The U.S. leads the world in developing biomedical innovations, but it often falls short in delivering them to patients. The FDA approved the first PCSK9 inhibitor to treat cardiovascular disease in 2015; today less than 1% of eligible patients receive them. It’s a similar story with CAR-T therapy to treat blood cancers, first approved in 2017; today less than 20% of eligible patients receive them. Everyone from federal research agencies to new biomedical startups are rushing to capitalize on the new capabilities that AI brings to R&D. But unless we fix the breakdowns that keep our healthcare system from getting new innovations to patients, we’ll see a widening gap between what science and technology can create and what our healthcare system can deliver.
Virtually everyone working in healthcare is used to thinking in terms of a patient’s journey through the system. But what about the product’s journey? The healthcare system should not take years to deliver an FDA-approved product to its intended patient population. It undermines health outcomes, wastes resources, and threatens the sustainability of both biomedical innovation and care delivery. This is a complex problem that calls for a multi-stakeholder redesign of the system. An emerging concept called “biomedical health efficiency” offers a critical design tool when success can only be achieved by stakeholders working together rather than alone.
Biomedical health efficiency fosters system-level improvements that can optimize a product’s path from FDA approval to the patients that product was developed to benefit. Instead of point solutions or top-down policy mandates, improving biomedical health efficiency requires establishing a collaborative process where insurers, system administrators, biopharmaceutical companies, pharmacies, and patient groups agree on common points of measurement for determining how efficiently the system delivers product types for a given disease.
A collaborative approach is crucial for a few reasons. First, there is no magic bullet for a malfunctioning system. To improve biomedical health efficiency, multiple solutions across the system must be designed and implemented in coordinated ways. Further changes will be needed over time, too, as new diagnostics, therapeutics and knowledge emerge. Second, different actors in the healthcare system have their own goals and incentives—and each holds a unique perspective. Any solution that one actor might propose for making the system more efficient inevitably favors their own interests, with no guarantee of improving the overall system.
But bringing actors together to focus on the shared challenge of delivering a target product type to its intended patients, it becomes possible to understand and then align incentives toward a net improvement in system-wide efficiency. This process should be driven by three questions: First, how do we design a system that delivers the greatest beneficial impact from the biomedical product in question? Second, how do we align incentives across the healthcare system’s various players to fuel implementation and scaling? And third, what do we measure to know we’re making progress? The goal is to get stakeholders to co-create a shared set of outcome measures that works for all of them, and then let them self-organize in new ways to achieve those metrics.
Consider Alzheimer’s disease, where innovation has recently made early diagnosis and medical treatment possible for the first time since the disease was classified over a century ago. Recently the FDA approved a second blood biomarker test that can help clinicians arrive at a clinical diagnosis before symptoms become obvious, prompting possible treatment with one of two FDA-approved therapies that slow progression of the disease in early-stage patients. The numbers of Americans living with Alzheimer’s is projected to increase from 7 million to 13 million by 2050. As every moment counts with this progressive, debilitating disease, it’s essential to design efficient systems for delivering these interventions to people who can benefit, when they can benefit.
Individual stakeholders are doing what they can to introduce these innovations to patients. But without intentionally coordinating across these silos, these efforts will fail to produce the efficient system needed to reach patients at scale. Defining a shared metric, like “increasing the number of treatment-eligible patients offered disease modifying therapies for Alzheimer’s” would fuel coordination and alignment across the healthcare system.
To skeptics, this may sound beyond reach. But in 2020, Operation Warp Speed demonstrated what is possible when players from across the biomedical ecosystem—people and institutions with different, often conflicting incentives—co-design a system that speeds the development, regulatory approval, manufacturing and distribution of a new medical product. To be sure, the hardest part is getting players to the design table. That said, there is nothing like a shared existential threat—be it a pandemic or unexpected funding cuts coupled with massive national policy volatility that threaten patients’ lives —to motivate competitors and adversaries to work together to drive improved paths forward.
Similarly, when high cost, high value cell and gene therapies entered the market, they posed a different sort of existential threat to stakeholders. While they offered durable and potentially curative effects for patients, the financial risk to payers and the commercial risk to biopharmaceutical companies posed a challenge to biomedical health efficiency. The FoCUS project led the design and derisking of a number of new payment models, never before used in healthcare, which have since been deployed globally.
We can have the best science in the world—but it means little if most patients have to wait years to gain access to approved preventives and treatment. Through coordinated approaches to system redesign, we can cut healthcare waste and inefficiency and nurture a healthcare system that keeps pace with biomedical innovation.
Gigi Hirsch, MD, is Director of the Center for Biomedical Systems Design at Tufts University, where she leads the New Drug Development Paradigms (NEWDIGS) initiative.
Murray Aitken, MBA, MCom, is the Executive Director of the IQVIA Institute for Human Data Science.
