Orphan diseases represent a collection of disorders that afflict fewer than 200,000 individuals for any single disease type, yet there are more than 7,000 distinct orphan diseases. The Orphan Disease Center (ODC) in the Perelman School of Medicine was founded in 2011 to promote and fund research in rare diseases globally, beyond Penn, making a difference for these under-served patient populations. A unique aspect of ODC is that it is a ‘Center without walls’ which sources and contributes to developing transformative therapies through the use of platform technologies and of research strategies that can be deployed across multiple rare disease programs. The main feature is a pilot grant program which provides $8M annually to over 35 global institutions. Reporting to the Deputy Director of ODC, your goal will be to provide international leadership in establishing research agendas in the rare disease that are a high priority of the ODC. You will build strong partnerships with key stakeholders, including patient advocacy groups, partnering academic institutions and key opinion leaders in related diseases within Latin and South America by organizing international scientific symposiums related to diseases of interest to the program of excellence, partnering with ODC staff to host these events. You will partner with the Programs of Excellence (POE) to connect your local contacts with the appropriate Director of Therapeutic Development.
Proactively and compliantly engage with regulators/government agencies within the region to stay up-to-date on clinical development regulations, emerging as the Subject Matter Expert for Latin/South America policy for the ODC. You will partner with Directors of Therapeutic Development by contributing to the strategic plans to establish clinical development for novel therapeutics within your region, advising on local regulatory requirements and cultural customs. You will leverage regional stakeholders in guiding your advisement for the development of this plan. In executing this plan, you will undoubtedly be required to have extensive interactions with biopharmaceutical industry partners, knowledgeable on the development of novel therapeutics and will need you to travel to biomedical conferences. You will be partnering with the POE’s to establish regional working groups within each disease to address research and clinical practice gaps and focus on filling those gaps to further the development of effective treatments.
Joining the ODC offers you with unique opportunity to contribute to revolutionizing the practice of medicine for these under-served patient groups.
Internal Number: 38931
About University of Pennsylvania
Penn has a long and proud tradition of intellectual rigor and pursuit of innovative knowledge, begun by Benjamin Franklin in 1740. That tradition lives today through the creativity, entrepreneurship, and engagement of our faculty, students, and staff. Academic life at Penn is unparalleled, with an undergraduate student body of 10,000 from every U.S. state and around the world. The entering class of 2015 is the most talented and diverse in Penn's history. Consistently ranked among the top 10 universities in the country, Penn welcomes an additional 10,000 students to our top-ranked graduate and professional schools.