Those diagnosed with a rare disease forever remember those initial moments. Shock, of course, followed by questions. Is there a cure? Or at least a treatment?
In the vast majority of cases, the answer is, sadly, “no.” Of the more than 7,000 rare diseases, 95 percent lack an FDA-approved treatment.
My company is dedicated solely to identifying, developing, and delivering life-changing therapies for rare diseases. And despite the many challenges we’ve faced — the path in rare disease is never easy — we’ve made encouraging progress toward treating rare kidney disease.
But last year, a new obstacle emerged — thanks to policymakers in Washington.
A provision of the Inflation Reduction Act (IRA) would needlessly close down some of the most promising avenues of rare disease research. Had the law been in place just a few years ago, the breakthroughs achieved might not have been possible.
Fortunately, lawmakers have a chance to correct this with the ORPHAN Cures Act, a bipartisan reform currently before Congress. It would remove a critical barrier facing companies wanting to invest in rare disease research, giving hope to those affected for a life-changing treatment, or even a cure.
My commitment to rare disease research started two decades ago, when my doctor said I was the seventh person he had ever known to be diagnosed with a rare form of cancer. I was also informed that if surgery didn’t eliminate the tumor, there was nothing they could do. Drug treatments simply weren’t effective.
As I quickly learned, my experience is all too common. By definition, a rare disease afflicts fewer than 200,000 Americans — that’s less than 1/10th of 1 percent of America’s population. Some of these conditions affect only a few dozen patients.
Finding investors willing to finance the development of treatments for such small populations can be incredibly difficult.
Despite these impediments, my company, Travere Therapeutics, has earned FDA accelerated approval for a rare kidney disease treatment and is pursuing a second approval for a different rare kidney disease.
These successes might not have been possible under a law like the IRA.
The IRA’s drug-pricing program allows the federal government to dictate the price Medicare pays for certain brand-name drugs. Recognizing that this policy would likely discourage investment, lawmakers took care to exempt rare disease therapies, also known as “orphan drugs,” from government price setting.
Unfortunately, this exemption only applies to medicines approved for a single disease. Any orphan drug approved for two or more diseases — even if it is a rare disease with no treatment — becomes eligible for government-mandated price setting. It’s this feature of the law that is so destructive.
One common reason companies can decide to pursue rare disease treatments is the promise that a successful drug might prove effective for multiple conditions. For instance, a treatment that successfully defeats one type of rare cancer might work on another type of tumor, or a drug that addresses one autoimmune condition might prove similarly effective on others. When a disease is rare, scientific progress that saves lives often depends on this approach.
Drug innovation is a highly risky enterprise, after all. It can cost billions of dollars to develop a new drug, particularly when you take into account the cost of unsuccessful programs. And only about 12 percent of new medicines that enter clinical trials ultimately earn approval.
The possibility that a line of research might produce a treatment for more than one disease helps reduce some of that risk, making it easier to attract investors. So by discouraging work on multiple rare diseases, the IRA creates significant new obstacles to developing innovative new medicines.
The provision actually deters companies from searching for new uses for drugs they’ve already developed. After all, why would a company perform expensive clinical trials to investigate new potential uses in a rare disease if a second approval from the FDA will only lead to price controls? A recent study by the Health Capitol Group from the Rare Disease Company Coalition showed that “limiting the average drug in the clinic to just one orphan indication would reduce the number of expected patients treated by over 24%.”
Congress can prevent this from happening by passing the ORPHAN Cures Act. The reform lightly revises the IRA so that all orphan drugs — and not just single-disease therapies — are exempt from the law’s pricing program.
It’s not often that a minor change in a law’s text can save countless lives. But that’s why the ORPHAN Cures Act enjoys such broad, bipartisan support. For the sake of the 30 million Americans living with a rare disease, Congress needs to pass this bill as soon as possible and clear the way for medical discoveries that patients can’t live without.
Eric Dube is the president and CEO of Travere Therapeutics.