The Future of Gene Therapy

People were scandalized, but they should have known better. Medical science and the pharmaceutical industry are businesses. They exist to make a profit. If they did not make a profit they would not long be in business. And they would not be doing the groundbreaking research that has given us gene therapy.

Evidently, they make more money from extended treatments than from one-shot miracle cures. Recently, the New York Times reported that some people might have to take anti-depressants for life, At least, SSRIsy are presumed to provide a therapeutic benefit. Psychoanalysis, on the other hand, does not produce any real therapeutic benefit, but has parlayed its failures into endless treatment. It may not be good for you, but it is a good business model.

Similar questions are now being raised about gene therapy. When a Goldman Sachs analyst asked whether one-shot cures for extremely rare genetic conditions could make for good business, people were horrified. They did not know that many pharmaceutical manufacturers choose to dedicate their research efforts to diseases that afflict more, not fewer people. This feels reasonable and caring. Why cure two people when you can find a drug that cures two million.

But it also assumes that we cannot do everything. Yet, if your child is one of two people with a genetically based illness, I doubt that you will feel consoled by the fact elementary economics tells us that we must choose where we put limited research dollars.

Anyway, the truth, as reported by the MIT Technology Review (via Maggie’s Farm) is that pharmaceutical manufacturers either avoid new gene therapies or price them at millions of dollars a treatment—passing the buck to insurance companies. And thus, dividing the risk. After all, if treatment costs millions, and if only three patients need the treatment, insurance companies will probably not have too much trouble paying it out. If it costs millions of dollars and millions of people have the illness, it’s a different story.

And yet, for the pharmaceutical manufacturer, spending millions to develop a treatment for an illness that afflicts a half-dozen people is not good business, no matter who is paying for it, and no matter how much they charge.

The Review explains a recent decision by GlaxoSmithKline:

Just today, we saw GlaxoSmithKline sell off its pipeline of gene therapies for rare disease to a London startup called Orchard Therapeutics for a 20 percent stake in the young company.

The treatments Glaxo didn’t want were bona fide miracles: one-and-done cures that replace a broken gene and save a life.

One was Strimvelis, a therapy for a rare immune deficiency that’s been curing kids outright.

Another, still in development, is a DNA fix for a devastating childhood disorder called metachromatic leukodystrophy, which affects the brain. Victims lose their ability to talk, walk, and think before losing their lives.

The economic problem is that companies can run out of patients as they’re cured. Or they may not have enough of them in the first place, if the gene therapies treat exceedingly rare diseases.

For instance, only a couple of dozen kids each year in the US and Europe are diagnosed with the type of “bubble boy” disease that Strimvelis treats, called ADA-SCID. So even at a price of $665,000, Glaxo didn’t see that the drug was going to be much of a business.

Or else, consider the case of Novartis:

Other gene cures could be made more valuable if prices are a high enough. On April 9, Novartis said it would pay almost $8.7 billion to acquire AveXis, owner of a gene therapy that’s been curing kids with spinal muscular atrophy.

That disease is 20 times as common, so there's a larger market. Yet Novartis’s projections that the treatment could bring in several billion a year in revenue also suggest that the company might charge unheard-of prices, perhaps $2.25 million according to Wall Street bankers.

Should we consider it fortunate that spinal muscular atrophy is 20 times more common than “bubble boy” disease? It’s a grim calculus, made even more grim by our knowledge that a large part of medical expenses today involve end-of-life care.

Recently, we learned that Barbara Bush had decided to forgo any further medical care in favor of comfort or palliative care. And we recently reported on Barbara Ehrenreich’s new book, wherein she suggests that she is done with preventative tests.

The treatments provided in the last six months of life extend life a little and perhaps make it more livable… even though many of these treatments do not even do that. And now, we spend fortunes on everyone’s grandparents and are wondering about how we are going to pay for an expensive treatment for an illness that afflicts a dozen children. It seems easy until you ask: how many diseases afflicting a very small number of individuals exist?

It feels like an easy question, but it is likely more difficult than we think. At the least, it’s worth raising.