Published on November 21, 2017
Thanksgiving week in the U.S., many patients, like myself, are thankful for the development of medications that have kept us alive, with a good quality of life. But the debate and discussions and politicking go on regarding pricing, insurance coverage, and so on. And precision medicine is only going to make things worse.
Don’t get me wrong. Precision medicine is a good thing. As we wrote here the week of September 25th, it targets specific attributes of our individual diseases making treatment more effective, often with fewer side effects, and also helps us avoid medicines that just won’t work because of the genetic structure of our disease. All of that is great news, and Patient Power is part of that growing trend in medicine.
But that creates a simple economic dilemma that I raise for your consideration. How do we pay for large costs for developing treatments for small groups of patients?
Take the example of Luxturna, a new gene therapy awaiting approval for a certain form of blindness (IRD). It is expected to be priced in the million-dollar range. Seems outrageous.
But the market is small, with possibly as few as 3,000 to 6,000 patients who carry the precise gene mutation worldwide. As we wrote about CAR-T drugs in September, that’s a very small number of patients to cover the development costs.
An argument against paying that price, in an article we read this week, is just outrageous: “One of the biggest barriers for reimbursement is that patients with IRD don’t cost healthcare systems much money currently—they simply go blind.”
Is that really part of our healthcare equation? If it costs too much, let them go blind?
We’ve already seen another high-priced gene therapy drug pulled from the market in Europe after treating just one patient. The company said the cost of manufacturing each dose is so high that it will now save $2 million a year by withdrawing the drug from the market. But what about the patients? What happens to the others who might benefit from the therapy?
Getting back to Luxturna, a self-appointed institute with anti-pharma leanings has determined that the expected price point “makes this unlikely to be a cost-effective intervention at commonly used cost-effectiveness thresholds.”
Simply put, it’s damn expensive. We agree, it is expensive, and that’s the dilemma that applies to cancer drugs too.
- Is there a threshold that says the cost of saving a life (or saving someone’s sight) is too high? Those of you joining us from the UK know that NICE has done just that and won’t pay for cancer medications that are available in the U.S. if they cost “too much.” Those of you in Canada know paying for a cancer medication varies from province to province, which means your healthcare may actually depend on where you live. Is that what we want here in the U.S.?
- Do we tell private companies not to expect to recover costs, not to mention making even a small profit, and therefore discourage anything that’s not for a huge population?
- Do we leave it to the NIH or academia? Those groups do basic research but don’t do the research and testing required to bring a drug to market, and where would they get the money to do that anyway?
- We should lower development costs for these drugs, but how low can it go?
It’s not enough to just call pharmaceutical companies greedy—perhaps some are, but without profits there are no investors and without investors there’s no money to develop these new drugs. It’s not enough to just blame insurance companies and say they should shell out large payments at once. But it’s also not enough to say to patients we have the technology, we have the know-how, but we’re not going spend the money to save your life. We find that hard to accept when a football commissioner is expecting a salary of $50 million plus benefits.
What we suggest, as we have before, is to look at the total healthcare bill—hospitals, doctors and drugs. Look at offsets, such as when a drug can reduce hospitalizations. Look at economic benefits when those of us with cancer get treatments that keep us in the workplace contributing financially. And above all, don’t fall for easy answers to a complex situation.
Beyond that, these are decisions we as a society have to make. We would love to hear your thoughts and suggestions and for you to share them, so this is truly a patient perspective.
Wishing you and your family the best of health!
Cancer Patient and Co-Founder, Patient Power LLC
Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.
Your site is AWESOME! Thank you all so much for this incredible resource to families who are in crisis/affected by cancer.