This Week at FDA: House holds user fee markup, CDER’s ARC for rare diseases

This Week at FDAThis Week at FDA | 13 May 2022 |  By 

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug and medical device regulation and what we’re reading from around the web. This week, we saw legislation to reauthorize the FDA’s user fee programs advance in the House. We also learned that FDA is standing up a new program to speed the development of treatments for rare diseases with unmet medical need. Plus, we take a look at a new GAO report on FDA’s efforts to make tests available during the COVID-19 pandemic.
The biggest FDA-related news this week was the House Energy & Commerce Health subcommittee’s markup of legislation to reauthorize the agency’s user fee agreements. The legislative package was released last week after lengthy negotiations between FDA and industry. In a unanimous vote on Tuesday, the subcommittee advanced the legislation to the full committee, which Politico reports will consider the legislation next week. The Senate is still reportedly still working on its version of the package. Our former colleague Zachary Brennan has more on the markup at Endpoints.
The Center for Drug Evaluation and Research (CDER) on Thursday launched a new program dubbed Accelerating Rare disease Cures (ARC) to bolster the development of treatments that meet unmet medical needs for rare diseases. The center-wide effort is being led by the CDER Rare Diseases Team. “In its first year, CDER’s ARC Program will focus on strengthening internal and external partnerships with stakeholders and will engage with external experts to help identify solutions for the challenges in rare disease drug development,” CDER said.
We’re also reading this Government Accountability Office (GAO) report on FDA’s efforts to make tests available during the COVID-19 pandemic. The report highlights the need for policy to address testing shortfalls in future public health emergencies.
Drugs & biologics
This week, FDA approved the first oral form of Radicava (edaravone) to treat amyotrophic lateral sclerosis (ALS). Previously, the drug was only available via intravenous (IV) infusion. The drug’s sponsor, Mitsubishi Tanabe Pharma said the new dosage form offers patients more “flexibility in how they take their medicine.”
The agency also posted an overview of the recent Catalyst Pharms., Inc. v. Becerra ruling, which impacts the statutory interpretation of orphan drug development incentives, namely orphan drug exclusivity (ODE). “Under the Catalyst decision, the first company to gain approval for any use for a drug that has been designated for a rare disease will, in most cases, have seven years of exclusivity for its drug for the entire rare disease. Specifically, the court held that under the statute, ODE blocks approval of another company’s application for the same drug for the entire disease or condition for which the drug is granted orphan-drug designation, regardless of whether the drug was approved only for a narrower use or indication,” FDA explained. The agency said it believes the decision will “adversely affect children … and other populations that are typically studied later in drug development.”
FDA officials also detailed the agency’s efforts to improve the domestic supply of molybdenum-99 (Mo-99) made without highly enriched uranium (HEU), enabling a sufficient supply of the medically important radioactive isotope for the first time without reliance on foreign production. Due to the isotope’s short half-life of 66 hours, it cannot be readily stockpiled. “Through FDA and [the Department of Energy’s National Nuclear Security Administration] DOE/NNSA combined efforts, Mo-99 is now sufficiently produced from low enriched uranium (LEU) for medical imaging. This means the U.S. no longer needs to send HEU to nuclear reactors in other countries to produce Mo-99. As such, the U.S. has banned sending HEU overseas for the purpose of medical isotope production, further advancing national security objectives.”
We’re also looking at this warning letter posted on Tuesday that details good manufacturing practice (GMP) violations at Brigham and Women’s Hospital in Boston, MA related to the hospital’s production of positron emission tomography (PET) drugs.
Next month, FDA will host a public meeting on the financial transparency and efficiency of its user fee programs for prescription drugs, generics and biosimilars.
Plus, we heard from Endpoints that BridgeBio has arranged to sell its priority review voucher to an unnamed buyer for $110 million.
Medical devices
While the user fee legislation makes its way through Congress, the Center for Devices and Radiological Health (CDRH) has released a summary of public comments and an explanation of changes made to the MDUFA V agreement. The document details revisions made to several of the agreement’s provisions, including the additional of performance goals for the Total Product Life Cycle (TPLC) Advisory Program (TAP Pilot), postmarket safety, and aspects of the five-year agreement.
The center also issued a notice reclassifying human immunodeficiency virus (HIV) serological diagnostic and supplemental tests and HIV nucleic acid (NAT) diagnostic and supplemental tests into class II (special controls).


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