A new study has found that publicly funded cancer clinical trials have saved cancer patients more than three million years of life, with each life year gained costing just $125 of federal funding. Abi Millar finds out more.
Publicly funded trials have long been viewed as an integral part of the clinical research landscape. While the majority of trials are financed by pharmaceutical companies, this can lead to an emphasis on profit, and in turn a skew towards certain types of research. Publicly sponsored trials, then, might be seen as a way to fill in the gaps, exploring areas that may not be a priority for industry.
This is particularly important in fields like oncology. As Dr Richard Schilsky, senior vice president and chief medical officer at the American Society of Clinical Oncology (ASCO), points out, trials of this nature have addressed many key questions in cancer management.
“Publicly funded trials have been responsible for most modern advances in treatment of cancer in children, in life-saving adjuvant therapy after surgery, in combining modalities of treatment such as drugs and radiation, in studying rare cancers and in developing effective cancer prevention strategies,” he says. “They have also rigorously evaluated purported advances in cancer care that were popularised by single individuals or centres. In some cases, they have proven these advances ineffective, sparing patients from the cost and side effects of useless treatments.”
Drop in trials
These benefits might be seen to speak for themselves. However, in recent years the balance has shifted. Between 2006 and 2014, the number of clinical trials funded by the US National Institutes of Health (NIH) dropped from 1,376 to 1,048, while the number funded by commercial entities surged from 4,585 to 6,550.
According to Dr Stephen Ehrdhart, the epidemiologist who compiled these findings, the drop is likely due to two factors: flat NIH funding levels, and more researchers competing for the same cash pool.
“My concern is that independent trials are on the decline and that means we have less high-quality data to inform public health that are not influenced by commercial interests,” he told Bloomberg in 2015.
Of course, it is not a given that fewer publicly funded trials means a lower overall quality of research. And it could be argued that investing in trials – which may or may not yield positive results – represents a waste of money for taxpayers. However, a new paper, published in JAMA Oncology in June, has suggested that clinical trials are indeed a valuable target for government funding.
The study, conducted by SWOG and presented at the 53rd annual meeting of the American Society of Clinical Oncology, found that SWOG’s publicly funded cancer trials have saved patients more than three million years of life in total. What’s more, each year of life gained cost just $125 of federal funding.
“With limited dollars to invest, funding agencies have to assess the value of different research organisations and processes, and make trade-offs between funding different approaches to cancer,” says study author Dr Joseph Unger, a SWOG biostatistician and a researcher at Fred Hutchinson Cancer Research Center. “In this context, our estimate of $125 per life year gained suggests that the National Cancer Institute’s (NCI’s) network groups are great value.”
SWOG is a worldwide network of researchers who design and conduct cancer clinical trials. Sponsored primarily by the NCI, the group has approximately 12,000 members across the US, and around 80 trials now active. Since it was founded in 1956, it has enrolled more than 200,000 patients.
“Because our trial networks represent publicly funded research programs, their studies address research questions that focus on issues important to patients,” says Unger. “Many of these research questions may not be top priorities of industry, which focus more on new drug development.”
Quantifying the benefits
While this has brought clear benefits over the years – not least 14 new cancer drugs and more than 100 changes to the standards of cancer care – Unger wanted to quantify exactly what the impact has been on survival rates.
He identified 193 completed trials, of which 23 showed a statistically significant increase in survival due to the new therapy. Using a statistical model with various inbuilt assumptions (that the treatments would become the standard of care, that their effects would last five years and that all future patients would benefit), he calculated how many years of life had been gained by patients. The total was 3.34 million.
“We used a counterfactual modelling approach, by which I mean, we estimated how many life years would have been lived by cancer patients with versus without the new treatments, and calculated the difference,” says Unger. “The study estimated population life-years gained from the 23 treatment trials through 2015 by mapping the effect of the new treatments onto the US cancer population.”
He also calculated the return on investment for SWOG’s trials, by dividing the total NCI investment by 3.34 million.
“We estimated that the total federal investment in SWOG treatment trials during the course of its 60 year history was $418 million, or about $7 million per year on average over 60 years to the U.S. taxpayer. For the average taxpayer of course, this is a very small amount of money,” says Unger.
The general consensus
So what kind of conclusions can be drawn from Unger’s research? While the model could stand be further refined (Unger plans to extend it to include groups other than SWOG) there are lessons to be gleaned from its very existence. Namely, the value of federal funding is not a purely ideological question – it comes down to maths, not conjecture.
Only last year, KPMG produced a report assessing the economic impact of the NIHR Clinical Research Network’s activities in the UK. It found the research had added £2.4bn to GDP and generated almost 39,500 jobs.
In fact, the research community has been near unanimous in commending public funding. As Dr Frank Hulstaert, senior researcher at the Belgian Federal Healthcare Knowledge Centre (KCE), points out, the model can make good economic sense if the money is allocated appropriately.
“The evidence available strongly suggests that publicly funded pragmatic practice-oriented clinical trials can provide a positive return on investment provided the selection of the research question is done properly, the trial is conducted in a professional way and the results are used by healthcare decision makers to change daily practice,” he says.
Dean Baker, co-director of the Center for Economic and Policy Research, believes that government-funded research might negate the need for patent monopolies, which lead to spiralling drug costs for patients. He has a clear proposition for how it might be done.
“I think publicly funded trials are a great idea. However the government should buy up the rights to the drug being tested in advance, so that the drug is then placed in the public domain and can sold as a generic, if the tests prove successful,” he says. “This offers the advantage of full transparency in the testing process, as all results would be public, subject to restrictions to patient confidentiality. It would also mean the next great cancer drug will be available for a few hundred dollars, rather than a few hundred thousand dollars.”
Of course, we are unlikely to see a huge uptick in federal research funding any time soon, given the lack of political momentum – Baker feels it may fall to wealthy philanthropists to set the ball rolling. However, it seems clear that the mechanisms that are already in place have many advantages for patients.
“Publicly funded trials through the NCI’s networks use an existing infrastructure of many top researchers and institutions to conduct trials of great breadth and diversity,” says Unger. “They provide patients greater access to trials that are not strictly related to new drug development.”
This article appears in the September 2017 edition of Pharma Technology Focus