Rare Disease Day: the Power of Diverse Voices and Collective Collaboration

Today is Rare Disease Day, an opportunity to recognize that one in 10 people around the world are living with a rare disease — 80 percent of which are genetic. These diseases disproportionality affect children and their families. For those who don’t know, there are about 6,000 rare diseases and fewer than 10 percent have approved therapies.

I am passionate about fostering awareness about rare diseases because of my own experiences as a rare disease parent advocate. This also led me to work on the first campaign for Rare Disease Day in the U.S., for the National Organization for Rare Disorders (NORD). The theme we developed, “Alone we are Rare, Together we are Strong” was all about using our collective energy to change the system and drive innovation by having the voices of the rare disease community included at every level.

While there is still much work to be done, we have seen amazing progress. In the last 20 years, the FDA has approved 599 orphan products to treat rare diseases.

For perspective, prior to the 1983 enactment of the Orphan Drug Act, only 38 orphan products existed.  Today, there are more than 700 medicines in the current biopharmaceutical pipeline for rare diseases.

In addition to treatment, there has been positive movement for other interventions and resources. Companies have started including patients and caregivers as part of their development process which in turn has benefited companies commercially. We are also seeing rare disease and mainstream pharma companies adding Chief Patient Officers or Chief Patient Advocates — many of whom came up through an advocacy path and are now sitting at leadership tables. There’s no doubt, having the patient perspective included as part of the executive decision-making process changes the conversation.

I have also witnessed this change within the HBA. Evolving what leadership looks like and who sits at the table is central to our global United Force for Change community.  By recognizing the rare disease community’s challenges and opportunities, we can learn from them. Last year at the Woman of the Year event, I spoke about my personal journey as a rare disease caregiver as well as being a diversity champion to expand the table to include more voices to drive innovation —together. You can hear the speech here.

According to Korn Ferry, while 80 percent of the healthcare and social assistance workforce are women, fewer than 20 percent of those women are in healthcare leadership roles. At the HBA, we are working tirelessly to address this disparity and bring our knowledge and experience to the forefront to help patients. On multiple levels — individually, collectively and systemically — we are developing inclusive leadership tables through experiential learning, mentoring, networking and education programs, engaging with our corporate partners through our ambassador programs and HOPE programs, and working to enact macro changes through our Gender Parity Collaborative.

On this day today, we recognize the hard work that has been done and acknowledge the hard work that lies ahead in the rare disease arena. We also stand strong as a united network to ensure diverse voices have a seat at every healthcare leadership table.

#showyourstripes #rarediseaseday #raredisease

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Authored by Wendy White, Interim CEO, Healthcare Businesswomen's Association.

Wendy White is a purpose-driven and strategy-minded entrepreneur with a successful track record of innovation and building and growing companies. She is also a sought-after speaker and thought leader in rare disease inspired by the challenge of integrating the patient voice and emerging technologies into care development and delivery to improve patient outcomes.

Wendy currently serves on several boards and advisory boards including as Board Director for Ceek Women's Health—a fast growing, privately held, for-profit company that makes a medical device named by TIME magazine as one of the top inventions in 2020. 

In 2017 she was recognized by PharmaVoice with a Red Jacket lifetime achievement award as one of the most inspiring people in the pharmaceutical industry.