When the Economics Evaporate: A Persistent Challenge for Rare-Disease Patients
By ALDP Co-founders Michael Glassner and Jason Young
Last week brought a small but genuinely encouraging piece of news.
Blood Cancer United announced that it had acquired the remaining supply of Luvelta, an experimental cancer therapy that Sutro Biopharma stopped actively developing last year. The nonprofit will make the drug available free of charge through a compassionate-use program for children with a rare and aggressive form of acute myeloid leukemia who may benefit from it.
Patient advocate Nancy Goodman, founder of Kids v Cancer, called the arrangement “a terrific model for pediatric cancer drug development because the drug has been de-risked and so much has been invested in the drug in terms of research and development.”
We agree. Not because this story is unique, but because it isn’t.
Every debate about prescription drug pricing eventually arrives at the same question: What happens to patients with rare diseases when the economics become difficult? The answer is more complicated than many people realize, and it matters because those patients are often cited in debates over drug pricing, affordability boards, and transparency measures.
A challenge larger than any one company
The Luvelta story is not really about one drug or one company. It’s about a reality that patients with rare diseases, their families, and the organizations that support them have understood for decades.
Developing treatments for very small patient populations is hard. Clinical trials are harder to recruit for. Manufacturing costs are spread across fewer patients. Investors face greater uncertainty. Even when a therapy shows promise, the commercial pathway may be difficult.
That is why Congress created the Orphan Drug Act in 1983 and why Americans across the political spectrum generally support incentives for companies willing to pursue treatments for rare diseases. Those incentives have helped produce hundreds of therapies that might never have existed otherwise. But they do not eliminate a fundamental challenge: sometimes the economics simply stop working. When that happens, patients still need treatment.
We’ve seen this before
Luvelta is not the first example. In Europe, a gene therapy called Strimvelis was developed for ADA-SCID, an immune disorder so severe that even a common cold could prove fatal for a child living with it. After changing corporate ownership and strategic priorities over time, the therapy’s rights were ultimately transferred back to the Telethon Foundation, the nonprofit organization that had originally helped support its development.
Telethon became the first nonprofit in the world to manufacture and distribute a gene therapy after commercial interest had faded. That story wasn’t primarily about pricing policy. It wasn’t about transparency requirements. Nor was it about state-level affordability boards.
It was about a challenge that predates all of those debates: how to sustain treatments for extremely small patient populations when traditional market incentives become insufficient.
Why this matters now
Rare-disease patients are frequently invoked in debates about prescription drug pricing. Those concerns should be taken seriously. Patients facing rare diseases often have fewer treatment options, fewer alternatives, and more at stake than almost anyone else in the healthcare system.
But stories like Luvelta and Strimvelis are a useful reminder that the greatest threat to access is not always the one being discussed. Sometimes access challenges emerge because a promising therapy never reaches the market. Sometimes they emerge because development stops. Sometimes they arise because serving an extremely small patient population proves economically difficult, even with incentives designed to encourage rare-disease development. These realities need to be understood.
What ALDP believes
Americans for Lower Drug Prices has never argued that patients with rare diseases should bear the burden of broader drug-pricing reforms. We support continued innovation; incentives that encourage research into rare diseases; policies that preserve patient access to therapies serving small populations; and transparency.
Understanding how drug prices are set does not prevent innovation. Knowing how a price was determined does not stop a medicine from being prescribed. Transparency is not the same thing as restricting access.
In fact, greater transparency is likely to help policymakers better understand the genuine economic challenges associated with developing therapies for rare diseases and distinguish those challenges from broader pricing practices elsewhere in the market.
Those are distinctions that matter in such a consequential debate.
Looking ahead
The most encouraging part of the Luvelta and Strimvelis stories is not that a nonprofit acquired a drug. It’s that patients were not forgotten when the commercial pathway became uncertain.
Blood Cancer United and Telethon Foundation saw value where traditional market incentives no longer pointed in the same direction. That should not be viewed as an indictment of industry. Markets and nonprofits play different roles, and both are important.
But it is a reminder that protecting patients with rare diseases requires more than slogans, more than assumptions, and more than invoking vulnerable families in policy debates. It requires a clear-eyed understanding of the challenges they face. And sometimes, as the Luvelta and Strimvelis stories remind us, those challenges begin long before anyone starts arguing about drug prices.
Editor’s note: Readers interested in learning more about rare diseases, orphan drugs, and how those terms are used in drug-pricing debates can read ALDP’s companion explainer, “Understanding Rare Diseases, Orphan Drugs, and the Drug Pricing Debate.”