This Week at FDA: FDA eases digital health oversight, MDUFA negotiation update, 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, FDA updated two guidances aimed at reducing oversight of digital health products and published meeting minutes from its user fee negotiations.

FDA has updated two guidances to ease its oversight of general wellness devices and clinical decision support (CDS) software in a move intended to bolster the adoption of artificial intelligence (AI)-enabled digital health products. The agency said that the changes will provide product developers with more flexibility and predictability in bringing products to market, while ensuring that medical-grade products continue to be regulated.

FDA has scheduled several important public meetings in the next few weeks, including hosting an information session on 21 January to engage researchers in discussing the development of research proposals to advance generic drug development. Agency scientists are expected to outline their research needs for FY 2026 during the meeting.

Emily Morris, a mathematical statistician in the Division of Biometrics IV at CDER's Office of Biostatistics, is scheduled to give a presentation on the key challenges of designing a Phase 3 trial in a rare disease setting and how statistics can help inform design decisions on January 15. She will also discuss the use of adaptive designs.

As stakeholders are expected to begin conforming to FDA's Quality Management System Regulation (QMSR) starting on 2 February, the agency is holding a town hall meeting to discuss what they can expect on 14 January. The final rule updating the regulation was published two years ago and is intended to align more closely with the regulatory expectations used by other regulatory authorities.

The Centers for Disease Control and Prevention (CDC) announced earlier this week that it was reducing the number of vaccines recommended as part of the childhood vaccination schedule based on consultations with peer nations and advice from HHS executives, including FDA Commissioner Marty Makary. It no longer broadly recommends the hepatitis A, hepatitis B, meningococcal, rotavirus, and influenza vaccines for all children, and only recommends a single dose of the human papillomavirus (HPV) vaccine; instead, the CDC now recommends some of the vaccines only for high-risk populations or “based on shared clinical decision-making” with healthcare providers.

Drugs & biologics

The Center for Drug Evaluation and Research (CDER) has updated its manual of policies and procedures (MAPP) on posting new safety information or potential risks from FDA's Adverse Events Reporting System (FAERS). The update is intended to clarify and align the agency's policies to with its newly identified safety signal (NISS) process.

CDER published meeting minutes from December between FDA and drug industry representatives as they negotiate the next Prescription Drug User Fee Amendments (PDUFA VIII) reauthorization deal. Notably, the agency recommended that it keep the current real-world evidence (RWE) pilot program in the next iteration of the user fee program to collect more information. The agency also responded to industry inquiries about topics such as agency employees dedicated to rare disease, incorporating regulatory science in their decision-making, and the use of patient-experience data (PED).

Agios's drug Aqvesme (mitapivat) was approved by FDA to treat anemia in adults with alpha- or beta-thalassemia. The agency noted that it is the first oral treatment option for patients with beta-thalassemia and the first drug approved to treat patients with alpha-thalassemia.

Omeros' injectable drug Yartemlea (narsoplimab-wuug) was approved to treat patients two years and older with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). The agency noted that it was the first drug approved for the treatment of TA-TMA.

Sanofi's Cablivi (caplacizumab-yhdp) was approved by FDA to treat patients 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. The drug was first approved in 2019 to treat adults with aTTP and was also approved by the Chinese National Medical Products Administration (NMPA) last month for patients 12 years of age and older with the rare blood disorder.

Cytokinetics's drug Myqorzo (aficamten) was approved by FDA to treat adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. The treatment for the rare inherited heart condition had initially received orphan drug designation and breakthrough therapy designation by the agency.

Shield Therapeutics' drug Accrufer (ferric maltol) was approved by FDA to treat patients 10 years and older with iron deficiency. The drug was first approved for adults in 2019, and the agency notes it is the first drug approved for children with the condition.

Boehringer Ingelheim's drug Jascayd (nerandomilast) was approved to treat adults with progressive pulmonary fibrosis (PPF). The drug was previously approved to treat idiopathic pulmonary fibrosis (IFP) and was eventually given breakthrough therapy designation for PFF.

Medtech

The Center for Devices and Radiological Health (CDRH) published meeting minutes from its December meetings with medtech industry representatives as they negotiate the next Medical Device User Fee Amendments (MDUFA VI) reauthorization deal. Notable among the discussions is a proposal by FDA to launch a new iteration of the Total Product Lifecycle Advisory Program (TAP 2.0), which would include advisors with both regulatory and non-regulatory experience, as well as regulatory and non-regulatory service lines, to facilitate more interaction with stakeholders.

CDRH also proposed additional IT tools to enable more automation in electronic submission, real-time validation of user fees, and facilitate more secure interactions between the agency and manufacturers. The center also emphasized international harmonization by exploring harmonized pre-market reviews, working with other regulatory authorities on issues such as supply chain resilience, increasing the frequency of meetings of global importance, and expanding outreach to support regulatory capacity building.

This Week at FDA: FDA eases digital health oversight, MDUFA negotiation update, and more

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