Why I view advocacy as essential to building a rare disease company

Why I view advocacy as essential to building a rare disease company

When I joined Saniona as President and Chief Executive Officer last year, I had several urgent priorities. I needed to advance our clinical programs, raise money, establish our new corporate office in the U.S., and build an executive team. Amid all of this, one of my highest priorities was to ensure that Saniona is incorporating the voices of people with rare diseases and their caregivers into everything that we do, globally and equitably. Today, on Rare Disease Day, I wanted to share why this was such a critical area of focus for me from the very start.

I chose to put a strong focus on patient advocacy and engagement early in Saniona’s growth transformation because there is a special bond between a rare disease community and a company that is discovering and developing rare disease therapies. Medicines cannot be developed without the courageous patients and their families who enroll in clinical trials. Patients are putting their trust in our science and innovation so, it is up to us to earn that trust each and every day. Earning that trust begins with listening to and learning from patients and their families, and then incorporating those insights into our programs, studies, and culture. In fact, to ensure that we incorporate patient experiences into everything we do, we recently developed guiding principles on engaging patient advocacy groups that are aligned with our core values of putting people first, innovation with impact, and integrity always. We worked with patient advocacy organizations to develop these principles, which you can read more about here.   

Today, I’m proud to say that we have begun living up to these principles through collaborating with a number of patient advocacy organizations in areas that are meaningful to them. These include participating in a clinical trials educational webinar hosted by the Prader-Willi Syndrome Association USA; learning about living with hyperphagia in hypothalamic obesity directly from survivors through a meet & greet hosted by the Raymond A. Wood Foundation; and sharing insights learned from advocacy groups like the International Prader-Willi Syndrome Organisation (IPWSO) and the Foundation for Prader-Willi Research (FPWR) with not just our clinical trial team, but all employees at Saniona. We also include patient advocacy insights as a session for all new employees at Saniona so everyone can appreciate the incredible responsibility we have to the families that we serve. 

In the greater rare disease community, we are collaborating with organizations like Global Genes and NORD on raising awareness and creating educational materials to support the rare disease communities.

This is of particular importance for people with rare diseases, because while there are over 7,000 rare diseases, fewer than 10% have FDA-approved treatments.

And of course, our involvement with the rare disease community won’t stop after we bring a therapy to market. Saniona will be committed to working with advocacy groups to assist patients with getting access to our therapies along with resources and tools that will help address the multiple needs that come with living with a rare disease. 

Every year, on the last day of February, I’m always encouraged to see how patient advocacy organizations and the rare disease community rally to raise awareness of the struggles of living with these conditions and the need for treatment options. While we’ve seen progress over the years, by continuing to work together, we can go further in improving the lives of the millions of people around the world living with a rare disease. 

Last month marked my one-year anniversary at Saniona. When I look back at how many important insights the advocacy community has shared with us, and how these insights have helped to inform our business strategy and decisions, I cannot imagine starting and running a business without this focus. And for that, I am incredibly grateful to you, the rare disease community.

John Walter

CEO | Strategic Thinker | Venture Philanthropist | Helping Nonprofits Expand, Thrive and Change Patient's Lives

3y

Thank you for your commitment to our space!!!

Harella A Halevy

Healthcare communications expert | Bringing health to life

3y

So interesting and inspiring - turning goals into practice and company aspirations into patients’ hopes is admirable. Saniona is lucky to have you as CEO

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