I started the year with optimism, and there is good news to revel in, but I’d be lying if I said there weren’t also problems on the horizon. There are certainly plenty of reasons for insomnia in the news, but what’s keeping me awake at night in the healthcare field is this: we’re looking at an absolute avalanche of new treatments, many for rare disorders, all hitting the health care system in the next ten years. Which is absolutely great news in theory, but how is the health care system, already badly broken, going to handle it, both in the U.S. and around the world? Having a treatment or cure is worthless if patients can’t get it.
There are three big issues with the explosion of new treatments in the pipeline: 1. clinician knowledge/education, 2. cost to the system, and, 3. related to both clinician knowledge and the cost, patient access to the new and expensive treatments.
Clinicians are already overwhelmed with the information they need to know. The XLH community has direct experience with the oft-cited statistic that it takes seventeen years for a new scientific discovery to go from “bench (researcher’s lab) to bedside (patient’s access). It took 18 years for the discovery of FGF23 as the cause of XLH to be transformed into an approved treatment (burosumab), and now, almost six years after that approval, only a small percentage of the worldwide patients (especially adults) can actually access the treatment, even though everyone acknowledges it’s both safe and effective.
The XLH experience also demonstrates how overwhelmed general clinicians are with the rapidly expanding world of scientific knowledge. It’s perhaps a silly example, but even the clinicians whose offices are involved in the administration of burosumab injections are getting the commercial name wrong, calling it Crystiva or Crystivia (like the sweetener stevia?) instead of Crysvita. It’s not a complicated name, unlike many that they prescribe regularly, so I have to believe the error is due to information overload, that it’s just one too many things to remember, when they’re already maxed out, and aren’t dealing with it multiple times a day.
Now, imagine the rapid introduction of thousands of new treatments over the next five to ten years, probably all with bizarre names derived from gene locations. In theory, there could be one new treatment for the majority of the estimated seven thousand rare disorders, since they tend to have genetic causes. That’s clearly way too much information for a general practitioner to learn, but it’s even a lot for a specialist to keep track of, since they’ll need to know not just the name, but the dosages and the contraindications and the potential interactions with other treatments. And remember that specialists may treat a whole group of rare disorders, not just one. How are they going to cope with all of this new information? Will patients need to do even more to bring new treatments to the attention of their clinicians, and if so, will clinicians listen to us? How are patient advocates/groups going to reach all the affected patients in their communities to educate them about new treatments in a timely manner?
Second, how is the health care system going to absorb the cost? Gene/cell therapy is expected to be extraordinarily expensive. It holds the promise of a “one and done” treatment, an actual cure, but the assumption is that it will also have a commensurately high price tag. I’ve seen estimates in the three to five million dollar range for the sickle cell treatment that was recently approved. Again, the XLH community knows about expensive treatment, with burosumab costing in the vicinity of a million bucks every three to four years, and no likelihood of the price coming down through competition or a generic version. In the U.S. that means that insurers absorb the bulk of the cost (giving them incentives to deny coverage any way they can), while in other countries, the national health care system (taxpayers) absorbs the cost. One of the reasons why insurers are willing, however reluctantly, to pay for expensive rare-disorder treatments now is that there are relatively few of us (most rare diseases have no treatment options), so it’s a tiny blip in their bottom line. But imagine all seven thousand or so rare disorders that currently have no treatment suddenly have a multi-million-dollar cure. Insurers will go from paying for the occasional quarter-million-dollar-a year patient to potentially paying three million bucks (or more) apiece for ten percent of its insured population. Sure, there may be savings down the road (a cured patient won’t need ongoing treatment), but that’s a lot of money all at once, just in the hope of avoiding future expensive surgeries, etc. I’m not suggesting you should feel sorry for the sad, bankrupt insurance company of the future, but it’s going to be a struggle. Insurers aren’t going to simply roll over and die, so something will have to change about the way health care is paid for, and that massive a change won’t be easy.
And that brings me to the third concern: how will this explosion in new treatments affect patient access to state-of-the-art treatments/cures? Assuming clinicians even know about the options, will a doctor who only has one XLH patient really have enough information to decide between, say, burosumab treatments for life and a one-time gene therapy cure? And from the financial perspective, how will the new treatments/cures be paid for? Will insurers, suddenly overwhelmed with thousands of new, multi-million-dollar treatments change the rules for coverage of rare disorders? Will they decide, as is already happening in some countries, to weigh the likely exorbitant cost of gene/cell therapy against how much the patient is suffering, and only pay for the cure if they decide the suffering is worth the insanely expensive treatment? And how will patients prove how much they’re suffering? That outcome seems likely, considering how many people view health care as a luxury, not a right.
Have I given you insomnia yet? Gene/cell therapy is, I believe, literally revolutionary for medicine, for patients, and also for health care systems. Which is incredibly exciting for scientists and individual patients. But there will be challenges, and we need to identify them now, so we aren’t taken by surprise in a few years.
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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