Students Learn How to Advance Therapies for Rare Diseases

Ian Phillips

Three years ago, Dr. Tim Coté, director of the Food and Drug Administration's Office of Orphan Products Development, challenged a group of KGI students to fill out an Orphan Drug Designation Application in less than two hours to illustrate the simplicity of the process.

"Indeed KGI students performed incredibly," Cotè said during a third orphan drug application workshop for KGI students. "I was stunned."

What started out as an academic exercise for Master of Bioscience (MBS) students by the Center for Rare Disease Therapies at KGI has led to a series of two-day workshops in which FDA officials meet with pharmaceutical and biotech companies, academics and patient advocates and walk them through the orphan product application process step-by-step.

The first industry workshop, held at KGI in February this year, resulted in 18 applications being filed.

"I figured if the students can do it in two hours, industry can do it in two days," Cotè said.

The goal of the workshops is to encourage more companies and individuals to pursue development of drug therapies for people who are suffering from rare diseases, which affect nearly 30 million Americans, or approximately 1 out of 10 people. Of the 7,000 identified rare diseases, many have no treatments. For those that can be treated, the cost to the patient is often prohibitive.

The Orphan Drug Act of 1983 offers special incentives to develop these products because there is not a lot of profit in developing a drug for a disease that may afflict less than 100 people.

More than 70 students gathered for the KGI student Do-A-Designation Workshop in late October where Coté, conducting a webinar from Maryland, talked about the evolution of orphan drugs in the United States.

Since the Orphan Drug Act of 1983 was passed, 361 orphan drugs have received full market approval, and more than 2,250 have been designated orphan drugs, Coté said.

By comparison, from 1973 to 1982, only 10 new drugs for rare diseases had been approved. In 2008, 38% of new drugs approved by the FDA were orphans, and Coté ee xpects 2010 to be a record year in terms of orphan-drug approvals.

To receive orphan drug designation, the proposed therapy must target a rare disease (one with a patient population of 200,000 or less) and it must show promise for treating the rare disorder.

After an orphan drug designation application is approved, the next step is clinical trials with the goal of gaining marketing approval. Orphan drug designation protects the drug developer by promising market exclusivity for seven years. The developer also receives 50% tax credits on the cost of clinical trials and is exempt from drug review fees of $1.5 million.

An added advantage, Coté said, is that orphan drug designations are listed on the FDA website, where venture capitalists routinely check for investment opportunities.

Following Coté's presentation, Dr. Mathew Thomas and Dr. Erica K. McNeilly, visiting FDA health science administrators, conducted the workshop. They gave students an overview of the application before dividing them into groups of six. Students had less than two hours to prepare their applications with materials provided by the FDA.

Ryan LaRanger (MBS '11) said he thought the workshop was an excellent opportunity to interface with the FDA.

"As someone interested in management consulting, I found this workshop to be quite helpful because while I will not likely work for the FDA, my future clients certainly will need to work with the FDA," LaRanger said.

"Seeing that the FDA is making attempts to be more transparent indicates that that aspect of my job will become easier as time goes on." 

Wendy Milling (MBS '12), who plans to work for a biotech company after she graduates, said she found the workshop very helpful in revealing the types of information on the application that is important to the FDA.

"Biotech companies often develop orphan drugs, and the rise of personalized medicine will almost certainly facilitate the orphan drug trend," Milling said. "For that reason, knowledge of the advantages and constraints of the orphan drug designation process is likely to come in handy in the future."

Professor Ian Phillips, PhD, director of KGI's Center for Rare Disease Therapies, said, "I feel that this is one of the more important student experiences we offer. To meet with FDA officials one-on-one is unique for students at any university, college or academic unit."

"This interaction with the Office of Orphan Product Development has given KGI a new reputation at the national level in regulatory affairs and in rare diseases," Phillips said. "The student workshops are already starting to have an impact."

Second-year MBS student Silviya Meletath, who attended a student workshop in October 2009, helped win an Orphan Drug Designation for BPT Pharmaceuticals in Irvine for a drug that shows promise in treating pediatric Multiple Sclerosis.

"Silviya recommended we seek the designation and provided invaluable help in preparing the application prior to the KGI workshop. She also worked with us directly for both days of the workshop and was critical to developing the successful application," said Lynn Foster, BPT Pharmaceuticals' CEO.

Multiple Sclerosis is most commonly diagnosed in young adults. It afflicts about 450,000 people in the U.S. and about 2.5 million worldwide. But pediatric Multiple Sclerosis only afflicts about 20,000 young people under the age of 18, and it is for this small population that the drug, ProEnzy, will be used under the Orphan Drug Designation.