Health

FDA’s Accelerated Approval Pathway: Separating Fact From Fiction

Today, you can go from hero to villain instantaneously. The Food and Drug Administration was rightfully applauded for efficiently reviewing COVID-19 vaccines for safety and efficacy. Just months later, the FDA is being booed for approaching another fatal disease with a sense of urgency.

Criticism immediately followed FDA’s approval of the first treatment for Alzheimer’s disease in nearly two decades. The criticism isn’t limited to the treatment but includes the accelerated approval pathway that FDA relied on.

Accelerated approval is an FDA review pathway limited to treatments for serious and often fatal illnesses that currently have few, if any, options. Patients benefit from faster access to promising medicines.

Accelerated approval originated in 1992 when HIV/AIDS was destroying lives and communities. Rather than basing its review on whether a medicine prevented death, which could take years while people died, the FDA looked at a surrogate endpoint and evaluated potential medicines on their ability to reduce viral load.

The concept worked — reducing viral load did prevent death. Medicines for HIV/AIDS were approved faster, deaths plummeted and HIV/AIDS became a manageable illness. In 2012, Congress authorized expanded use of accelerated approval for rare or life-threatening diseases or conditions.

Accelerated approval serves two important purposes. First, it provides much faster access for patients without compromising FDA’s rigorous, essential safety and efficacy regulatory reviews. Second, it creates an incentive for investment in new therapies for some of the most serious illnesses including those affecting very small patient populations.

Since those early days, more than 250 drugs have been approved through the pathway —extending and improving countless lives of people living with different cancers, rare diseases, and other serious illnesses.

The attacks on accelerated approval may be cresting after the Alzheimer’s treatment was greenlit, but they were already underway. If these criticisms negatively affect policy, they will hurt people with severe health challenges and further complicate their care.

Consider that the Medicaid and CHIP Payment and Access Commission recently recommended that manufacturers pay higher rebates to Medicaid for accelerated approval drugs than they do for conventionally approved medications. And despite specific guidance otherwise, some states may restrict access to these drugs in Medicaid by claiming that they are not “fully” FDA approved and are too expensive.

Given the stakes, these facts are important:

  • Accelerated approval is full, rigorous FDA approval. The standards for data are as high for accelerated approval as for conventional pathways, as current and former FDA commissioners have emphasized.
  • The science around surrogate endpoints is accepted and appropriate.
  • Patients and caregivers are willing to accept uncertainty in the face of inevitable disease progression. For example, when it comes to Duchenne muscular dystrophy, studies show a willingness to accept risk and uncertainty in exchange for potentially slowing disease progression and muscle loss. Patient testimonies and data demonstrate similar tolerance across other rare diseases. Patient perspectives appear to have been left out of the public debate over the Aduhelm approval.
  • EveryLife Foundation’s National Economic Burden of Rare Disease Study included 379 rare diseases affecting 15.5 million people and estimated that the overall economic impact in 2019 exceeded $966 billion. Costs absorbed directly by affected families accounted for nearly 60 percent of overall costs.
  • There is strong evidence that accelerated approval drugs are not driving health care costs. An analysis recently published in the American Journal of Managed Care found that these drugs accounted for less than 1 percent of yearly Medicaid outlays between 2007 and 2018, and added negligibly to spending growth.

By design of accelerated approval, it will take time to fully understand how effective Aduhelm will be against Alzheimer’s, but we must not let the debate undercut a program with a proven track record of saving and extending lives.

In parallel, we must pay attention to the overall impact of the accelerated approval pathway. For diseases for which accelerated approval is applied, we have a public health crisis and must deploy all resources to solving them. In diseases where there was once “nothing to do” for patients, we now have opportunities to slow or reverse disease progression, collect data and evaluate therapeutic impact over time, improve patient outcomes, and encourage scientific advances.

The accelerated approval pathway is working as intended: delivering better health to those in dire need. We must value and protect it.

 

Pat Furlong is the founding president and CEO of Parent Project Muscular Dystrophy, the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy.

Annie Kennedy serves as the chief of policy & advocacy for the EveryLife Foundation for Rare Diseases, a patient advocacy coalition dedicated to impactful, science-driven policy.

Ken Thorpe, Ph.D., is chairman of the Partnership to Fight Chronic Disease and a Robert W. Woodruff professor and chair of the Department of Health Policy & Management in the Rollins School of Public Health at Emory University.

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