming experts of their condition, sponsors must also strive to understand and collaborate with the patients they wish to engage.”
Our “Focus 5” eBook series, in which we share the insights of five thought leaders on a variety of industry topics and trends, has been incredibly well-received, and we’re pleased to issue the latest installment in the series on Rare Disease Research. Patient advocates, researchers and physicians from NORD, Xenon Pharmaceuticals, Landon Pediatric Foundation, Rare Genomics Institute and RareShare.org all weigh in on where we are today and what we can do to help advance research.
A few weeks ago, we attended NORD’s 7th Annual Rare Disease Day in Boston. People impacted by rare diseases, including patients and patient advocates, industry thought leaders and legislators, gathered at the State House to help raise awareness of the impact rare diseases have on the lives of millions of people in the US and around the world.
Late last month, BBK hosted the webinar Rare Disease 20/20: A Look at the Challenges and Opportunities in Rare Disease Clinical R&D giving our listeners the opportunity to gain insights from thought leaders in rare disease patient advocacy and orphan drug development.
Part I of our blog series on adaptive patient recruitment focused on the importance of early support. Part II looked at how to best analyze investigator resources to optimize patient recruitment efforts, including cultivating integrated relationships. Today we wrap up our rare disease series on recruitment with two final tactics that can yield strong results.
Topics: Rare Disease
As we discussed in part I of our blog series on adaptive patient recruitment, the rare disease community is constantly striving to invest in developing new treatments. While there is a huge unmet need in this area, there are recruitment tactics that effectively provide support. Beyond the introduction of an early support program, which Jaime Cohen discussed in our previous post, there are additional strategies that can assist in higher recruitment numbers for rare disease. In part II of our series on rare disease we explore further.
There is a huge unmet need in the rare disease community to invest in developing treatments. According to the US advocacy group Global Genes, there are more than 7,000 distinct rare diseases affecting 350,000,000 people worldwide and only 400 approved therapies. How can we adapt traditional recruitment strategies to target the right patients? The goal is to create tailored adaptive strategies aimed at extending reach and building relationships. There are many ways in which this can be done. In this first post of a series focused on adaptive recruitment for rare disease, I will explore the introduction of an early support program.