Editor’s note: This story is part of our 2022 PharmaVoice 100 feature.
Our honoree: Nina Wachsman
Title: CEO, co-founder, Know Rare
The company’s focus: Know Rare, a technology-based rare disease company that connects patients with research and experts, educational resources and insights to help others along their journey, is a family affair. The company was started by Wachsman and her son Jake upon learning that their grandson and nephew was diagnosed with an ultra-rare disease, which opened their eyes to the gap between rare disease patients trying to access research and clinical trial sponsors trying to recruit patients.
Together, mother and son set out to build the Know Rare technology platform, but recognized it also needed a human touch “to help these reluctant, frightened, hard-to-find patients and caregivers who are struggling to understand their disease and the 'unknowns' of participating in a rare disease trial,” one of her nominators says.
Wachsman’s journey: When confronted with the life-altering news about her grandson, Wachsman and her family were frustrated by the lack of information, resources and education that they desperately needed to start to answer some of the biggest questions they had ever encountered. With passion, tenacity and an understanding that technology could be a great enabler, Wachsman leaned into her more than 30 years of marketing experience and a vast network to build out Know Rare.
As one of her nominators says Wachsman is a passionate and tireless provider of a “too-often-difficult-to-find critical pathway for those patients suffering with rare diseases and who need direction and support for their families. A thought leader in the rare diseases' community, [Wachsman] initiates and collaborates with key organizations that can help support and guide rare disease patients and caregivers through unchartered territory.
Her impact: By imagining the possibilities, and then making it happen at a pace that works for all, Wachsman is having a positive impact on the rare disease community — “one rare disease patient enrolled at a time.”
“[Wachsman’s] tenacity, creativity, strategic agility in combination with her breadth of experience result in patients participating in clinical trials that offer hope and insights in a range of rare diseases,” a nominator says.
Why she is inspiring: Wachsman’s compassion is her calling card and has led her to help families around the world find the resources they need during some of their darkest hours.
“I am driven to cultivate collaborations with other rare disease groups or researchers.”
CEO, co-founder, Know Rare
While struggling with the grief of learning her son had a severe form of a rare disease that would be life limiting, “[Wachsman] inspired me to rethink my career as a nurse practitioner to support my new found passion for supporting other mothers of rare disease children and progressing the science,” a nominator says. “With Know Rare, she is addressing the issue of connecting rare disease patients to clinical trials — allowing doctors to focus on what they do best and patients to find hope and possible treatments. She is helping progress science, and uplifting the communities of rare disease patients and clinicians. Personally, she has inspired me to shift my career in a meaningful way. She truly cares about the population her company serves and the people she employs. As a nurse, a mother, and someone impacted by a rare disease, I am so grateful to Wachsman.”
Wachsman’s leadership style is rooted in humanity and bolstered by deep strategic and scientific know-how.
“She understands how family and life need to weave into our workday. This leadership style brings out the best in the team...encouraging cooperation, compassion, and a persistence of our vision to support rare patients and families get the information they need to take the 'unknowns' away, and provide people with the ability to live a little more comfortably with their disease,” a nominator says.
In her own words: “Rare disease, in general, is ‘terra incognita’ for the most simple things like diagnostics, causes, epidemiology and treatments.”