A Small Fix to the IRA Will Give Hope to Patients With Rare Diseases
For the roughly 30 million Americans living with a rare disease, the chances of finding an effective treatment are tragically slim.
Our healthcare system defines a rare condition as one affecting fewer than 200,000 patients around the country. Such small patient populations create enormous financial and practical barriers to developing cures and therapeutics.
Unfortunately, one element of last year's Inflation Reduction Act (IRA) is contributing to the challenge. As written, the law's provisions strongly discourage research into treatments for rare conditions -- and also discourage making the most out of the few treatments that do emerge from our labs.
The good news is that Congress can undo this unintended consequence by passing the ORPHAN Cures Act, bipartisan legislation introduced in the House and the Senate. The legislation would remove an unnecessary barrier to medical progress that Congress probably never intended to erect in the first place.
This is an issue I take very personally -- both as a physician and a leader of companies with years of first-hand experience bringing breakthrough medicines to patients -- including one of the most successful treatments for sickle cell disease yet discovered.
Developing medicines that treat rare diseases like sickle cell -- or "orphan drugs," as they're sometimes known -- is an extraordinarily difficult task. The science is especially challenging because we tend to know less about the etiology, development and progress of rare diseases.
No less daunting than the scientific challenges are the financial challenges. Investors are generally unwilling to spend billions of dollars developing a product that benefits so few patients. As a result, funding for these treatments is often scarce, and progress dispiritingly slow.
Right now, just 5% of rare diseases have an FDA-approved treatment. What's needed are government policies that incentivize work on the other 95% -- as well as improvements on the orphan drugs we already have.
Congress has long recognized this problem. In 1983, lawmakers passed the landmark Orphan Drug Act, which created additional financial incentives for developing treatments for rare diseases. We should be expanding those incentives. Unfortunately, the IRA as crafted does the opposite.
In order to reduce Medicare's costs, the IRA allows the government to demand price controls on a range of prescription drugs paid for through the program. So as not to stand in the way of orphan drug development, lawmakers attempted to exclude these medicines from the price-setting program -- but with one important exception. The rare-disease carve-out doesn't extend to treatments approved for more than one rare disease.
In other words, an orphan drug approved to treat one disease is exempt from Medicare price cuts, but if a scientist discovers a second rare disease application for the same drug, they face a steep financial penalty through loss of the exemption.
As a result, the IRA discourages researchers from investigating whether existing orphan drugs have additional uses or at best, it incentivizes delaying launching the drug as long as possible. This would deal an enormous blow to the rare-disease research community.
Of the more than 280 orphan drugs approved since 2003, nearly a quarter were later approved to treat additional diseases. Some medicines earned approval for four or more follow-on indications.
These are potentially life-saving discoveries that may not have occurred under the pricing system created by the IRA.
Lawmakers can fix this problem right now simply by passing the ORPHAN Cures Act. Introduced by Reps. John Joyce (R-PA) and Wiley Nickel (D-NC) in the House and Sens. John Barrasso (R-WY) and Tom Carper (D-DE) in the Senate, the two-page bill amends the IRA's drug-pricing section by changing the words "only one rare disease" to "one or more rare diseases." With that revision, the legislation vastly increases the odds of treating and curing many of the thousands of rare diseases that currently lack effective medications.
It's hard to believe the IRA's architects actually intended to derail research into rare diseases. Yet that's exactly what's happening. Lawmakers can reverse course and correct the problem by passing the ORPHAN Cures Act.
Dr. Ted W. Love is Chair of the Biotechnology Innovation Organization's Board of Directors, and former President and CEO of Global Blood Therapeutics.
