When rare disease became a part of their family’s life, mother and son, Nina and Jake Wachsman, realised there was a disconnect between patients and researchers recruiting for clinical studies. They decided to combine their professional experiences along with their personal drive to improve the lives of those with a rare disease, and founded Know Rare

RARE entrepreneur series: meeting the beating hearts behind the RARE brands

Know Rare’s mission is to empower more people to know more about rare disease. We want to share whatever we learn about the new research and researchers pioneering new treatment approaches to create a better future. We will use whatever resources we have to help facilitate the connection of people with rare diseases to research and researchers.

1.

What was the driving force in starting your own business in the rare disease space? Was there an unmet need you were responding to?

We happened into the rare disease space when two things happened: first, a former colleague had just become the head of the new products division at a company specialising in rare disease, and he asked me to help with the recruitment of three of their rare disease studies, in which they were struggling to find patients. Then, my grandson (Jake’s nephew) was born with an ultra rare disease, and suddenly my daughter was finding other friends and friends of friends who had children born with a rare disease. They reached out to her, because they knew of my background in pharma, and wanted me to help them find clinical studies for their children, since there was no treatment available. They were very motivated to join a study, often because they wanted to get under the care of the specialists they knew were conducting the study, or simply because they were not satisfied with the status quo and wanted to feel they were leaving no stone unturned in the care of their children. I tried to help them navigate clinicaltrials.gov, to explain the phases of studies, and what the endpoints meant, but despite emailing several of the contacts left for the studies, none of them were able to connect with a researcher. That’s when I realised there was a terrible gap: researchers wanted to enrol patients but couldn’t find them, while patients wanted to join studies but couldn’t figure out how. So Jake and I decided to put together what we knew about clinical studies with who we knew was looking for them: and we started Know Rare.

2.

How does your business benefit the rare disease community?

We are not a pharma company, but members of the community, and we want to help
people with rare diseases who are looking for other options for themselves or their children find them. We try our best to find studies or resources for anyone who contacts us, whether we have a sponsor for a study or not. When we have a sponsored study it’s easier, because we have the contact information for the researcher and the study site, and the sponsor has encouraged the site to accept outside patients from Know Rare. This allows access to research and the leading thought leaders in the disease state to others outside of that hospital or organisation, allowing for more equitable recruitment.

3.

What advice, if any, did you get when setting up your business? Has there been anyone in particular who has been pivotal in supporting your business?

We were lucky to have a lot of former clients and colleagues who have provided guidance and insight to us right from the start and became our founding board members or joined our advisory board. We have gained from a wide range of experience and expertise, from clinicians to finance to clinical operations. We realised you can’t build an effective business in clinical trials unless you understand the patient, the sponsor, and the research teams points of view, and luckily, we got them. We also benefited from a collaboration with the Rare Advocacy Movement (RAM) who helped shape many aspects of our programme so that it would be sensitised and accessible to the diverse communities they represent. It has always been our goal to enable more diverse representation in clinical trials.

4.

How do you manage the demands of running a business with your own health needs, those of someone you care for, or those of your employees?

That is really tough, especially with those in my age group, who have elderly parents with dementia, who are always one step away from a crisis. Since I am partners with my son, he helps cover for me when I’m out due to their health needs. Our company is also built on being sensitised to special needs, and many of our staff have chronic conditions, or children with chronic conditions who need the flexibility of work hours and the understanding for when infusions or loss of a caregiver happen.

5.

What advice do you have for someone starting their own business?

Don’t be afraid, and don’t worry about every little thing. Take one step at a time, and realise that when something happens, you’ll just find a way to deal with it. On an operational side, make sure you document processes and standard operating procedures (SOPs), so if a key employee leaves or you need to scale up and add more employees, you can train them and they have a road map to follow so everything will continue to run smoothly.

6.

What are the most rewarding aspects of establishing and running your own business?

For years I worked for a global agency where I brought in two thirds of the business, but I essentially was building a business for someone else. Now, I reap what I sow. I work on things I want to work on, and on my schedule. Those are the major benefits.

7.

What would you consider to be the greatest achievements of your business thus far?

Hearing from moms that we have helped get into a study, who now post how well their child is doing on a new medication. Finding out our support has helped ‘save’ a study that was ready to be scrapped because of a lack of recruitment.

8.

What advice would you give someone considering working in the rare disease space?

Get real. You have to be authentic, and be careful of what you promise. There have been too many disappointments in the rare space from studies that initially gave great hope and then failed. Also be careful how you communicate—be sensitive to the skepticism and the sense of disenfranchisement from people whose have learned their disease is out of the experience of most doctors.

9.

What are your hopes for the future of your business?

That we can scale it up more and provide more tools to benefit the rare community. Next up for us is a journaling app for rare, Healthstories, which helps caregivers note events to
enable a better conversation with their specialists about their patient’s status between
visits.

10.

If you hadn’t founded Know Rare, what was Plan B?  What did your 10-year-old self want to be?

I’d be retired now, dedicating my time to writing my historical mysteries full time. Now I have to write my books and short stories early in the morning and on weekends to make my publisher’s deadlines. Jake, my son, already tried his first dream, as a sports agent for NBA players. I dragged him into the healthcare space, which he likes much better. He feels he is really making a difference in the world with what we’re doing.