In the rare disease space, the patient advocate or foundation leader is increasingly looking for solutions to their own concerns on how to make new treatments a reality for loved ones. But carving new pathways to cures requires strategic mechanisms to collaboration in line with the new social values-driven 3.0 era. Yet research has long ignored the role of servant leadership in delivering the collaborative mechanisms needed for the delivery of cures and treatments to the rare disease stakeholder.
Rare diseases pose particular challenges to patients who are affected, to the clinicians who care for them, and to the investigators who study their conditions. Although individually uncommon, rare diseases are common in the aggregate, with approximately 7,000 described rare diseases affecting 25 to 30 million US adults. Challenges posed to affected individuals and their families largely regard being diagnosed, receiving optimal care, and affording disease-specific medications. Challenges facing clinicians who care for affected individuals include gaining knowledge and experience in caring for such patients, and the availability of local experts and of expert guidelines. Finally, challenges to investigators regard the difficulty and expense of assembling large cohorts of affected individuals for study, and garnering funding for research (Stoller, 2018). Fortunately, in the face of these challenges, the steadfast resolve of patient advocates and foundation leaders to enhance care and generate new knowledge has fostered a large rare disease community space to offset these challenges. Although further progress is surely needed, successes to date include the formation of powerful patient advocacy groups which have brokered collaborations between the patient, scientific communities, the government, and pharma/device communities in service of detection, optimal care, and research. However, we still face the unsettling reality of having only 5% of rare diseases with FDA approved treatments.
When I started in the rare disease space, I felt overwhelmed by the mountainous information and the labyrinth of drug development byways and highways. I felt like I was in high school cramming for a major test; only the results were of far greater consequence than a mere passing grade. In my quest for knowledge and understanding, I came across Chasing Miracles: The Crowley Family Journey of Strength, Hope, and Joy. The story of John Crowley, a father who builds a pharmaceutical company to develop cures for children’s rare disease, was both inspiring and tantalizingly heroic. In 2016, at a conference, I was elated to run into John and share with him my own efforts to bring a treatment for Jansen’s disease - a debilitating skeletal disease I had passed down to my two boys. As John bent down to listen to the little lady in the wheelchair, I wondered about the many similar narratives he must have encountered as a leader in the rare disease space. Narratives bound by grueling pain and anguish, but also of swashbuckling bravery. It dawned on me that we were all on a similar quest, some more ahead on the road, and some just starting. But all of us with a common destination.
Earlier this year, I was invited to be a part of Amicus’s Healing Beyond Disease, a global, Amicus employee-driven initiative inspired by people living with rare diseases and their families. Healing Beyond Disease aims to enhance and expand the company’s support for patients, families and advocacy organizations through innovative volunteerism, philanthropy, and mentorship. Being able to not just share my story, but to also get input from industry leaders on the best pathway forward was vital. Vital because knowledge is power in a space that thirsts information and understanding. Rare disease patients, caregivers and leaders do not need to reinvent the wheel, but rather learn quickly from others gone before us. Not only did I come away from my visit feeling empowered by the experience, but I felt a strong sense of community – a feeling that I wasn’t alone.
“The most important roles for a leader are to set a vision and inspire others,” John says. “By articulating a clear vision, hiring the best people possible, and encouraging them to do everything they can to achieve a common mission, the inspiration part falls into place.” I think what strikes me most about these words and what sets people like John apart is the belief in the power of a common mission. The central desire to help others was woven into the fabric of everyone I had the good fortune to meet.
While leadership is deemed a central aspect in spurring treatments and novel pathways, the leader whose mission and values tend toward a greater involvement and co-responsibility with broader society takes on the position of a servant leader. This unique leadership approach which, as captured by its name, focuses on serving others (Liden et al., 2014), including the most vulnerable in society (Greenleaf, 1977). And who fits the bill more than the rare disease patient. When there is a true concern that extends beyond an organization itself to meet the well-being of patient, other stakeholders, and society in a wider sense (Greenleaf, 1977; Barbuto and Wheeler, 2006), great change can be made.
The rare disease space is a complex adaptive system. Power is the main currency in a traditional system, whereas influence is the main currency in a complex adaptive system. Therefore, leadership models must adapt accordingly in the rare disease space and in drug development arenas, so that power is shared and change is embraced. A true collaborative pathway to cures can only happen when servant leadership along with transformational power merge.
Although servant leadership has gained momentum over the years, few studies have been done to explore the concept of servant teachers in the rare disease space. Leaders who incorporate the principles of servant leadership into the practice of drug development have a powerful gift that inspires patient advocates and foundation leaders to achieve and do great things with the support of the servant teacher. Servant teaching can be conveyed in the community setting by creating an environment where patients feel comfortable, appreciated, and supported in their learning.
The principles of servant leadership can guide visionary leaders to consider all facets: service to the community, patient care, and relationships built on respect and compassion. The rare disease leader’s supportive direction and vision maintains the unity and common purpose of the rare community. This organizational approach engages the community and promotes thoughtful insight and opportunities for establishing compassionate collaborative partnerships in all settings. It reminds us of the work that together we set out to do: bring cures to those who need it.
Barbuto J. E., Wheeler D. W. (2006). Scale development and construct clarification of servant leadership. Group Organ. Manag. 31 300–326. 10.1177/1059601106287091
Greenleaf R. K. (1977). Servant Leadership: A Journey in to the Nature of Legitimate Power and Greatness. New York, NY: Paulist Press.
Liden R. C., Wayne S. J., Liao C., Meuser J. D. (2014). Servant leadership and serving culture: influence on individual and unit performance. Acad. Manag. J. 57 1434–1452. 10.5465/amj.2013.0034
Stoller, J. (2018). The Challenge of Rare Diseases. Chest, 153(6), 1309-1314.