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The Center for Rare Diseases educates students to become biotech industry leaders who understand rare diseases and recognize the need for remedies.

Advocacy, Education, and Science to Improve Lives

Individually, rare diseases affect relatively few Americans. Collectively, they cause millions of people and their families to suffer. For many of these diseases, no treatment is available. For some, drugs or other therapies exist, but patients are unable to access them because the cost is too high.

KGI鈥檚 Center for Rare Disease Therapies addresses these issues. It raises awareness of rare diseases and promotes promising treatments. The center educates students to become biotech industry leaders who understand rare diseases and recognize the need for remedies. It brings together all of the parties who can make a difference and find solutions: patient advocacy groups, pharmaceutical and biotech companies, government agencies, research institutions, and nonprofit organization.

The center dedicates its advocacy, education and science to a singular purpose: making more treatments available to improve the lives of people with rare diseases.

The Center鈥檚 Mission

The mission of the Center for Rare Disease Therapies is to advocate new therapies for rare diseases and to increase awareness of rare diseases through education, science, and ethical business practices. The center acts as a nonprofit catalyst that interacts with government agencies like the FDA鈥檚 Office of Orphan Product Development, patient advocates such as the National Organization for Rare Disorders and the Pharmaceutical Research and Manufacturers Association (PhRMA).

In the long term, the center aims to be a national and global resource for expertise, training, and information related to rare diseases.

About Rare Diseases

Rare Diseases Affect Millions of Americans

The Orphan Drug Act of 1983 defines a rare or orphan disease as one diagnosed in fewer than 200,000 people in the United States. This may seem like a small number relative to the overall U.S. population, but with more than 7,000 different rare diseases, the numbers add up.

In all, 25 million Americans-nearly 1 out of every 10 people in the country-suffer from a rare disease. Their conditions range from the more familiar to those largely unknown, including:

• Cystic fibrosis, a chronic disease of the lungs and digestive system
• Muscular dystrophy, a progressive muscle disease
• Lupus, a chronic inflammatory disease
• Infantile spasm, an early childhood epilepsy
• Spinal cord degeneration
• Inherited metabolic inclusion disease, a condition that can destroy muscle cells
• Uncommon cancers

Treating Rare Diseases is an Economic Challenge

Bringing a new drug or therapy to market requires a significant investment of time and money. In exchange, companies expect to make a profit. But when no more than 200,000 people need a product, companies lack a financial incentive to pursue drug discovery, development and approval. As a result:

• Research is limited. Many companies have discovered compounds or technologies that might be useful in treating rare diseases but haven鈥檛 continued their research because of the limited market for any products they might develop.
• Treatments are few. About 1,800 drugs and other therapies have been identified or approved. Yet only 350 are commercially available today. Despite financial incentives created through the Orphan Drug Act of 1983 to encourage pharmaceutical companies to develop new therapies, patients with most of the 7,000 identified rare diseases remain without any treatment at all.
• Cost is an issue. Even when drugs and other therapies are commercially available, their cost can be high. People who might otherwise access treatment simply can鈥檛 afford it.

KGI鈥檚 Center for Rare Disease Therapies brings attention to the impact and severity of rare diseases in the United States. The center elevates the need for treatment to a problem of national importance that calls for an effective response.

The Center's Work

Unlike organizations that focus on a single disease, KGI鈥檚 Center for Rare Disease Therapies functions as a nonprofit think tank addressing the problems arising from all rare diseases. The Center partners with the pharmaceutical and biotech industry, government agencies, patient advocacy groups, research institutions, and nonprofit organizations to:

• Increase awareness of rare diseases and their impact
• Revive promising therapies and encourage research to uncover new ones
• Promote drug development and commercialization
• Train a workforce prepared to find solutions for treating rare diseases

The Center for Rare Disease Therapies draws on KGI鈥檚 strengths, talents and distinctions. KGI鈥檚 extensive industry relationships, faculty with industry experience and classes focused on industry鈥檚 business, scientific and ethical challenges help the center perform its work and fulfill its mission.

Advocating for Treatments

The center ensures that rare diseases receive the attention that can lead to finding therapies for people who have no remedy available to them today. In advocating for more treatments, the center:

• Promotes the search for new therapies and helps to identify those with the most promise
• Reaches out to drug companies, government agencies, research institutions, and patient advocacy groups to encourage them to communicate with one another and work together to combat rare diseases
• Assesses the potential usefulness of research and orphan drugs that companies didn鈥檛 pursue or make available for sale
• Works with patient advocacy groups to document the prevalence of rare diseases and devise strategies to promote the development and commercialization of treatments
• Helps to prepare and file orphan drug designations
• Heightens awareness of rare diseases to gain broad public support for the development of new, more affordable therapies

Educating Students and Others About Rare Diseases

The Center for Rare Disease Therapies is an integral part of a KGI education. It ensures KGI students become professionals who understand rare diseases. It also enables those already working in the biotech industry and in research to learn more about rare diseases. Specifically, the center involves student teams in industry-sponsored, year-long projects with companies specializing in rare disease therapies

Teams of four to five students work with an industry liaison and a faculty mentor to complete their Team Master鈥檚 Project by examining the technical, regulatory, and business aspects of rare diseases. It also:

• Provides summer internships for students with patient advocacy groups, pharmaceutical and biotech companies, nonprofit organizations and government agencies such as the U.S. Food & Drug Administration鈥檚 Office of Orphan Products Development (OOPD)
• Teaches students about all aspects of rare diseases, including the market size, orphan drug discovery, the orphan drug application process, regulation and public policy
• Develops case studies on rare diseases that bring together science, business and ethics
• Presents workshops and symposia
• Disseminates key findings and other information through conferences and papers on topics such as the economics of rare diseases and strategies to address cost issues

Tackling Rare Diseases Through Science

Being part of KGI enables the Center for Rare Disease Therapies to go beyond scientific research to also focus on how scientific discovery leads to a commercial product.

The center benefits from its relationship with KGI scientists whose research may be instrumental in finding new treatments for rare diseases. KGI faculty and researchers are examining promising gene therapies as well as biomarkers, the chemicals or proteins that signal the presence of disease. They are also actively engaged in drug discovery. By turning to scientific colleagues at KGI and other university research institutions, the Center for Rare Disease Therapies moves closer to its goal of developing a clinically approved therapy for a rare disease within the next three to five years.