New Team Masters Projects Include Emphasis on Rare Diseases
While this year's Team Masters Projects (TMPs) at KGI cover a wide spectrum of topics, four TMPs focus specifically on rare diseases, reflecting an increasing interest among pharmaceutical companies in exploring new business models for developing medicines to treat rare conditions—also known as "orphan drugs."
According to the U.S. Food and Drug Administration (FDA), an orphan disease is a disorder that affects fewer than 200,000 patients in the U.S.; in Europe, an orphan disease is defined as a life threatening or chronically debilitating condition that affects no more than five in 10,000 people. While individually these conditions affect only a small number of patients, rare diseases are among the most serious of all illnesses and impact millions of patients and their caregivers around the world.
Pfizer, the world's largest pharmaceutical company, is sponsoring its first TMP, with a team of KGI students led by Professor Tim Coté, MD, MPH—who recently joined the KGI faculty after serving as director of the FDA's Office of Orphan Products Development. The students are comparing the clinical development paradigms that have led to regulatory approvals of orphan vs. non-orphan drugs in several key disease areas. This project will help the company refine drug development strategies for rare diseases and better assess technical and regulatory risk, which in turn may inform business strategies at large pharmaceutical companies such as Pfizer.
"About 2,500 orphan drug designations have evolved into almost 400 drugs and biologic products that have been developed and marketed since the passage of the Orphan Drug Act in 1983," notes Coté. "Traditionally, early stage development has taken place within small biotechnology companies, with large pharmaceutical companies adding important expertise during the regulatory process, and in delivering products to patients."
Coté adds that large pharmaceutical companies have traditionally focused on developing therapies to treat broad patient populations. However, scientific and regulatory advances have created new opportunities for large pharmaceutical companies to shift their focus toward targeted therapeutics for small patient populations. As large companies expand their focus in this space, they can use their significant resources—such as global scale, regulatory expertise and relationships with leading clinicians and researchers—to drive significant progress.
"Although the transition to focus on orphan product development is not without challenges, investing in treatments for rare diseases can be a viable business model for large companies as well," adds Coté.
Of the partnership with Pfizer, he says, "This is an exciting new relationship for KGI and one that we think will be fruitful for both Pfizer and our students."
"Pfizer understands the devastating impact of rare diseases on patients and caregivers, and we welcome the opportunity to collaborate with academic partners such as KGI to advance our strategy for the development of new treatments for rare conditions and to share complementary skills and expertise," said Dr. Cara Cassino, vice president of Pfizer's Specialty Care Medicines Development Group.
The partnership builds on Pfizer's strong foundation in rare diseases, offering marketed products treating 17 orphan indications in the U.S.
In addition to Pfizer, other companies sponsoring a rare-disease-related TMP this year are: Amylin, whose project aims to map the current landscape for the genetic testing and diagnosis of rare and ultra-rare metabolic diseases; AVI Biopharma, which has the goal of generating a database of global orphan or rare diseases based on specific inclusion criteria; and Sigma-Tau, which is focusing on development of a prototype pharmaco-economic evaluation process to determine the economic and qualitative effects of a medication.
"This is an exciting area that is attracting a lot of attention from pharmaceutical companies," adds Diana Bartlett, KGI assistant vice president and director of corporate partnerships. "With KGI's Center for Rare Disease Therapies, our institute has extensive expertise and a strong network within the rare disease space."
The capstone of the Master of Bioscience degree program, the Team Masters Project was founded in 2001. The number of projects per year has nearly tripled over the past decade—from six or seven in the early years to this year's record-breaking number of 18 projects.