The US Food and Drug Administration (FDA) and similar agencies in other industrialized countries get politically punished more for approving drugs that turn out to have unexpected side effects. At the same time they few rewards for taking risks to approve drugs that might turn out to deliver large benefits. The willingness of politicians to criticize the FDA when drugs turn out to cause unexpected harm probably plays a role in a very large reduction in the rate at which the FDA approves drugs.
Why do terminally ill patients have to wait so long to get access to the only treatments that hold any promise of saving their lives? And why is it not their right to decide?
The problem has been magnified in recent years as the number of new drug approvals has fallen dramatically. The FDA approved just 16 new drugs last year, and is on pace to approve only 18 this year. That's down from a high of 53 in 1996 and 39 in 1997.
After a few high-profile drug scares, such as the 2004 withdrawal of Vioxx from the market, FDA officials have become gun-shy about approving new products. After all, the agency receives scathing criticism from Congress and the press when an approved drug turns out to be more risky than expected -- but rarely for keeping beneficial ones off the market.
This trend does not bode well for the development of rejuvenation therapies. The FDA will hold off approval of an anti-cancer drug for people who have a fatal disease. Never mind that people who have a fatal disease are going to die anyway. The FDA won't let people take a risk when they have little to lose. That makes no sense to me.
Rejuvenation therapies are going to treat that fatal disease called aging. Absent those therapies we are all going to die from complications of aging. Weakened immune systems will allow cancer cells to grow and spread. Weakened hearts and clogged arteries will lead to heart attacks and strokes. Aged brains and probably aged immune systems will allow junk to accumulate in our brains leading to neural cell death and eventual death from Alzheimer's or other brain diseases.
Faced with rising risks of death combined with increasing pain and disablement people should be given wider latitude to try new and unproven therapies. Once diagnosed with terminal illnesses people should be freed from government mandated restrictions on available therapies.
Here's another example of how better medical treatments could save time, boost incomes, boost economic output, and raise quality of life. The costs of raising kids with developmental defects is very high and if we could only avoid these defects in babies we would gain a great benefit.
New research suggests that the average household with children with autism not only spends thousands of dollars toward educational, behavioral and health care expenses each year, but also suffers from a lesser-known cost that hits them up front – a sizeable chunk of missed household income, perhaps as much as $6,200 annually.
The study, published in April’s edition of Pediatrics, paints a more detailed financial picture of how expensive life can become for parents of children with an autism spectrum disorder.
“To our knowledge, this is the first U.S. study that examines this front half of the ‘money in, money out’ equation,” said economist Guillermo Montes, Ph.D., the study’s lead author and a senior researcher at the Children’s Institute, a not-for-profit organization affiliated with the University of Rochester, where he also serves as a faculty member in the division of General Pediatrics. “To collect data on expenses is fairly straightforward– it’s a survey report. But projecting earning potential and then stacking that against actual income requires complex statistical modeling.”
The study is based on data from the National Household Education Survey on After School Programs and Activities in 2005, a telephone questionnaire that drew on parents of more than 11,000 children, kindergarten-age through eighth grade. Parents reported if their child had an autism spectrum disorder, or ASD (that is, if he or she had ever been diagnosed with autism or a pervasive developmental disorder), their total household income and their highest level of education. Several other demographic details were collected, including the parent’s age, type of family (two parent or otherwise) and whether they lived in an urban or rural locale.
The economic benefits of medical research are very high and I think medical research is underappreciated for its benefits. Costs of health problems of mind and body show up in lots more ways than just high medical bills or inability of a sick person to go to work. Families pay big tolls when a child has major disabilities.
The federal Office for Human Research Protections has ruled that it is perfectly fine for hospitals to use checklists to remind doctors and nurses to wash their hands and follow other sanitary procedures — provided the goal is to improve the quality of care given to patients. But if those hospitals want to analyze what impact the checklists might have in reducing infections, that counts as research and they must first seek approval from institutional review boards.
So if no checklists are used that is okay. Or if checklists are always used that is okay. But don't dare try to run a comparison between using and not using checklists with data collected to compare the results. That's experimentation and that isn't allowed without going thru a bureaucratic process.
Japanese manufacturers made huge strides in quality by making lots of changes in their processes aimed at eliminating sources of error and defects. This philosophy of continuous improvement works best when the obstacles to changes are few and where the consequences of changes can be measured. A deep embrace of this approach would do wonders for improving the quality of medical care - quality improvements that are sorely needed both for patient health and to control costs.
Mind over matter can only work by smart minds figuring out how to better manipulate matter.
A patient’s positive or negative emotional state has no direct or indirect effect on cancer survival or disease progression, according to a large scale new study. Published in the December 1, 2007 issue of CANCER, a peer-reviewed journal of the American Cancer Society, the study found that emotional well-being was not an independent factor affecting the prognosis of head and neck cancers.
We need immune therapies and gene therapies against cancer. We need more science and more biotechnology. Wishful thinking won't cut it.
Update: Spending more on therapies don't help increase survival times much either.
A new study finds that survival for elderly patients with lung cancer has changed little despite large increases in healthcare expenditures for lung cancer treatment. The study by Harvard University, National Cancer Institute, and National Bureau of Economic Research researchers, published in the December 1, 2007 issue of CANCER, a peer-reviewed journal of the American Cancer Society, finds that average life-expectancy rose by less than one month between 1983 and 1997, while costs rose by over $20,000 per patient.
But the study period ended in 1997. The bigger effects of the money come in the longer term.
Lung cancer remains the top cause of cancer death in the United States, with an estimated 160,390 deaths expected to occur in 2007.
The U.S. spends more than five billion dollars a year on detection, determining the disease severity, and treatment of lung cancer. This is a significant increase over the last few decades, mirroring similar increases in general healthcare spending over the same period.
But throwing lots of money at treatments has one really beneficial effect: The lure of that money encourages drug companies and venture capitalists to spend money to develop newer and better treatments. Big markets attract new players. In a way it is disappointing that only $5 billion a year spent on lung cancer in the United States. All else equal I'd rather that the venture capitalists would see lung cancer as a $25 billion a year market. Fortunately the other forms of cancer also cost billions to treat. So we are probably looking at tens of billions a year spent on all forms of cancer. Lots of money for drug companies and VCs to chase.
The Bush Administration proposed fiscal year 2008 budget for the National Institutes of Health will once again lag behind the rate of inflation and cause an inflation-adjusted cut on US federal biomedical research funding.
Meanwhile, funding for the National Institutes of Health, which oversees medical research, would rise nearly 2 percent to about $28.7 billion.
Biomedical research funding will deliver more benefit per dollar spent than any other form of government spending. Eventually this research is going to lead to the reversal of aging and the end of death from aging.
In inflation adjusted dollars US federal government funding of all research peaked in 2003 and has been declining since then.
Bush's budget is 4.2% bigger than last year's. He raises defense spending by more than 10%. Spending on veterans and foreign aid soars by double digits. There's more money for programs ranging from Pell college grants to national parks.
Defense and homeland security will get $658 billion or about 22 times as much as the NIH budget for biomedical research. Just the increase in defense and war spending will amount to about twice as much as the total NIH budget. I do not feel better defended and better served by that defense and war spending increase. I figure it will result in a much lower average life expectancy than we'd get if the money was spent on medical research. Worse, the war spending creates big figure costs such as maimed soldiers who'll earn less and need government programs to help care for them.
The war budget amounts to over 5 NIH budgets.
The total war budget of $163 billion, sought in the 2007 fiscal year, is projected to be $141 billion in 2008 and just $50 billion in 2009, far enough in the future that the estimate is little more than a place holder.
Think about that. Pull the troops out of Iraq and free up money to increase biomedical research by a factor of 5. The war does nothing to make us safer and probably has a net negative effect on our security. We could instead spend money on research that'll cure all the diseases we are going to get as we age. Eventually the money will produce biotechnologies that allow us to rejuvenate our bodies.
Due to inflation the National Institutes of Health are awarding far fewer research grants.
Funding for biomedical research, which has been flat for several years, may now begin to grow. The House proposal would give NIH a 2% increase this year, adding $620 million to the current budget of $28.6 billion. The austerity since 2003 has taken a toll, NIH officials say, as inflation significantly eroded NIH's buying power and reduced the number of new and competing grant awards from 10,300 to fewer than 9100.
The percentage of grant proposals funded has been dropping.
Five years ago, 27 percent of scientists' research proposals got NIH funding, according to agency data.
By last year, that dropped to 16 percent. The success rate for Pioneer Grants - which finance groundbreaking approaches to biomedical problems - is now below 5 percent.
Scientific and technological knowledge, once discovered, delivers us benefits year after year into the future. The sooner we get the knowledge the more total benefit we'll get from that knowledge. We spend less than 1 percent of the US federal budget on biomedical research. That's a mistake. We could derive greater benefit from a much bigger effort.
A Plos Medicine article reviews the sources of cancer funding in the European Union and the United States and finds Europe is greatly lagging in per capita spending in cancer research from funding sources which are not for-profit businesses. The US federal government's National Cancer Institute alone (not the only source of cancer research funding at the federal level) spends more than two and a half times the total spent by all non-commercial sources for cancer research in Europe.
In our survey, we identified 139 noncommercial funding organisations that collectively spent
1.43 billion on cancer research for the year spanning 2002–2003. Absolute spending in 2002/2003 on cancer research varied widely across the EU, ranging from
In Euros the 3.6 billion for the US National Cancer Institute is more than two and a half times the 1.43 spent by all European noncommercial sources.
The EU spends a greater proportion of its cancer research funding on cancer biology than does the US (41% compared with 25%). The US spends a greater proportion of its cancer research funding on research into prevention and treatment than does the EU (prevention, 9% in the US compared with 4% in the EU; treatment, 25% in the US compared with 20% in the EU) (Figure 2). Data published by the US National Cancer Institute has been fully validated, whereas the EU uses self-reported, top-level CSO categories for 62% (n = 74) of the organisations from which financial data was obtained. The size of the two pie charts in Figure 2 is representative of the sizes of the annual budgets: in 2002/2003, the US National Cancer Institute spent
But wait, the gap is even bigger.
The average per capita spent across the entire EU (including European Commission and Trans-European Organisation spending) was
I would be happy to see Europe try to seriously compete with the United States in biomedical science funding. I would be happy to see Europe act more pro-life and anti-death and less lame and pathetic. We would all benefit if the European countries tried as hard as America to conquer cancer and a large variety of other old age killers. Do I even need to mention that the general advances in biomedical science and technology that come from research on diseases of old age will inevitably produce biotechnologies we need for rejuvenation therapies?
In his entry in his "Scream this from the rooftops" series Alex Tabarrok came across a research paper with evidnce that European drug price controls are causing Europeans to produce far less commercially funded medical advances as well.
EU countries closely regulate pharmaceutical prices whereas the U.S. does not. This paper shows how price constraints affect the profitability, stock returns, and R&D spending of EU and U.S. firms. Compared to EU firms, U.S. firms are more profitable, earn higher stock returns, and spend more on research and development (R&D). Some differences have increased over time. In 1986, EU pharmaceutical R&D exceeded U.S. R&D by about 24 percent, but by 2004, EU R&D trailed U.S. R&D by about 15 percent. During these 19 years, U.S. R&D spending grew at a real annual compound rate of 8.8 percent, while EU R&D spending grew at a real 5.4 percent rate. Results show that EU consumers enjoyed much lower pharmaceutical price inflation, however, at a cost of 46 fewer new medicines introduced by EU firms and 1680 fewer EU research jobs.
Europeans, like most of the rest of the world, are freeloading off of US medical research funded by our federal government, states, private foundations, and private sector companies. We would all benefit if they stepped up to the plate and spent on medical research as much as Americans do.
While many complain about the continuing rise of health care costs which far exceeds the overall rate of inflation, in a New York Times op-ed piece Marginal Revolution blogger and economist Tyler Cowen argues high US health care costs accelerate the rate of medical research and development and saves many lives in the longer run.
But the American health care system may be performing better than it seems at first glance. When it comes to medical innovation, the United States is the world leader. In the last 10 years, for instance, 12 Nobel Prizes in medicine have gone to American-born scientists working in the United States, 3 have gone to foreign-born scientists working in the United States, and just 7 have gone to researchers outside the country.
Tyler says European-style government cost controls would save money in the short run but slow innovation and therefore shorten lives and cost more in the long run. I agree.
Europeans fund a small fraction of the medical research that Americans fund.
In real terms, spending on American biomedical research has doubled since 1994. By 2003, spending was up to $94.3 billion (there is no comparable number for Europe), with 57 percent of that coming from private industry. The National Institutes of Health’s current annual research budget is $28 billion, All European Union governments, in contrast, spent $3.7 billion in 2000, and since that time, Europe has not narrowed the research and development gap. America spends more on research and development over all and on drugs in particular, even though the United States has a smaller population than the core European Union countries. From 1989 to 2002, four times as much money was invested in private biotechnology companies in America than in Europe.
Dr. Thomas Boehm of Jerini, a biomedical research company in Berlin, titled his article in The Journal of Medical Marketing in 2005 “How Can We Explain the American Dominance in Biomedical Research and Development?” (ostina.org/downloads/pdfs/bridgesvol7_BoehmArticle.pdf) Dr. Boehm argues that the research environment in the United States, compared with Europe, is wealthier, more competitive, more meritocratic and more tolerant of waste and chaos. He argues that these features lead to more medical discoveries. About 400,000 European researchers are living in the United States, usually for superior financial compensation and research facilities.
Americans do not live longer than people in other countries in part because the innovations that get funded in America get used around the world. In Canada and some European countries drugs are sold for lower prices than in the US. So drug companies make most of their profits and therefore get most of their revenue to fund research by selling products in the United States. Effectively the United States is subsidizing medical research for the rest of the world.
What I'd like to see: Policies should be aimed at automating the delivery of care. The large armies of nurses, technicians, office workers, and other deliverers of health care rarely innovate. Automation of their work would increase the rate of innovation by freeing up money and labor to do more research and development.
The problem we have is that the high price of medical care funds both innovation and waste. The number of dollars that goes to innovation is relatively small as compared to the dollars that go to delivering care using existing technology with lots of labor. High prices of drugs provide an incentive for drug companies to develop new drugs and other better treatments. But high costs for labor do not provide as much of an equivalent incentive to innovate to improve medical industry labor productivity. The demand for medical care is too inelastic due to the ways medical care is paid for.
I'd like to see much larger budgets for government-funded medical research. Currently the US federal and state governments are increasing their medical care spending faster than the rate of inflation while increasing medical research spending is growing more slowly than the rate of inflation (one of the unappreciated costs of the very expensive Iraq war btw). 18 out of 19 NIH institutes were proposed for budget cuts for fiscal year 2007. Note that if their budgets were maintained at the same level of nominal dollars they'd get cut about 3% due to inflation. This is exactly the wrong direction. The $2 billion per week burn rate of the Iraq war would more than quadruple federal research spending if the war was ended and the money shifted to research. That would save many lives.
Government funded research spending is a small fraction of government funded health care. Medicaid alone was projected at $338 billion in costs for 2006. Add in Medicare which is projected to be $450 billion in fiscal year 2007 and the total cost of just those two health programs (i.e. not including costs of federal employee health insurance, VA hospitals, etc) is about $800 billion. That's about 27 times the amount spent on federally funded medical research and the ratio is rising.
Right now, one of the reasons why we have a long-term fiscal problem is that health care costs, themselves, are projected to grow way above the rate of inflation. We're projecting Medicare costs will grow out over the course of the budget window about 9 percent per year.
This points to a problem in Tyler's analysis: Yes, huge medical funding has accelerated medical research, past tense. But the costs are getting so huge that cost controls are getting placed on medical expenditures and those cost controls will cut into the profits for new drugs and treatments far more than they cut into care delivered with existing technology. At the same time, government funding of research is dropping. We are therefore at risk of a gradual decrease in both public and private funding of medical research and development.
Seems to me we need policies that will make research and development a rising fraction of total money spent on health care. One idea: Make NIH spending a fixed percentage of Medicare spending. When Medicare spending rises 9% then the far smaller NIH spending should rise as well. How about making NIH spending 10% of Medicare spending?
Another suggestion: How about big prizes for achievements that increase labor productivity in health care? For example, how about a multimillion dollar prize for the first surgical team that removes 10 gall bladders in a record setting time and then another prize for the next time that bests the previous record time? Also, how about multimillion dollar prizes for the first totally robotic surgery for each of several popular types of surgery? $10 million and $20 million dollar prizes are very small potatoes compared to the trillions spent on health care. But the innovations that the prizes would spur would pay back in the tens and hundreds of billions of dollars.
Stroke costs of an aging population will be enormous in the next 45 years.
St. Paul, MN -- With projected costs of ischemic stroke in the United States expected to top $2.2 trillion dollars by 2050, the American Academy of Neurology (AAN) is urging Congress to further increase funding for the National Institutes of Health (NIH).
This estimate is probably a big overestimate because of the advances in biomedical technology that will occur between now and 2050. Stem cells therapies will start doing artery repairs certainly by 2025 and probably sooner. Also, drugs will come on the market that raise cardiovascular health improving HDL cholesterol and will work synergistically with drugs that lower the harmful types of cholesterol. Also, drugs that target different ways to lower general cholesterol will come on the market as well. Plus, gene therapies and drugs that improve vein and artery health by rejuvenating stem cells and other vascular cells will also make it onto the market by the 2020s.
Having said all that, this report in Neurology still serves the useful purpose of pointing out just how expensive each of the major diseases are in a single affluent country. The international costs are of course much higher. The size of these costs argue for greater efforts to develop treatments that will prevent stroke as well as other diseases. Each disease case avoided amounts to a large economic cost avoided as well as an increase in life expectancy and health.
A study published August 16, 2006 in the online edition of Neurology, the scientific journal of the AAN, found the total cost of stroke from 2005-2050, in 2005 dollars, is projected to be $1.52 trillion for non-Hispanic whites, $379 billion for African Americans and $313 billion for Hispanics.
"With the cost of stroke reaching $2.2 trillion, it is essential the NIH have the resources to halt this impending epidemic," said Catherine M. Rydell, CEO and Executive Director of the AAN. "The NIH has the ability to perform the research that can save countless lives and billions of dollars in health care costs if Congress would adequately fund its mission. The AAN will continue to work with our partners at the American Stroke Association, a division of the American Heart Association, and others to stress to Congress the importance of funding NIH."
The AAN is strongly supporting a budget increase of five percent, or $1.4 billion, to bring overall funding for the NIH FY-07 budget appropriation to $30 billion.
The researchers claim that rising rates of obesity and diabetes might cause an increase in the rate of stroke. This argument is plausible in the short to medium term.
"Doing the right thing now ultimately could be cost-saving in the future, but we have a long way to go before all Americans receive adequate stroke prevention and emergency stroke care," she says. "If our society is not going to do it for the right reasons, perhaps we can do it because it's going to be obscenely expensive."
Brown and her colleagues say their $2.2 trillion estimate is extremely conservative, because it is based on current rates of the conditions that put people at higher risk of stroke -- such as diabetes, cardiovascular disease and obesity. Such conditions are projected to become even more common in the future.
The $2.2 trillion estimate includes the cost of everything from ambulances and hospital stays to medications, nursing home care, at-home care and doctor's visits. They also include lost earnings for stroke survivors under age 65, based on current median salaries for each ethnic group. Earnings of those over 65 weren't included.
The aging of Western populations will force raises in retirement ages. Earnings losses from stroke therefore are probably underestimated by their assumption of only counting people under 65 as workers.
What can Americans do to decrease this looming bill? No matter what their age or ethnicity, individuals can cut their own risk of a future stroke by quitting smoking, losing weight, eating healthily, exercising, and keeping their blood pressure, cholesterol levels and any heart-rhythm problems under control, says Brown.
Meanwhile, doctors and hospitals can do a better job of providing preventive care and screening to patients with high blood pressure, clogged arteries and heart-rhythm problems. And, they can improve their use of a post-stroke drug called tPA.
We should strive to eat healthy diets. But we should also press for acceleration of the development of stem cell therapies, gene therapies, microfluidics, and other avenues of research that will lead to enormously more powerful therapies. We need rejuvenation therapies based on Strategies for Engineered Negligible Senescence (SENS). SENS therapies will make stroke and heart attacks extremely rare.
The economic pay-off of medical research will be enormous when cures for cancer are developed. A couple of academic researchers claim that a cure for cancer would have an economic value of $50 trillion for Americans alone. Add in the value of the cure to other industrialized societies that the total value of the cure likely exceeds $100 trillion.
A new study, to be published in a forthcoming issue of the Journal of Political Economy, calculates the prospective gains that could be obtained from further progress against major diseases. Kevin M. Murphy and Robert H. Topel, two University of Chicago researchers, estimate that even modest advancements against major diseases would have a significant impact – a 1 percent reduction in mortality from cancer has a value to Americans of nearly $500 billion. A cure for cancer would be worth about $50 trillion.
"We distinguish two types of health improvements – those that extend life and those that raise the quality of life," explain the authors. "As the population grows, as incomes grow, and as the baby-boom generation approaches the primary ages of disease-related death, the social value of improvements in health will continue to rise."
Many critiques of rising medical expenditures focus on life-extending procedures for persons near death. By breaking down net gains by age and gender, Murphy and Topel show that the value of increased longevity far exceeds rising medical expenditures overall. Gains in life expectancy over the last century were worth about $1.2 million per person to the current population, with the largest gains at birth and young age.
"An analysis of the value of health improvements is a first step toward evaluating the social returns to medical research and health-augmenting innovations," write the authors. "Improvements in life expectancy raise willingness to pay for further health improvements by increasing the value of remaining life."
Murphy and Topel also chart individual values resulting from the permanent reduction in mortality in several major diseases – including heart disease, cancer, and diabetes. Overall, reductions in mortality from 1970 to 2000 had an economic value to the U.S. population of $3.2 trillion per year.
In 2005 the US economy produced $12.4 trillon worth of goods and services. So the value of a cancer cure equals over 4 years of US economic output.
The enormous economic value of curative treatments for cancer and similar magnitude economic value for cures for other major killers such as stroke and heart disease mean we can get huge the future returns on investment in public spending for basic biomedical research. This means increased biomedical research funding by governments is pretty easy to justify when viewed in economic terms. Yet in order to fund a war, pork, and other wastes the Bush Administration has sought to cut biomedical research spending in inflation-adjusted terms and even proposed a freeze in nominal dollar terms (which means that real research spending goes down by the rate at which inflation goes up).
The numbers bandied about above understate the coming return on decades of basic biomedical research. Rejuvenation therapies will lengthen working careers and brain rejuvenation will boost productivity for most years worked. Minds which have both youthful vigor and the knowledge and skills accumulated from decades of work will achieve much greater feats and operate at much higher levels of productivity.
Because the rate of advance of research can not be forecasted accurately I think there's a tendency on the part of policy makers and the public to underestimate the future return on biomedical research and in other forms of research as well. Our accumulating body of knowledge is going to reach a critical mass at some point in the next 50 years where the vast majority of diseases become curable and replacement or rejuvenation of worn aged body parts becomes commonplace. We ought try much harder to make that day come sooner.
Medical research will pay off with cures to diseases and eventually full body rejuvenation within the lifetimes of some of us alive right now and yet George W. Bush wants to freeze the NIH budget.
In stark contrast to his initiative for physical sciences [ScienceNOW, 1 February and 3 February], President Bush today proposed a budget freeze for the National Institutes of Health (NIH) in 2007, holding its funding steady at $28.6 billion. The proposal, part of the President's overall budget request to Congress, is drawing concern and even outrage from biomedical research advocacy groups, who worry that NIH is losing ground after its budget was doubled from 1999 to 2003. Now the budget proposal, which curbs domestic discretionary spending while boosting funding for national defense, must wind its way through Congress before being approved in some form later this year.
This is being penny wise and pound foolish. A freeze is really a cut by whatever the rate of inflation turns out to be. So medical research is getting cut 2% or 3%. Yet medical research is, in my opinion, the best value per dollar of government spending.
The Bush Administration proposes to not allow biomedical research funding to keep up with inflation and therefore is effectively proposing cuts in biomedical research funding.
Under the president's request, the budget of the National Institutes of Health, which doubled from 1998 to 2003, would rise by 0.7 percent, to $28.7 billion next year. That is much less than what would be needed to keep pace with the costs of biomedical research, which are rising more than 3.5 percent a year.
For the National Science Foundation, Mr. Bush will request $5.6 billion in 2006, an increase of 2.4 percent, budget documents show. Mr. Bush requested an increase last year as well, but Congress ended up making a small cut in the agency's budget for this year.
At the Food and Drug Administration, buffeted in recent months by concerns about drug safety, the budget would increase by 4.5 percent, to $1.9 billion.
Aside on the FDA budget increase: What we need are more drugs in the drug pipeline, not more regulation of the drug development process.
The Bush Administration announced almost a year ago that funding of most categories of research was not going to keep up with inflation. The latest announced NIH budget increase for FY 2006 is even smaller than the 2% increase for fiscal year 2005.
The US federal budget is running a large deficit. Cuts have to be made. But medical research is a penny-wise pound-foolish place to cut spending. What we need are better treatments that are more cost-effective. Advances in medical science and in supporting technologies will some day yield treatments that are both much cheaper and much more effective. During transition phases early and less effective treatments cost more than not being able to do anything at all to treat a disease. But more advanced treatments that attack problems at the root level will inevitably cost less.
If we delay efforts to find cheaper and more effective ways to treat diseases then the avoidable cost in the long run of using less effective and more expensive treatments is going to dwarf the sums saved by cuts in medical research.
Stanley Kurtz lays out a gloomy future due to medical spending increases for an aging population.
The Congressional Budget Office estimates that the combined cost of Medicare and Medicaid alone will consume a larger share of the nation’s income in 2050 than the entire federal budget does today. By 2050, the combined cost of Social Security, Medicare, Medicaid, and interest on the national debt will rise to 47 percent of gross domestic product — more than double the level of expected federal revenues at the time. Without reform, all federal spending would eventually go to seniors. Obviously, the system will correct before we reach that point. But how?
The industrialized countries with aging populations are faced with a future of much higher taxes and benefits cuts as well. The unfunded liabilities for the care of old people are literally orders of magnitude larger than the amount spent on medical research. Take that $28.7 billion dollar figure cited above for the NIH research budget. Sound like a large number? The new Bush Administration proposal for the fiscal year 2006 budget is $2.57 trillion dollars for an economy that will be somewhere in the neighborhood of approximately $12.5 trillion per year based on an increase from the fourth quarter of 2004.
Current-dollar GDP -- the market value of the nation's output of goods and services -- increased 5.3 percent, or $152.1 billion, in the fourth quarter to a level of $11,967.0 billion. In the third quarter, current-dollar GDP increased 5.5 percent, or $157.4 billion.
But, barring cuts in entitlements, in 2050 Medicare and Medicaid alone may cost proportionately more than all of the US federal government today. So their costs alone could become be about 2 orders of magnitude more than is currently spent by the US federal government on medical research.
Of course government spending is only one portion of total medical spending. So even if medical entitlements programs are scaled back the portion of total GDP that goes to medical costs could be much higher than it is today.
While projections from current trends yield a bleak financial situation in 2050 it seems reasonable to assume that medical science is going to advance greatly by 2050. Even without higher levels of funding for medical research the rates of advance of biotechnology and biomedical science are likely to produce effective and cheap treatments by 2050. Some of those advances will produce cheaper treatments. Therefore I am less concerned about financial crises brought on by aging populations in the Western industrialized countries in 2050 than I am in the medium term, say 2025. We need to make medical advances come sooner so that we can cut costs, reduce the incidence of degenerative diseases, and increase the number of years people can work before Western countries get caught up in economic stagnation caused by higher taxes.
Here is another financial way to state my basic argument: Western governments have committed themselves to provide enormous quantities of medical care in the coming decades that those governments can not afford to deliver. The political debate on how to handle these commitments tends to center around whether to raise taxes, cut benefits, impose price controls, or inject more market forces into medical care. All four of those responses have either substantial downsides or insufficient upsides or both. Some of the approaches will even make the problem worse while also producing lower living standards. Let us consider each potential response in turn.
First off, taxes can be increased. But taxes raised beyond some point cease to produce a net revenue gain because people will respond to high tax rates by working less. This will slow economic growth and therefore in the long run will reduce the size of the underlying economy available to be tapped to pay for old age benefits. The United States could end up like European countries that have slower economic growth, lower per capita GDPs, higher taxes, and lower labor market participation rates.
Benefits cuts are still not happening in the United States and still seem a distant prospect. In fact, Republican President Bush added a new expensive drugs benefit a couple of years ago. No politician wants to propose benefits cuts and therefore government finances will get worse before cuts become politically feasible. Yet benefits cuts seem inevitable in the 2010s, 2020s, and beyond since the working population will oppose tax increases that are large enough to pay for all promised benefits.
Price controls are really backdoor benefits cuts. Price controls will produce decreases in the quality of service and will produce queues and rationing of the sort seen in Canada and Britain. Price controls also (and most importantly in my view) reduce incentives for development of new treatments by reducing the profits from new treatments. Hence price controls will make the financial crunch even worse by delaying the development of cost effective treatments.
Then there is the injection of more market forces into medicine. I'm all for this and favor health savings accounts for this reason. However, my judgement is that market forces alone can not prevent medical costs from becoming a huge weight on living standards because the public is going to demand governments to pay for medical bills that individuals can not afford. We are therefore politically limited in how much market forces will be allowed. Also, markets transfer the bulk of the benefits of innovation to customers and therefore underreward and underfund innovation. Markets provide even worse incentives for funding the scientific research needed to develop better medical treatments. If the government, as such a huge buyer of medical care, wants to benefit from innovative cost-saving biotechnology then the government will need to fund more research that will enable the development of that biotechnology.
Given that the four major alternatives discussed above have major downsides why not consider science as a potential solution? After all, science will eventually produce solutions that cheaply cure or prevent all the major diseases. The only question is when. Acceleration of the rate of advance could not only reduce the size of future liabilities but could also have the very attractive added benefit allowing us all to get healthier and stay healthier for much longer.
The biggest reason I can see for why medical research isn't taken seriously as a policy tool to solve the problem of unfunded old age medical care entitlements is that it is hard to predict the rate of advance of medicine. If a government wants to build a road across a continent it can get fairly realistic cost and time projections. Then the government can set out to build the road fairly confident that the goal can be accomplished in a time frame and for a cost not too far from original projections. But science by its very nature seems unpredictable. We have been pursuing the development of cancer cures for decades with tens of billions spent to date and still can't cure most cancers.
But the unpredictable nature of science ought to be considered in light of the substantial downsides of all the alternatives. We have a huge problem with aging populations. The problem doesn't get the attention it deserves in part because tallies of numbers of tens of trillions of dollars of liabilities and descriptions of trends in dependency ratios of workers to retirees and children are rather bleak to discuss and depressing to ponder. Debate on the issue easily deteriorates into a partisan battle about the New Deal and the proper role of government. Ideologues are quick to run to take up defensive positions against perceived ideological attacks by the other side, leaving them ill-disposed to think through rational analyses of the problems posed by aging populations. Does the US Social Security old age retirement program face a financial crisis? Perceived (and in some cases real) attacks on its very moral legitimacy prevents the financial problems of Social Security from being discussed rationally in too many cases. Ditto for programs for medical care for the aged. We need to move beyond the ideological sparring and look for better solutions.
Precisely because scientific research takes a long time to produce desired results we need to greatly increase the push to advance biomedical science and biotechnology now. We can't wait until the budget deficits are even larger, taxes are even higher, medical care rationing has become commonplace, the economy is stagnating, and the task of finding money to allocate to research is even more difficult. As things stand now medical treatment spending is going up faster than inflation while medical research spending is going up more slowly than inflation. Medical research spending ought to rise as fast as medical treatment spending rises.
My modest proposal for funding medical research: Change the major medical entitlements programs to require that 10% of all medical entitlements budgets go to fund medical research. Then when medical entitlements spending inevitably goes up medical research spending will go up proportionately. Yes, that will make the financial numbers for the medical entitlements programs look worse in the short run. But the money thereby spent will produce much larger savings for those programs in the longer run and will also produce treatments that will lead to great improvements in the health of the vast majority of people.
The Scientist has additional details about the NIH budget.
The proposed NIH budget would provide $15.5 billion for new (competing) and continuing (non-competing) research project grants, a 0.4% increase of $56 million. This would fund about 38,746 total projects, 402 less than this year. The average new research project grant would be funded at $347,000, about the same amount as in FY 2005.
One question I have: Is the current allocation of money in the NIH between staff researchers and grant-funded researchers optimal? My guess is that less funding for NIH staff and more funding for academic researchers would produce more total research progress. Anyone have any useful insights into this?
Scott Gottlieb M.D., recently departed US Food and Drug Administration (FDA) Director of Medical Policy Development, a practicing physician and fellow at the American Enterprise Institute, argues that the FDA is making cancer drug approval harder and more costly.
Even more concerning, there is also a whiff of caution coming from the agency's cancer division--the Oncology Drug Advisory Committee (ODAC)--where delays on the approval of new drugs can have a dramatic impact on the lives of patients who are suffering from terminal disease.
At a meeting last month, outside advisers to ODAC as well as rank-and-file FDA medical reviewers expressed criticism of the applications they are seeing and a desire to clamp down on the number of cancer drugs qualifying for accelerated approval. Accelerated approval regulations allow the FDA to approve products based on preliminary test results, with the proviso that the company continues with clinical trials after the drug is marketed.
The chair of the FDA's cancer drug advisory committee rejected the idea that cancer drugs should be allowed onto the market if they are reasonably safe and have some degree of effectiveness (known as "efficacy") with the understanding that oncologists will determine their value through routine use of the drug.
Higher costs per drug developed translate into fewer drugs being developed at any one time and fewer new drugs available in the future.
The FDA is moving toward making it harder for a new drug to be approved if it can not be demonstrated to work better than the off-label use of an existing drug.
The other point of contention at the cancer advisory meeting was whether it is appropriate for the FDA to require drugs up for accelerated approval for treatment of a specific kind of terminal cancer to prove that they were superior to other drugs being used off-label to treat the same cancer.
The debate turned on the FDA's consideration of Inex Pharmaceutical's application for accelerated approval of Marqibo vincristine sulfate liposome injection to treat relapsed, aggressive non-Hodgkin's lymphoma (NHL). The FDA noted that no products have been approved for the indication, and suggested that the committee consider whether a number of products that are used off-label for the indication should be considered "available therapy." By suggesting that ODAC consider off-label uses as available therapies, the FDA dramatically raised the bar for approval of Marqibo.
Imagine you have relapsed NHL. Suppose there is an existing drug that works against it for some though not all people. Would you want the FDA to hold another drug off the market that also works for some but not all people? Or do you think you should have the right to choose between the two drugs or even to take both of them?
I believe that once someone has been given a diagnosis of a fatal disease that they should be given the equivalent of a "get out of the FDA drug approval jail free card". In other words, once your days are numbered due to a specific diagnosed disease you should be free to take any experimental therapy and drug and biotech companies should be free to sell you any experimental therapy without the FDA being able to stop them.
A smaller step in the right direction would be to replace the existing membership of the above mentioned Oncology Drug Advisory Committee (ODAC) with a new membership made up of people with diagnosed cancer. There are plenty of scientists, epidemiologists, and medical doctors walking around today with diagnosed cancers. So the committee would not have to lack for expertise. But what it would cease to lack is a great sense of urgency and a sense that patients should be given more choices.
If you are going down the elevator for the final check-out from from the big hotel of life why should the government have any power to prevent your trying any therapy imaginable on your way down? Someone close to me is dying from metastatic cancer and I do not understand why the government should have any power at all over what experimental treatments someone such as this person might try. The government can not protect people from death. The government can not regulate death out of existence. But the government can and does impose such costs and obstacles on the drug development process that the rate of development of new drugs is greatly slowed and the date at which various diseases become curable gets pushed much farther into the future than it needs to be.
Last night Charlie Rose conducted a group interview of Robert Klein, campaign chair for the Calfornian Proposition 71 embryonic stem cell funding initiative, Brook Byers, partner at venture capital firm Kleiner Perkins Caufield Byers, and Susan Desmond-Hellman, president of product development for Genentech. The general area of discussion was about biotechnology and medicine. These three interviewees all agreed on one very important point: only innovation can solve the problems caused by high and rising health care costs. It was gratifying to see these figures make an argument that is familar to readers of my blogs. Scientific and technological advances will be the ultimate solutions to the rapidly rising costs of medical care.
The view that innovation is the solution to our health care cost problems is not a new one. The late Lewis Thomas M.D., who was director of the Memorial Sloan-Kettering Cancer Center and the author of many essays on biology, was making that argument decades ago. Later in his life in 1992 Thomas repeated the argument that only scientific advances can produce the treatments that will make the major diseases of our time cheap to treat.
To be sure, we do have some spectacular surgical achievements in the headlines--the transplantation of hearts, kidneys, livers, and the like--but these are what I have called halfway technologies, brought in to shore things up after the still- unexplained diseases of these organs have run their course. And these measures, plus the new advances in diagnostic precision, account for a large part of the escalating costs of health care today. It seems obvious, to me anyway, that the only practical policy for bringing down those costs will be by more and more basic research in biomedical science, in the hope and expectation that we can then begin to understand, at a deep level, the underlying events in human disease. Sooner or later I am confident that this will be accomplished, and I hope for the sooner.
In one of his Lives Of A Cell books (this is an old memory, forget which one, probably Lives Of A Cell: Notes Of A Biology Watcher) published some time in the 1970s Thomas noted that before the advent of effective treatments of tuberculosis the many tuberculosis sanitariums were very expensive to operate. But he pointed out that once effective drugs against TB were developed the vast bulk of the sanitariums were quickly shut down as their patients were quickly and cheaply cured of TB. Thomas contended that expensive treatments are expensive because they fail to fix the underlying causes of diseases and that just about any really effective treatment is going to be pretty cheap. I agreed with him then and decades later I've grown only more confident that he correctly saw the fundamental problem (far more important than tort law, regulations, tax law, government entitlements programs, or market failures) that that is the root of high medical costs.
"In my own early professional life when I was an intern on the wards of Boston City Hospital the major threats to human life were tuberculosis, tetanus, syphilis, rheumatic fever, pneumonia, meningitis, polio, septicemia of all sorts. These things worried us then the way cancer, heart disease, and stroke worry us today. The big problems of the 1930s and 1940s have literally vanished."
A few of those diseases have made a come-back of sorts. But all are still problems of much lower orders of magnitude than they were a century ago. But what is most important to note is that when effective treatments were found for them the costs of preventing and treating them became a small fraction of the costs that those diseases previously inflicted on their victims, families of victims, and the rest of society.
Aside: One surprise in the Charlie Rose show discussion came from the venture capitalist Brook Byers. Byers says there are multiple groups in the United States and England that are working on nanoscale-level devices for very rapidly and cheaply sequence whole human genomes. Byers expects this problem to be solved in a few years.
The solution to the problem of cheap DNA testing will lead to much lower costs for drug development. How? Well, one way is that during clinical trials it will be possible to use smaller sets of trial participants to check out which genetic patterns each drug works best and worst with. Also, many drugs are held off of the market because they are too harmful to some subsets of society who have particular genetic sequences. Clinical trial participants who have adverse reactions to new drugs will be able to have their DNA sequences checked to see if genetic causes for the adverse reactions can be identified. Then drugs that would otherwise never be sold to the public can be approved for use with subsets of the population who are genetically compatible with those drugs. We will each have drugs that we can't use due to some genetic variations we each have. But more drugs will be able to make it onto the market than can be approved today.
The ability to quickly identify which genetic sequences make some people have adverse reactions to a given drug or to have much higher responsiveness to a drug (e.g. because their liver doesn't break it down as quickly) will also allow drug developers to more quickly and easily puzzle out the mechanisms by which the drugs vary in how they interact with different people. The knowledge of the genetic variations involved in drug response differences will allow drug developers to build animal models of genetic variations that cause different reactions to drugs and to design drugs that eitehr work with a larger set of genetic variations or to design different drugs for different genetic variations.