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Last year, smaller companies led in new drug approvals, according to a recent OutsourcingPharma.com article featuring Ken Getz, director of sponsored research at Tufts University. Notably, nearly half of these new drugs were for the treatment of rare diseases, demonstrating a shift within the industry away from a focus on scaling infrastructure.*

CEO and Founding Principal of BBK Worldwide, Joan F. Bachenheimer, responds to the article, explaining that “the precision aspects of clinical trial discovery have remapped the patient experience. The more targeted the investigational therapy, the more individualized the orientation becomes toward patient recruitment and engagement. It’s logical that the search for patients with specific biomarkers begets an orientation centered on individual needs versus a mass orientation."

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.