Patient Advocacy in the Rare Disease Community

Worldwide, over 440 million persons are living with rare diseases (1) , including an estimated 30 million Americans (2). On February 29th, the world will raise awareness for this community on Rare Disease Day.

Rare Disease Day is a great opportunity for those of us who work in the rare disease community, including those of us at Mallinckrodt, to not only bring attention to the existence of these rare diseases but also the daily challenges faced by patients, families, caregivers and the community.

As a pharmaceutical company, we not only strive to offer treatments for underserved patients with rare diseases but we also have the ability, and the responsibility, to reach out to the rare disease community and find opportunities to collaborate.

Mallinckrodt has had the honor to partner with advocacy organizations focused on rare diseases such as Neimann-Pick type C, sarcoidosis, nephrotic syndrome, cutaneous T-cell lymphoma, hepatic renal syndrome-type 1, infantile spasms and myositis.  It is through these partnerships that we are able to foster an exchange of information, understand the challenges faced by patients, explore the caregiver role, and ultimately, identify and support initiatives that can improve the lives of patients.

Another role that we have taken on is educating the patients that we serve on the resources that are available. While medications and treatments are an important part of the healthcare journey, they are by far, not the only thing that our patients need to live their lives to the fullest. We believe that by connecting patients and caregivers to organizations that can offer support groups, education, advocacy and funding for research, we can bring a more holistic approach to care.

We are just one member of this amazing rare disease community and we know that there is still a lot of work to do to address the gaps and challenges faced daily by those with rare disease. So on this Rare Disease Day, we honor the patients who face their disease with dignity, strength and hope, the families and caregivers who work tirelessly to support their loved ones, the health care providers who share their expertise and never stop working to find the right diagnosis and the best treatments, and to the communities including the regulatory and legislative champions who continue to fight for pathways that improve care.  You all inspire us, and we will remain committed, on this day and every day, to continue the work.

For more information please visit

  1. Wakap, Stéphanie Nguengang, et al. “Estimating Cumulative Point Prevalence of Rare Diseases: Analysis of the Orphanet Database.” Nature News, Nature Publishing Group, 16 Sept. 2019,
  1. “Rare Disease Day at NIH 2020.” National Center for Advancing Translational Sciences, U.S. Department of Health and Human Services, 21 Feb. 2020,