This Week at FDA: MDUFA V public meeting, CMS’ Aduhelm decision, and more

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 look at upcoming FDA meetings to discuss the Medical Device User Fee Amendments (MDUFA V) program and a US-Canada joint consultation on several International Council for Harmonisation (ICH) guidelines. We also look at the Centers for Medicare and Medicaid Services (CMS) coverage decision for Aduhelm.
 
Following FDA’s decision to grant accelerated approval to Biogen’s Aduhelm (aducanumab) last year, the Centers for Medicare and Medicaid Services (CMS) finalized its controversial coverage decision for the drug that would limit coverage to patients enrolled in clinical trials. Prior to the decision, multiple lawmakers and the pharmaceutical industry expressed concerns about the coverage decision, arguing that CMS would be overstepping its authority and mandating duplicative trials of the drug.
 
This week, FDA officially announced a virtual public meeting to discuss the MDUFA V program on 19 April after releasing its draft commitment letter last month. The meeting will feature presentations from FDA and industry groups, as well as representatives of patient, consumer, and scientific organizations.
 
FDA will also host a joint meeting with Health Canada to gather feedback on several ICH topics on 11 May, including E11A, Q9(R1), Q2/Q14, E14/S7B and Q3D(R3). Each of the topics recently reached milestones in the formal ICH process. FDA also released the final version of ICH E8(R1) General Considerations for Clinical Studies this week, and opened a consultation on the M7(R2) addendum on the application of the M7 guideline to the calculation of compound-specific acceptable intakes.
 
The agency also released a draft questions and answers guidance on the use of whole slide imaging in nonclinical toxicology studies conducted in compliance with good laboratory practice (GLP). The draft guidance is jointly issued by all of FDA’s product centers and the Office of Regulatory Affairs.
 
Drugs and biologics
 
The Center for Biologics Evaluation and Research (CBER) also released updated data on two of its designations programs – fast track and the regenerative medicine advanced therapy (RMAT) designation – providing the latest look at how those designations are faring within the center. The RMAT designation data is broken up by requests received, withdrawn and rescinded designations, and product approvals. To date, just three products with RMAT designation have been approved, the most recent of which was Stratatech’s StrataGraft.
 
This week, Fierce Biotech reported that FDA’s Oncology Center of Excellence (OCE) is planning yet another project, this one labelled Project FrontRunner, that would look at ways to bring accelerated approvals to earlier stages of cancer treatment. “We really want people to be looking at accelerated approval not in the most refractory populations,” Pazdur told Fierce, “but let’s move these drugs up to an earlier disease setting as their first approval in randomized studies.”
 
The agency also announced a workshop on 6 May to discuss the roll of phytosterols in parenteral nutrition-associated liver disease (PNALD)/intestinal failure-associated liver disease (IFALD).
 
This week, our former colleague Zachary Brennan at Endpoints gives us another look at the thousands of pages of documents pertaining to FDA’s review of the Pfizer-BioNTech COVID-19 vaccine. While the documents have turned up “few surprises,” they offer a look at some of the behind-the-scenes details that go into a regulatory submission that aren’t usually made public.
 
FDA’s Office of Surveillance and Epidemiology (OSE) has released its 2021 annual report. The report details the office’s response to the COVID-19 pandemic, and its efforts to detect, assess, prevent and manage risks related to pharmaceuticals.
 
Medical devices
 
On Monday, the Biden administration said that Medicare Part B beneficiaries will be able to get eight free over-the-counter COVID-19 tests each month as the pandemic drags on. “Testing remains a critical tool in mitigating the spread of COVID-19, and we are committed to making sure people with Medicare have the tools they need to stay safe and healthy,” said CMS Administrator Chiquita Brooks-LaSure.
 
We’re also reading in STAT that Sens. Elizabeth Warren (D-MA) and Chuck Grassley (R-IA) have introduced a bill that would require FDA to issue regulations on OTC hearing aids within 30 days of its passage. The senators expressed frustration that the agency is behind on delivering the regulation.
 
In safety-related news, FDA sent a letter to healthcare providers alerting them to changes in reprocessing methods for certain Karl Storz urological endoscopes, as the “current reprocessing instructions … are inadequate and are being changed.” The agency said the company’s cystoscopes, ureteroscopes, cystourethroscopes and ureterorenoscopes are affected by the issue. The agency stressed that high-level disinfection methods or liquid chemical sterilization methods should not be used, and that the affected endoscopes should be sterilized after each use according to the sterilization methods called for in the specific device’s instructions for use.