that matter to patients, including functional status, quality of life, or length of life.”
The media coverage of this event will, if past reporting is any indication, ignore the science, ignore the perspectives and courage of the patients who have benefited from Exondys, who have found the medicine to be a source of freedom and hope. Instead, it will focus on the fact that patient groups receive money from companies who have developed such medicines.
In doing so, such journalists are seeking to marginalize the plight of people with rare, degenerative conditions, tarring them as mere tools of the evil Big Pharma while exalting groups such as ICER, ally the while ignoring the massive amount of funding ICER receives from former Enron trader, John Arnold. Indeed, some journalists are being paid from money John Arnold has given their publications.
The demonization of small patient groups as somehow being mere mouthpieces for drug companies is consistent with the narrative that pharma funding of organizations who would otherwise have no voice is illegitimate. And it also sidelines a more serious discussion of whether patients believe Exondys work.
The fact is, ICER ignores the value of orphan drugs and media coverage of funding enables such willful ignorance. ICER claims that though Exondys has “ been on the market for three years and yet we still found notably inadequate data on patient outcomes.”
Bet on journalists such as Kate Sheridan from STAT, Peter Loftus from the Wall Street Journal and Jonathan Saltzman from the Boston Globe accepting ICER’s assessment at face value and devoting more time attacking the patients who are justified in taking exception to such smears.
Indeed, these same reporters will side with ICER. By demonizing patient groups for receiving pharma funds, these journalists legitimize ICER’s refusal to take into account the impact of such medicines on patients and their families.
While ICER is welcome to conduct its assessment of our product or any other orphan medicine using its own set of value and methods, I know for a fact that ICER excludes many, if not most, of the information
and considerations that insurers, doctors and patients took into account in determining how Exondys should be reimbursed.
ICER assumes that everyone with DMD lives with the disease in the same way. Indeed, ICER favors those with more treatable conditions and those with greater potentials for health—be it in terms of functioning or longevity. Its founder noted in an article entitled “Which Orphans Will Find a Home: The Rule of Rescue in Resource Allocation for Rare Diseases” that there is no apparent obligation to rescue identifiable rare disease patients based on a duty of rescue within personal morality.”
In evaluating the value of Exondys and other medicines for rare diseases, ICER claims to take into account that to people with the most serious rare diseases a small improvement in well-being is important. Yet in determining this value – on behalf such patients – ICER states: “The opportunity cost of supporting the use of ultra-orphan drugs necessitates that patients with a more common disease, for which a cost-effective treatment is available, are denied treatment.”
In other words, ICER’s assessment of orphan drugs is shaped by its desire as Pearson writes, to “ensure that an undue burden is not placed on others for the sake of a few.” More specifically, ICER claims that paying for orphan drugs means “…we’re siphoning off resources for other things we need like better schools and more resources for local police, roads and bridges. “
These assertions are morally repugnant and factually misleading. Why should the cost of orphan drugs be pitted against spending on other forms of care for most other patients? Why not pit the high cost of hospitalization relative to medicines for so-called common diseases? What about the nearly $400 billion that is spent each year on ineffective or needless care? Indeed, to the extent that ICER does not evaluate whether each additional dollar spent on police, roads and bridges generate more benefit using
the same methods it applies to people with rare diseases, such trade-offs are both arbitrary and ideological.
Ultimately, what ICER fails to measure is what better health provides people living with orphan diseases: human dignity that is made possible when every individual, no matter how rare their condition has the same opportunity to live a full and long life. The price of Exondys reflects the size of the community with DMD and an indication of the importance of that social contract. But it also measures what ICER does not: The effect of increasing the freedoms – the capabilities – to choose to do and be more of what people value.
As Nobel Prize-winning economist Amartya Sen points out: groups that weigh the cost and benefit of helping people with serious illnesses against the total cost of health care, do so “without taking any direct interest in freedom, rights, creativity or actual living conditions. To insist on the
mechanical comfort of having just one homogeneous “good thing” would be to deny our humanity as reasoning creatures.”
Instead of focusing on the freedoms generated by medical innovation, ICER and its journalistic allies measure medical innovation in terms of how much money it “siphons off” from governmental and insurance budgets.
It does not measure “What is each person able to do and to be?" In other words, ICER measures an average health benefit (in QALY units) that has nothing to with increasing the capabilities and
opportunities available to each person.
A measure of what advances in medicine contribute to increasing our capabilities is focused on choice or freedom. Rather than budget impact, the value of medicines should be measured by how they contribute to each individual’s ability to do or be what they hope for. And unlike ICER, both the approach and methods of measuring value must be inclusive, taking into account that the capability achievements that are central for people are different in quality, not just in quantity.
Rejecting a price or price increase using the ICER standard is tantamount devaluing the lives of people such institutions are responsible for. ICER’s use of standard cost-benefit analysis does not capture the tragic choices people with rare disease must live each day. It does not capture the daily and accumulated costs of living with a fatal or degenerative disease that people with good health don’t have to bear. Nor does it capture what living a life with more capabilities means to individuals or our nation. The media is complicit in ICER’s implementation of soft eugenics.
Finally, ICER suggests limiting spending on any medicines if the total amount goes over a certain percentage of our GDP. This is not the first time some have made the GDP a measure of what’s important and ultimately valuing individuals in terms of whether they add or subtract to that total. Before the Nazis did so to justify sterilizing and killing people with hemophilia, depression, spina bifida or DMD, private foundations in the United States spent millions to support a similar program of extermination to save money and ensure that a small group of patients doesn’t impose a drain on society.
In 1968 Robert F. Kennedy discussed the danger of this approach. His words are as important today as they were over a half-century ago:
“Too much and for too long, we seemed to have surrendered personal excellence and community values in the mere accumulation of material things. Our Gross National Product, now, is over $800 billion dollars a year, but that Gross National Product - if we judge the United States of America by that - that Gross National Product counts air pollution and cigarette advertising, and ambulances to clear our highways of carnage. It counts special locks for our doors and the jails for the people who break them. It counts the destruction of the redwood and the loss of our natural wonder in chaotic sprawl. It counts napalm and counts nuclear warheads and armored cars for the police to fight the riots in our cities….
Yet the gross national product does not allow for the health of our children, the quality of their education or the joy of their play. It does not include the beauty of our poetry or the strength of our marriages, the intelligence of our public debate or the integrity of our public officials. It measures neither our wit nor our courage, neither our wisdom nor our learning, neither our compassion nor our devotion to our country, it measures everything in short, except that which makes life worthwhile. And
it can tell us everything about America except why we are proud that we are Americans.”
Ultimately medicines for rare diseases should be measured by the latter set of standards. And journalists who savage rare disease groups should care how they stack up against that moral measure. My guess is they won’t and will continue to give ICER the legitimacy it requires to advance an inherently evil agenda.