World’s most expensive drug could be free for some in lottery

Novartis may give away a new drug with a price tag above $2 million for free to some patients using a lottery-style program. The drug, Zolgensma, is a cure for a deadly inherited disease, spinal muscular atrophy (SMA), and the world’s most expensive drug.

It was approved by the FDA in May 2019. The news of the lottery system was first reported by The Wall Street Journal.

The high price tag brought attention to the drug when it was approved earlier this year, though Novartis maintains the price is on par with other gene therapies. U.S. medication prices cost about 300% more than the global median. In addition, Zolgensma is intended only for a small population affected by SMA. The drug could be free for a small population outside the U.S. under the global managed-access program.

Still, Novartis’s approach is different, as most companies that give away experimental drugs not approved in other countries do so under compassionate use programs that consider requests on a case-by-case basis. High demand for Zolgensma led Novartis to the lottery-style system. If production allows, Novartis aims to distribute 100 free doses annually.

See the full story below:

Amy Baxter

Amy joined TriMed Media as a Senior Writer for HealthExec after covering home care for three years. When not writing about all things healthcare, she fulfills her lifelong dream of becoming a pirate by sailing in regattas and enjoying rum. Fun fact: she sailed 333 miles across Lake Michigan in the Chicago Yacht Club "Race to Mackinac."

Around the web

Given the precarious excitement of the moment—or is it exciting precarity?—policymakers and healthcare leaders must set directives guiding not only what to do with AI but also when to do it. 

The final list also included diabetes drugs sold by Boehringer Ingelheim and Merck. The first round of drug price negotiations reduced the Medicare prices for 10 popular drugs by up to 79%. 

HHS has thought through the ways AI can and should become an integral part of healthcare, human services and public health. Last Friday—possibly just days ahead of seating a new secretary—the agency released a detailed plan for getting there from here.