Gene therapy is expensive. But so are misleading attacks.

by Anthony Davies

**I am aware that I’m posting this on the dawn of the American election event that is Super Tuesday, 2020. I do not intend my comments here as any sort of endorsement of any of the candidates. Instead, I consider this is an equal opportunity point of clarification. Politicians the world over simplify information in order to get attention, true, but misinformation is a more dangerous game.**

I suppose we who work in cell & gene therapy¹ should be honored, in some backwards fashion, to have been singled out for criticism in a tweet from a U.S. presidential candidate earlier this year. Regardless of source or political leaning, however, I refuse to accept staggering inaccuracies from those who wish to lead. Before a presumptive Democratic nominee is decided and the talking points of the 2020 American presidential election solidify, I’d like to take the opportunity to correct this particular set of misunderstandings.

I am speaking of Bernie Sanders’ January tweet about BioMarin’s upcoming therapy to treat haemophilia, in which the Senator threatens BioMarin’s patent and takes aim at what he mischaracterizes as the “world’s most expensive drug.” (Pay attention also to his reference to this new therapy costing $2-3M “a pop.”)

First, to acknowledge the obvious. Are healthcare costs in the U.S. a disaster? Yes. Americans’ very existence is a blur of crowdfunding requests, medical bankruptcies, a huge uninsured population, and an even larger “insured” population that cannot afford to use that insurance.

But does that mean that all expensive therapies are to blame? No. If we are to critique life-saving measures and drug innovation, we need to do so responsibly and with precision rather than exploiting the public with inflammatory, misleading soundbites. Using BioMarin’s haemophilia therapy as our starting point, then, let’s examine three facets of this issue: (a) the relative costs of treating the disease, (b) the role the patent plays in our society, and (c) where reform is both more necessary and more appropriate.  

First, let’s consider (a), the cost of treatment options for haemophilia A. This condition—in which a patient can bleed to death from even a small cut due to their blood’s inability to clot—affects approximately 20,000 Americans and is currently treated with an enzyme replacement therapy. That therapy costs our healthcare system $270k per year, per patient. A patient with this condition will live an average of 70 years (post-diagnosis) if the disease is managed.² Multiply $270k by 70 years and we get a lifetime price tag of $18.9M per patient.

In contrast, BioMarin’s therapy (for which results during clinical trials have been overwhelmingly and consistently positive²) costs $2-3M. Period. End of story. The difference between these types of drugs is that a gene therapy is a one-and-done treatment, not something that a patient continually receives. So, while it certainly is much more expensive per “pop,” that one “pop” is all it takes.³

Imagine a tweet in which a political figure hailed a new drug that would allow us to treat six or nine patients (and more effectively, at that) for the previous cost of treating one patient. The reception would be overwhelmingly positive, and for very good reason. But reviling Big Pharma garners more attention, more likes and retweets, and so the disinformation spreads.

On to (b), aka, the concept of revoking a patent, another popular political talking point. I have two arguments against this consideration: a conceptual one and a concrete one.  

Conceptually, we know why patents exist: to fuel innovation by rewarding innovators, giving them the chance to recoup their costs as well as make a profit. (“Profit” has developed negative associations, but it is only a bad word when it’s misused: when one profits unfairly at the expense of the many. A life-saving, money-saving drug does the opposite.)

Patents are not a new concept designed to further line the pockets of the rich, nor do they cause a monopoly; they are, instead, one of the linchpins of our civilization. The concept of patents and intellectual property dates back at least to the 1400s; almost every element of our daily experience was once patented. Nearly 600 years of human history, at least, is predicated on an understanding that those who innovate will be compensated for their discoveries. If we start punishing innovation, it goes away, and with it goes our ability to move our knowledge and our cures forward for the good of all.  

Now, for the concrete argument against removing patents. If a patent for such an extraordinarily complex therapy is revoked, no competitor will be able to copy the process in a reasonable time frame. And if they were able to? Well, that’s where the laborious but critical proof of equivalence comes in: no generic is allowed to be sold unless it can prove itself comparable to the original product. That hurdle exists to protect the patient/consumer against false claims for inadequate products. (Your drugstore ibuprofen works just like your Advil® does, which is why it’s safe to purchase and use it in the same manner.) So, if a cheaper generic version wouldn’t be accessible anyway, why bother to unnecessarily disincentivize innovation?

And finally, on to (c): options I would recommend in order to affect more meaningful and lasting change.

• First, pharmaceutical companies are on a pricing spree that must be tamped back. In 2019 alone, the average price hike was over 10%, or five times inflation. Some degree of increase is necessary, of course—I would even argue that increasing beyond the rate of inflation is appropriate given that with each passing day on the market, the efficacy of a drug is proven further. But 10% annually has gone too far.
• Those same companies are employing sleight-of-hand to extend the life cycle of patented drugs. Just as I firmly believe that patents must be protected, I also intensely respect the limitations of a patent. If a company fakes their way to keeping that patent longer than they should, then they are taking wildly unfair advantage of the market and of the patients they claim to be serving.
• Related to the two points above, drugs cost more in the U.S. than in other countries. U.S. regulations correctly prize safety above all, but do not adequately emphasize cost-effectiveness…and our drug companies face limited competition. And within the U.S., the prices for any given Rx lack transparency and, above all, consistency.

We can do better, and we must. The cost of being unwell in this country has spun wildly out of control.

But in order to take appropriate measures to stop Americans from devastating consequences (both financial and health-related), we must hold our politicians and ourselves to a higher standard. We must do the work of becoming informed and insist that those who speak for us—whether on the right or on the left—do the same.

¹ Do I work in this, the space that generates the most expensive therapies around? Yes, I do. And I do so because cell and gene therapy is where we can save lives in a more efficacious and ultimately cost-effective manner. Show me the cell or gene therapy that isn’t designed to ultimately save money vs. a current treatment and…well, you can’t, because there isn’t one.

² The current enzyme therapy option still saves lives, but it leaves room for improvement beyond pricing alone. The patients in BioMarin’s trial had 16.5 “bleeding incidents,” (in which a patient’s blood fails to adequately clot) per year on enzyme replacement therapy prior to treatment with ValRox, vs. less than one per year in each of the three years after treatment.

³ This assumes that the effects of the drug last throughout the patient’s lifetime, which is, to be clear, still only an informed expectation because not enough time has passed for the first patients treated to live out the next six or seven decades. But for lifetime treatments, it would be miserably irresponsible to wait decades to treat other patients whose lives are at stake today, so the FDA has a process for approving such treatments if the results are sufficiently clear.
And, by the way, BioMarin’s clinical results are better than enzyme replacement therapy’s (see ² above) and reach the financial break-even point within eight years of the patient’s life.