We need the Accelerated Approval pathway to enable breakthrough drugs for diseases without alternative treatments, explains today’s new episode of the I am BIO Podcast.
What is Accelerated Approval? Created to counter AIDS/HIV in 1992, Accelerated Approval (AA) has allowed nearly 300 new drugs, most for rare cancers, to come to market, says podcast host and BIO CEO Rachel King.
How it works: Drugs targeting untreatable diseases are greenlit by the Food and Drug Administration (FDA) “…based on substantial evidence that a surrogate, or intermediate endpoint, is reasonably likely to predict clinical benefit. This pathway allows for approval when a medicine has shown a change to a biologic or physical response.” says BIO Chief Scientific Officer Dr. Cartier Esham. After entering the market, further FDA-mandated clinical trials are then conducted to confirm the drug’s effectiveness.
The majority of AA drugs gain full approval within 5 years, notes Duane Schulthess, CEO of research firm Vital Transformation. Drugs that take longer usually treat rare diseases, for which tests are challenging, and can take years.
Sometimes biomarkers are better: “In many rare diseases, they are a more accurate measure of underlying disease” than the standard of a patient simply getting better, says Emil Kakkis, CEO of Ultragenyx, which specializes in rare disease development.
But AA is under attack: The Centers for Medicare & Medicaid Services (CMS) recently limited coverage of two AA drugs for Alzheimer’s, questioning FDA’s science-based decision. And though AA drugs are required by FDA to confirm overall effectiveness a few years after entering the market, some lawmakers would shorten that timeline. This would have a chilling affect on the AA pathway, confirmatory trials can often take years for a variety of legitimate reasons, such as low enrollment.
A strengthened AA means hope: John Crowley, who founded Amicus Therapeutics to find a cure for his children’s rare disease, says that 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 just like it did in HIV AIDS for the next 10, 20 years of research for many new treatments."
Listen: The new season of the I am BIO Podcast is available via Apple, Google, Spotify, or wherever you get your podcasts, with new episodes released every other Tuesday.
More Health News:
The Wall Street Journal: Drug middlemen accused of price fixing by Ohio attorney general
“The lawsuit filed Monday by the Ohio attorney general in a state court alleges Cigna Group, Humana Inc., and Prime Therapeutics LLC shared pricing and other information gathered by a Swiss subsidiary to gain leverage during negotiations with drugmakers for rebates.”
U.S. Chamber of Commerce: Patient Access Report
“The price controls included in the Inflation Reduction Act will have a detrimental impact on biopharmaceutical innovation. Government intervention in price setting undermines the innovation ecosystem that empowered the U.S. to become one of the most innovative countries in the world.”
