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Guest opinion: The cases for irrational research

This photo provided by Novartis shows Zolgensma. The one-time gene therapy developed by Novartis, Zolgensma, will cost $2.125 million. It treats a rare condition called spinal muscular atrophy, or SMA, which strikes about 400 babies born in the U.S. each
This photo provided by Novartis shows Zolgensma. The one-time gene therapy developed by Novartis, Zolgensma, will cost $2.125 million. It treats a rare condition called spinal muscular atrophy, or SMA, which strikes about 400 babies born in the U.S. each year. The therapy, given in a one-hour infusion, was approved for children under age 2. (Novartis via AP)

The Food and Drug Administration recently approved a novel, one-time treatment for the rare, muscle-wasting disease of childhood, spinal muscular atrophy, for which manufacturer Novartis is expected to charge $2.1 million per dose. From the standpoint of individual patients and their families, this potential cure for an otherwise fatal disease is clearly progress. But should the company be able to charge such a price — which will likely be billed to insurers in intervals over five years and then passed along via increased premiums to all health care consumers? And, more fundamentally, are such drugs cost effective? Should they be developed at all?

The driving force behind the development of treatments for most rare diseases in the United States is the Orphan Drug Act of 1983. This seminal statute aimed at addressing the dearth of research on diseases affecting relatively small numbers of patients — defined by the law as 200,000 Americans. To remedy this shortage, the ODA incentivized manufacturers with a combination of generous tax breaks, fast-track approval provisions and a seven-year period of market exclusivity, all with the goal of making such investments worthwhile for drug companies. One of the consequences are high cost treatments that benefit small numbers of patients. Prior to 1983, only 38 such drugs had received FDA approval. According to Shailin Thomas and Arthur Caplan, writing in JAMA, 503 such drug were approved between 1983 and August 2018.

Novartis’s drug, Zolgensma, offers an extreme example — although not as extreme as one might sense from media depictions, because spinal muscular atrophy is not that rare of a disorder. According to the SMA Foundation, the incidence is roughly 1 in 6,000 to 10,000 births — about half as many as cystic fibrosis or Duchenne’s muscular dystrophy. So this is not a treatment that will benefit a few dozen infants, but possibly many thousands.

Whether the treatment is cost effective — and how society measures that concept — is another matter entirely. The nonprofit Institute for Clinical and Economic Review reported that Zolgensma would be cost-effective for the more severe type of SMA under one common measure (quality adjusted life years) at a price of $310,000-$900,000 per treatment. These figures are lower than the proposed price tag — but not disproportionate to other pharmaceutical markups.

Leading commentators have recently raised concerns about an incentive system that encourages research on rare conditions at the expense of treatments for far more common diseases. What sense does it make, one might ask, to cure SMA when the same research dollars might be spent funding potential cures for Alzheimer’s disease or Type 2 diabetes. And where is the logic in paying millions for Zolgensma when the funding might save many more lives if expended upon flu shots and mammograms and smoking cessation programs? The individuals who do not receive the smoking cessation treatment are “invisible victims,” because they often do not think of themselves as having been refused lifesaving care, as would the SMA patients if insurers balked at paying for Zolgensma.

Maybe the strongest argument for expensive orphan drugs is that Americans do not want their health care dollars spent in purely rational ways. Our society gladly pays for costly, high stakes treatments like heart transplants and hepatitis cures that will save fewer lives than if those dollars were devoted to public health measures. We donate to send surgeons to Latin America to repair cleft palates in toddlers when the cost of one operation might provide nutritional support to an entire village. To be blunt, we do not wish to live in a society governed by the rational dictates of health care economists.

Fortunately, there is a stronger argument to defend such costly efforts — that our society does not have to choose between curing common diseases and rare ones. Research need not be a zero-sum game. In the wealthiest nation in the history of the world, it is mostly a matter of getting our priorities straight: Do we want to live longer, healthier lives? Or do we prefer more discretionary spending for consumer gadgets? We would be far wiser to persuade the American public, and their political leaders, to increase the size of the research pie, rather than arguing over the size of the individual slices.