![]() Rare disease families are faced with numerous challenges in health literacy. Importance of Improving Health Literacy across Rare Diseases It is through the tremendous work of patient advocates and through collaborations with industry that we can better understand the complexities of a rare disease to effectively contribute and advance new therapeutic developments. It provides comprehensive genomic profiling at multiple points during the study which will both provide critical information to the patient and develop unique data to help understand how cancer changes over time. TRACK is unique in that it is a clinical study fully initiated and managed by an advocacy foundation. with no need to travel or change their treating physician. TRACK incorporated remote consenting so patients can enroll from anywhere in the U.S. Jim Palma, Executive Director of the TargetCancer Foundation, discussed the study called TRACK (Target Rare Cancer Knowledge), a rare cancer precision medicine clinical trial. Eric Sherman, a medical oncologist at Memorial Sloan Kettering Cancer Center, shared “Advocacy groups bring people together where progress otherwise wouldn’t be able to happen.” Together, industry was able to better collaborate with the patient community to design a trial that was more inclusive and addressed the challenges around mobility by reducing visits outside of the home and added the flexibility of blood draws in the home, ultimately increasing patient participation in the trial.Īnother panel discussed advocacy as a driving force in advancing progress in rare disease research −especially in rare cancers. Missling shared an example where leveraging the caregivers in the Rett patient community enabled them to identify gaps in understanding the challenges of patients with Rett syndrome. I had the opportunity to moderate a session on “Industry and Advocacy Collaborations ─ Making it a Win-Win.” Panelists Nadia Bodkin, PharmD, Rare Advocacy Professional and Co-Founder of the Rare Advocacy Movement (RAM), and Christopher Missling, Ph.D., President & CEO of Anavex Life Sciences, Corp., shared the importance of learning the stakeholder’s goals and objectives, as well as encompassing all stakeholders involved in the patient community, including caregivers and care partners. The importance of industry and academia collaboration with patient advocacy groups was apparent throughout Rare Disease Day 2021 and was shared across several sessions. Here are some key messages from the event: Collaboration is Key “The last year has seen a historic…accuracy in the development of therapeutics and vaccines for COVID-19 and the urgency we have seen in the public and private sector is the kind of urgency that we feel in the rare disease community every day.” Christopher Austin, Director of NCATS, commended the rare disease community on their commitment to progressing research for rare disease patients and expounded on the importance of rare disease treatment. COVID-19 has taught us that we are all still connected regardless of where we live.ĭr. Most importantly, this day enabled us to reinvigorate our passion to advance treatments for rare disease patients and reminds us that the patient is the central point of our work. Rare Disease Day 2021 was an opportunity to collaborate, learn from one another, and celebrate the work that continues to move forward. ![]() The energy and passion researchers, physicians, regulators, and patient advocates harness to work together was clear. Earlier this month, I participated in the virtual Rare Disease Day 2021 activities hosted by the National Institutes of Health (NIH) Clinical Center and National Center for Advancing Translational Sciences (NCATS) as part of the global observance to focus the world’s attention on this important subject. They also face additional stressors associated with the current global health crisis posed by COVID-19 over the past year. Individuals living with rare diseases have a lot to navigate with regards to the availability of, and access to, life-saving medications. Wednesday, MaUBC’s Senior Director of Patient & Physician Services Shazia Ahmad attended the virtual Rare Disease Day 2021 on March 1 Collaboration, Health Literacy, & Technology: A Patient Advocate’s Perspective From NIH’s Rare Disease Day 2021
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