ROI from clinical research can suffer when too many research organizations seek to develop treatments for similar conditions and indications, making it more difficult for any one pharma company to achieve medical and financial success. Financial analysts note that one-third of R&D spending is going into oncology and inflammatory conditions, and that pipelines also are over-crowded with potential treatments for melanoma and rheumatoid arthritis. Yet research programs have shrunk for new antibiotics, as well as for treatments for heart disease and central nervous systems disorders, which affect larger patient populations.
Much of the enthusiasm for developing new cancer therapies lies in the ability to target population subsets, which often supports designation as an orphan drug and/or breakthrough therapy. Such status reduces regulatory fees and opens the door to added advice from FDA experts and speedy approval, based on small, limited studies.
In some cases, single-arm studies may be sufficient to demonstrate activity, even though such limited trials provide little information on safety, explained Richard Pazdur, irector of the Office of Hematology and Oncology Products in the Center for Drug Evaluation & Research (CDER). Pazdur observed at the FDA/CMS Summit in December that he was not a “big fan” of single-arm studies a few years ago. But for a drug with a 50% response rate for a condition with no existing effective therapy, a limited study may be enough. However, the emergence of more oral cancer therapies for chronic use may require sponsors to look more at safety, Pazdur added, noting that clinical trials for such drugs have to capture toxicities and examine what is a tolerable dose.