By MacKay Jimeson

In a proclamation last year, President Joe Biden said, “in the next 30 years, the number of Americans with Alzheimer’s is expected to reach nearly 14 million, straining families and our health care system. Fortunately, we are on the cusp of life-saving advances that can forever change the course of the disease.” There is little doubt that neurodegenerative diseases like Alzheimer’s are one of the major epidemics of this generation. Improvements in medical treatments and care are desperately needed to change the trajectory of both the human and financial burden.

The President is right. After several major failures a decade ago in Alzheimer’s treatments, scientists, industry, and regulators are developing a better understanding of the disease and creating a new generation of medicines. Sadly, the federal government and our health insurers are more conflicted on who should have affordable access to these medicines.

Surprising many last week, the Veterans Health Agency (VHA) agreed to cover Leqembi for patients over 65 with early Alzheimer’s symptoms and elevated levels of amyloid plaque. This bold step broke from the healthcare payer orthodoxy established by the Centers for Medicare and Medicaid Services (CMS), who want to restrict access, despite the greenlight given by the Food & Drug Administration (FDA).

Leqembi was approved in January by the FDA using the Accelerated Approval pathway. The FDA uses Accelerated Approval when a medicine is intended to treat a serious condition and there is clinical evidence that it works, but more time or research is needed to gain traditional approval.

Alzheimer’s patients have clumps of sticky plaque on the brain called Beta Amyloid. Many Neuroscientists and regulators agreed that reducing amyloid plaque in the brain in early Alzheimer’s patients is a measure that can be used for Accelerated Approval. Follow-on confirmatory clinical trials are required for full approval. Leqembi demonstrated this effect in phase 2 clinical trials.

The purpose of Accelerated Approval is to approve medicines faster with the intent of giving patients access sooner. Our federal government is failing to deliver on the last part of the promise, or at least it is highly conflicted.

CMS says they need more evidence to consider covering drugs like Leqembi, but that is nonsense.  In an era that emphasizes value-based agreements, risk-adjusted contracts can be established with manufacturers that encourage evidence generation over the lifecycle of a medicine. These contracts can be designed to reward treatments that are demonstrating meaningful value to patients and caregivers over time, or, alternatively, offset any financial loss to the payer in the unfortunate case where a treatment fails to deliver on its promise.

The reality is that CMS tangled themselves in knots to avoid covering Aduhelm, another Alzherimer’s therapy that the FDA granted Accelerated Approval. The Aduhelm approval was viewed as controversial due to the data and unconventional engagement between regulators and industry. CMS created a policy for Medicare last year to only cover new biologic, anti-amyloid treatments for Alzheimer’s disease that receive traditional FDA approval – not Accelerated Approval.  Medicare patients can receive “enhanced access and coverage” if they participate in CMS-approved studies, but participation in these studies can be burdensome for patients and caregivers.

For Leqembi, these restrictions are pointless. The manufacturers already completed their phase 3 trial, published the data and in January submitted their application for traditional approval. In fact, in the phase 3 clinical trial, Leqembi became the first medicine that showed Alzheimer’s can be slowed down by reducing beta amyloid – a major advance in science. The FDA accepted the submission for regulatory review in March and signaled a decision will come in early July.

VHA seems to understand this. What’s the hold up at CMS?

For Alzheimer’s patients and their caregivers, time directly impacts quality of life. As an agency that is predominantly responsible for the health of Americans over the age of 65, CMS has been irresponsible to stand by a highly restrictive policy that is preventing patients from receiving treatment.  By not paying for Leqembi – which the manufacturers say will cost $26,500 annually – CMS will save in the short term, but not necessarily in the long term.

Today 6.7 million Americans age 65 or older are estimated to be living with Alzheimer’s dementia. By 2050, it is estimated that number will double. The Alzheimer’s Association has projected that the introduction of a therapy in 2025 that delays onset of the disease by five years would reduce the number of patients in the severe stage of Alzheimer’s from 6.5 million to 3.6 million in 2050.

Even if Leqembi only offers a modest delay in disease progression it is still a major advance for science and for patients. The past 20 years were hard for Alzheiemer’s patients and caregivers with emotional ups and downs of multiple large-scale clinical trial failures. Scientists were undeterred and learned from every setback. The persistence has started to pay off with a new generation of Alzherimer’s treatments being approved. We are starting to crack the Alzheimer’s nut and what we learn from these new approvals creates more promise that greater advances are on the horizon.

Alzheimer’s Disease is only the tip of the iceberg. There is a hunger among patients, industry and some regulators to break down past barriers to approval in difficult disease areas. Regulatory science is advancing in response to the needs of patients, who are suffering from diseases with few or no treatments. To beat many complex diseases a more flexible regulatory process is needed to succeed. Public-Private Partnerships, like the Critical Path Institute, are identifying new and scientifically valid ways to get cutting-edge medicines approved faster by the FDA. As a result, collecting evidence and financing treatment will need to evolve. CMS and private payers need to be equally responsive to patients and catch up.

The biologics division of the FDA recently announced an “Operation Warpspeed” for Rare Disease, which will focus mainly on cell and gene therapy. The FDA expects that starting in 2025 they will review 10-20 of these cutting-edge treatments annually. Predictably, payers are already crying foul. They say they cannot afford to pay for these medicines and that they will have to exclude patients.

For government and private health insurers it is time to evolve or die. The 20th Century health insurance model was designed to cover less expensive medicine to routinely manage chronic conditions. The 21st Century is different.  Advances in science and medicine are arriving that could not be imagined 30 years ago.  Innovation in how we finance healthcare should follow suit.

Addressing major diseases like Alzheimer’s should be a national priority and we should expend consistent, patient-centered decisions across federal agencies. The numbers of people impacted by Alzherimer’s will only grow. Government, industry, scientists, and payers must systematically work towards advancing science and technology, developing better care and treatments, and financing the needs of patients and caregivers.  Will it be expensive? Yes. But, better medicines reduce demand for health services and the burden of disease. That makes advancing science and medicine our best hope to improve human health and wellbeing, along with bending the cost curve over the long term.

MacKay Jimeson is the executive director of the Patient Access and Affordability Project at Patients Rising. He was previously an executive at Pfizer and an aide to former Florida Governor Jeb Bush.