On November 28-29, 2012, the American Conference Institute (“ACI”) held in Boston, Massachusetts, its inaugural conference: “Orphan Drugs and Rare Diseases: Maximizing Opportunities and Overcoming Stumbling Blocks in the Designation and Development Process“. The Conference was well attended and featured a pre-conference boot camp on the Orphan Drug Act as well as a post-conference master class on overcoming clinical trial challenges and proving the safety and efficacy for orphan drugs.
One highlight of the conference was Timothy R. Cote, M.D., M.P.H., Principal, Cote Orphan Consulting (Director of FDA’s Office of Orphan Drug Products Development (2007-2011)). Cote provided a back drop for how the Orphan Drug Act came about, emphasizing that while for each individual disease the afflicted patient population is small, when all of the orphan diseases are pooled together, actually the population is sizable and a rapidly-growing area. Dr. Cote described orphan drug development as a “high touch” field where the need for new therapies are often driven by families with afflicted members and typical “big pharma” strategies are less effective. Calling it “scrappy not crappy” science, Cote explained that unlike big pharma projects that involve thousands of patients and hundreds of millions of dollars to develop new therapies, orphan drugs often can be developed for under $10 million–in part because there are so few patients who are candidates for clinical studies and treatment. Cote said while approximately 60% of orphan drug designation requests are approved, the success stories are the products that obtain new drug application approval and win the highly-coveted seven-year exclusivity for the first orphan indication (“a horse race”). More importantly, the basic research required to find cures for orphan drugs often provides valuable medical knowledge in how our bodies work, which he called “pharma karma.”
Another highlight of the conference was Christopher-Paul Milne, D.V.M., M.P.H., J.D., Associate Director, Tufts Center for the Study of Drug Development, Tufts University. Milne said that while the initial orphan market was thought of as relatively small, there are often higher rates of return than more “mainstream” diseases, particularly for diseases with more options for treatment. One phenomena that he tracked was the “ultra orphans,” where the orphan population for treatment, which is restricted to less than 200,000 when applied for orphan drug designation in the United States, can get closer to the 200,000 number, and sometimes can expand to more mainstream numbers, if the therapy is later found to have non-orphan treatment options. While certain disease areas such as various cancers have good orphan drug representation, other therapeutic areas, such as neurology, have had less success stories, Milne reported. Milne added that amidst the tension of competition versus collaboration, it has often been important for unlikely partners to work together, with the help and motivation of patient groups, such as the National Organization for Rare Diseases (“NORD”).