Life without disease should be the goal of the healthcare system and should be accessible to all.
10 years ago, in my first year out of medical school, I learned that we cannot outwork a broken system. Taking care of a 16–year-old girl in the hospital suffering from pain with every heartbeat and breath taught me that.
I will never forget when her mother was finally able to purchase the device we needed. The sad news was that her daughter had just passed on. Upon relaying the news, I expected grief. Instead, she expressed relief that her daughter was no longer suffering. We failed that girl in not providing a miracle. A miracle she should not have needed.
This was the first of many of my patients that required miracles. Not only biological miracles but also miracles of access, equity, and affordability. Let us make a system where miracles can be adopted and accessed so quickly that everyone has their fair share opportunity to the best outcomes. And if possible, live a life without disease.
I suspect your reaction to healthcare services may have been different to the others despite housing, education, and health being considered human rights. When it comes to receiving the best, in healthcare we don’t add the hurdle of meritocracy.
The current US healthcare system is struggling with potential imminent insolvency and misaligned economic incentives. The insolvency pressures stem from the inability to sustainably finance the multi-million dollar, single-dose cures that are increasingly coming on-line and the growing burden of financing the management of chronic diseases in the elderly through a social welfare mechanism in an aging population. This problem is further compounded by the inability to economically align on long-term health outcomes of the younger population due to consumer/patient portability in the system. We are unable to pay for a world without disease even if we had the clinical interventions to do so.
Monogenic disorders (disorders due to single-gene defects) are individually rare but as a group are present in 6% of the population. If we were able to cure all monogenic disorders—assuming a single payment of an average list price of US$ 2.6 million per cure and a target population of 6% of the annual birth population of 3.6 million—it would cost us US$ 561 billion per year. Generic or competitive entry may facilitate discounting. However, three generic competitors gets us to US$ 450 billion and eight gets us to US$ 110 billion. If we take it from a whole population perspective we get US$ 51, 41, and 10 trillion respectively. Our collective ability to sustainably finance cures is a bottleneck that will likely cause friction in therapeutic access, adoption, and innovation, leading to an addressable suboptimal health state of a significant proportion of the population.
There is currently no efficient financial mechanism to attribute and reward market participants that improve health outcomes, especially for long-term horizons. Because all participants are not rewarded on some appropriate basis derived from a consensus metric, each well meaning participant has created their version of bringing value to the system. These perspectives are many times antagonistic and not cooperative. As the market has been orienting towards value being defined in metrics related to improved patient outcome and cost-effectiveness, now is an appropriate time to create a mechanism where we effectively attribute and reward improvement. One stakeholder of note is the payor. They make their ROI by decreasing expenses of enrollees, i.e. passively. And do not benefit when the patient moves to another payor. This issue of portability results in decreased incentives to operate based on long-term horizons for patients.
As the US aims to optimize cost-effective financing of health, we believe the privatized model of US healthcare is a feature and not a bug. Solving how we sustainably finance genetic therapies is an opportunity to unlock the benefits for patients of an aligned self-regulating competitive market.
We believe a viable solution needs to be able to:
