Instead, the rare disease community finds itself under assault as little more than front groups for pharma companies who – critics claim – want the freedom to bankrupt our health care system by charge hundreds of thousands of dollars for medicines that aren’t that safe or effective.
The critics are attacking patient groups for strategic reasons: shut down patient groups or at least neutralize their influence and their organizations – anointed as objective and expert – will consolidate their power over the development, pricing and use of new medicines.
One of the leaders of this movement is Steven Pearson, the founder of the Institute for Clinical and Economic Review – an organization that claims it’s “a trusted non-profit organization that evaluates evidence on the value of medical tests, treatments, etc.” Pearson claims the influence of patient groups will drive our health care system and economy into bankruptcy. As the number drugs for rare diseases for small groups of patients increase, their higher prices (relative to medicines for common medical conditions) will become unaffordable. And the driving force is the ability of rare disease groups to advocate for new medicines.
Or as he puts in an article entitled, “Which Orphans Will Find a Home? The Rule of Rescue in Resource Allocation for Rare Diseases,” publicity can be a powerful and important tool for advocacy groups, “but it is not an appropriate ethical justification for coverage of particular orphan drugs over others.” Pearson writes, our nation needs a framework that will restrain “society’s desire to help those weakest among us, especially when their small numbers allow us to see them as unique individuals.”
Armed with this noble sentiment (and about $5 million from health insurers and the Laura and John Arnold Foundation) Pearson is positioning ICER to develop this ‘value framework.’
In May, ICER will be meeting with patient groups and others to recommend “fair prices that reflect the value of orphan drugs to patients and the health system to allow for broader insurance coverage for innovative new treatments.” (It is targeting Spinraza™ (a new drug for spinal muscular atrophy and Exondys-51™ for Duchenne muscular dystrophy (DMD))
Pearson believes that a “bright line between what constitutes a fair claim on health benefits and what does not will be difficult to draw.”
To ICER and Pearson, that bright line is $150000 for an additional year of life. Most new medicines for rare diseases are expensive and don’t save health insurers money. And ICER measures value from the insurer or government health program perspective. So most new orphan drugs would have been discounted more than 90 percent of the rebated price a medicine just to stay behind Pearson’s bright line.
Next, ICER sets a limit of $915 million on what should be spent on each new drug. It multiplies the price of the drug by the number of people who could benefit. Going over the cap mean that many people with rare diseases will be denied access to a growing array of new medicines. ICER uses these bright lines to “improve affordability” with changes to pricing, payment, or patient eligibility
ICER justifies such limits because beyond that cap it “…we’re siphoning off resources for other things we need like better schools and more resources for local police, roads, and bridges. “
These claims of budgetary Armageddon are overhyped. Between 2007 and 2014, orphan drugs have increased as a percentage of spending on drugs (from about 2 percent to 4 percent in Europe and 5 percent to 8 percent in the United States) even as the percentage spent on drugs has remained the same. And a study by Dr. Frank Lichtenberg shows new medicines for rare diseases are reducing the number of life years lost by about five percent a year.
Conversely, ICER’s limits on access will hurt people and rob them of their lives. Lichtenberg’s study found in France, which took longer to pay for fewer orphan drugs relative to the US, the number of deaths declined by 1.8 percent.
It is precisely our moral sense to save lives in immediate danger and at any expense that sustains humanity and economic progress. We need more orphan drugs not just because more people will be able to enjoy life and live longer. It’s because civilization is enriched when we provide people who are marginalized because of their medical condition the opportunity to contribute to our well-being and happiness.
Pearson and ICER are a threat to that moral vision. They threaten the remarkable advances in medicine that Rare Disease Day celebrates, made possible in large part by the patient advocacy groups that ICER seeks to replace.