Study Finds Underinvestment in Long-Term Cancer Research
Pharmaceutical firms underinvest in long-term research to develop new cancer-fighting drugs due to the greater time and cost required to conduct such research, according to a newly published study authored by Massachusetts Institute of Technology (MIT) economists. These findings were published by Budish et al in the American Economic Review.
Specifically, drugs to treat late-stage cancers are less costly to develop than drugs for earlier-stage cancers, partly because the late-stage drugs extend people's lives for shorter durations. This means that the clinical trials for such drugs get wrapped up more quickly, too—and provide drug manufacturers more time to control patented drugs in the marketplace.
“There is a pattern where we get more investment in drugs that take a short time to complete and less investment in drugs that take a longer time to complete,” said Heidi Williams, PhD, MSc, Class of 1957 Career Development Assistant Professor of Economics at MIT.
The social cost is significant: Researchers estimate that the lack of investment in longer-term drugs resulted in a loss of 890,000 life-years among people diagnosed with cancer in the year 2003 alone. The study also suggested three policy adjustments that might produce more long-term research on anticancer drugs.
The finding “doesn't mean that the private firms are doing anything wrong,” Dr. Williams added, given the incentives they face. However, she observed, “The public sector is more willing to invest in these long-term projects than is the private sector,” suggesting that new policies could produce more types of drugs for patients.
Study Findings
To conduct the study, the researchers analyzed 4 decades of data from a variety of comprehensive sources, including the National Cancer Institute and the U.S. Food and Drug Administration (FDA). In all, the study encompassed over 200 subcategories of cancers detected at different stages of development.
Before reaching their conclusions, however, the researchers had to establish that the tendency toward short-term drug research was due significantly to the shorter duration of clinical trials. They concluded this, in part, by studying what happens when clinical trials do not use mortality to establish effectiveness but instead use “surrogate endpoints” (biomarkers) that stand as proxies for eventual outcomes that help to estimate how effective the drugs will be.
By examining the historical data, the researchers found that in some cases where surrogate endpoints were allowed in cancer research clinical trials—including many types of leukemias—there were relatively more trials and money poured into research, other things being equal.
“When you have good surrogate endpoints, you see a dramatic increase in research development investment, which means lives saved,” said Benjamin Roin, JD, Assistant Professor of Technological Innovation, Entrepreneurship, and Strategic Management at the MIT Sloan School of Management.
Suggested New Approaches
The researchers suggest three new policy approaches that could spark the development of a greater number of long-term cancer drugs.
The first is continued use of surrogate endpoints—or at least initially, more research to find out whether wider use of surrogate endpoints for additional cancers is valid.
A second possible policy change is more public funding of research and development for anticancer drugs, since such funding is free of short-term, private-sector shareholder pressure to produce returns.
These first two points are related, Dr. Williams emphasized, noting, “No individual private firm wants to come in and provide all of the evidence that you need to validate a surrogate endpoint, because once one is validated, that's going to be used by all of the firms on the market.”
A third potential new policy, suggested by the researchers, would be changing the terms of drug patents, which typically run from the time of patent filing, to run from the time when the drug hits the market. That said, the FDA can currently grant exemptions that lengthen drug patents to account for the time research development takes.
Dr. Roin, an expert in patent law and biomedical innovation, said future congressional changes to this policy are “extremely realistic, although [such changes] would take time.”
Dr. Williams emphasized that, as economists, she and her colleagues are only advocating the use of surrogate endpoints where they have been accepted by the consensus within mainstream medicine. As it happens, surrogate endpoints have been validated and used regarding heart disease through the Framingham Heart Study, a major, multidecade project, although their incorporation into cancer research has been more gradual.
This research was supported by the National Institute on Aging, the National Institutes of Health, and the National Science Foundation.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
