Arnold-Funded Sickle Cell Report Filled With Errors

August 20, 2019 1:40 pm

Last week we examined misstatements made by Patients for Affordable Drugs (P4AD) founder and President David Mitchell, including a claim about taxpayer-funded research. It will come as no surprise that Mitchell’s rhetoric permeates a recent P4AD report on research into treatments for sickle cell disease.


Just as Mitchell did, the report misunderstands the nature of government research and industry research. Government researchers conduct basic science – they do not develop novel drugs. (In fact, to our knowledge, the federal government never has fully developed a Food and Drug Administration-approved drug.)


Industry then conducts applied science. Both components are extremely important, but the latter is what brings new treatments to market. Once more, we go back to John LaMattina’s explanation (because it is so good): basic research is the first mile of a marathon. Applied research is the last 25.2 miles. 


Federal law not only allows for this relationship; it encourages it. Under legislation enacted in 1980, private entities may access basic inventions made during federally-funded research in order to conduct applied research and development. The law, which was passed with broad bipartisan support, enables the work done in government labs to be developed into real-life products, including approved therapies that reduce suffering, lengthen the lives of some patients and cure others.


As an example, let’s take the discovery by the National Institutes of Health of a new gene in Alzheimer’s progression. This discovery – again, while incredibly important – is not a drug. Inventing a drug, and ensuring it is effective and safe, will take many more years and millions more in investment. (But we agree: a cure for Alzheimer’s cannot come fast enough.)


Another way to look at it is that basic research from the NIH creates a hypothesis for future work. Testing that hypothesis is high risk and capital intensive, and thus can only happen found outside a resource-restricted environment like a government lab.


Besides misunderstanding (or at least deliberately ignoring) the difference between basic and applied research, the P4AD report ignores how much is spent on basic research versus applied.


Average basic research grants from the NIH generally are less than a half a million dollars. Within those grants, it is very difficult to estimate how much is directly attributed to the basic biology and understanding of the disease being investigated. In contrast, a company might spend a billion and a half dollars in direct costs to develop a single drug – much of this spent in the clinical trials phase.


Here is another way to calculate the number: taking into account the entire NIH budget, which includes construction costs, education, overhead, and other administrative expenses, the total amount that could potentially be attributed to basic research is $33 billion. That is less than half of what the pharmaceutical sector spends on research and development alone every year.


In addition to overlooking this point, the P4AD report makes enormous assumptions. Most of the grants listed in the paper are so broad that there is no way to break down what portion the funding went to viral gene therapy development for sickle cell. For example, a grant titled “Developmental Biology of Human Erythropoiesis” is listed for $50.9 million dollars spread over 33 years. This grant could cover anything in developmental biology related to blood cells. Another grant, “Gene Therapy for Alzheimer’s Disease” is being counted for the sickle cell drug development even though it provides funding for Alzheimer’s research.


Patients for Affordable Drugs is not the only Arnold-funded entity that is attempting to undermine researchers trying to find treatments for sickle cell. The Institute for Clinical and Economic Review (ICER) announced last week that it “plans to assess the comparative clinical effectiveness and value of crizanlizumab (Novartis) and voxelotor (Global Blood Therapeutics) for the treatment of sickle cell disease.”


As this blog post explains, ICER likely will not put much stock in what patients with sickle cell have to say about industry innovation. But we will. Check out this video to discover what one patient told us.


Many companies – including those looking for innovative medicines to address sickle cell disease – have invested hundreds of millions of dollars in research and do not yet have a single drug on the market. That’s why news outlets call the search for these treatments “a gamble.”


Arnold-funded organizations can continue to ignore this risk, but U.S. policymakers should not.