The Accelerated Approval pathway was established by the U.S. Food and Drug Administration (FDA) in the 1990s to boost R&D for HIV/AIDS, and the pathway has assisted the U.S. biotech industry in making remarkable advances over the last 30 years.
Today, the pathway is under attack – but we need it to expedite FDA approval for medications that address critical illnesses with no therapies and to enable breakthrough drugs for diseases without alternative treatments, explains today’s new episode of the I am BIO Podcast.
Accelerated Approval, which allows the FDA to speed approval of drugs for patients with serious and life-threatening conditions, “has allowed nearly 300 new drugs, most for rare cancers, to come to market,” says Rachel King, President and CEO of the Biotechnology Innovation Organization (BIO), and host of the podcast.
For some patients, it is “the difference between life and death,” she notes. “We have treatments for only about 5% of rare diseases.” “The accelerated approval pathway is a critical tool to find cures and treatments faster,” King adds.
How Accelerated Approval works
As BIO’s Chief Scientific Officer, Dr. Cartier Esham, explains on the podcast, “this pathway allows for approval when a medicine has shown a change to a biologic or physical response.”
This provides early access to treatments for patients with serious and life-threatening conditions.
She notes that drugs targeting untreatable diseases that the FDA greenlights don’t need to immediately prove they cure a disease and can enter the market if shown to impact a biomarker – like increasing T cells in HIV patients. She stressed, however, that Accelerated Approval drugs “are approved using the same gold standard for safety and effectiveness as any other medicine.”
According to Emil Kakkis, CEO of Ultragenyx, which specializes in rare disease development, biomarkers are sometimes even better: “in many rare diseases, they are a more accurate measure of underlying disease” than the standard of a patient simply getting better.
Kakkis believes “the biomarker framework of the accelerated approval pathway could transform the drug development process for rare diseases.”
John Crowley, who founded Amicus Therapeutics to find a cure for his children’s rare disease, says, “biotechnology is just a great big word that, for many people, just means hope.”
“What’s really exciting about the potential for accelerated approval is not just to get approval for a medicine today but to lay the groundwork,” Crowley noted, so many new treatments will eventually lead to a cure.
Accelerated Approval is under attack
Questioning FDA’s science-based decision, the Centers for Medicare & Medicaid Services (CMS) recently limited coverage of two Accelerated Approval drugs for Alzheimer’s only to patients enrolled in clinical trials.
This is problematic because it introduces uncertainty regarding FDA decisions and because CMS does not have the scientific expertise to make this kind of determination, King says.
Kakkis believes Accelerated Approval, unfortunately, “is not being used very often in rare disease,” calling further limitations “misguided.” From his perspective, Accelerated Approval should be strengthened, not weakened.
Per Duane Schulthess, CEO of Vital Transformation, a health research firm, most Accelerated Approval drugs gain traditional approval quickly. Drugs that take longer to get approved usually treat rare diseases, for which tests are challenging.
And though Accelerated Approval drugs must prove overall effectiveness a few years after entering the market, some lawmakers would shorten that timeline with “a bill that was looking at putting a hard cap of five years for a data collection part” of the approval.
If proposals to limit the program were implemented, per Schulthess, “it’s really gonna have an overwhelmingly deleterious impact on small orphan drugs, which is where we need the most innovation right now and is often where the best science is going.”
As King concluded, “It’s our responsibility to strengthen and broaden it and ask regulators to use it more frequently so that we can win the race against time for so many people in need.”