The news this week that the Food and Drug Administration (FDA) approved Aduhelm to treat Alzheimer’s disease has been met with hope, but also skepticism in terms of the drug’s clinical safety and efficacy, and its cost. We’d like to offer a different perspective — a “forest through the trees” view — from America’s pharmacy benefit managers, PBMs, which are positioned squarely at the intersection of access and affordability for patients.
The approval of Aduhelm feels personal for so many people, including my family. My father suffered and passed away from Alzheimer’s just three years ago. He followed my grandfather and uncle, who passed away from early onset of the disease in his 50s. This new treatment gives me hope for emerging science — that it will drive the development of drugs that will allow individuals living with Alzheimer’s and their loved ones to have longer, healthier, and happier lives together.
The inspiration borne of innovation is not lost on PBMs. We welcome breakthrough treatments that give patients hope and higher-quality lives. Our job — on behalf of employers, unions, and government programs providing coverage to patients — is to enable access to innovations while balancing the safety, efficacy, and clinical appropriateness with the cost and value they provide.
The approval — and price point — of Aduhelm is not the healthcare industry’s first experience with a medication perceived to be a financial back-breaker for the health care system. The speculation of the budget-busting costs swirling around the introduction of a Hepatitis C cure, Sovaldi, and the PCSK-9 cholesterol-lowering drugs was largely unrealized because providers, physicians, and PBMs worked to strike the balance between cost and access for patients.
PBMs are recognized for leveraging market competition to negotiate discounts and rebates on prescription drugs. Our companies also negotiate value- and outcomes-based contracts for drugs that have quantifiable outcomes. Because the use of prescription drugs can lead to avoidance of more expensive procedures, paying for a positive outcome makes sense, and it is these common-sense cost measures that are the focus of much of our work and which are already being explored in the case of Aduhelm. These arrangements are a key element of our three-part plan for addressing high drug costs.
What is less well known is that PBMs also serve an important role as the hub connecting multiple aspects of the health care system that serves patients. Connecting different aspects of health care provides clinicians and health plan sponsors a line of sight into patients’ total care — from medical to pharmacy treatment. PBMs are not only benefit managers, but care managers on behalf of patients.
This coordination of multiple stakeholders working together is an example of how new medications become accessible to patients, and a way that PBMs focus on safety. In the case of a drug like Aduhelm, this coordination is all the more essential. Aduhelm was granted accelerated approval, a pathway designed to provide earlier patient access to potentially valuable therapies where there is an unmet need, and where there is expectation of clinical benefit but also uncertainty. That uncertainty includes a safety profile in which 40% of phase three clinical trial participants experienced brain swelling or bleeding. This new drug may represent a breakthrough for patients, but it also may not be for everyone. That is why the tools used by PBMs are so essential.
For example, PBMs empower patients and prescription drug prescribers to understand the known safety risks, side effects, efficacy, potential for drug-drug interactions, and clinical appropriateness of any prescription drug treatment. Giving doctors the ability, in real-time, to weigh the risks and benefits of prescribing medications for any given individual patient results in the right drug being prescribed at the right time.
For a drug like Aduhelm, PBMs’ care coordination work is designed to complement the FDA’s accelerated approval parameters to help physicians navigate a decision-making process that may be complicated by the additional evidence that must be generated as the FDA balances early access to this treatment against the fundamental considerations of safety and efficacy.
Much has been and will be said about this new treatment for Alzheimer’s. PBMs have a responsibility to leverage our care management tools to help both patients and federal regulators balance appropriate access and safety while additional evidence is generated about the risks and benefits of this drug. Our companies are ready to do what we do best — help patients get safe, appropriate and affordable access to the treatments they need.