Today, the Senate could determine the fate of millions of Americans suffering from rare diseases.
That might sound hyperbolic. But it's no exaggeration. Lawmakers have an opportunity to include a simple bipartisan reform in the budget reconciliation bill that the Senate is hoping to finalize today.
The reform would strongly incentivize biotech companies to invest in rare disease research -- at virtually no cost to taxpayers. Patients' health, and even their lives, depend on senators including the provision in the bill.
The measure in question is the ORPHAN Cures Act. It would fix a provision in the Inflation Reduction Act that has unintentionally caused investment into rare disease treatments to plummet.
The IRA allows Medicare to negotiate the price of brand-name drugs after they've been on the market for a set number of years. But it carved out an exception for "orphan drugs" -- treatments for rare diseases that affect fewer than 200,000 Americans -- since lawmakers recognized that price controls would discourage investment in this high-risk, often low-reward area of research.
But the IRA only exempts drugs approved for a single rare disease. If a company finds that the same therapy could help patients with a second rare disease, the drug becomes subject to price negotiations -- often before companies have recovered their R&D costs. That's a huge disincentive to pursue additional research, even when the science is promising.
And the damage is already evident. One analysis found a 48% decline in research into additional indications for orphan drugs since the IRA passed. That's not a statistic. That's children, families, and adults being told there's no hope -- not because the science failed, but because the incentive to pursue it was stripped away.
The ORPHAN Cures Act would fix this by exempting all orphan-only drugs from IRA price controls, regardless of whether they treat one rare disease or several. It also clarifies that the countdown to negotiation eligibility doesn't begin until a drug is approved for non-orphan use.
This reform would make an enormous difference for the 30 million Americans living with rare diseases, 95% of which have no FDA-approved treatments at all. Yet it represents a rounding error in the federal budget over the next decade. According to a Congressional Budget Office estimate, the ORPHAN Cures Act would increase federal spending by about $500 million a year, on average, over the next ten years.
That annual cost is roughly what the government spends every 38 minutes.
And in the long-run, failing to treat rare diseases would prove more expensive. It'd mean higher emergency care costs, greater burdens on caregivers, and shorter lives for patients.
Patients across the country are hopeful that the Senate will urgently move the Orphan Cures Act forward -- let's hope lawmakers make the right choice.
Pam Squires is the executive director of the Eosinophilic & Rare Disease Cooperative.
That might sound hyperbolic. But it's no exaggeration. Lawmakers have an opportunity to include a simple bipartisan reform in the budget reconciliation bill that the Senate is hoping to finalize today.
The reform would strongly incentivize biotech companies to invest in rare disease research -- at virtually no cost to taxpayers. Patients' health, and even their lives, depend on senators including the provision in the bill.
The measure in question is the ORPHAN Cures Act. It would fix a provision in the Inflation Reduction Act that has unintentionally caused investment into rare disease treatments to plummet.
The IRA allows Medicare to negotiate the price of brand-name drugs after they've been on the market for a set number of years. But it carved out an exception for "orphan drugs" -- treatments for rare diseases that affect fewer than 200,000 Americans -- since lawmakers recognized that price controls would discourage investment in this high-risk, often low-reward area of research.
But the IRA only exempts drugs approved for a single rare disease. If a company finds that the same therapy could help patients with a second rare disease, the drug becomes subject to price negotiations -- often before companies have recovered their R&D costs. That's a huge disincentive to pursue additional research, even when the science is promising.
And the damage is already evident. One analysis found a 48% decline in research into additional indications for orphan drugs since the IRA passed. That's not a statistic. That's children, families, and adults being told there's no hope -- not because the science failed, but because the incentive to pursue it was stripped away.
The ORPHAN Cures Act would fix this by exempting all orphan-only drugs from IRA price controls, regardless of whether they treat one rare disease or several. It also clarifies that the countdown to negotiation eligibility doesn't begin until a drug is approved for non-orphan use.
This reform would make an enormous difference for the 30 million Americans living with rare diseases, 95% of which have no FDA-approved treatments at all. Yet it represents a rounding error in the federal budget over the next decade. According to a Congressional Budget Office estimate, the ORPHAN Cures Act would increase federal spending by about $500 million a year, on average, over the next ten years.
That annual cost is roughly what the government spends every 38 minutes.
And in the long-run, failing to treat rare diseases would prove more expensive. It'd mean higher emergency care costs, greater burdens on caregivers, and shorter lives for patients.
Patients across the country are hopeful that the Senate will urgently move the Orphan Cures Act forward -- let's hope lawmakers make the right choice.
Pam Squires is the executive director of the Eosinophilic & Rare Disease Cooperative.
