Infants with spinal muscular atrophy may soon have two treatment options.
The Food and Drug Administration is poised to approve a second gene treatment for infants with SMA. With a price tag of $1.5 million to $5 million, it will be the most expensive drug on the planet. Above, a girl with SMA and her mother take a walk.
In this case, it has featured claims and counterclaims of safety and cost effectiveness at the pinnacle of U.S. biopharmaceutical development. It spawned a Washington public relations campaign of mysterious origin, featuring what independent communications experts characterized as coordinated op-ed columns by former Cabinet secretaries warning about the safety of the Novartis therapy.
How the medical system handles these questions of safety, effectiveness and price will set a precedent for future battles. The FDA is poised to review more applications for gene therapies for inherited diseases such as hemophilia and Duchenne muscular dystrophy. Neither drug works uniformly in children, and even the best outcomes leave kids with some muscle weaknesses. Still, Novartis is promoting its drug as a bigger breakthrough.
The company is highlighting the drug’s positive safety profile, which is not only strong but also far better understood . At the same time, the company is attempting to sow doubt about the safety of the Novartis drug by highlighting the new product’s small clinical trials. Biogen also is raising questions about two infant deaths in those trials.“Forever is great if you’re getting nothing but benefit, and forever is not so good if there are safety issues,’’ Alfred Sandrock Jr.
The scientific issues were drawn into the political sphere in recent months by high-profile authors: Tom Price, President Trump’s former secretary of health and human services, and Bill Richardson, former energy secretary in the Clinton administration. Their names appeared atop two of five published columns that made strongly similar assertions about potential health dangers associated with the Novartis treatment.
Then Price’s article, also citing Gelsinger’s death and warning of the risks of gene therapy, appeared in U.S. News & World Report on April 10. Price, who was HHS secretary for less than a year before he resigned in 2017 amid questions about his government-paid travel, asserted the Novartis treatment should not be “rushed to market unchecked.’’
Sandrock, Biogen’s chief medical officer, said he was not aware of the articles and had no knowledge of how they came into existence. Later, Biogen, through a spokeswoman, offered a partial denial. Richardson did not respond to additional questions about who, if anyone, asked him to write the article, where he got the information it contained, or if he had any contact with an intermediary who could have been acting as an agent for a company.
Rare-disease treatments typically are not the sort of drugs that trigger accusations of price-gouging in Congress, like unwarranted spikes in generic prices. Also, with only a few hundred new patients a year, the costs are not going to break the health-care bank.
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