Are We Ready for the Cure for Cancer?

The entire healthcare system is built around the idea that we can detect, diagnose, and manage disease effectively for patients. This pandemic has been a stress test of that capability, and we have roundly failed that test. If we can’t do those “easy” things—test for the virus, provide medical care for those that need it, even just get people the right PPE—how are we possibly going to absorb major new technologies into the system? 

COVID-19 has been a massive disruption to our system. But there’s another one coming. We’re in the middle of a fundamental shift in healthcare which will require major change across the entire landscape: our increasing ability to fix the underlying cause of disease by manipulating biology directly into new therapeutics. These are the most complicated, but potentially most impactful, medicines humankind has ever seen. 

This new category of therapeutic works by engineering the fundamental unit of disease itself—a cell or gene—into medicines that live inside our body. These “living medicines” could, in theory, actually cure many of our most devastating diseases. With gene therapy, we can correct the genetic error that causes sickle cell anemia instead of giving patients monthly blood transfusions for the rest of their lives. CRISPR, now a Nobel-winning gene editing innovation, is being developed as a potential treatment for several diseases, including COVID-19. And by engineering cells for CAR T therapy, we can reprogram a patient’s own T cells so that their immune system attacks specific cancer cells, melting the cancer away instead of dosing people with chemotherapy that poisons cells indiscriminately. If we can’t effectively route face masks to healthcare workers, how will we route engineered cells to a patient dying of cancer?

If we can’t effectively route face masks to healthcare workers, how will we route engineered cells to a patient dying of cancer?

Living medicines are pouring into our system at an astonishing rate: the Food and Drug Administration (FDA) is currently overseeing 800 active applications for such cell and gene therapies, and by 2025, could approve 10-20 each year. The arrival of this new category of medicines is a seismic event that will shake up the entire system. Nearly everything about how we evaluate, manufacture, deliver, administer, and pay for our healthcare will need to change in response. As our medicines become programmable, we will need to reprogram our healthcare system to absorb them—otherwise, our innovations will overwhelm our infrastructure. 

As with all new vaccines and medicines, the first order of business is of course the need to make sure they are safe—recent deaths in gene therapy clinical trials serve as a grim reminder of this sacred obligation.  So we need new approaches for evaluating the risk and benefit of these complex therapies to get them to the patients who need them. When editing DNA, we can’t make any “typos”; when engineering immune system cells, the immune system can’t overheat. Once we inject CRISPR machinery to edit genes into the human body, there’s no turning back. So we test and introduce these powerful tools into the human body very carefully. We do it outside the body first, “checking our work” there, or editing cells within contained areas, like the eye. Regulators like the FDA are also creating new frameworks for clinical trials: testing multiple cell therapy candidates faster; trials that adapt based on “real world” findings; even smaller trials—just a few cures can go a long way towards proving a drug actually works. 

As our medicines become programmable, we will need to reprogram our healthcare system to absorb them—otherwise, our innovations will overwhelm our infrastructure.

Once we know these living medicines are safe, we have to produce them at scale. These medicines can’t be mass produced like pills and capsules, and we still have many manufacturing hurdles to overcome. For example, the way we get gene therapies into the human body right now is by using a modified virus (adeno-associated virus, AAV) as a delivery vehicle. Viruses are notoriously finicky; the process of “growing” them requires deep domain specialization and technical customization, and purification and quality control are critical. Making complex medicines like these at scale requires dedicated, specialized facilities. Some companies and medical centers are investing billions of dollars to push the bounds of the industry’s manufacturing capabilities; other institutions are banding together into public-private partnerships. That’s just the beginning of what’s needed to create enough supply for a very desperate demand.

Then we need to get these medicines into the hands of patients. You can’t just pick up reprogrammed cells at your corner pharmacy; these medicines are made from you, for you—they are as much medical procedures (think cellular surgery) as they are medicines, so they require an incredibly sophisticated supply chain. CAR T therapy for cancer treatment involves complex “vein-to-vein” logistics: removing a patient’s cells at a hospital, sending them to the manufacturer for reprogramming and regrowing, sending them to the hospital for infusion into the patient. The process is the product. The chain of custody has to be rigorously maintained—those cells must remain alive, and you definitely can’t have one patient getting someone else’s cells. 

We absolutely must make sure that the benefits of powerful new medical technologies like these don’t belong only to a select few. That’s not access at all.

When it comes to saving lives, the last mile is everything. So how do we get these medicines to patients? As of 2018, only 40 medical centers in the U.S. were authorized to administer CAR T. Medical centers will need to retrofit their systems, invest in software, and embrace other technologies that fully capture both the cell’s and the patient’s journey. Otherwise, only patients with access to a select few medical centers will receive those life-saving medicines. We absolutely must make sure that the benefits of powerful new medical technologies like these don’t belong only to a select few. That’s not access at all.

And then, access isn’t the same as affordability. The hard truth is, our current system is not well set up to pay for cures. These therapies are expensive to make and deliver. CAR T costs (including fees for hospital stays and supportive care) could total more than $1 million per patient. Gene therapy Zolgensma made headlines as the most expensive medicine in history, at $2.1 million per patient. With all the living medicines in development, it’s not going to hold that title for long. Pricing these therapies accounts for the past R&D that went into them, the present costs of making them, and the future costs to the healthcare system they avoid. 

But what’s the value of a cure? We need new ways to value, pay for, and share the financial burdens and benefits of these “one-and-done” medicines. Instead of price per dose, nonprofits like the Institute for Clinical and Economic Review (ICER) think about value per life. We’re already seeing new payment models—like installment models with performance guarantees (if it doesn’t work, you don’t pay), a sort of “mortgage for a cure.” Or, if you’re feeling lucky, even a lottery. We could even be seeing the Netflix-ication of medicine, where we essentially pay for a subscription to an engineered gene or cell that lives and works tirelessly inside our bodies to cure disease.

The sad reality is even if we had the cure for cancer in hand tomorrow—or if we discover we can program our immune cells to fight off COVID-19—our current system couldn’t handle it.

The sad reality is even if we had the cure for cancer in hand tomorrow—or if we discover we can program our immune cells to fight off COVID-19—our current system couldn’t handle it. Every new technology introduces second-order effects, and more questions: What happens when a therapy becomes part of your own biology? Who’s responsible for payments if a patient changes insurance? What new ecosystems might form around all this?  

Living medicines made of cells and genes are blurring lines all across the industry: from medicines as products to procedures, from paying per pill to mortgages for health, from managing care to creating real cures. But even the most effective therapies don’t work if they can’t reach patients. Innovation needs new infrastructure: cars needed roads and highways; telephones needed cables and wires. All of the stakeholders in the healthcare system—regulators, manufacturers, payors, and providers—need to prepare. 

The cures are coming. Now is the time to build those roads. Or we may find that we aren’t able to take our medicine.