An orphan disease - also called a rare disease – is defined as any condition that affects less than 200,000 people in America. There are over 7,000 distinct orphan diseases today, affecting one in every ten Americans. Because these conditions are so variable and may only affect a few hundred people, diagnosis and treatment is complicated. Specialist physicians are scarce and therapies are even fewer in number. In fact, for 95% of the people with an orphan disease, there is no FDA-approved medication, treatment, or cure. Madeline hopes to change this.
For her capstone thesis project, Madeline is working in conjunction with corporate sponsor Horizon Pharma plc to assess the market potential of a new pharmaceutical product. This medication would be considered a “new therapeutic entity”, which is a recent innovation in the drug industry. Madeline is analyzing the feasibility and business case for taking an existing technology and applying it to new disease areas to truncate the timeline from benchtop to rollout. “This means that we can develop and deliver the treatments that people desperately need in an expedited fashion,” says Madeline. She continues, “The project is both fascinating and gratifying because the direct result of accelerating this innovation cycle is a tangible improvement in someone’s physical health.”
Madeline’s thesis experience thus far has been rewarding not only in working towards that goal – but also in the process. “I researched orphan diseases for two and a half years at the Boler-Parseghian Center for Rare and Neglected Diseases during my undergraduate studies, but getting to see this side of the industry gives me an entirely new perspective of the drug creation process,” Madeline states. While she is able to contribute her background in working with orphan diseases, she also states that the professional skills sets she has learned in the process will translate well into future careers.
Currently, the biopharmaceutical industry is at the epicenter of innovation. “The translation from concept into a marketable product is complicated, but there is so much new energy and focus in this area that it is an exciting space to be in right now.” The momentum she refers to is the recent uptick in orphan drug development. Today, one-third of FDA drug approvals are for rare diseases. Legislative measures such as the Orphan Drug Act now provide the economic incentives to pharmaceutical companies to pursue the risky and expensive process of attempting to design a new drug.
Madeline’s ultimate goal for the project is to design and validate the business model for marketing one of the existing drugs in Horizon’s portfolio for a novel use. She will analyze the biochemical mechanism of the drug in the body, the financial implications of rounds of FDA testing, product cost and price modeling, intellectual property concerns, and the commercialization strategy if rollout is deemed appropriate. In the end, she intends to give a recommendation to the senior executives of the company on whether or not the endeavor is too risky.
Through ESTEEM, Madeline is confident that her project will continue to champion the rare disease community at large.